StemCell Therapy

CARLSBAD, Calif., Aug. 14, 2017 (GLOBE NEWSWIRE) -- International Stem Cell Corporation (OTCQB:ISCO) (www.internationalstemcell.com) (ISCO or the Company), a California-based clinical stage biotechnology company developing novel stem cell-based therapies and biomedical products, today provided a business update and announced operating results for the three and six months ended June 30, 2017.

Over the period ended, we have substantially improved our business indicators, including a significant decrease in our cash burn, said Andrey Semechkin, PhD, Co-Chairman and CEO of ISCO. Additionally, we are successfully continuing with phase I Parkinsons disease clinical trial. Based on our success in the current clinical trial, we are working towards commencing phase II of the traumatic brain injury, for which a more detailed update will be released shortly.

Year-to-Date Financial Highlights

Recent Clinical Trial Highlights

About International Stem Cell Corporation

International Stem Cell Corporation is focused on the therapeutic applications of human parthenogenetic stem cells (hpSCs) and the development and commercialization of cell-based research and cosmetic products. ISCO's core technology, parthenogenesis, results in the creation of pluripotent human stem cells from unfertilized oocytes (eggs). hpSCs avoid ethical issues associated with the use or destruction of viable human embryos. ISCO scientists have created the first parthenogenetic, homozygous stem cell line that can be a source of therapeutic cells for hundreds of millions of individuals of differing genders, ages and racial background with minimal immune rejection after transplantation. hpSCs offer the potential to create the first true stem cell bank, UniStemCell. ISCO also produces and markets specialized cells and growth media for therapeutic research worldwide through its subsidiary Lifeline Cell Technology (www.lifelinecelltech.com), and stem cell-based skin care products through its subsidiary Lifeline Skin Care (www.lifelineskincare.com). More information is available at http://www.internationalstemcell.com.

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Safe harbor statement

Statements pertaining to anticipated developments, expected clinical studies (including timing and results), progress of research and development, and other opportunities for the company and its subsidiaries, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates,") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, regulatory approvals, need and ability to obtain future capital, application of capital resources among competing uses, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the company's business, particularly those mentioned in the cautionary statements found in the company's Securities and Exchange Commission filings. The company disclaims any intent or obligation to update forward-looking statements.

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International Stem Cell Corporation Announces Operating Results for the Three and Six-Months ended June 30, 2017 - GlobeNewswire (press release)

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ASCTCs most advanced product is an assay that can detect, very early in the drug development pipeline, drug candidates that will ultimately fail because of their toxicity to tissue stem cells.The companys patent portfolio contains biotechnologies that solve the two main technical problems production and quantification.In addition, the portfolio includes novel technologies for isolating cancer stem cells and producing iPSCs.

Boston, MA (PRWEB) August 29, 2014

A major challenge before new biotechnology start-up companies, especially ones in the biotech start-up dense realm of Boston-Cambridge, is gaining visibility that can lead to important strategic alliances and able investors. James Sherley, the Director of Bostons Adult Stem Cell Technology Center, LLC (ASCTC), has made increasing the local and national visibility of his company an important priority since he started the company in September 2013.

In addition to a social media marketingcampaignlaunched earlier in July of this year, Director Sherley has targeted research and development conferences both nationally andinternationallyto increase industry awareness of ASCTCs unique portfolio of intellectual property available for licensing and its current commercial development targets. The company is focused on producing two products to address two important needs in drug development and regenerative medicine, respectively, that it is uniquely positioned to address.

ASCTCs most advanced product is an assay that can detect, very early in the drug development pipeline, drug candidates that will ultimately fail because of their toxicity to tissue stem cells. ASCTC developed the new technology in partnership withAlphaSTAR, Corporation, located in Long Beach, California. Currently, such lurking drugs are not detected until after expensive animal testing, more expensive clinical trials, or worse, after marketing. Director Sherley refers to the second product as, A future of pounds and pounds of normal adult tissue stem cells. The company holds a patented technology for mass production of human tissue stem cells. The initial production target is human liver stem cells that can be used to make mature human liver cells for use in drug development and to support liver transplant patients. The company also holdspatentsfor production of pancreatic stem cells and hair follicle stem cells.

The sponsor the 2014 Stem Cells & Regenerative MedicineConference, in Boston, September 15-16, Terrapinn, Inc., invited ASCTC to attend as a VIP guest. Although ASCTC will not make a formal presentation at this conference, Director Sherley will participate in a roundtable discussion on the topic, Articulating value for up-and-coming regenerative medicine, stem cell and cell-based therapies.

Later in September (22-24), Director Sherley will present one of the selected Next Generation Presentations for new companies atBioPharm America 2014, also taking place in Boston. In addition to the public presentation, ASCTC will also participate in confidential partnering meetings with potential investors and strategic alliance partners arranged by conference organizers.

In October, Director Sherley will present to a primarily academic research audience a more detailed accounting of ASCTCs computer simulation technology for quantifying tissue stem cells in culture. This technology is the basis for the companys new assay for tissue stem cell toxicity. Director Sherley is particularly interested in the response from several experts in tissue stem cell growth dynamics who are invited speakers. The symposium, which will take place at Rhode Island Hospital, a medical affiliate of Brown University in Providence, has the goal of presenting emerging disruptive research in the area of Novel Stem Cells and Vesicles. Director Sherley is a member of the symposium organizing committee.*****************************************************************************************The Adult Stem Cell Technology Center, LLC(ASCTC) is a Massachusetts life sciences company established in September 2013. ASCTC Director and founder, James L. Sherley, M.D., Ph.D. is the foremost authority on the unique properties of adult stem cells. The companys patent portfolio contains biotechnologies that solve the two main technical problems production and quantification that have stood in the way of successful commercialization of human adult tissue stem cells for regenerative medicine and drug development. In addition, the portfolio includes novel technologies for isolating cancer stem cells and producing iPSCs. Currently, ASCTC is employing its technological advantages to pursue commercialization of mass-produced therapeutic human liver cells and facile assays that are early warning systems for drug candidates with catastrophic toxicity due to adverse effects against adult tissue stem cells.via

During the Second International Adult Stem Cell conference at the Vatican, a boy who had his windpipe replaced with one grown using his own stem cells won the Pontifical Hero Award for his courage. Ciaran Finn-Lynch, 14, was born withlong-segment tracheal stenosis,a condition that resulted in a narrow windpipe and made it hard for him to breathe.The operation involved taking a donor trachea and seeding it with stem cells taken from Ciarans bone marrow. The result of the procedure was that after six months, his trachea looked almost normal. Further more, the operation used his own cells, preventing the need for anti-rejection drugs.

Vatican Honors Boy for Courage During Stem Cell Trachea Transplant Operation that Used His Own Cells

During the Second International Adult Stem Cell conference at the Vatican, a boy who had his windpipe replaced with one grown using his own stem cells won the Pontifical Hero Award for his courage.

Ciaran Finn-Lynch, 14, was the second person to receive the award, and he made the trip from Northern Ireland to the Vatican to receive it.

Ciaran is a shining example of what this result has shown, said his father, Paul Finn, in an April 12 interview with CNA.

His mother, Colleen Finn, said we need to have faith in God to get through all of this.

This has made our faith stronger because we need more and more prayers all the time, she added.

Ciaran was born with long-segment tracheal stenosis, a condition that resulted in a narrow windpipe and made it hard for him to breathe.

He had a major transplant surgery to rebuild his trachea when he was two years-old.

Doctors placed metal stents to hold his windpipe open and he went without any major issues until he was 10 years-old.

One day after school, the stents that had been placed in his windpipe started to cut into his aorta, the main blood vessel coming out of his heart.

He was taken to intensive care at Belfast Hospital and then later transferred to Londons Great Ormond Childrens Hospital.

He had several operations but he had more bleeding from his stents, said Doctor Paolo De Coppi, head of the surgery unit at University College Londons Institute of Child Health, during the April 12 morning session of the conference.

The leader of our team didnt know what to do next, but an option was to do an operation done before on an adult in Barcelona. But we didnt have the time to do that, De Coppi explained.

But we did something similar and it was a quite difficult operation, he said.

The operation involved taking a donor trachea and seeding it with stem cells taken from Ciarans bone marrow.

The result of the procedure was that after six months, his trachea looked almost normal.

Ciaran is doing really well and I think he has a chance to become a rock star, since he plays the drums so well, De Coppi commented after showing a video of Ciaran playing with a band.

Ciaran told CNA that it felt good to receive the award and that he was happy with his life.

His father noted that the stem cells have been a great contribution to Ciarans procedure.

What weve heard here these last couple of days (at the conference) has been amazing, knowing theyre talking about building other organs, Paul Finn said.

Ciarans mother noted that she was happy that her son is not on any medication, since the operation used his own cells, preventing the need for anti-rejection drugs.

You just have to keep going on for him, and you cant show that youre scared or teary and you just have to put a brave face on, said Colleen.via

Parkinson's: Adult Stem-Cell Use Proves Successful Once Again!

LifeNews.com reports the results were published in the February issue of the Bentham Open Stem Cell Journal. Dr. David Prentice, a fellow with the Family Research Council, says the research features only one patient.

The gentleman was treated with stem cells into only half of his brain, and he went almost five years (without symptoms), he explains. Now his symptoms did start to return after that, and obviously hed like the other half of his brain treated.

The patients motor skills improved by over 80 percent in the first five years after the procedure. Prentice says he was able to have an active lifestyle. During that time he was traveling all around the world and living a full life, he points out.

David Prentice (FRC)No human embryos were killed in the research. They used the gentlemans own adult stem cells, so obviously theres no chance of transplant rejection, no tumors, Prentice notes, and of course, adult stem cells really work in patients.

UCLA researchers will now expand their work to 15 humans.via

Revolutionaryfindings in study by researchers in Korea suggest the first real breakthrough toward preventing Alzheimer's and helping millions of patients and families by relieving its symptoms. Researchers announced this week the results of a study that suggests an astounding possibility: adult stem cells may not only have a positive effect on those suffering from Alzheimer's disease, theycanprevent the disease.Using fat-derived adultstem cells from humans,adipose-derived mesenchymal stem cells, researchers were able to cause Alzheimer's disease brains in animal models to regenerate. For the first time in history, stem cells were used toidentify the mechanism that is key to the treatment of Alzheimer's disease, and demonstrated how to achieve efficacy as well as prevention of the symptoms of Alzheimer's with adult stem cells, a "holy grail" of biomedical scientists for decades.

In the first study of its kind, researchers at Korea's leading university and the RNL Bio Stem Cell Technology Institute announced this week the results of a study that suggests an astounding possibility: adult stem cells may not only have a positive effect on those suffering from Alzheimer's disease, theycanprevent the disease.Using fat-derived adultstem cells from humans [scientific term:adMSCs, orhuman, adipose-derived mesenchymal stem cells], researchers were able to cause Alzheimer's disease brains in animal models to regenerate. The researchers, for the first time in history, used stem cells toidentify the mechanism that is key to treatment of Alzheimer's disease, and demonstrated how to achieve efficacy as well as prevention of the symptoms of Alzheimer's with adult stem cells, a "holy grail" of biomedical scientists for decades.

Alzheimer's disease, the most common form of dementia (loss of brain function), is the 6th leading cause of death, and affects 1 in 8 people -- more than breast cancer. As of 2010, there were 35.6 million people with Alzheimer's disease in the world, but this number is expected to double every 20 years. It is estimated that the total cost of Alzheimer's is US$604 billionworldwide, with 70% of this cost in the US andEurope. To put that in perspective, Alzheimer's care costs more than the revenues of Wal-Mart (US$414 billion) and Exxon Mobil (US$311 billion), according to the British World Alzheimer's Report of ADI. The cost of Alzheimer's is at the top of health economists' list of the disorders of aging that could topple nations' entire economies, and that regularly ruin not only the lives of patients but of their relatives.

According to the results of this first major study, Alzheimer's may soon be a preventable disease, or even a thing of the past. Equally important, the safety human administration of the kind of adult stem cells used in this experiment has been established in multiple articles and government-approved clinical trials.

THE RESEARCH:

The study was jointly led by SeoulNational UniversityProfessorYoo-Hun Suhand RNL Bio Stem Cell Technology Institute (SCTI) director Dr.Jeong-Chan Ra.

The researchers and their teams injected stem cells into mice genetically designed to have the core symptoms and physiology of Alzheimer's disease. They were able to identify that these human stem cells, derived from adipose tissue, behave in a very special way when injected into the tail vein of mice subjects. The cells migrated through the blood brain barrier, thought by many to be impossible for adult stem cells to cross, and went into the brain. In fact,fluorescent labeled cells were monitored for distribution in subjects and the team identified that the infused cells migrated throughout the bodiesincluding brainexcept the olfactory organ, and therefore confirmed that IV infused stem cell can reach to the brain across the blood brain barrier.

The team infused human adipose stem cells intravenously in Alzheimer model mice multiple times two weeks apart from three month to 10 month.Once there, the mice who received cells improved in every relevant way: ability to learn, ability to remember, and neuropathological signs. More important, for the first time ever, Alzheimer model mice showed the mediation of IL-10, which is known for anti-inflammation and neurological protection.

The team also found that stem cell restored special learning ability from Alzheimer model subjects with great reduction of neuropathy lesions.This was found using tests used for Alzheimer's disease: behavioral assessment. In assessment it was found, amazingly, that stem cells' therapeutic effect on Alzheimer's disease was tremendous. This was also found in pathological analysis. The key though was prevention: the scientists showed that stem cells, when infused into Alzheimer's mice, decreased beta amyloid and APP-CT, known to cause brain cell destruction, leading to dementia and Alzheimer's disease. In the lab it was clear that stem cells increased neprilysin, which hydrolyzes toxic proteins. No other compound or treatment has ever suggested so strongly the potential to prevent, as well as stop, this epidemic of incurable dementia sweeping across suffering patients and their families.

Stopping Alzheimer's disease, let alone preventing it, is the focus of thousands of researchers worldwide. Speaking of their breakthrough discovery,Professor Yoo-Hun Suh, who led the study, said, "It is a ground breaking discovery that such a simple method as IV injection of the safest autologous adipose stem cells, without causing any immune rejection, or any ethical issues, opened a new door to conquering Alzheimer's disease, one of the most horrible, expensive and incurablediseases of our time." Joining him, leader of the RNL Bio Stem Cell Technology InstituteDr.Jeong-Chan Rasaid, "It has never been more clear that it is an ethical imperative for governments to provide patients with incurable diseases with their right to participate not only in studies like this but in therapies with such obvious potential, once they have been tested as many times for safety as has our technology." Both scientists stressed that the real breakthrough in their complex research is the prevention of the onset of symptoms.

Specifically, stem cells grafted in the brain, in another part of the study, were identified to induce cell division and neuro differentiation of endogenous neuro progenitor cells around the hippocampus and its surrounding cells and increase in great deal the stability of dendrites and synapses. Stem cell also contributed various anti-inflammatory and neuro growth factors, especially increased the expression of IL-10. This again suppressed apoptosis of brain neurons, the prevention effect against Alzheimer's disease.

Dr. Ra of RNL Bio noted that, "RNL Bio has already completed government-approved clinical trials confirming the efficacy of RNL Bio stem cells in the management and treatment of other diseases, including osteoarthritis, limb ischemia, and progressive hemifacial atrophy (Romberg's disease)."

This study was published in a recent volume of the renowned, peer-reviewed U.S. medical journalPLOS ONE. Images, plans for future efforts, and impact on this crushing disease will be discussed when the scientists discuss the details of this revolutionary study in a press conference inSeoulonSeptember 27th.via

SOURCE RNL BIO CO., LTD.

Type 2 Diabetes Trial Using Mesoblasts Proprietary Adult Stem Cells Yields Positive Results

MELBOURNE, Australia, June 18, 2014 Results from the Phase 2 trial of Mesoblasts proprietary adult stem cells in type 2 diabetes patients have been presented at the scientific sessions of the American Diabetes Association annual meeting.

Type 2 diabetes and its complications are considered to have an underlying immunological component associated with excessive pro-inflammatory cytokines.

The immunomodulatory properties of Mesoblasts Mesenchymal Precursor Cells (MPCs) provided the rationale for conducting the study.

The Phase 2 randomized, single-blind, placebo-controlled, dose escalation trial was conducted across 18 U.S. sites. The trial evaluated the effects of a single intravenous infusion of 0.3, 1.0 or 2.0 million MPCs/kg or placebo over 12 weeks in 61 patients who were inadequately controlled on metformin alone or with one other glucose-lowering agent. Mean diabetes duration was 10 years.

The key findings from the study:

The study investigators concluded there was sufficient evidence to support further evaluation into the use of MPCs in type 2 diabetes and its complications, and to explore further the effects of MPCs on disease mechanisms.

Multiple Sclerosis - Italians Lead Clinical Trial Testing Safety and Effectiveness of Stem Cell Transplantation MS Patients

(ANSA) Boston, September 9 Mesenchymal stem cell therapy to treat multiple sclerosis so far appears safe and without side effects, according to data released Tuesday and obtained through clinical trials on patients as part of the international Mesems project coordinated by University of Genoa neurologist Antonio Uccelli.

The results were announced ahead of the World Congress on Treatment and Research in Multiple Sclerosis opening in Boston Wednesday through Saturday. The Mesems project involves researchers from nine countries Italy, Spain, France, Britain, Sweden, Denmark, Switzerland, Canada and Australia.

It is the first large phase II international multicentre clinical trial to determine the safety of a consensus treatment protocol established by the International Mesenchymal Stem Cells Transplantation Study Group to obtain information on its effectiveness on multiple sclerosis patients.

So far, 81 patients have been involved in the project half of the 160 needed for the whole clinical trial. About 73 or 90% of those involved in blind testing were given at least one injection with mesenchymal therapy or got a placebo while 51 or 63% were given both injections and 27 33% completed the study.

The promising result is that so far none of these 27 people have suffered significant adverse events, which means that, so far, the treatment appears to be safe, said Uccelli. The neurologist warned that caution is necessary and that the effectiveness of the therapy can only be determined once the study is completed in 2016.

Uccelli however added that preliminary studies on animals have persuaded researchers that mesenchymal stem cells can halt inflammation on the central nervous system and probably succeed in protecting nervous tissue, even repairing it where damage is minor. Out of the 81 patients recruited so far, 28 are Italian and 10 of them have completed the study, Uccelli said, adding that all patients over the past year did relatively well except for one who was treated with placebo.

The neurologist expressed the hope that data in 2016 will give final confirmation that the therapy is effective so we can take the subsequent step with a larger phase III study aimed at demonstrating the role of stem cells as neurorepairers. Meanwhile Genoas bioethics committee has approved a two-year extension of the project, which will be called Mesems Plus, to verify, beyond the year of observation provided for by Mesems, the long-term safety of treatments in the study and the potential insurgence of adverse events in all those treated, said Uccelli.

A study last week showed statin use increases the risk of diabetes by 46 per cent, and those who take the cholesterol-lowering drugs are more than twice as likely to develop Parkinsons disease in later life than those who do not.The Parkinsons research carried out over 20 years suggests cholesterol may have a vital role in protecting the brain and nervous system.The findings have alarmed experts who say if applied to the number of Britons deemed eligible for statins it couldequate to 150,000 extra patients with Parkinsons.The work has also fuelled concerns that statins, now recommended for up to half the adult population over 50 by government drug policy adviser the National Institute for Health and Care Excellence, may be doing many patients more harm than good.

Dr Kailash Chand, deputy chairman of the British Medical Association, was speaking following research which found those who take the cholesterol-lowering drugs are more than twice as likely to develop Parkinsons disease in later life than those who do not.

A study last week showed statin use increases the risk of diabetes by 46 per cent.

It has led to calls to end to the widespread use of the drugs.

The Parkinsons research carried out over 20 years, and involving nearly 16,000 people, suggests cholesterol may have a vital role in protecting the brain and nervous system.

The findings have alarmed experts who say if applied to the number of Britons deemed eligible for statins it could equate to 150,000 extra patients with Parkinsons, a central nervous system disorder affecting one in 350 mostly older people.

The work has also fuelled concerns that statins, now recommended for up to half the adult population over 50 by government drug policy adviser the National Institute for Health and Care Excellence, may be doing many patients more harm than good.

Doctors used to prescribe the drugs only to those who had a 30 per cent or greater risk of suffering a heart attack within a decade, but this was lowered to 20 per cent in 2005.

A 10-year plan has been introduced to reduce this further and include low-risk patients who have just a 10 per cent chance of a heart attack within a decade.

Nice believes this could save thousands of lives.

Other studies have shown a link between the cholesterol-lowering drugs and potentially disabling side effects including cataracts, diabetes, muscle pains, fatigue and memory loss.

Researchers warn that the mass roll-out of statins leaves 150,000 people at risk

Dr Chand, who suffered debilitating muscle pains while taking statins, said of the Parkinsons research:

This research has been done over a considerable amount of time and on a considerable number of people and it is very worrying.

Speaking in a personal capacity, he added:

"The risks of side-effects of these drugs are far greater than any potential benefits and it is high time these drugs were restricted in the low-risk population" - Dr Kailash Chand

Dr Xuemei Huang, who led the research, recently published in the journal of Movement Disorders, expressed concerns about the widespread prescription of statins.

If we blanket prescribe statins to people we could be creating a huge population of people with neurological problems.

Does mother nature create cholesterol for a reason?

"I think doctors are over-enamoured with statins and think it is a cure-all.

But the body is not just for the heart, it is also for the brain.

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Don Margolis - Adult Stem Cell Research

Viacyte, privately-held, leading regenerative medicine company, announced today that the first patients have been implanted with the PEC-Direct product candidate, a novel islet cell replacement therapy in development as a functional cure for patients with type 1 diabetes who are at high risk for acute life-threatening complications. The first implant procedures of the clinical trial took place at the University of Alberta Hospital in Edmonton, Alberta, and the UC San Diego School of Medicines Altman Clinical Trials Research Institute. The goal of the open-label clinical trial is to evaluate the PEC-Direct product candidate for safety and definitive evidence of efficacy. In the coming months, the company expects to expand the trial to additional centers including the University of Minnesota and other sites in the US and Canada.

The first cohort of type 1 diabetes patients is receiving multiple small-format cell-filled devices called sentinels in order to evaluate safety and implant viability. These sentinel units will be removed at specific time points and examined histologically to provide early insight into the progression of engraftment and maturation into pancreatic islet cells including insulin-producing beta cells. A second cohort of up to 40 patients is expected to begin enrolling later this year to evaluate both safety and efficacy. The primary efficacy measurement in the trial will be the clinically relevant production of insulin, as measured by the insulin biomarker C-peptide, in a patient population that has little to no ability to produce endogenous insulin at the time of enrollment. Other important endpoints will be evaluated including injectable insulin usage and the incidence of hypoglycemic events. ViaCytes goal is to demonstrate early evidence of efficacy in the first half of 2018 and definitive efficacy 6 to 12 months later.

Islet transplants have been used to successfully treat patients with unstable, high-risk type 1 diabetes, but the procedure has limitations, including a very limited supply of donor organs and challenges in obtaining reliable and consistent islet preparations, said trial investigator James Shapiro, MD, PhD, FRCSC, Director of the Clinical Islet Transplant Program, University of Alberta. An effective stem cell-derived islet replacement therapy would solve these issues and has the potential to help a greater number of people.

Patients with high-risk type 1 diabetes complications, such as hypoglycemia unawareness, are at constant risk of life-threatening low blood glucose, said Jeremy Pettus, MD, investigator in the clinical trial and Assistant Professor of Medicine at UC San Diego. The PEC-Direct islet cell replacement therapy is designed to help patients with the most urgent medical need.

The PEC-Direct product candidate is being developed for type 1 diabetes patients who have hypoglycemia unawareness, extreme glycemic lability, and/or severe hypoglycemic episodes. It is estimated that about 140,000 people in Canada and the US have such high-risk type 1 diabetes. In addition to providing an unlimited supply of cells for implantation, the PEC-Direct approach has other potential advantages relative to cadaver islet transplants such as delivering a more consistent product preparation under quality-controlled cGMP conditions, and a more straightforward and safe mode of delivery.

The clinical trial is being supported in part by JDRF, the leading global organization funding type 1 diabetes research. JDRF remains dedicated to accelerating the delivery of beta cell replacement therapies to the T1D community, and we commend ViaCyte in its announcement of the first patients to be implanted with the PEC-Direct islet cell replacement therapy, said Derek Rapp, JDRF President and Chief Executive Officer. JDRF is excited to support this clinical development given its potential to help those people with type 1 diabetes that need it the most those at high risk of life-threatening acute complications. JDRF and ViaCyte share a continuing commitment to realizing the potential of beta cell replacement strategies to deliver insulin independence without immune suppression for people living with type 1 diabetes, and ultimately, at JDRF we hope this will move us forward in fulfilling our vision of a world without type 1 diabetes.

There are limited treatment options for patients with high-risk type 1 diabetes to manage life-threatening hypoglycemic episodes, said Paul Laikind, PhD, President and Chief Executive Officer of ViaCyte. We believe that the PEC-Direct product candidate has the potential to transform the lives of these patients and we are excited to move closer to that goal with the initiation of clinical evaluation announced today. This also represents a step towards a broader application of the technology. We remain fully committed to developing a functional cure for all patients with insulin-requiring diabetes. To that end, we are hard at work on next-generation approaches as well, and expect the work with PEC-Direct to further advance our knowledge and drive progress.

In addition to JDRF, the California Institute for Regenerative Medicine (CIRM)s Alpha Clinic, the Sanford Stem Cell Clinical Center, the JDRF Canadian Clinical Trials Network (CCTN), the Stem Cell Network, and Alberta Innovates Health Solutions (AIHS) are all providing support for the trial.

About the PEC-Direct Product Candidate

The PEC-Direct product candidate delivers stem cell-derived pancreatic progenitor cells, called PEC-01 cells, in a device designed to allow direct vascularization of the cells in the device. After implantation, these cells are expected to become mature human islet tissue including well-regulated beta cells producing insulin on demand. The direct vascularization of the implanted cells is expected to allow for robust and consistent engraftment but will necessitate the use of maintenance immune suppression therapy.

About ViaCyte

ViaCyte is a privately-held regenerative medicine company developing novel cell replacement therapies as potential long-term diabetes treatments to reduce the risk of hypoglycemia and diabetes-related complications. ViaCytes product candidates are based on the derivation of pancreatic progenitor cells from stem cells, which are then implanted in a durable and retrievable cell delivery device. Once implanted and matured, these cells are designed to secrete insulin and other pancreatic hormones in response to blood glucose levels. ViaCyte has two products in clinical development. The PEC-Direct product candidate delivers the pancreatic progenitor cells in a non-immunoprotective device and is being developed for type 1 diabetes patients who have hypoglycemia unawareness, extreme glycemic lability, and/or recurrent severe hypoglycemic episodes. The PEC-Encap (also known as VC-01) product candidate delivers the same pancreatic progenitor cells in an immunoprotective device and is being developed for all patients with diabetes, type 1 and type 2, who use insulin. ViaCyte is headquartered in San Diego, California. The Company is funded in part by the California Institute for Regenerative Medicine (CIRM) and JDRF.

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Pouch of stem cells implanted in trial to cure type 1 diabetes - Next Big Future

(HealthDay News) -- An antibiotic treatment intended to lower stem cell transplant patients' risk of developing a respiratory complication appears to have backfired.

French researchers explored the potential of administering the antibiotic azithromycin before and after stem cell transplantation to limit the risk for a condition known as bronchiolitis obliterans syndrome (BOS).

A significant number of blood cancer patients who undergo allogeneic stem cell transplant are at risk for developing BOS. It's a potentially deadly complication in which airflow to the lungs becomes progressively obstructed, turning routine breathing into an ordeal. Allogeneic transplants are ones where genetically similar, but not identically matched, material is transplanted.

The new investigation had to be stopped prematurely -- after just over a year. Preliminary indications suggested that the random pool of allogeneic stem cell transplant patients treated with antibiotics were actually more likely to develop BOS than those who were not getting azithromycin.

Dr. Henry Fung serves as vice chair of hematology and oncology at the Fox Chase Cancer Center in Philadelphia.

Although he was not a part of the current investigation, Fung was familiar with the results of the study and said, "in the absence of new scientific rationale on using azithromycin, a similar study should not be repeated."

Azithromycin "will not benefit patients undergoing an allogeneic stem cell transplant," Fung concluded.

Initially, the French investigation planned to include nearly 500 French blood cancer patients, average age 52. The patients began the allogeneic transplant process at one of 19 French bone marrow transplant facilities at some point between 2014 and 2015.

Fung, who's also director of the Fox Chase Cancer Center-Temple University Hospital Bone Marrow Transplant Program, noted that "allogeneic stem cell transplant is a life-saving procedure for many patients with blood cancers."

However, "the success ... [is] limited by long-term complications," he explained.

One complication is a condition known as graft-versus-host disease. This occurs when newly transplanted bone marrow and/or stem cells are seen by the patient's body as foreign material. The immune system then starts attacking the transplant.

The other is BOS.

And, BOS, "once diagnosed, is usually irreversible with no effective treatment available," Fung said.

Following allogeneic stem cell transplantation, roughly 4 percent to 6 percent of patients will develop BOS, the study authors said. That figure rises as high as 14 percent among those patients who also develop graft-versus-host disease.

The French researchers were led by Dr. Anne Bergeron from the Saint-Louis Hospital in Paris. The team noted that prior research had indicated that using the antibiotic azithromycin as a preventive therapy effectively reduced BOS risk among patients undergoing a lung transplant, during which BOS risk typically goes up.

To see whether the same would be true among stem cell transplant patients, the French study participants were randomly assigned to be treated with either azithromycin (250 milligrams three times a week for two years) or a placebo (dummy pill).

However, by December 2016 the investigation was halted, even before all the participants had completed their two-year antibiotic or placebo regimen.

The cited reason was an "unanticipated imbalance" in the incoming results. More people than anticipated in the group that had been getting the antibiotic were developing BOS.

The study findings were published in the Aug. 8 issue of theJournal of the American Medical Association.

More information

There's more about BOS at theRare Clinical Diseases Research Network.

SOURCES: Henry C. Fung, M.D., vice chair, hematology and oncology, Fox Chase Cancer Center, and director, Fox Chase Cancer Center-Temple University Hospital Bone Marrow Transplant Program, Philadelphia; Aug. 8, 2017,Journal of the American Medical Association

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Antibiotic Doesn't Prevent Lung Complication After Stem Cell Transplant - KARK

SAN DIEGO, Aug. 1, 2017 /PRNewswire/ -- ViaCyte, Inc., a privately-held, leading regenerative medicine company, announced today that the first patients have been implanted with the PEC-Direct product candidate, a novel islet cell replacement therapy in development as a functional cure for patients with type 1 diabetes who are at high risk for acute life-threatening complications. The first implant procedures of the clinical trial took place at the University of Alberta Hospital in Edmonton, Alberta, and the UC San Diego School of Medicine's Altman Clinical Trials Research Institute. The goal of the open-label clinical trial is to evaluate the PEC-Direct product candidate for safety and definitive evidence of efficacy. In the coming months, the company expects to expand the trial to additional centers including the University of Minnesota and other sites in the US and Canada.

The first cohort of type 1 diabetes patients is receiving multiple small-format cell-filled devices called sentinels in order to evaluate safety and implant viability. These sentinel units will be removed at specific time points and examined histologically to provide early insight into the progression of engraftment and maturation into pancreatic islet cells including insulin-producing beta cells. A second cohort of up to 40 patients is expected to begin enrolling later this year to evaluate both safety and efficacy. The primary efficacy measurement in the trial will be the clinically relevant production of insulin, as measured by the insulin biomarker C-peptide, in a patient population that has little to no ability to produce endogenous insulin at the time of enrollment. Other important endpoints will be evaluated including injectable insulin usage and the incidence of hypoglycemic events. ViaCyte's goal is to demonstrate early evidence of efficacy in the first half of 2018 and definitive efficacy 6 to 12 months later.

"Islet transplants have been used to successfully treat patients with unstable, high-risk type 1 diabetes, but the procedure has limitations, including a very limited supply of donor organs and challenges in obtaining reliable and consistent islet preparations," said trial investigator James Shapiro, MD, PhD, FRCSC, Director of the Clinical Islet Transplant Program, University of Alberta. "An effective stem cell-derived islet replacement therapy would solve these issues and has the potential to help a greater number of people."

"Patients with high-risk type 1 diabetes complications, such as hypoglycemia unawareness, are at constant risk of life-threatening low blood glucose," said Jeremy Pettus, MD, investigator in the clinical trial and Assistant Professor of Medicine at UC San Diego. "The PEC-Direct islet cell replacement therapy is designed to help patients with the most urgent medical need."

The PEC-Direct product candidate is being developed for type 1 diabetes patients who have hypoglycemia unawareness, extreme glycemic lability, and/or severe hypoglycemic episodes. It is estimated that about 140,000 people in Canada and the US have such high-risk type 1 diabetes. In addition to providing an unlimited supply of cells for implantation, the PEC-Direct approach has other potential advantages relative to cadaver islet transplants such as delivering a more consistent product preparation under quality-controlled cGMP conditions, and a more straightforward and safe mode of delivery.

The clinical trial is being supported in part by JDRF, the leading global organization funding type 1 diabetes research. "JDRF remains dedicated to accelerating the delivery of beta cell replacement therapies to the T1D community, and we commend ViaCyte in its announcement of the first patients to be implanted with the PEC-Direct islet cell replacement therapy," said Derek Rapp, JDRF President and Chief Executive Officer. "JDRF is excited to support this clinical development given its potential to help those people with type 1 diabetes that need it the most those at high risk of life-threatening acute complications. JDRF and ViaCyte share a continuing commitment to realizing the potential of beta cell replacement strategies to deliver insulin independence without immune suppression for people living with type 1 diabetes, and ultimately, at JDRF we hope this will move us forward in fulfilling our vision of a world without type 1 diabetes."

"There are limited treatment options for patients with high-risk type 1 diabetes to manage life-threatening hypoglycemic episodes," said Paul Laikind, PhD, President and Chief Executive Officer of ViaCyte. "We believe that the PEC-Direct product candidate has the potential to transform the lives of these patients and we are excited to move closer to that goal with the initiation of clinical evaluation announced today. This also represents a step towards a broader application of the technology. We remain fully committed to developing a functional cure for all patients with insulin-requiring diabetes. To that end, we are hard at work on next-generation approaches as well, and expect the work with PEC-Direct to further advance our knowledge and drive progress."

In addition to JDRF, the California Institute for Regenerative Medicine (CIRM)'s Alpha Clinic, the Sanford Stem Cell Clinical Center, the JDRF Canadian Clinical Trials Network (CCTN), the Stem Cell Network, and Alberta Innovates Health Solutions (AIHS) are all providing support for the trial.

About the PEC-Direct Product Candidate The PEC-Direct product candidate delivers stem cell-derived pancreatic progenitor cells, called PEC-01 cells, in a device designed to allow direct vascularization of the cells in the device. After implantation, these cells are expected to become mature human islet tissue including well-regulated beta cells producing insulin on demand. The direct vascularization of the implanted cells is expected to allow for robust and consistent engraftment but will necessitate the use of maintenance immune suppression therapy.

About ViaCyte ViaCyte is a privately-held regenerative medicine company developing novel cell replacement therapies as potential long-term diabetes treatments to reduce the risk of hypoglycemia and diabetes-related complications. ViaCyte's product candidates are based on the derivation of pancreatic progenitor cells from stem cells, which are then implanted in a durable and retrievable cell delivery device. Once implanted and matured, these cells are designed to secrete insulin and other pancreatic hormones in response to blood glucose levels. ViaCyte has two products in clinical development. The PEC-Direct product candidate delivers the pancreatic progenitor cells in a non-immunoprotective device and is being developed for type 1 diabetes patients who have hypoglycemia unawareness, extreme glycemic lability, and/or recurrent severe hypoglycemic episodes. The PEC-Encap (also known as VC-01) product candidate delivers the same pancreatic progenitor cells in an immunoprotective device and is being developed for all patients with diabetes, type 1 and type 2, who use insulin. ViaCyte is headquartered in San Diego, California. The Company is funded in part by the California Institute for Regenerative Medicine (CIRM) and JDRF. For more information on ViaCyte, please visit http://www.viacyte.com and connect with ViaCyte on Twitter and Facebook.

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SOURCE ViaCyte, Inc.

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ViaCyte Announces First Patients Implanted with PEC-Direct Islet Cell Replacement Therapy in International Clinical ... - Markets Insider

Springs Integrated Health offers leading-edge, all-natural medical care. The center provides services intended to get to the root of patient issues and deliver real, lasting results in the simplest, most effective way. Instead of covering up symptoms with medications, the clinic breaks down health into obtainable goals that can optimize the wellness of each and every patient. Services include chiropractic, hormone lab testing, physical rehabilitation, Supartz therapy, trigger point therapy, FAR infrared sauna, stem cell therapy and more.

There was a time when stem cell therapy was out of reach for most people, but it has become increasingly accessible in recent years; and a go-to solution for a range of physiological complications. Stem cells are blank cells in the body that can become any tissue, whether that be knee tissue, bone tissue, cartilage, organ tissue stem cells can become whatever they are closest and nearest to, said Tiffany Graham, DC of Springs Integrated Health. So when you inject them into a joint thats damaged the body is always healing itself anyway it can create new tissue where there has been damage.

Stem cell therapy has been used for decades in Europe, and in the United States has been used by Peyton Manning, Tiger Woods, and many NFL players and other professional athletes. Although stem cells were initially reserved for the ultra wealthy, they have since become both affordable and accessible; and many patients are opting for them over lengthy and expensive knee and shoulder surgeries. One in 400 total knee replacements result in fatal infection, and those that do not end up in infection still prove to have an extensive recovery time. Stem cell therapy is safe and quick, and people can feel results in as little as one week. Further, the company Springs Integrated Health utilizes for stem cells has given more than 50,000 injections with zero side effects and zero adverse reactions.

There are two different types of stem cells. The first is adult stem cells, where patients take their own bone marrow, fat or blood; spin it down; and re-inject into the joints. Thats not what is used at Springs Integrated Health, because its a long procedure that can be very painful and expensive. The second is amniotic stem cells, which are from donated placental tissue. This tissue comes from mothers who have planned c-sections, and who have elected to donate their placenta to science. The stem cells have been thoroughly tested and are clear of all antigens, so there is no risk for rejection or infection. The stem cells used at Springs Integrated Health are 100 percent ethically-sourced, and are not embryonic stem cells, which come from aborted fetal tissue, said Graham.

Rick Paine is a beaming example of the efficacy of stem cell therapy. He is an avid runner and hiker, and coached swimming at the University of Nebraska for 17 years. He was also an Olympic Head Coach in Australia in 2000. Eight years ago, he wore his left knee out and had to get a knee replacement, and it took two to three years to recover. About two years ago, his right knee was becoming worn out, and he did not want to go through the another knee surgery, because it was a very unpleasant experience for him. He was seeing an active release therapist who was helping, but he still had trouble with downhill on hikes, walking on the golf course and doing the everyday activities that make him happy.

Paine had been seeing Dr. Graham for about a year and a half before deciding to commence with stem cell therapy for his torn medial meniscus on the right knee in November 2016. At first I was skeptical, but I thought, lets give it a shot, Paine said. The procedure was quick and pretty painless, and it only took about a month after the injection for my knee to feel really good. He cautions that although the knee may feel great in a month or less, its essential to still take it easy, and give the tissue time to grow before becoming physically active.

Before I got stem cells, I couldnt squat to pick up a ball on the golf course but since getting the stem cells I can definitely do that. Im 65 years old, and a surgery would have taken me out of hiking for two to three years, but with this, it was only three months until I was hiking again, said Paine. We took X-rays a few months ago, and there has been significant improvement in my knee. I didnt expect to see that, I thought it was too good to be true, but Im living proof that stem cell therapy works.

Paine shared that now, eight months after his procedure, his knee still feels perfect. He admits that its not like having a brand new knee, but he has no issues whatsoever with downhill, uphill or bending down. I knew I wanted to do at least one more 14er, and didnt think it would be possible, but stem cells have definitely allowed me to do that. Paine is now gearing up for a 12 mile hike from Crested Butte to Aspen, a hike he couldnt have even considered before stem cells. I wish we had stem cells way back when, because it would have saved a lot of athletes careers.

To receive a complimentary consultation, or to attend an upcoming, free informational seminar at Springs Integrated Health, call 719-301-6649 or visit SpringsIntegratedHealth.com.

Springs Integrated Health is located at 1712 W. Uintah St., Colorado Springs. Hours are Monday, 10 a.m. to 12:30 p.m. and 3 to 6 p.m.; Tuesday, 3 to 6 p.m.; Wednesday, 10 a.m. to 12:30 p.m. and 3 to 6 p.m.; Thursday, 3 to 6 p.m.; and Friday 8 to 10 a.m.

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Stem Cell Therapy can provide a surgery-free solution to knee and shoulder issues - Colorado Springs Gazette

Stem cell therapy isn't approved to treat heart failure in the U.S., but dozens of clinics nationwide advertise the treatments anyway, often charging thousands of dollars for procedures that may not be safe or effective, a new study suggests.

Researchers found 61 centers offering stem cell therapies for heart failure as of last year in the U.S. alone, including five that claimed to have performed more than 100 procedures. Only nine centers required copies of patients' medical records and just one facility said it had a board certified cardiologist on staff.

NEUROLOGIST BACKS FAMILY'S FIGHT TO OVERTURN TEEN'S DEATH CERTIFICATE

"We simply do not know anything about the quality of the treatment delivered at these centers," said senior study author Dr. Paul Hauptman director of heart failure at Saint Louis University Hospital.

"These centers are not regulated in any way," Hauptman said by email.

Almost 6 million Americans have heart failure, and it's one of the most common reasons older adults go to the hospital, according to the American Heart Association.

It happens when the heart muscle is too weak to effectively pump enough blood through the body. Symptoms can include fatigue, weight gain from fluid retention, shortness of breath and coughing or wheezing. Medications can help strengthen the heart and minimize fluid buildup in the body.

While some experimental stem cell therapies for heart failure are currently being tested in late-stage human trials, none have won approval from the U.S. Food and Drug Administration.

CTE FOUND IN BRAINS OF 110 OUT OF 111 DECEASED NFLERS

In theory, after a transplant, stem cells could permanently become part of the diseased heart and either help grow new healthy heart tissue or tell existing cells to work better, said Paul Knoepfler, a cell biology researcher at the University of California Davis School of Medicine in Sacramento who wasn't involved in the study.

It's also possible stem cells could temporarily visit the heart and stimulate a positive response in cells already there, he said.

Even though there's no conclusive proof yet that any stem cell treatments are safe and effective for heart failure, centers contacted for the study charge an average of $7,694 for each treatment using patient's own stem cells and $6,038 for each procedure with donor stem cells.

In one instance, though, a clinic staff member said, "If you have a million dollars to spend we will set you up with weekly infusions."

Hauptman's team had used a standard script when contacting each center, asking about the stem cell treatment itself, medical exams before and afterward and pricing.

Among the other responses they received from clinic staff were remarks such as, "If you know anyone that can start an IV, a neighbor that is a nurse for example, we can send you the stem cells and that person can administer them to you" and "We hope you don't believe your doctor when they tell you there is nothing they can do, you were smart to call us."

None of the sites in the study discussed what methods they used to isolate or identify stem cells, though most claimed to use patients' cells and 24 said they got cells from fat tissue.

Most centers claimed to deliver cells intravenously, researchers report in JAMA Internal Medicine.

"This approach has been associated with complications such as stroke, in which infused cells block blood vessels in the brain," said Douglas Sipp, a researcher at RIKEN Center for Developmental Biology in Kobe, Japan, who wasn't involved in the study.

"The biggest risk is that patients will waste their money, time and hopes on an unnecessary and useless invasive procedure," Sipp said by email.

DENTIST FACES CHARGES AFTER 4-YEAR-OLD SUFFERS BRAIN DAMAGE

If any stem cell treatment did ultimately prove safe and effective enough to win FDA approval, it would likely offer a significant improvement over the limited treatment options currently available, said Leigh Turner, a researcher at the University of Minnesota Center for Bioethics who wasn't involved in the study.

But it's impossible to say what patients would get at unregulated clinics offering unapproved stem cell therapies, Turner said by email. In at least two cases unrelated to the current study, patients died after getting stem cell procedures at a clinic in Florida, and in another case at a different Florida clinic, a woman went blind, Turner noted.

"Clinics marketing stem cell treatments to patients suffering from heart failure might be administering anything from slurries of mixed cells, some of which might be stem cells, to nothing more than cellular debris," Turner said. "Often one can only speculate."

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Dozens of US clinics sell unproven stem cell therapies for heart failure - Fox News

Submitted photoA picture of Linus and Leonard Huiras in their early 20s. Both brothers died of symptoms related to Dyskeratosis Congenita, a rare hereditary disease that affects a small number of people worldwide.

Robin Huiras decided long ago that rather than sit and feel sorry for herself, she could do the exact opposite and raise awareness for the rare disease that she was born with.

Huiras life changed at age 10 when she received the news that she had Dyskeratosis Congenita (DC), a disease that affects less than one in a million people worldwide. Its a hereditary disease that was passed on to her from her fathers side of the family.

The daily battle of fighting a rare disease that is capable of destroying the human body leaves her with few options: Battle the disease quietly or help educate as many people as she can and raise money to better research it.

Huiras, who turned 40 this past March, is a 1995 New Ulm Cathedral graduate. Her dad, Linus Huiras died in June 1991 at age 43 from complications of the disease. Her uncle and Linus twin brother Leonard died in 1985 at the age of 37, also from complications of the disease. Her cousin, Joe Huiras (Leonards son), a 2002 graduate of Sleepy Eye St. Marys, also is battling the disease.

She is passionate about educating others about the disease. She became involved in a non-profit charity called Dyskeratosis Congenita, Outreach, Inc. and in 2013 she became president and helped the group do a number of activities, including sponsoring family camps and publishing a medical guidebook on the disease.

Submitted photoCousins Robin Huiras and Joe Huiras together last December. Both have Dyskeratosis Congenita, a rare hereditary disease.

She helped the group raise money and this year, for the second year in a row, she is organizing a golf tournament at the Sleepy Eye Golf Club and that will be held on Aug. 19. Last year the tournament raised more than $7,000 for the group and shes hoping to double the size of the tournament from last year.

About the disease:

Raising awareness for DC is Huiras main goal. Those with the disease usually have an increased chance of developing several life-threatening conditions, including disorders that impair bone marrow function. These disorders disrupt the ability of the bone marrow to produce new blood cells.

While the disease is capable of affecting many organs throughout the body, areas that have high rates of cell reproduction are mainly affected.

A lot of people think of it almost as an aging disease, where our aging process for our skin and our organs are accelerated, Huiras said. People that have DC a lot of times have bone marrow failure because our blood cells are one of the areas in our body that reproduce really quickly. Ninety percent of people that have DC have bone marrow failure.

Submitted photoGolfers in last years Huiras Golf Tournament Fundraiser in Sleepy Eye. The tournament raised more than $7,000 for Dyskeratosis Congenita research.golf tournament group photo cutlineParticipants in last years Huiras Golf Tournament Fundraiser in Sleepy Eye. The tournament raised more than $7,000 for Dyskeratosis Congenita research.

They are also at higher than average risk for blood cancers and have a higher risk of developing other cancers, especially cancers of the head and neck.

Oral cancer is huge, different blood cancers, different lymphomas can happen, she said. Scientists are interested in researching the disease because it can tell them a lot about the process of cancer.

Huiras noted that there are no cures for many of the complications, but there are some treatments that can slow the progression.

A lung transplant or a bone marrow transplant can be a cure for a symptom, but there is no cure for the disease, she said. Scientists are really interested in studying it, especially because of this concept of aging.

Huiras said that the diagnosis is usually made early on in a persons life. Something as simple as frequent bloody noses can be a warning sign.

Usually what happens is people get anemia and most times it happens when a person is a child, she said. There are cases when people find out when theyre an adult, they just have less severe variety of it. Kids might get bloody noses a lot, or kids might just get sick all the time and a lot of patients have poor immune systems.

Her own personal battle:

Huiras is no stranger to the complications of DC. Having battled the disease her whole life, shes had her fair share of struggles that shes encountered, none of them being an easy fix.

She started having chronic headaches at age 10 and so she went to Mayo Clinic and they did a bone marrow biopsy. There, they made the diagnosis of DC after seeing her cells were abnormal.

Despite having the disease, her teenage years were fairly normal, but she did have a few problems. She started taking prednisone daily when she was 17.

I bruised easy and my biggest thing was I couldnt run, she said. When we did the mile in gym class, that was the hardest thing for me to do.

In 1999 at the age of 22, she had an internal bleeding episode and she nearly died before she finally got to the hospital. This was a life-changer for her and she knew she had to make better decisions if she was going to continue to battle the disease.

I wasnt taking care of myself and I wasnt making the best choices, she said. I did finally go to the hospital and they were surprised I was walking and still alive. Then in my 20s, I was actually transfusion dependent, so every two months Id have to go in and get a liter of red blood cells.

She was prescribed anabolic steroids and the drugs helped her depleted bone marrow make red blood cells for a short time, but eventually those stopped working.

In December of 2005 at the age of 28 and out of options, she had a stem-cell transplant. A younger brother, who is not affected by the illness, was her stem cell donor.

She has two children and neither have DC. Her first child was born in 2008 and her second in 2012. In 2010, she had eye tear duct surgery and in 2012, she started having mild hip pain. This led to eventually having her hip joints replaced, one in January of 2017 and the other in April of 2017.

Robins battle is far from over. But shes doing fine and shes turning her attention again to helping bring awareness to the disease.

Im certainly much better than I was before, she said. I wouldnt say Im at 100 percent, but its so minor compared to the horror show that was going on before.

More than just a golf

tournament:

Robins life isnt the only in her family thats been dramatically impacted by DC. Her cousin has been fighting symptoms of the disease for nearly a decade. A stem cell transplant in 2012 at Fairview University Hospital in Minneapolis has been followed by medical ups and downs. In November 2016 Joe was diagnosed with two forms of cancer.

Not only will the Huiras golf tournament bring attention to the disease, but it will directly supporting Joe and his family, which fits the mission of DC Outreach, Huiras said.

Many of the people competing are family members, but there are many others who attended last years event to help raise money for DC.

While that was satisfying for Huiras, it was also a time where friends and families gathered to share stories of her dad and uncle.

It was like a family reunion, but what was amazing about it was we had never had an opportunity to really talk about my dad and my uncle Leonard in a way in that everyone was acknowledging of what they went through, and how it wouldve made them to know that their kids were working together to keep their memory alive, she said. It was special to have everyone in the same room to have everyone talk about their lost brothers and that was really awesome.

Some of the symptoms of

Dyskeratosis Congenita

Skin, nail and mouth changes: Abnormal dark discoloration of the skin, nails may fall off, white, thickened patches of on mucous membranes of th mouth (oral leukoplakia).

Bone marrow failure: Individuals can develop bone marrow failure marked by deficiency of the three types of blood cells (red cells, white cells, platelets)

Leukemia and cancer: Individuals can develop leukemia, especially of the head and neck.

Lung disease: Often found in patients with DC, it usually develops later than the skin abnormalities and bone marrow failure.

Excessive watery eyes

Excessive sweating of the palms and soles of the feet

Cavities and tooth loss

Narrowing of the esophagus

Urinary tract anomalies

Liver disease

Information came from: https://rarediseases.org/rare-diseases/dyskeratosis-congenita/#symptoms

2017 Huiras

Golf Tournament

August 19, Sleepy Eye Golf Course

If you go:

The tournament itself is a nine-hole scramble on August 19 at Sleepy Eye Golf Club. Registration begins at 8 a.m. And shotgun start is 8:45. You can call the Sleepy Eye Golf Club ahead of time or show up the day of to participate in the event.

The event itself costs $50 and that includes golf cart and lunch. There were 44 golfers last year who competed and there are spots for 72 this year. Money goes toward DC research and Joe Huiras medical expenses.

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The fight of her life - NUjournal

Stem cell therapy isnt approved to treat heart failure in the US, but dozens of clinics nationwide advertise the treatments anyway, often charging thousands of dollars for procedures that may not be safe or effective, a new study suggests.

Researchers found 61 centers offering stem cell therapies for heart failure as of last year in the US alone, including five that claimed to have performed more than 100 procedures. Only nine centers required copies of patients medical records and just one facility said it had a board certified cardiologist on staff.

We simply do not know anything about the quality of the treatment delivered at these centers, said senior study author Dr. Paul Hauptman director of heart failure at Saint Louis University Hospital.

These centers are not regulated in any way, Hauptman said by email.

Almost 6 million Americans have heart failure, and its one of the most common reasons older adults go to the hospital, according to the American Heart Association.

It happens when the heart muscle is too weak to effectively pump enough blood through the body. Symptoms can include fatigue, weight gain from fluid retention, shortness of breath and coughing or wheezing. Medications can help strengthen the heart and minimize fluid buildup in the body.

While some experimental stem cell therapies for heart failure are currently being tested in late-stage human trials, none have won approval from the US Food and Drug Administration.

In theory, after a transplant, stem cells could permanently become part of the diseased heart and either help grow new healthy heart tissue or tell existing cells to work better, said Paul Knoepfler, a cell biology researcher at the University of California Davis School of Medicine in Sacramento who wasnt involved in the study.

Its also possible stem cells could temporarily visit the heart and stimulate a positive response in cells already there, he said.

Even though theres no conclusive proof yet that any stem cell treatments are safe and effective for heart failure, centers contacted for the study charge an average of $7,694 for each treatment using patients own stem cells and $6,038 for each procedure with donor stem cells.

In one instance, though, a clinic staff member said, If you have a million dollars to spend we will set you up with weekly infusions.

Hauptmans team had used a standard script when contacting each center, asking about the stem cell treatment itself, medical exams before and afterward and pricing.

Among the other responses they received from clinic staff were remarks such as, If you know anyone that can start an IV, a neighbor that is a nurse for example, we can send you the stem cells and that person can administer them to you and We hope you dont believe your doctor when they tell you there is nothing they can do, you were smart to call us.

None of the sites in the study discussed what methods they used to isolate or identify stem cells, though most claimed to use patients cells and 24 said they got cells from fat tissue.

Most centers claimed to deliver cells intravenously, researchers report in JAMA Internal Medicine.

This approach has been associated with complications such as stroke, in which infused cells block blood vessels in the brain, said Douglas Sipp, a researcher at RIKEN Center for Developmental Biology in Kobe, Japan, who wasnt involved in the study.

The biggest risk is that patients will waste their money, time and hopes on an unnecessary and useless invasive procedure, Sipp said by email.

If any stem cell treatment did ultimately prove safe and effective enough to win FDA approval, it would likely offer a significant improvement over the limited treatment options currently available, said Leigh Turner, a researcher at the University of Minnesota Center for Bioethics who wasnt involved in the study.

But its impossible to say what patients would get at unregulated clinics offering unapproved stem cell therapies, Turner said by email. In at least two cases unrelated to the current study, patients died after getting stem cell procedures at a clinic in Florida, and in another case at a different Florida clinic, a woman went blind, Turner noted.

Clinics marketing stem cell treatments to patients suffering from heart failure might be administering anything from slurries of mixed cells, some of which might be stem cells, to nothing more than cellular debris, Turner said. Often one can only speculate.

See the article here:
More than 60 US clinics have sold unproven stem cell therapies for heart failure - New York Post

A transplant can cure end-stage liver cirrhosis - hardening of the liver - but not all patients have this option.

For those who are not eligible for a transplant, an alternative may be in sight.

A clinical trial was launched yesterday to explore the use of stem cells to reverse liver cirrhosis.

In the study conducted by a multi-centre team led by the National University Hospital (NUH), doctors aim to determine if stem-cell therapy can improve liver function. Stem cells will be taken from a patient's own bone marrow and will be isolated and injected directly into the patient's liver to initiate the repair.

The $2.6 million Phase III trial will use biopsy and clinical measurements of liver function to test the efficacy, effectiveness and safety of the stem-cell treatment.

The study is funded by the National Medical Research Council, and a total of 46 patients will be recruited. It will run for four years, and patients will not need to bear the costs of stem-cell treatment.

Liver cirrhosis is caused by diseases such as chronic hepatitis B and non-alcoholic fatty liver disease. A liver transplant provides a definitive cure to end-stage cirrhosis. However, in Singapore, less than 5 per cent of end-stage liver cirrhosis patients receive a liver transplant.

The number of people on the waiting list for a liver transplant has been increasing over the years, according to statistics from the Ministry of Health.

Last year, there were 57 on the waiting list, up from nine in 2007. There are around 50 waiting for a liver transplant this year.

Many patients do not fulfil the eligibility criteria for a transplant because of other health complications or because they are above the age limit of about 70 years.

Ms Jac Low, 44, who works in the engineering sector, is optimistic. Her 70-year-old mother suffers from liver cirrhosis and is waiting to enter the trial.

"This brings new hope to patients, and my mother is happy to learn about it," said Ms Low.

While similar therapy treatments have been conducted overseas in countries such as Egypt and India, they have not been fully evaluated for efficacy.

Associate Professor Dan Yock Young, a senior consultant in the division of gastroenterology and hepatology at NUH, said: "We are conducting the study in a systematic and scientific manner in order to get definitive evidence of the effects of the treatment."

He said stem-cell therapy is not a substitute for a liver transplant but provides an option for those who are not eligible for one.

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Can't go for liver transplant? There's hope still, Health News & Top ... - The Straits Times

Fear becomes a constant companion when ones child is desperately ill.

Kim Lahti-Scranton knows this all too well as her daughter Jane is fighting for her life at BC Childrens Hospital.

As much as Janes journey is all-consuming, Kim is helping a desperate woman to get young people to donate bone marrow in order to find a better match for her son, Noah, who is in critical need of a bone marrow transplant soon.

The Scranton family is living in Vancouvers Ronald McDonald House and shares a kitchen with 17-year-old Noah Stoltes family.

Noah survived a first round of cancer when he was 10. In April, the family was given the terrifying news that he had relapsed.

His mom thought they were going to find a match within family; theyre 100 per cent Dutch, says Kim, noting family members are usually the best choice, but his three sisters are only a 50 per cent match.

Out of the worldwide pool of donors, there are two people who are a 75 per cent genetic match and are being considered, one of whom lives in Europe. The lower the match, the more chance there is for complications and a less than optimal outcome.

There is a time crunch as Noah needs to have a transplant sometime in September and it can take a couple of months from registration to donation.

One of Kims nephews is an NHL player, who used his connections to help Noahs aunt interest 85 people in becoming bone marrow donors in Edmonton.

Unable to get help from Canadian Blood Services to organize a similar drive in Vancouver because of staffing issues, Kim and Noahs mother, Stacey VanderLee Stolte, went to a Vancouver Canadians game to raise awareness and hopefully encourage people to sign up to be on the registry for stem cell or bone marrow donation.

We were met with a whole range of responses from people who were incredibly receptive, to people who completely ignored us and everything in between, says Kim. We got some interesting comments like no kid would want my bone marrow, Ive done too many drugs, to other people saying no because they thought if they were a match that donating bone marrow would be too painful.

Dr. Lucy Turnham, a clinical associate who oversees the outpatient oncology clinic at BC Childrens Hospital, has performed Janes procedures and says males between the ages of 17 and 35 are the best resource.

Its one of the most wonderful charitable donations you can do, Turnham says, noting many people never receive a call. It has a huge impact on a patient who has no other options.

Turnham explains that donors are educated before they do a swab. If they are told they are a match, they can still say no at any time. They then undergo further testing and maybe counselling at that point, and can still back out an any time.

If they had said yes and the patient begins treatment to get rid of the diseased cells prior to transplant, and then the donor backs out, the patients life is at serious risk, so it is important to know what you are getting into before saying yes, Kim stresses. If the donor says yes to the procedure, they are told beforehand when the treatment would start and told when the last chance to back out would be.

Stem cell donations can be made in two ways: the donor is hooked to a machine with IVs in each arm. Blood is taken from one arm, stem cells are removed, the blood is replaced through the IV in the other arm where the body grows more stem cells.

Or, the donor is anesthetized, a needle is placed into the hip bone from the back and part of the bone marrow is sucked out.

You might be a little bit sore afterwards, but for less than 24 hours, Turnham says, noting women can be donors but not if they have been pregnant. Young bone marrow is more robust and we regenerate marrow and blood all the time.

Becoming a donor is not a speedy process for people living in smaller communities, but would-be donors can receive a swab kit through the mail and return it postage-free.

Marc Plante, a representative with the national office of Canadian Blood Services, says people can go to http://www.blood.ca and proceed to the Stem Cells tab at the top of the page.

If you have an opportunity to save a life and just put up with a couple of days of discomfort, I would do it in a heartbeat, says Kim, who must also face the reality that Jane could one day be in the same dilemma. If most people had the opportunity to save a life, they would do it.

The Scrantons have been living at Ronald McDonald House for several months, while Jane receives treatment.

Shes as good as can be expected; shes very compromised and we need to make sure shes not exposed to anything, says Kim, noting the first, and this, the fourth phase of treatment, are considered to be the toughest. She has nothing to fight off infection.

There is another terrifying aspect to this disease.

As well as living with the fear of potential relapse, some of the chemo drugs being used to treat Jane increase the risk of heart disease and cause secondary cancer, neuro-cognitive issues and behavioural issues.

We dont know if its coming or what it will be, but we dont have a choice, we have to save her life now and deal with the consequences later, Kim says. Youre just kind of waiting for the other shoe to drop, for things to take a turn, its not fun. And while Jane will require a lot of follow-up assessment in the years to come, the tough little hero turns six on Aug. 12.

She has everything she needs; I just want people to sign up for the bone marrow registry or donate blood, Kim says, noting she is grateful for the communitys support throughout the ordeal.

If you cant donate but would like to help Jane celebrate, you can take birthday cards to the Salmar Grand Theatre where manager Daila Duford will make sure they get to their destination.

Read this article:
Bone marrow transplant last chance - Revelstoke Review

All of our blood vessels are lined with a type of cell called endothelial cells. To form vessels, individual endothelial cells begin to create empty holes in themselves, called vacuoles. They then connect with other endothelial cells that have done the same thing and the linked vacuoles form tubes, which ultimately become capillaries. Here, the researchers took endothelial cells and mixed them with either fibrin -- a protein involved in blood clotting -- or a semi-synthetic material called gelatin methacrylate (GelMA), which can be easily 3D-printed. When mixed with fibrin, the endothelial cells formed tubes fairly easily, but that wasn't the case with the GelMA. However, when the researchers added in another type of cell, a stem cell found in bone marrow, the endothelial cells were then able to form tubes in the GelMA.

"We've confirmed that these cells have the capacity to form capillary-like structures, both in a natural material called fibrin and in a semi-synthetic material called gelatin methacrylate, or GelMA," Gisele Calderon, the lead author of the study, said in a statement, "The GelMA finding is particularly interesting because it is something we can readily 3D print for future tissue-engineering applications."

The benefits of this method over others include cells that can be patient-specific, reducing the risk of immune system complications, and growth environments that are well suited for organ and vasculature growth -- they're reproducible, not likely to induce immune responses and help boost cell growth and vessel development. Along with making 3D-printed organs more viable, this method will also allow for the development of tissue that could make for more effective and efficient drug testing. In a statement, Jordan Miller, whose lab the work was done in, said, "Preclinical human testing of new drugs today is done with flat two-dimensional human tissue cultures. But it is well-known that cells often behave differently in three-dimensional tissues than they do in two-dimensional cultures. There's hope that testing drugs in more realistic three-dimensional cultures will lower overall drug development costs."

You can watch a video of the cells beginning to form tubes here and Calderon explaining her work in the video below.

Read the original here:
Researchers are closer to working capillaries in 3D-printed organs - Engadget

SINGAPORE - The use of stem cells to reverse liver cirrhosis - or the hardening of the liver - is being explored in a clinical trial.

Conducted by a multi-centre team led by the National University Hospital (NUH), doctors aim to determine if stem cell therapy can improve liver function.

Previously, liver cirrhosis, caused by various diseases such as chronic hepatitis B and non-alcoholic fatty liver disease, was thought to be irreversible.

A liver transplant provides a definitive cure to end-stage cirrhosis.

However, in Singapore, less than 5 per cent of end-stage liver cirrhosis patients receive a liver transplant.

The number of people on the waiting list for a liver transplant has been increasing over the years, according to statistics from the Ministry of Health.

In 2007, there were nine on the waiting list, compared with 57 last year. There are around 50 waiting for a liver transplant this year.

Also, many patients do not fulfil the eligibility criteria to receive a liver transplant due to other health complications or being above the age limit of 70 years.

The $2.6 million study, which was launched on Tuesday (July 11),is funded by the National Medical Research Counciland 46 patients will be recruited for it. It will run for four years and patients will not need to bear the costs of the stem cell treatment.

Stem cells will be taken from a patient's own bone marrow and will be isolated and injected directly into the patient's liver to initiate the repair.

Similar therapy treatments have been conducted overseas in countries such asEgypt and India, although they have not been fully evaluated for efficacy.

Associate Professor Dan Yock Young, a senior consultant in the division of gastroenterology and hepatology at NUH, said: "We are conducting the study in a systematic and scientific mannerto get definitive evidence of the effects of the treatment."

He also notes that the stem cell therapy is not a substitute for a liver transplant. "This treatment is not intended to pull patients off the waiting list, but provide an option for those who are not eligible for a transplant."

Read more:
Clinical trial for stem-cell therapy to reverse liver cirrhosis - The Straits Times

A few small scars on Ashli Stempels lower back are the only evidence that a drill burrowed into her hipbone last year at Brigham and Womens Hospital in Boston. The surgery was to harvest the stem cells in her bone marrow to save her older brother Andrew Stempels life.

At age 27, Andrew was diagnosed with cancer of the white blood cells called Hodgkins lymphoma. Donating her bone marrow so that Andrews body could manufacture healthy blood cells, was a small price to pay to give him a shot at survival, Ashli says.

Since the transplant last August after years of treatment and testing Andrew has been cancer-free and Ashli now volunteers periodically in their hometown of Greenfield, where she serves on the Town Council, to spread awareness about this life-saving treatment.

Our bodies are a cure for some cancers, says Ashli Stempel on a recent Saturday as she handsout sign-up forms atGreenfields Energy Park for the Be A Matchnational donor registry. If even one person joins the registry that is awesome.

Its a sunnyday and Stempel, 30,wearing a black and white spaghetti-strap dress stands behind a booth smiling and talking to passersby.

Everybody wants to cure cancer, but I think not everybody understands that we, ourselves, can be the cure for some types of cancers, she says. I can say that I killed cancer and I am pretty excited about that.

In the hollow spaces in a bodys bones, stem cells inside the bone marrow tissue work to create red blood cells, which feed oxygen to the organs, and make white blood cells to fight infections. The bone marrow also produces blood platelets to help form clots but when a cancer of the blood like, leukemia or lymphoma strikes, these life-supporting systems are thrown out of whack, leaving the bodys immune system unable to fight diseases, infection or the cancer.

Chemotherapy and radiation also can kill off bone marrow tissue, leaving patients with more damage to their immune systems, says physician assistant Susanne Smith, donor services clinician at Dana-Farber/Brigham and Womens Hospitals Cancer Center in Boston.

When transplanted into a cancer patients bloodstream, stem cells, a precursor to all the immune system cells in the body, colonize the bones and help fight any remaining cancer, says Smith.

In many cases (a transplant) is the only cure for a leukemia or lymphoma diagnosis chemotherapy can only get a patient so far, says Mary Halet, director of community engagement at the Be The Match Registry, the Minneapolis organization that manages the largest bone marrow registry in the world. But first, a patient must find a tissue match, that is, a donor who has a similar protein marker called the human leukocyte antigen, which is found on most cells in the body.

There are up to 14,000 patients every year who could benefit from a bone marrow transplant, but many of these people will not receive a donation, says Halet. In most cases, the patient will not finda tissue match in his or her own family andmust seek help from a stranger, she says. A patients likelihood of finding a matching bone marrow donor ranges from 66 percent to 97 percentdepending on ethnic background. White patients have a 97 percent chance of finding a match, while black patients only find a match 66 percent of the time.The difference reflects the complexity of the tissues makeup and the number of donors.

Thats why Halets organization promotes recruitment events like the one Ashli Stempel held in Greenfield.

Stempel says she was ecstatic when she found out that she was a match for her brother. She was in her late 20s at the time, a bubbly woman working in communications at Smith College in Northampton, who grew up in a close-knit family.

Her brother, who was working as a retail manager in the Boston area, had discovered a bump on his collarbone.

I woke up one morning and there was a non-painful lump, Andrew Stempel says.

He ignored it for as long as he could before seeing a doctor who diagnosed it as a swollen lymph node caused by Hodgkins lymphoma.

Cancer is a very scary word. I think what you learn going through it is that it is not such a scary word, you can survive, says Ashli Stempel.

The Stempel family had seen that firsthand years earlier when Andrew and Ashlis mother, Deborah, recovered from breast cancer.

Still, that didnt lessen the anxiety for Andrew. As soon as the doctor said the word cancer, he says, his life started to unravel with a battery of experimental drugs, chemotherapy and radiation.

In the begining there was a lot of uncertainty, he says.

Even through his cancer went into remission after a year, doctors did not expect it to remain that way without high doses of chemo or radiation. The plan was to do a bone marrow transplant for long-term survival.

Still, using donated bone marrow meant taking the risk that Andrews body would reject it, which could be fatal.

So, doctors first wanted to try using Andrews own tissue. That would require removing some of his bone marrow, treating it and then injecting it back into his bloodstream.

Within months of the procedure, however, Andrews cancer returned, indicating to doctors that his body wasnt strong enough to fight it on its own.

Ashli was tested via a mouth swab and Andrew was relieved to learn that she was a tissue match.

I was just overwhelmed with happiness, he says.

Ashli went through a month-long screening process to ensure that she was healthy enough to be a donor. People who have infectious diseases like HIV or hepatitis cannot be donors, nor can those with immune systems weakened by autoimmune diseases. Doctors also prefer to use bone marrow from young donors under the age of 44, says Halet. The registry wont accept donors over 60.

When we are young, our immune systems are at their healthiest and the older we get the less robust they are, she says.

It took two years from the time Ashli first learned she was a match for her brother for the transplant to take place.

Not long aftershe woke up from the surgery, Ashli saw the bone marrow that had been taken from her, a two-literjug ofmilky, red liquid. It was whisked away to another partof the hospital where it ended up in a drip bag connected to a vein in Andrews arm.

Doctors saw hisred and white blood cell counts go up immediately after the transplant.

My sisters cells were working, he says. It was amazing.

Even though the transplant was a success, Andrew had to stay in the hospital for a month. Chemotherapy had caused sores in his mouth, he lost his ability to taste food along withhis appetiteand he droppednearly 30 pounds.

It was tough, day to day, but progressively got better, he says.

Since he was essentially receiving a new immune system, like a newborn baby he also had to be shielded from germs, says Ashli.

When his wife, Meghan Stempel, came to visit him, she needed to wear a facemask and gloves. Even when he returned home, he had to be careful. Hetook a year off from his job to recover, spending many afternoons resting on the couch watching TV. After spending months working to building hisstrength back up,he says, most of his weakness has subsided.

I feel a thousand times better, he says.

He is now cancer free and is returning to hisjob as a retail managerat Sherwin Williams this week.

Following her operation, Ashli took off a few weeks from her job in communications at Smith College, but was back on her feet within a couple days. Her hips were sore which meant limping around the house for a short time.

I was in pain, of course, she says. But its a quick recovery.

A few weeks ago Ashli decided to signup for the national bone marrow donor registry through Be A Matchto donate for a second time.

Her name will stay in the system for the foreseeable future. A match could come up or it might never.

Maybe I will be called on to do it again, who knows?

To learn more about becoming a bone marrow donor or to sign up for the registry, go tobethematch.org.

Potential donors can fill out an online form and the registry will mail a mouth swab kit, which can be returned by mail.

If called, a potential donor will undergo a series of blood tests which will evaluate the suitabililty and safety of the match. Though doctors say risks are low for donors, possible complications include infection and bleeding.

Once a donor is cleared, the transplant procedure could occur within a few weeks or a few months, depending on a recommendation from the patients doctor.

The bone marrow transplant is an outpatient procedure for the donor.Recovery time is only a few days anddonors are typically back to their normal routine in two to seven days.

Donors are told their commitment means being willing to devote up to 30 hours spread over four to six weeks to attend appointments and give the donation.

All medical costs for the donation procedure are covered by the National Marrow Donor Program, which operates the Be The Match Registry, or by the cancer patients medical insurance.

Sometime travel is required. Most travel expenses are covered by Be The Match.

See the article here:
Ashli Stempel helped save her brother's life. She hopes to inspire others. - GazetteNET

Hematopoietic stem cell transplantation (HSCT) is the transplantation of multipotent hematopoietic stem cells, usually derived from bone marrow, peripheral blood, or umbilical cord blood.[1][2] It may be autologous (the patient's own stem cells are used), allogeneic (the stem cells come from a donor) or syngeneic (from an identical twin).[1][2] It is a medical procedure in the field of hematology, most often performed for patients with certain cancers of the blood or bone marrow, such as multiple myeloma or leukemia.[2] In these cases, the recipient's immune system is usually destroyed with radiation or chemotherapy before the transplantation. Infection and graft-versus-host disease are major complications of allogeneic HSCT.[2]

Hematopoietic stem cell transplantation remains a dangerous procedure with many possible complications; it is reserved for patients with life-threatening diseases. As survival following the procedure has increased, its use has expanded beyond cancer, such as autoimmune diseases.[3][4]

Indications for stem cell transplantation are as follows:

Many recipients of HSCTs are multiple myeloma[5] or leukemia patients[6] who would not benefit from prolonged treatment with, or are already resistant to, chemotherapy. Candidates for HSCTs include pediatric cases where the patient has an inborn defect such as severe combined immunodeficiency or congenital neutropenia with defective stem cells, and also children or adults with aplastic anemia[7] who have lost their stem cells after birth. Other conditions[8] treated with stem cell transplants include sickle-cell disease, myelodysplastic syndrome, neuroblastoma, lymphoma, Ewing's sarcoma, desmoplastic small round cell tumor, chronic granulomatous disease and Hodgkin's disease. More recently non-myeloablative, "mini transplant(microtransplantation)," procedures have been developed that require smaller doses of preparative chemo and radiation. This has allowed HSCT to be conducted in the elderly and other patients who would otherwise be considered too weak to withstand a conventional treatment regimen.

In 2006 a total of 50,417 first hematopoietic stem cell transplants were reported as taking place worldwide, according to a global survey of 1327 centers in 71 countries conducted by the Worldwide Network for Blood and Marrow Transplantation. Of these, 28,901 (57 percent) were autologous and 21,516 (43 percent) were allogeneic (11,928 from family donors and 9,588 from unrelated donors). The main indications for transplant were lymphoproliferative disorders (54.5 percent) and leukemias (33.8 percent), and the majority took place in either Europe (48 percent) or the Americas (36 percent).[9]

In 2014, according to the World Marrow Donor Association, stem cell products provided for unrelated transplantation worldwide had increased to 20,604 (4,149 bone marrow donations, 12,506 peripheral blood stem cell donations, and 3,949 cord blood units).[10]

Autologous HSCT requires the extraction (apheresis) of haematopoietic stem cells (HSC) from the patient and storage of the harvested cells in a freezer. The patient is then treated with high-dose chemotherapy with or without radiotherapy with the intention of eradicating the patient's malignant cell population at the cost of partial or complete bone marrow ablation (destruction of patient's bone marrow's ability to grow new blood cells). The patient's own stored stem cells are then transfused into his/her bloodstream, where they replace destroyed tissue and resume the patient's normal blood cell production. Autologous transplants have the advantage of lower risk of infection during the immune-compromised portion of the treatment since the recovery of immune function is rapid. Also, the incidence of patients experiencing rejection (and graft-versus-host disease is impossible) is very rare due to the donor and recipient being the same individual. These advantages have established autologous HSCT as one of the standard second-line treatments for such diseases as lymphoma.[11]

However, for other cancers such as acute myeloid leukemia, the reduced mortality of the autogenous relative to allogeneic HSCT may be outweighed by an increased likelihood of cancer relapse and related mortality, and therefore the allogeneic treatment may be preferred for those conditions.[12] Researchers have conducted small studies using non-myeloablative hematopoietic stem cell transplantation as a possible treatment for type I (insulin dependent) diabetes in children and adults. Results have been promising; however, as of 2009[update] it was premature to speculate whether these experiments will lead to effective treatments for diabetes.[13]

Allogeneics HSCT involves two people: the (healthy) donor and the (patient) recipient. Allogeneic HSC donors must have a tissue (HLA) type that matches the recipient. Matching is performed on the basis of variability at three or more loci of the HLA gene, and a perfect match at these loci is preferred. Even if there is a good match at these critical alleles, the recipient will require immunosuppressive medications to mitigate graft-versus-host disease. Allogeneic transplant donors may be related (usually a closely HLA matched sibling), syngeneic (a monozygotic or 'identical' twin of the patient - necessarily extremely rare since few patients have an identical twin, but offering a source of perfectly HLA matched stem cells) or unrelated (donor who is not related and found to have very close degree of HLA matching). Unrelated donors may be found through a registry of bone marrow donors such as the National Marrow Donor Program. People who would like to be tested for a specific family member or friend without joining any of the bone marrow registry data banks may contact a private HLA testing laboratory and be tested with a mouth swab to see if they are a potential match.[14] A "savior sibling" may be intentionally selected by preimplantation genetic diagnosis in order to match a child both regarding HLA type and being free of any obvious inheritable disorder. Allogeneic transplants are also performed using umbilical cord blood as the source of stem cells. In general, by transfusing healthy stem cells to the recipient's bloodstream to reform a healthy immune system, allogeneic HSCTs appear to improve chances for cure or long-term remission once the immediate transplant-related complications are resolved.[15][16][17]

A compatible donor is found by doing additional HLA-testing from the blood of potential donors. The HLA genes fall in two categories (Type I and Type II). In general, mismatches of the Type-I genes (i.e. HLA-A, HLA-B, or HLA-C) increase the risk of graft rejection. A mismatch of an HLA Type II gene (i.e. HLA-DR, or HLA-DQB1) increases the risk of graft-versus-host disease. In addition a genetic mismatch as small as a single DNA base pair is significant so perfect matches require knowledge of the exact DNA sequence of these genes for both donor and recipient. Leading transplant centers currently perform testing for all five of these HLA genes before declaring that a donor and recipient are HLA-identical.

Race and ethnicity are known to play a major role in donor recruitment drives, as members of the same ethnic group are more likely to have matching genes, including the genes for HLA.[18]

As of 2013[update], there were at least two commercialized allogeneic cell therapies, Prochymal and Cartistem.[19]

To limit the risks of transplanted stem cell rejection or of severe graft-versus-host disease in allogeneic HSCT, the donor should preferably have the same human leukocyte antigens (HLA) as the recipient. About 25 to 30 percent of allogeneic HSCT recipients have an HLA-identical sibling. Even so-called "perfect matches" may have mismatched minor alleles that contribute to graft-versus-host disease.

In the case of a bone marrow transplant, the HSC are removed from a large bone of the donor, typically the pelvis, through a large needle that reaches the center of the bone. The technique is referred to as a bone marrow harvest and is performed under general anesthesia.

Peripheral blood stem cells[20] are now the most common source of stem cells for HSCT. They are collected from the blood through a process known as apheresis. The donor's blood is withdrawn through a sterile needle in one arm and passed through a machine that removes white blood cells. The red blood cells are returned to the donor. The peripheral stem cell yield is boosted with daily subcutaneous injections of Granulocyte-colony stimulating factor, serving to mobilize stem cells from the donor's bone marrow into the peripheral circulation.

It is also possible to extract stem cells from amniotic fluid for both autologous or heterologous use at the time of childbirth.

Umbilical cord blood is obtained when a mother donates her infant's umbilical cord and placenta after birth. Cord blood has a higher concentration of HSC than is normally found in adult blood. However, the small quantity of blood obtained from an Umbilical Cord (typically about 50 mL) makes it more suitable for transplantation into small children than into adults. Newer techniques using ex-vivo expansion of cord blood units or the use of two cord blood units from different donors allow cord blood transplants to be used in adults.

Cord blood can be harvested from the Umbilical Cord of a child being born after preimplantation genetic diagnosis (PGD) for human leucocyte antigen (HLA) matching (see PGD for HLA matching) in order to donate to an ill sibling requiring HSCT.

Unlike other organs, bone marrow cells can be frozen (cryopreserved) for prolonged periods without damaging too many cells. This is a necessity with autologous HSC because the cells must be harvested from the recipient months in advance of the transplant treatment. In the case of allogeneic transplants, fresh HSC are preferred in order to avoid cell loss that might occur during the freezing and thawing process. Allogeneic cord blood is stored frozen at a cord blood bank because it is only obtainable at the time of childbirth. To cryopreserve HSC, a preservative, DMSO, must be added, and the cells must be cooled very slowly in a controlled-rate freezer to prevent osmotic cellular injury during ice crystal formation. HSC may be stored for years in a cryofreezer, which typically uses liquid nitrogen.

The chemotherapy or irradiation given immediately prior to a transplant is called the conditioning regimen, the purpose of which is to help eradicate the patient's disease prior to the infusion of HSC and to suppress immune reactions. The bone marrow can be ablated (destroyed) with dose-levels that cause minimal injury to other tissues. In allogeneic transplants a combination of cyclophosphamide with total body irradiation is conventionally employed. This treatment also has an immunosuppressive effect that prevents rejection of the HSC by the recipient's immune system. The post-transplant prognosis often includes acute and chronic graft-versus-host disease that may be life-threatening. However, in certain leukemias this can coincide with protection against cancer relapse owing to the graft versus tumor effect.[21]Autologous transplants may also use similar conditioning regimens, but many other chemotherapy combinations can be used depending on the type of disease.

A newer treatment approach, non-myeloablative allogeneic transplantation, also termed reduced-intensity conditioning (RIC), uses doses of chemotherapy and radiation too low to eradicate all the bone marrow cells of the recipient.[22]:320321 Instead, non-myeloablative transplants run lower risks of serious infections and transplant-related mortality while relying upon the graft versus tumor effect to resist the inherent increased risk of cancer relapse.[23][24] Also significantly, while requiring high doses of immunosuppressive agents in the early stages of treatment, these doses are less than for conventional transplants.[25] This leads to a state of mixed chimerism early after transplant where both recipient and donor HSC coexist in the bone marrow space.

Decreasing doses of immunosuppressive therapy then allows donor T-cells to eradicate the remaining recipient HSC and to induce the graft versus tumor effect. This effect is often accompanied by mild graft-versus-host disease, the appearance of which is often a surrogate marker for the emergence of the desirable graft versus tumor effect, and also serves as a signal to establish an appropriate dosage level for sustained treatment with low levels of immunosuppressive agents.

Because of their gentler conditioning regimens, these transplants are associated with a lower risk of transplant-related mortality and therefore allow patients who are considered too high-risk for conventional allogeneic HSCT to undergo potentially curative therapy for their disease. The optimal conditioning strategy for each disease and recipient has not been fully established, but RIC can be used in elderly patients unfit for myeloablative regimens, for whom a higher risk of cancer relapse may be acceptable.[22][24]

After several weeks of growth in the bone marrow, expansion of HSC and their progeny is sufficient to normalize the blood cell counts and re-initiate the immune system. The offspring of donor-derived hematopoietic stem cells have been documented to populate many different organs of the recipient, including the heart, liver, and muscle, and these cells had been suggested to have the abilities of regenerating injured tissue in these organs. However, recent research has shown that such lineage infidelity does not occur as a normal phenomenon[citation needed].

HSCT is associated with a high treatment-related mortality in the recipient (1 percent or higher)[citation needed], which limits its use to conditions that are themselves life-threatening. Major complications are veno-occlusive disease, mucositis, infections (sepsis), graft-versus-host disease and the development of new malignancies.

Bone marrow transplantation usually requires that the recipient's own bone marrow be destroyed ("myeloablation"). Prior to "engraftment" patients may go for several weeks without appreciable numbers of white blood cells to help fight infection. This puts a patient at high risk of infections, sepsis and septic shock, despite prophylactic antibiotics. However, antiviral medications, such as acyclovir and valacyclovir, are quite effective in prevention of HSCT-related outbreak of herpetic infection in seropositive patients.[26] The immunosuppressive agents employed in allogeneic transplants for the prevention or treatment of graft-versus-host disease further increase the risk of opportunistic infection. Immunosuppressive drugs are given for a minimum of 6-months after a transplantation, or much longer if required for the treatment of graft-versus-host disease. Transplant patients lose their acquired immunity, for example immunity to childhood diseases such as measles or polio. For this reason transplant patients must be re-vaccinated with childhood vaccines once they are off immunosuppressive medications.

Severe liver injury can result from hepatic veno-occlusive disease (VOD). Elevated levels of bilirubin, hepatomegaly and fluid retention are clinical hallmarks of this condition. There is now a greater appreciation of the generalized cellular injury and obstruction in hepatic vein sinuses, and hepatic VOD has lately been referred to as sinusoidal obstruction syndrome (SOS). Severe cases of SOS are associated with a high mortality rate. Anticoagulants or defibrotide may be effective in reducing the severity of VOD but may also increase bleeding complications. Ursodiol has been shown to help prevent VOD, presumably by facilitating the flow of bile.

The injury of the mucosal lining of the mouth and throat is a common regimen-related toxicity following ablative HSCT regimens. It is usually not life-threatening but is very painful, and prevents eating and drinking. Mucositis is treated with pain medications plus intravenous infusions to prevent dehydration and malnutrition.

Graft-versus-host disease (GVHD) is an inflammatory disease that is unique to allogeneic transplantation. It is an attack of the "new" bone marrow's immune cells against the recipient's tissues. This can occur even if the donor and recipient are HLA-identical because the immune system can still recognize other differences between their tissues. It is aptly named graft-versus-host disease because bone marrow transplantation is the only transplant procedure in which the transplanted cells must accept the body rather than the body accepting the new cells.[27]

Acute graft-versus-host disease typically occurs in the first 3 months after transplantation and may involve the skin, intestine, or the liver. High-dose corticosteroids such as prednisone are a standard treatment; however this immuno-suppressive treatment often leads to deadly infections. Chronic graft-versus-host disease may also develop after allogeneic transplant. It is the major source of late treatment-related complications, although it less often results in death. In addition to inflammation, chronic graft-versus-host disease may lead to the development of fibrosis, or scar tissue, similar to scleroderma; it may cause functional disability and require prolonged immunosuppressive therapy. Graft-versus-host disease is usually mediated by T cells, which react to foreign peptides presented on the MHC of the host.[citation needed]

Graft versus tumor effect (GVT) or "graft versus leukemia" effect is the beneficial aspect of the Graft-versus-Host phenomenon. For example, HSCT patients with either acute, or in particular chronic, graft-versus-host disease after an allogeneic transplant tend to have a lower risk of cancer relapse.[28][29] This is due to a therapeutic immune reaction of the grafted donor T lymphocytes against the diseased bone marrow of the recipient. This lower rate of relapse accounts for the increased success rate of allogeneic transplants, compared to transplants from identical twins, and indicates that allogeneic HSCT is a form of immunotherapy. GVT is the major benefit of transplants that do not employ the highest immuno-suppressive regimens.

Graft versus tumor is mainly beneficial in diseases with slow progress, e.g. chronic leukemia, low-grade lymphoma, and some cases multiple myeloma. However, it is less effective in rapidly growing acute leukemias.[30]

If cancer relapses after HSCT, another transplant can be performed, infusing the patient with a greater quantity of donor white blood cells (Donor lymphocyte infusion).[30]

Patients after HSCT are at a higher risk for oral carcinoma. Post-HSCT oral cancer may have more aggressive behavior with poorer prognosis, when compared to oral cancer in non-HSCT patients.[31]

Prognosis in HSCT varies widely dependent upon disease type, stage, stem cell source, HLA-matched status (for allogeneic HSCT) and conditioning regimen. A transplant offers a chance for cure or long-term remission if the inherent complications of graft versus host disease, immuno-suppressive treatments and the spectrum of opportunistic infections can be survived.[15][16] In recent years, survival rates have been gradually improving across almost all populations and sub-populations receiving transplants.[32]

Mortality for allogeneic stem cell transplantation can be estimated using the prediction model created by Sorror et al.,[33] using the Hematopoietic Cell Transplantation-Specific Comorbidity Index (HCT-CI). The HCT-CI was derived and validated by investigators at the Fred Hutchinson Cancer Research Center (Seattle, WA). The HCT-CI modifies and adds to a well-validated comorbidity index, the Charlson Comorbidity Index (CCI) (Charlson et al.[34]) The CCI was previously applied to patients undergoing allogeneic HCT but appears to provide less survival prediction and discrimination than the HCT-CI scoring system.

The risks of a complication depend on patient characteristics, health care providers and the apheresis procedure, and the colony-stimulating factor used (G-CSF). G-CSF drugs include filgrastim (Neupogen, Neulasta), and lenograstim (Graslopin).

Filgrastim is typically dosed in the 10 microgram/kg level for 45 days during the harvesting of stem cells. The documented adverse effects of filgrastim include splenic rupture (indicated by left upper abdominal or shoulder pain, risk 1 in 40000), Adult respiratory distress syndrome (ARDS), alveolar hemorrage, and allergic reactions (usually expressed in first 30 minutes, risk 1 in 300).[35][36][37] In addition, platelet and hemoglobin levels dip post-procedure, not returning to normal until one month.[37]

The question of whether geriatrics (patients over 65) react the same as patients under 65 has not been sufficiently examined. Coagulation issues and inflammation of atherosclerotic plaques are known to occur as a result of G-CSF injection. G-CSF has also been described to induce genetic changes in mononuclear cells of normal donors.[36] There is evidence that myelodysplasia (MDS) or acute myeloid leukaemia (AML) can be induced by GCSF in susceptible individuals.[38]

Blood was drawn peripherally in a majority of patients, but a central line to jugular/subclavian/femoral veins may be used in 16 percent of women and 4 percent of men. Adverse reactions during apheresis were experienced in 20 percent of women and 8 percent of men, these adverse events primarily consisted of numbness/tingling, multiple line attempts, and nausea.[37]

A study involving 2408 donors (1860 years) indicated that bone pain (primarily back and hips) as a result of filgrastim treatment is observed in 80 percent of donors by day 4 post-injection.[37] This pain responded to acetaminophen or ibuprofen in 65 percent of donors and was characterized as mild to moderate in 80 percent of donors and severe in 10 percent.[37] Bone pain receded post-donation to 26 percent of patients 2 days post-donation, 6 percent of patients one week post-donation, and <2 percent 1 year post-donation. Donation is not recommended for those with a history of back pain.[37] Other symptoms observed in more than 40 percent of donors include myalgia, headache, fatigue, and insomnia.[37] These symptoms all returned to baseline 1 month post-donation, except for some cases of persistent fatigue in 3 percent of donors.[37]

In one metastudy that incorporated data from 377 donors, 44 percent of patients reported having adverse side effects after peripheral blood HSCT.[38] Side effects included pain prior to the collection procedure as a result of GCSF injections, post-procedural generalized skeletal pain, fatigue and reduced energy.[38]

A study that surveyed 2408 donors found that serious adverse events (requiring prolonged hospitalization) occurred in 15 donors (at a rate of 0.6 percent), although none of these events were fatal.[37] Donors were not observed to have higher than normal rates of cancer with up to 48 years of follow up.[37] One study based on a survey of medical teams covered approximately 24,000 peripheral blood HSCT cases between 1993 and 2005, and found a serious cardiovascular adverse reaction rate of about 1 in 1500.[36] This study reported a cardiovascular-related fatality risk within the first 30 days HSCT of about 2 in 10000. For this same group, severe cardiovascular events were observed with a rate of about 1 in 1500. The most common severe adverse reactions were pulmonary edema/deep vein thrombosis, splenic rupture, and myocardial infarction. Haematological malignancy induction was comparable to that observed in the general population, with only 15 reported cases within 4 years.[36]

Georges Math, a French oncologist, performed the first European bone marrow transplant in November 1958 on five Yugoslavian nuclear workers whose own marrow had been damaged by irradiation caused by a criticality accident at the Vina Nuclear Institute, but all of these transplants were rejected.[39][40][41][42][43] Math later pioneered the use of bone marrow transplants in the treatment of leukemia.[43]

Stem cell transplantation was pioneered using bone-marrow-derived stem cells by a team at the Fred Hutchinson Cancer Research Center from the 1950s through the 1970s led by E. Donnall Thomas, whose work was later recognized with a Nobel Prize in Physiology or Medicine. Thomas' work showed that bone marrow cells infused intravenously could repopulate the bone marrow and produce new blood cells. His work also reduced the likelihood of developing a life-threatening complication called graft-versus-host disease.[44]

The first physician to perform a successful human bone marrow transplant on a disease other than cancer was Robert A. Good at the University of Minnesota in 1968.[45] In 1975, John Kersey, M.D., also of the University of Minnesota, performed the first successful bone marrow transplant to cure lymphoma. His patient, a 16-year-old-boy, is today the longest-living lymphoma transplant survivor.[46]

At the end of 2012, 20.2 million people had registered their willingness to be a bone marrow donor with one of the 67 registries from 49 countries participating in Bone Marrow Donors Worldwide. 17.9 million of these registered donors had been ABDR typed, allowing easy matching. A further 561,000 cord blood units had been received by one of 46 cord blood banks from 30 countries participating. The highest total number of bone marrow donors registered were those from the USA (8.0 million), and the highest number per capita were those from Cyprus (15.4 percent of the population).[47]

Within the United States, racial minority groups are the least likely to be registered and therefore the least likely to find a potentially life-saving match. In 1990, only six African-Americans were able to find a bone marrow match, and all six had common European genetic signatures.[48]

Africans are more genetically diverse than people of European descent, which means that more registrations are needed to find a match. Bone marrow and cord blood banks exist in South Africa, and a new program is beginning in Nigeria.[48] Many people belonging to different races are requested to donate as there is a shortage of donors in African, Mixed race, Latino, Aboriginal, and many other communities.

In 2007, a team of doctors in Berlin, Germany, including Gero Htter, performed a stem cell transplant for leukemia patient Timothy Ray Brown, who was also HIV-positive.[49] From 60 matching donors, they selected a [CCR5]-32 homozygous individual with two genetic copies of a rare variant of a cell surface receptor. This genetic trait confers resistance to HIV infection by blocking attachment of HIV to the cell. Roughly one in 1000 people of European ancestry have this inherited mutation, but it is rarer in other populations.[50][51] The transplant was repeated a year later after a leukemia relapse. Over three years after the initial transplant, and despite discontinuing antiretroviral therapy, researchers cannot detect HIV in the transplant recipient's blood or in various biopsies of his tissues.[52] Levels of HIV-specific antibodies have also declined, leading to speculation that the patient may have been functionally cured of HIV. However, scientists emphasise that this is an unusual case.[53] Potentially fatal transplant complications (the "Berlin patient" suffered from graft-versus-host disease and leukoencephalopathy) mean that the procedure could not be performed in others with HIV, even if sufficient numbers of suitable donors were found.[54][55]

In 2012, Daniel Kuritzkes reported results of two stem cell transplants in patients with HIV. They did not, however, use donors with the 32 deletion. After their transplant procedures, both were put on antiretroviral therapies, during which neither showed traces of HIV in their blood plasma and purified CD4 T cells using a sensitive culture method (less than 3 copies/mL). However, the virus was once again detected in both patients some time after the discontinuation of therapy.[56]

Since McAllister's 1997 report on a patient with multiple sclerosis (MS) who received a bone marrow transplant for CML,[57] over 600 reports have been published describing HSCTs performed primarily for MS.[58] These have been shown to "reduce or eliminate ongoing clinical relapses, halt further progression, and reduce the burden of disability in some patients" that have aggressive highly active MS, "in the absence of chronic treatment with disease-modifying agents".[58]

Clincs performing HSCT includes Northwestern University and Karolinska University Hospital.

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Hematopoietic stem cell transplantation - Wikipedia

Stem cells have tremendous promise to help us understand and treat a range of diseases, injuries and other health-related conditions. Their potential is evident in the use of blood stem cells to treat diseases of the blood, a therapy that has saved the lives of thousands of children with leukemia; and can be seen in the use of stem cells for tissue grafts to treat diseases or injury to the bone, skin and surface of the eye. Important clinical trials involving stem cells are underway for many other conditions and researchers continue to explore new avenues using stem cells in medicine.

There is still a lot to learn about stem cells, however, and their current applications as treatments are sometimes exaggerated by the media and other parties who do not fully understand the science and current limitations, and also by clinics looking to capitalize on the hype by selling treatments to chronically ill or seriously injured patients. The information on this page is intended to help you understand both the potential and the limitations of stem cells at this point in time, and to help you spot some of the misinformation that is widely circulated by clinics offering unproven treatments.

It is important to discuss these Nine Things to Know and any research or information you gather with your primary care physician and other trusted members of your healthcare team in deciding what is right for you.

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Nine Things to Know About Stem Cell Treatments

In an effort to determine if stem cell therapy can prevent or improve a condition called "diabetic foot" caused by poor blood flow in patients with diabetes, a team of researchers in China has found that transplanting human placenta-derived mesenchymal stem cells (MSCs) into rats modeled with diabetes can affect blood vessel growth, potentially improving blood flow and preventing critical limb ischemia (CLI), a condition that results in diabetic foot and frequently leads to amputation.

The study will be published in a future issue of Cell Transplantation and is currently freely available on-line as an unedited, early epub at: http://www.ingentaconnect.com/content/cog/ct/pre-prints/content-ct-1594_liang_et_al

"CLI describes an advanced stage of peripheral artery disease characterized by obstruction of the arteries and a markedly reduced blood flow to the extremities. CLI is associated with high rates of mortality and morbidity, putting the patients at high-risk for major amputation," said study co-author Dr. Zhong Chao Han of the Beijing Institute of Stem Cells, Health and Biotech. "Mesenchymal stem cells are ideal candidates for transplantation because they have both angiogenic (potential to form new blood vessels) and immunomodulatory properties and are capable of differentiating into three different lineages. The utility of placenta-derived MSCs is poorly understood, so we sought to investigate the efficacy of combined regular therapy and cell therapy in treating diabetes-related CLI."

According to the researchers, human placenta was obtained from full-term cesarean section deliveries with written informed consent of the mother. The use of human-derived cells was approved by the Institutional Biomedical Research Ethics Committee of the Chinese Academy of Medical Science and Peking Union Medical College.

After injection into rats surgically modeled with CLI, the stem cells were traced and counted at various points in time. The researchers found that the stem cell counts decreased dramatically over time, but a few cells differentiated into vascular cells. The infused cells also secreted cytokines, which are small proteins secreted by cells that have a specific effect on the interactions and communications between cells.

"We believe that cytokines secreted by MSCs attract endothelial cells, a type of cells that make up the tissues lining the interior surface of blood vessels," said the researchers. "These cells participate in building new vascular tissues and also inhibit inflammation."

The researchers concluded that their experimental data implied that MSCs improved ischemia recovery in diabetic rats via direct cell differentiation and paracrine (protein-mediated) mechanisms, although the two mechanisms exist simultaneously. The paracrine mechanisms, said the researchers, were likely more important than direct cell differentiation.

"So far, MSC therapy represents a simple, safe and effective therapeutic approach for diabetes and its complications," the researchers concluded. "Our studies lay the groundwork for the transition from the experimental bench to the clinical bedside."

"Diabetes is becoming more prevalent across the globe and stem cell therapy may be a vital approach to serious vascular complications," said Dr. Maria Carolina Oliveira Rodrigues of the Ribeiro Preto Medical School - University of So Paulo, Brazil and section editor of Cell Transplantation. "Future studies should aim to expound upon previous findings in MSC transplantation studies and confirm the efficacy of placenta-derived MSCs for CLI."

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Transplantation with Human Placental Stem Cells Improves ...

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Guidelines for Preventing Infectious Complications among ...

Neurological complications (NC) after allogeneic hematopoietic stem cell transplantation (allo-HSCT) are common and life-threatening in most cases. They may involve either the central (CNS) or peripheral nervous system (PNS). The aim of this study was to describe incidence and characteristics of NC after reduced-intensity conditioning allo-HSCT (allo-RIC), an unexplored setting. For this purpose, we reviewed 191 consecutive patients who underwent this procedure at our institution between January 1999 and December 2006. The median follow-up for survivors was 48 months (3-98 months). RIC included fludarabine (Flu) 150 mg/m(2) in combination with busulfan (Bu) 8-10mg/kg (n=61), melphalan (Mel) 70-140 mg/m(2) (n=119), cyclophosphamide (Cy) 120 mg/kg (n=7), or low-dose total body irradiation (TBI) 2Gy (n=4). Graft-versus-host disease (GVHD) prophylaxis consisted of cyclosporine A (CsA) in combination with methotrexate (MTX; n=134) or mycophenolate mofetil (MMF; n=52). Twenty-seven patients (14%) developed a total of 31 NC (23 CNS and 8 PNS) for a 4-year cumulative incidence of 16% (95% confidence interval [CI] 11-23). CNS complications included nonfocal encephalopathies in 11 patients, meningoencephalitis in 5 patients, and stroke or hemorrhage in 4. PNS complications consisted of 5 cases of mononeuropathies and 3 cases of polyneuropathies. Drug-related toxicity was responsible for 10 of the 31 events (32%) (8 caused by CsA). Interestingly, 14 of the 23 CNS events (61%) and only 1 of the 8 PNS complications (13%) appeared before day +100 (P=.01). Overall, patients presenting NC showed a trend for higher 1-year nonrelapse mortality (NRM) (37% versus 20%, P=.08). In patients with CNS involvement, 1-year NRM was significantly worse (42% versus 20%, P=.02). CNS NC also had a negative impact on 4-year overall survival (OS; 33% versus 45%, P=.05). In conclusion, our study showed that NC are observed after allo-RIC and have diverse features. NC affecting the CNS have earlier onset and worse outcome than those involving the PNS.

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Early and late neurological complications after reduced ...

Journal of Pain Research 2015: 8: 437-447

Introduction: This was a prospective case series designed to investigate treatment for anterior cruciate ligament (ACL) tears using an injection of autologous bone marrow concentrate.

Methods: Consecutive adult patients presenting to a private outpatient interventional musculoskeletal and pain practice with knee pain, ACL laxity on exam, and magnetic resonance imaging (MRI) evidence of a grade 1, 2, or 3 ACL tears with less than 1 cm retraction were eligible for this study. Eligible patients were treated with an intraligamentous injection of autologous bone marrow concentrate, using fluoroscopic guidance. Pre- and postprocedural sagittal MRI images of the ACLs were analyzed using ImageJ software to objectively quantify changes between pre- and posttreatment scans. Five different types of measurement of ACL pixel intensity were examined as a proxy for ligament integrity. In addition pain visual analog scale (VAS) and Lower Extremity Functional Scale (LEFS) values were recorded at baseline and at 1 month, 3 months, 6 months, and annually postinjection. Objective outcomes measured were pre- to post-MRI measurement changes, as analyzed by the ImageJ software. Subjective outcomes measured were changes in the VAS and LEFS, and a self-rated percentage improvement.

Results: Seven of ten patients showed improvement in at least four of five objective measures of ACL integrity in their postprocedure MRIs. In the entire study group, the mean gray value, median, raw integrated density, and modal gray value all decreased toward low-signal ACLs (P=0.01, P=0.02, P=0.002, and P=0.08), indications of improved ligament integrity. Seven of ten patients responded to the self-rated metrics follow up. The mean VAS change was a decrease of 1.7 (P=0.25), the mean LEFS change was an increase of 23.3 (P=0.03), and mean reported improvement was 86.7%.

Conclusion: Based on this small case series, autologous bone marrow concentrate shows promise in the treatment of grade 1, 2, and possibly grade 3 ACL tears without retraction. Further investigation using a controlled study design is warranted.

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Orthopedic Stem Cell Research and Related Publications