StemCell Therapy

ScienceDaily (June 28, 2012) An international consortium of Huntington's disease experts, including several from the Sue & Bill Gross Stem Cell Research Center at UC Irvine, has generated a human model of the deadly inherited disorder directly from the skin cells of affected patients.

The re-created neurons, which live in a petri dish, will help researchers better understand what disables and kills brain cells in people with HD and let them gauge the effects of potential drug therapies on cells that are otherwise locked deep in the brain.

UCI scientists were part of a consortium that in 1993 identified the autosomal dominant gene mutation responsible for HD, but there is still no cure, and no treatments are available to even slow its onset or progression. The research, published online June 28 in the journal Cell Stem Cell, is the work of the Huntington's Disease iPSC Consortium. Participants examined several other cell lines and control cell lines to ensure that their results were consistent and reproducible in different labs.

"Our discovery will enable us for the first time to test therapies on human Huntington's disease neurons," said Leslie Thompson, UCI professor of psychiatry & human behavior and neurobiology & behavior, one of the world's leading HD experts and a senior author of the study. "This has been a remarkable time in HD research, with the advent of stem cell technologies that have allowed these scientific advancements. Also, having a team of scientists working together as a consortium has benefited the research tremendously and accelerated its pace."

Leslie Lock, a UCI assistant professor of developmental & cell biology and biological chemistry whose lab helped develop the induced pluripotent stem cells (iPSC), added: "It's exciting to be carrying out work that provides hope for HD patients and their families."

Thompson said that UCI scientists will use the new model to study the specific gene expression changes in human brain cells that trigger the onset of HD, helping them understand how these changes happen and how to correct them.

Huntington's disease afflicts about 30,000 people in the U.S. -- typically striking in midlife -- and another 75,000 carry the gene that will eventually lead to it. Caused by a mutation in the gene for a protein called huntingtin, the disease damages brain cells so that individuals with HD progressively lose their ability to walk, talk and reason. It invariably culminates in death. While rare, HD is the most common inherited neurodegenerative disease.

Alvin King, Malcolm Casale, Sara Winokur, Gayani Batugedara, Marquis Vawter and Peter Donovan of UCI contributed to the study.

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Human model of Huntington's disease created from skin's stem cells

Newswise Researchers at the Buck Institute have corrected the genetic mutation responsible for Huntingtons Disease (HD) using a human induced pluripotent stem cell (iPSC) that came from a patient suffering from the incurable, inherited neurodegenerative disorder. Scientists took the diseased iPSCs, made the genetic correction, generated neural stem cells and then transplanted the mutation-free cells into a mouse model of HD where they are generating normal neurons in the area of the brain affected by HD. Results of the research are published in the June 28, 2012 online edition of the journal Cell Stem Cell.

iPSCs are reverse-engineered from human cells such as skin, back to a state where they can be coaxed into becoming any type of cell. They can be used to model numerous human diseases and may also serve as sources of transplantable cells that can be used in novel cell therapies. In the latter case, the patient provides a sample of his or her own skin to the laboratory. We believe the ability to make patient-specific, genetically corrected iPSCs from HD patients is a critical step for the eventual use of these cells in cell replacement therapy, said Buck faculty Lisa Ellerby, PhD, lead author of the study. The genetic correction reversed the signs of disease in these cells the neural stem cells were no longer susceptible to cell death and the function of their mitochondria was normal. Ellerby said the corrected cells could populate the area of the mouse brain affected in HD, therefore, the next stage of research involves transplantation of corrected cells to see if the HD-afflicted mice show improved function. Ellerby said these studies are important as now we can deliver patient-specific cells for cell therapy, that no longer have the disease causing mutation.

Huntington's disease (HD) is a devastating, neurodegenerative genetic disorder that affects muscle coordination and leads to cognitive decline and psychiatric problems. It typically becomes noticeable in mid-adult life, with symptoms beginning between 35 and 44 years of age. Life expectancy following onset of visual symptoms is about 20 years. The worldwide prevalence of HD is 5-10 cases per 100,000 persons. More than a quarter of a million Americans have HD or are "at risk" of inheriting the disease from an affected parent. Key to the disease process is the formation of specific protein aggregates (essentially abnormal clumps) inside some neurons.

All humans have two copies of the Huntingtin gene (HTT), which codes for the protein Huntingtin (Htt). Part of this gene is a repeated section called a trinucleotide repeat, which varies in length between individuals and may change between generations. When the length of this repeated section reaches a certain threshold, it produces an altered form of the protein, called mutant Huntingtin protein (mHtt). Scientists in the Ellerby lab corrected the mutation by replacing the expanded trinucleotide repeat with a normal repeat using homologous recombination. Homologous recombination is a type of genetic recombination where two molecules of DNA are exchanged. In this case the diseased DNA sequence is exchanged for the normal DNA sequence.

Contributors to the work: Mahru An and Ningzhe Zhang are shared first authors of this study. Other Buck Institute researchers involved in the study include Gary Scott, Daniel Montoro, Tobias Wittkop, and faculty members Sean Mooney and Simon Melov. The work was funded by the Buck Institute and the National Institutes of Health.

About the Buck Institute for Research on Aging The Buck Institute is the U.S.s first and foremost independent research organization devoted to Geroscience focused on the connection between normal aging and chronic disease. Based in Novato, CA, The Buck is dedicated to extending Healthspan, the healthy years of human life and does so utilizing a unique interdisciplinary approach involving laboratories studying the mechanisms of aging and those focused on specific diseases. Buck scientists strive to discover new ways of detecting, preventing and treating age-related diseases such as Alzheimers and Parkinsons, cancer, cardiovascular disease, macular degeneration, diabetes and stroke. In their collaborative research, they are supported by the most recent developments in genomics, proteomics and bioinformatics. For more information: http://www.thebuck.org.

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Scientists Correct Huntington's Mutation in Induced Pluripotent Stem Cells

Main Category: Huntingtons Disease Also Included In: Stem Cell Research Article Date: 28 Jun 2012 - 9:00 PDT

Current ratings for: Huntington's Research Tool Developed Using Stem Cells

Cedars-Sinai scientists have joined with expert colleagues around the globe in using stem cells to develop a laboratory model for Huntington's disease, allowing researchers for the first time to test directly on human cells potential treatments for this fatal, inherited disorder.

As explained in a paper published June 28 on the Cell Stem Cell website and scheduled for print in the journal's Aug. 3 issue, scientists at Cedars-Sinai's Regenerative Medicine Institute and the University of Wisconsin took skin cells from patients with Huntington's disease and reprogrammed them into powerful stem cells; these were then made into the nervous system cells affected by the disease. Seven laboratories around the world collaborated to demonstrate the cells had hallmarks of Huntington's.

"This Huntington's 'disease in a dish' will enable us for the first time to test therapies on human Huntington's disease neurons," said Clive Svendsen, PhD, director of the Cedars-Sinai Regenerative Medicine Institute and a senior author of the study. "In addition to increasing our understanding of this disorder and offering a new pathway to identifying treatments, this study is remarkable because of the extensive interactions between a large group of scientists focused on developing this model. It's a new way of doing trailblazing science."

The Huntington's Disease iPSC Consortium united some of the world's top scientists working on this disease. Cedars-Sinai researchers took skin cells from a several Huntington's patients, including a six-year-old with a severe juvenile form of the disease. They genetically reprogrammed these tissues into induced pluripotent stem cells, which can be made into any type of cell in the body. The cells lines were banked by scientists at Cedars-Sinai and scrutinized by all consortium members for differences that may have led to the disease. These cell lines are now an important resource for Huntington's researchers and have been made available via a National Institutes of Health-funded repository at Coriell Institute for Medical Research in New Jersey.

Huntington's, known to the public, for example, as the cause of folksinger Woody Guthrie's death, typically strikes patients in midlife. It causes jerky, twitching motions, loss of muscle control, psychiatric disorders and dementia; the disease ultimately is fatal. In rare, severe cases, the disorder appears in childhood.

Researchers believe that Huntington's results from a mutation in the huntintin gene, leading to production of an abnormal protein and ultimately cell death in specific areas of the brain that control movement and cognition. There is no cure for Huntington's, nor therapies to slow its progression.

The consortium showed Huntington's cell deficits or how they differ from normal cells, including that they were less likely to survive cultivation in the petri dish. Scientists tried depriving them of a growth factor present around normal cells, or "stressing" them, and found that Huntington's neurons died even faster.

"It was great that these characteristics were seen not only in our laboratory, but by all of the consortium members using different techniques," said Virginia Mattis, a post-doctoral scientist at the Cedars-Sinai Regenerative Medicine Institute and one of the lead authors of the study. "It was very reassuring and significantly strengthens the value of this study."

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Huntington's Research Tool Developed Using Stem Cells

Public release date: 28-Jun-2012 [ | E-mail | Share ]

Contact: Nicole White nicole.white@cshs.org 310-423-5215 Cedars-Sinai Medical Center

LOS ANGELES (EMBARGOED UNTIL NOON EDT ON JUNE 28, 2012) Cedars-Sinai scientists have joined with expert colleagues around the globe in using stem cells to develop a laboratory model for Huntington's disease, allowing researchers for the first time to test directly on human cells potential treatments for this fatal, inherited disorder.

As explained in a paper published June 28 on the Cell Stem Cell website and scheduled for print in the journal's Aug. 3 issue, scientists at Cedars-Sinai's Regenerative Medicine Institute and the University of Wisconsin took skin cells from patients with Huntington's disease and reprogrammed them into powerful stem cells; these were then made into the nervous system cells affected by the disease. Seven laboratories around the world collaborated to demonstrate the cells had hallmarks of Huntington's.

"This Huntington's 'disease in a dish' will enable us for the first time to test therapies on human Huntington's disease neurons," said Clive Svendsen, PhD, director of the Cedars-Sinai Regenerative Medicine Institute and a senior author of the study. "In addition to increasing our understanding of this disorder and offering a new pathway to identifying treatments, this study is remarkable because of the extensive interactions between a large group of scientists focused on developing this model. It's a new way of doing trailblazing science."

The Huntington's Disease iPSC Consortium united some of the world's top scientists working on this disease. Cedars-Sinai researchers took skin cells from a several Huntington's patients, including a six-year-old with a severe juvenile form of the disease. They genetically reprogrammed these tissues into induced pluripotent stem cells, which can be made into any type of cell in the body. The cells lines were banked by scientists at Cedars-Sinai and scrutinized by all consortium members for differences that may have led to the disease. These cell lines are now an important resource for Huntington's researchers and have been made available via a National Institutes of Health-funded repository at Coriell Institute for Medical Research in New Jersey.

Huntington's, known to the public, for example, as the cause of folksinger Woody Guthrie's death, typically strikes patients in midlife. It causes jerky, twitching motions, loss of muscle control, psychiatric disorders and dementia; the disease ultimately is fatal. In rare, severe cases, the disorder appears in childhood.

Researchers believe that Huntington's results from a mutation in the huntintin gene, leading to production of an abnormal protein and ultimately cell death in specific areas of the brain that control movement and cognition. There is no cure for Huntington's, nor therapies to slow its progression.

The consortium showed Huntington's cell deficits or how they differ from normal cells, including that they were less likely to survive cultivation in the petri dish. Scientists tried depriving them of a growth factor present around normal cells, or "stressing" them, and found that Huntington's neurons died even faster.

"It was great that these characteristics were seen not only in our laboratory, but by all of the consortium members using different techniques," said Virginia Mattis, a post-doctoral scientist at the Cedars-Sinai Regenerative Medicine Institute and one of the lead authors of the study. "It was very reassuring and significantly strengthens the value of this study."

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Cedars-Sinai researchers, with stem cells and global colleagues, develop Huntingtons research tool

The stem cell bank that was a marquee piece of Governor Deval Patricks effort to bolster the life sciences industry will run out of funding at the end of the year and close, state and University of Massachusetts Medical School officials said Wednesday. The state invested $8.6 million in public funds to establish the bank at the medical schools Shrewsbury campus.

That decision in 2008 was seen then as a bold statement of support for research on human embryonic stem cells during a time when federal funding for work on the controversial cells was restricted. But advances in technology and changes to federal policies rapidly made the bank obsolete, state officials said.

The laboratory grew and stored human stem cells, which are capable of becoming any cell in the body, and made them available to scientists nationwide for use in experiments to study diseases such as diabetes and spinal cord injuries. When it is dismantled, several thousand vials of stem cellswill be sent back to the research centers where they originated, and the equipment will be given to other UMass labs.

Susan Windham-Bannister, president of the Massachusetts Life Sciences Center, a quasi-public agency that oversees the $1 billion life sciences initiative, defended the decision to initially fund the stem cell bank. She said there are many examples of technology that in hindsight are unnecessary, but at the time it was conceived, when the investment was made, it was absolutely state of the art. The center, she said, was one of them.

Originally, the bank was seen as a repository for embryonic stem cell lines that were being created but were not eligible for federal funding under Bush-era restrictions. The field has evolved significantly since then, with President Obamas loosening of restrictions on federal funding and the development of new technologies for making stem cells.

Still, stem cell banks are seen as useful by some. The California Institute for Regenerative Medicine, for example, is preparing to invest $10 million in its own stem cell banking initiative, and another $20 million to underwrite the creation of stem cells from patients with specific diseases.

Massachusetts Senate minority leader Bruce Tarr, Republican of Gloucester, said he was concerned that lawmakers had not been told the bank would close.

Given the fact that this is a resource that was created by an act of the Legislature, I would hope anyone seeking to change its status would consult with the Legislature, he said. The notion has always been we have been working hard to make Massachusetts a leader in stem cell research, and I dont know how ceasing the operations of the stem cell bank advances that goal.

Researchers who had developed and sent some of the 18 embryonic stem cell batches, called lines, that are currently available at UMass expressed their disappointment.

I think the closing of the UMass bank, where we had anticipated maintaining a lot of our lines, means we will have to come up with an alternative, said Dr. George Q. Daley, a stem cell scientist at Boston Childrens Hospital and the Harvard Stem Cell Institute who has sent about half a dozen stem cell lines to the bank. He said he received a call Tuesday from Joseph Laning, who joined UMass Medical School in 2010 to run the bank, alerting him that the bank would be closed.

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UMass stem cell lab to close

Public release date: 27-Jun-2012 [ | E-mail | Share ]

Contact: Bridget Dempsey b.dempsey@qmul.ac.uk 44-207-882-7927 Queen Mary, University of London

Scientists at Queen Mary, University of London have uncovered a link between two genes which shows how stem cells could develop into cancer.

The research, published in the online journal PLoS ONE, found a novel mechanism which could be the catalyst for stem cells changing into a tumour.

Dr Ahmad Waseem, a reader in oral dentistry at Queen Mary, University of London who led the research, said: "It was quite an unexpected discovery. We set out to investigate the role of the stem cell gene Keratin K15 which was thought to be a biomarker for normal stem cells.

"Through our research, we discovered there was link between K15 and the notorious cancer gene FOXM1. We found FOXM1 could target K15 to induce cancer formation."

Cancer develops when there is a problem with stem cells; the cells that carry out internal repairs throughout the human body. The loss of stem cell function leads to uncontrolled growth which ultimately develops into a tumour.

The team went through a process where they used extremely sensitive cell and molecular approaches to establish this link.

The study, funded by the Facial Surgery Research Foundation, Saving Faces, paves the way towards identifying new anti-cancer drugs which could be tailored towards cancer stem cells.

Consultant oral and maxillofacial surgeon Professor Iain Hutchison, founder of Saving Faces and co-author on the study, said: "We are excited about this finding as it could lead to more effective cancer drugs being developed to target cancer stem cells and prevent cancer recurrence."

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Scientists identify new cancer stem cell mechanism

Washington, June 28 : In a new study, researchers have successfully reversed diabetes in mice using stem cells, thereby paving the way for a breakthrough treatment for a disease that affects millions worldwide.

The research by Timothy Kieffer, from University of British Columbia in collaboration with scientists from the New Jersey-based BetaLogics, is the first to show that human stem cell transplants can successfully restore insulin production and reverse diabetes in mice.

Crucially, they re-created the "feedback loop" that enables insulin levels to automatically rise or fall based on blood glucose levels.

After the stem cell transplant, the diabetic mice were weaned off insulin, a procedure designed to mimic human clinical conditions.

Three to four months later, the mice were able to maintain healthy blood sugar levels even when being fed large quantities of sugar.

Transplanted cells removed from the mice after several months had all the markings of normal insulin-producing pancreatic cells.

"We are very excited by these findings, but additional research is needed before this approach can be tested clinically in humans," Kieffer said.

"The studies were performed in diabetic mice that lacked a properly functioning immune system that would otherwise have rejected the cells. We now need to identify a suitable way of protecting the cells from immune attack so that the transplant can ultimately be performed in the absence of any immunosuppression," Kieffer added.

The study has been recently published online in Diabetes. (ANI)

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Stem cells may help reverse diabetes

28.06.2012 - (idw) Ruhr-Universitt Bochum

RUB biologists have deliberately transformed stem cells from the spinal cord of mice into immature nerve cells. This was achieved by changing the cellular environment, known as the extracellular matrix, using the substance sodium chlorate. Via sugar side chains, the extracellular matrix determines which cell type a stem cell can generate. Influencing precursor cells pharmacologically so that they transform into a particular type of cell can help in cell replacement therapies in future says Prof. Dr. Stefan Wiese, head of the Molecular Cell Biology work group. Matrix modified Taking the fate of stem cells in hand RUB researchers generate immature nerve cells

RUB biologists have deliberately transformed stem cells from the spinal cord of mice into immature nerve cells. This was achieved by changing the cellular environment, known as the extracellular matrix, using the substance sodium chlorate. Via sugar side chains, the extracellular matrix determines which cell type a stem cell can generate. Influencing precursor cells pharmacologically so that they transform into a particular type of cell can help in cell replacement therapies in future says Prof. Dr. Stefan Wiese, head of the Molecular Cell Biology work group. Therapies, for example, for Parkinsons, multiple sclerosis or amyotrophic lateral sclerosis could then become more efficient. The team describes its findings in Neural Development.

Sulphate determines the fate of stem cells

Sodium chlorate acts on metabolism enzymes in the cell which attach sulphate groups to proteins. If these sulphates are not installed, the cell continues to form proteins for the extracellular matrix, but with modified sugar side chains. These chains in turn send out signals that define the fate of the stem cells. Stem cells can not only develop into nerve cells, but also form astrocytes or oligodendrocytes, which are, for instance, responsible for the mineral balance of the nerve cells or which form their insulation layer. What happens to the stem cells if the sulphate pattern is changed by sodium chlorate was examined by Dr. Michael Karus and his colleagues.

The RUB-laboratories of Prof. Dr. Stefan Wiese, Prof. Dr. Andreas Faissner and Prof. Dr. Irmgard Dietzel-Meyer collaborated for the study. Using antibodies, the researchers showed that cells which they had treated with sodium chlorate developed into nerve cells. They also analysed the flow of sodium ions into the cells. The result: treated cells showed a lower sodium current than mature nerve cells. Sodium chlorate thus favours the development of stem cells into nerve cells, but, at the same time, also inhibits the maturation - a positive side effect, as Wiese explains: If sodium chlorate stops the nerve cells in an early developmental phase, this could enable them to integrate into the nervous system following a transplant better than mature nerve cells would do.

Bibliographic record

M. Karus, S. Samtleben, C. Busse, T. Tsai, I.D. Dietzel, A. Faissner, S. Wiese (2012): Normal sulphation levels regulate spinal cord neural precursor cell proliferation and differentiation, Neural Development, doi:10.1186/1749-8104-7-20

Further information

Prof. Dr. Stefan Wiese, Molecular Cell Biology Work Group, Faculty of Biology and Biotechnology at the Ruhr-Universitt, 44780 Bochum, Germany, Tel. +49/234/32-22041 stefan.wiese@rub.de

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Taking the fate of stem cells in hand: RUB researchers generate immature nerve cells

Anda Berada di Sini : Dunia Berita

28 Jun, 2012 10:53 AM

Human Stem Cells Reverse Diabetes In Mice: Research

VANCOUVER, June 28 (Bernama) -- A new research has shown that human stem cell transplants can successfully restore insulin production and reverse diabetes in mice for the first time, China's Xinhua news agency reported.

The study, conducted by scientists from University of British Columbia (UBC) and the New Jersey-based BetaLogics, a division of Janssen Research & Development, LLC, could pave the way for a breakthrough treatment for the disease.

After the stem cell transplant, the diabetic mice were weaned off insulin, a procedure designed to mimic human clinical conditions, according to the study published online Wednesday in the journal Diabetes.

Three to four months later, the mice were able to maintain healthy blood sugar levels even when being fed large quantities of sugar.

"We are very excited by these findings, but additional research is needed before this approach can be tested clinically in humans," said Timothy Kieffer, one of the 13 authors and a professor from UBC.

Kieffer said that the studies were performed in diabetic mice that lacked a properly functioning immune system that would otherwise have rejected the cells.

He added that they now need to identify a suitable way of protecting the cells from immune attack so that the transplant can ultimately be performed in the absence of any immunosuppression.

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Human Stem Cells Reverse Diabetes In Mice: Research

Harvard Bioscience's "InBreath" Bioreactors Used in World's First Successful Regenerated Laryngotracheal Transplants

First Two Transplants Performed in Government-Approved Clinical Trial in Russia

HOLLISTON, Mass., June 26, 2012 (GLOBE NEWSWIRE) -- Harvard Bioscience, Inc. (HBIO), a global developer, manufacturer, and marketer of a broad range of tools to advance life science research and regenerative medicine, announces that its "InBreath" bioreactors were used for the world's first and second successful laryngotracheal implants, using synthetic laryngotracheal scaffolds seeded with cells taken from the patients' bone marrow. The surgeries took place at Krasnodar Regional Hospital in Krasnodar, Russia on June 19th and June 21st. The recipients of the implants, Julia T. and Aleksander Z., are recovering well. The implants in the procedures were grown in bioreactors developed by the regenerative medicine device business of Harvard Bioscience.

The transplants, which required more than a half-year of preparation, were performed on the first two patients enrolled in an ongoing clinical trial at Krasnodar Regional Hospital. The Russian Ministry of Health has approved a clinical protocol for an unlimited number of patients in this trial, all of which will involve trachea procedures.

Each bioreactor was specifically adapted by Harvard Bioscience to the clinical requirements for each patient. Each bioreactor was loaded with a synthetic scaffold in the shape of the patient's original organ. The scaffolds were then seeded with the patient's own stem cells. Over the course of about two days, the bioreactor promoted proper cell seeding and development. Because the patients' own stem cells were used, their bodies have accepted the transplants without the use of immunosuppressive drugs.

A photo accompanying this release is available at http://www.globenewswire.com/newsroom/prs/?pkgid=13437

The procedures are the result of a global collaboration involving organizations in the US, Sweden, Russia, Germany, and Italy:

-- The bioreactors were developed, manufactured and prepared by teams at Hugo Sachs Elektronik, a German subsidiary of Harvard Bioscience and at Harvard Bioscience, based in Massachusetts, U.S.A.

-- The scaffolds were created by US-based Nanofiber Solutions.

-- The principal transplant surgeon and main coordinator for both procedures was Dr. Paolo Macchiarini, Professor of Regenerative Surgery at Karolinska Institute in Stockholm.

Originally posted here:
Regenerative medicine pioneer continues changing lives with first successful laryngotracheal implants

HOUSTON, June 27, 2012 /PRNewswire/ --After an invitation from Celltex Therapeutics Corp., the Food and Drug Administration (FDA) visited the Celltex laboratory for two weeks in April, 2012. TheFDA studied Celltex operations in depth in accordance with the "good tissue practices" (GTP) standards, as it routinely does with inspections of facilities such as Celltex which are registered pursuant to 21 CFR Part 1271. In their close-out report given to Celltex on the last day of their visit, the FDA had 14 main observations that it requested Celltex resolve.

Celltex has worked closely withtheFDA both during its visit and since to provide requested details and documentationto answer its questions. We have resolved many of the FDA observations, and we are working to address the remainder. We have an open line of communication with the FDAand expect to maintain that in our cooperative relationship.

Celltex continues to provide stem cell banking and multiplication services without interruption and has not received any disciplinary action from the FDA.

Celltex's laboratory is currently operated by its licensing partner RNL Bio (dba Human Biostar), with lab technicians and scientists from RNL's Seoul, Korea headquarters. The main issues in the FDA observations arose from a language barrier. RNL scientists extensively document procedures, including validations, but they are recorded in Korean and were not able to be provided in English to FDA during its visit. Since the FDA's visit, the RNL procedures and other documents have been translated to English by an independent, professional translation service, and supplied to the agency. We are confident that the translated documents demonstrate the thoroughly validated scientific process that underpins the Celltex laboratory operations.

Celltex continues to strengthen its documentation and laboratory operations and has added to its staff Celltex personnel experienced in U.S. FDA compliance.

Some media reports and social media chatter suggest that Celltex is somehow acting illegally or providing unapproved treatments. These statements are inaccurate. Celltex is registered with the FDA as a facility that multiplies human cells and cellular products (HCT/Ps); in particular, adult mesenchymal stem cells. The FDA does not require a company to obtain FDA approval prior to distribution of its HCT/Ps. 21 CFR Part 1271. In addition, the FDA does not issue "licenses," so any reference that Celltex provides "unlicensed" procedures is inaccurate. Celltex's process for reproducing adult mesenchymal stem cells is legal, and there is no requirement that the cells be approved or licensed.

Celltex ensures that all of the cells it provides to physicians for therapeutic use are sterile, viable, intact mesenchymal stem cells. RNL's quality control scientists examine each patient's cells for their integrity and sterility prior to release, documenting those findings. Celltex and its partner RNL Bio process stem cells in a safe, sterile laboratory with procedures that ensure cell viability and integrity.

Celltex has taken the initiative to make autologous adult mesenchymal stem cell multiplication services available to physicians outside of academia for use with their patients. Celltex firmly believes in the great therapeutic potential for autologous mesenchymal stem cell multiplication services in regenerative medicine.

For more information, contact Celltex, 713-590-1000.

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Celltex Responds to Media Reporting on FDA Visit

Canadian scientists were able to reverse diabetes in mice with a human stem cell transplant, igniting hopes for a cure for the widespread disease -- caused by the failure of the pancreas to produce enough insulin to stabilize blood sugar levels -- in humans.

A paper outlining the work, led by Timothy Kieffer of the University of British Columbia and conducted in partnership with New Jersey-based company BetaLogics, appeared in the journal Diabetes on Tuesday.

Diabetic mice were weaned off of insulin after receiving the pancreatic stem cell transplant, which restarted the cycle in which insulin production rises or falls based on blood sugar levels. Three to four months later, the mice could maintain healthy blood sugar levels even after being fed a lot of sugar.

"We are very excited by these findings, but additional research is needed before this approach can be tested clinically in humans," Kieffer said in a statement on Tuesday.

The researchers cautioned that their study used mice that had a suppressed immune system, the better to prevent rejection of the transplanted cells.

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"We now need to identify a suitable way of protecting the cells from immune attack so that the transplant can ultimately be performed in the absence of any immunosuppression," Kieffer said.

In 2009, a different team of researchers led by scientists from the University of Sao Paulo in Brazil and Northwestern University reported in the Journal of the American Medical Association that they were able to successfully reverse type 1 diabetes by injecting 8 patients with some of their own stem cells.

Some studies have shown that this kind of stem cell transplantation is only a temporary fix - after anywhere between six months to three years, the insulin-producing cells are again attacked by the patient's immune system.

SOURCE: Rezania et al. "Maturation of Human Embryonic Stem Cell-Derived Pancreatic Progenitors into Functional Islets Capable of Treating Pre-existing Diabetes in Mice." Diabetes 27 June 2012.

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Diabetes Reversed In Mice Thanks To Stem Cell Transplant

VATICAN CITY, Italy, June 27, 2012 (GLOBE NEWSWIRE) -- Today, as part of an ongoing mission to advance scientific research on adult stem cell therapies and explore their cultural and ethical implications, Monsignor Tomasz Trafny of the Vatican's Pontifical Council for Culture, joined Dr. Robin Smith, CEO of NeoStem (NYSE MKT:NBS) and Chairman and President of the Stem for Life Foundation, and Dr. Max Gomez, trustee of the Stem for Life Foundation, to present the first copy of their forthcoming book, Our Stem Cells: The Mystery of Life and Secrets of Healing, to The Holy Father, Pope Benedict XVI.

The book is the result of a unique collaboration between the Vatican's Pontifical Council for Culture (via its charitable foundation STOQ International) and the Stem for Life Foundation, and will be available later this year. It includes a special address by His Holiness Benedict XVI, urging increased support and awareness for advancements in adult stem cell research in order to alleviate human suffering.

The book focuses on concepts discussed at the First International Vatican Adult Stem Cell Conference (2011) and presents the reader with an engaging, comprehensive overview of adult stem cells and their vital role in a future of regenerative medicine. In powerful, accessible language the book showcases a wide array of emerging adult stem cell breakthroughs, including their ability to repair damaged hearts and organs, restore sight, kill cancer, cure diabetes, heal burns and stop the march of degenerative diseases, such as Alzheimer's, multiple sclerosis and Lou Gehrig's disease.

"In addition to making the science easy to understand, we filled the book with here-and-now case studies on how adult stem cell therapies are already helping real people suffering needlessly from deadly and debilitating diseases and medical conditions," said Dr. Smith. "Not only does the book speak to the success of our historic partnership with the Vatican, but it sets the stage for our next events."

"This book promotes a powerful dialogue between scientific and religious communities," said Monsignor Tomasz Trafny. "This dialogue needs to find its expression within the important framework of searching for truth and being guided by the highest ethical values. We hope this book will help educate people throughout the world regarding the importance of ethical scientific research and help them understand they do not need to choose between their faith and science; but in fact, the two can work together to profoundly improve humanity."

To preorder the book, go to: http://www.stemforlife.org/ourstemcells

About the Stem for Life Foundation

Stem for Life Foundation (SFLF) is dedicated to improving the quality of life of millions of people suffering from dozens of painful and sometimes debilitating medical conditions by providing information and updates about adult stem cell research, therapy development and possible healthcare applications. SFLF focuses on educating the public, convening the best minds in adult stem cell medicine and research, supporting clinical research, and subsidizing adult stem cell collection and storage for those who need it most.

Understanding that adult stem cell research could lead to better treatments and possibly cures for chronic disease, as well as reduce health care costs and improve quality of life for those with chronic disease and disability, SFLF was established in 2007. SFLF's Board of Trustees and staff are deeply committed to expediting development of stem cell therapies that offer real hope to individuals suffering from a wide-range of life-threatening medical conditions.

About The Pontifical Council for Culture

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The Pontifical Council for Culture and the Stem for Life Foundation Present Groundbreaking Book on Adult Stem Cell ...

The Sugar Land company involved in Gov. Rick Perry's unlicensed adult stem-cell procedure is rife with basic manufacturing problems, according to the U.S. Food and Drug Administration.

In a report one expert called a blow to the entire adult stem-cell industry, the FDA found that Celltex Therapeutics Corp. cannot guarantee the sterility, uniformity and integrity of stem cells it takes from people and then stores and grows for eventual therapeutic reinjection.

"You have not performed a validation of your banking and thawing process to assure viability" of the stem cells, reads the April 27 report, meaning that the company cannot verify the cells are alive.

The FDA report, which followed an April 16-27 inspection of Celltex, was released under the Freedom of Information Act Monday to the Houston Chronicle and a University of Minnesota bioethicist who complained in February that Celltex is a potential danger to patients and not in compliance with federal law.

The report, partially redacted, was not accompanied by a warning letter.

A former FDA official who asked not to be identified, however, said the deficiencies - 79 in all, from incorrectly labeled products to failed sterility tests - are so serious that Celltex risks being shut down if it does not remedy the problems quickly.

Adult stem cells are cells in the body that multiply to replenish dying cells. Long used to treat leukemia and other cancers, they have shown promise for tissue repair in many other diseases in the last decade, although most scientists in the field consider them not ready for mainstream use.

Rules take effect July 8

Celltex has been in the public eye since it was revealed that Perry's Houston doctor treated him with his own stem cells during back surgery last July and in follow-up appointments. His stem cells were stored and grown at Celltex.

Perry subsequently called for Texas to become the nation's leader of adult stem cell medicine, which he touts as an ethical alternative to embryonic stem cells. Perry worked with his Houston doctor and a state representative to write legislation intended to commercialize the therapy in Texas.

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FDA report faults Houston stem-cell company

Optimal stem cell therapy delivery to damaged areas of the heart after myocardial infarction has been hampered by inefficient homing of cells to the damaged site. However, using rat models, researchers in France have used a magnet to guide cells loaded with iron oxide nanoparticles to key sites, enhancing the myocardial retention of intravascularly delivered endothelial progenitor cells.

The study is published in a recent issue of Cell Transplantation (21:4), now freely available online.

"Cell therapy is a promising approach to myocardial regeneration and neovascularization, but currently suffers from the inefficient homing of cells after intracavitary infusion," said Dr. Philippe Menasche of the INSERM U633 Laboratory of Surgical Research in Paris. "Our study was aimed at improving and controlling homing by loading human cord-blood-derived endothelial progenitor cells (EPCs) for transplant with iron oxide nanoparticles in order to better position and retain them in the hearts of myocardial-injured test rats by using a subcutaneously implanted magnet."

The researchers found that the cells were sufficiently magnetic to be able to be remotely manipulated by a magnet subsequent to implantation.

According to the researchers, an objective assessment of the technique to enhance the homing of circulating stem cells is the ability to track their fate in vivo. This was accomplished by visualization with MRI.

"We found a good correlation between MRI non-invasive follow-up of the injected cells and immunofluoresence or quantitative PCR data," said Dr. Menasche. The researchers concluded that further studies were needed to follow cell homing at later time points. They noted that the magnitude of homing they experienced may have been reduced by the relatively small number of cells used, owing to their large size and the subsequent risk of coronary thrombosis.

"In a rat model of myocardial infarction, this pilot study suggested homing of circulating stem cells can be improved by magnetic targeting and warrants additional benchwork to confirm the validity of concept," said Dr. Menasche. "There is also a need to optimize the parameters of targeting and assess the relevance of this approach in a clinically relevant large animal model."

"This study highlights the use of magnets to target transplanted cells to specific sites which could increase their regenerative impact. Factors to still be extensively tested include confirming the safety of the cells containing the magnetic particles and whether this process alters the cell's abilities" said Dr. Amit N. Patel, director of cardiovascular regenerative medicine at the University of Utah and section editor for Cell Transplantation.

More information: Chaudeurge, A.; Wilhelm, C.; Chen-Tournoux, A.; Farahmand, P.; Bellamy, V.; Autret, G.; Mnager, C.; Hagge, A.; Larghro, J.; Gazeau, F.; Clment, O.; Menasch, P. Can Magnetic Targeting of Magnetically Labeled Circulating Cells Optimize Intramyocardial Cell Retention? Cell Transplant. 21 (4):679-691; 2012.

Journal reference: Cell Transplantation

Continued here:
Magnet helps target transplanted iron-loaded cells to key areas of heart

COLORADO SPRINGS, Colo.--(BUSINESS WIRE)--

HemoGenix announced today that FDA CBER has given HemoGenix its first Master File Number for an in vitro blood stem cell potency, quality and release assay (HALO-96 PQR) (1)for cellular therapy products(2)used for stem cell transplantation purposes. HALO-96 PQR is the first commercially available stem cell potency assay for cellular therapy products. It incorporates the most sensitive readout available to measure changes in the cells energy source (ATP) as a function of the potential for stem cells to proliferate. Potency and quality of stem cell therapeutic products are required to be measured prior to use to help predict the engraftment of the cells in the patient. At the present time, tests such as cell number, viability and a stem cell marker called CD34 are routinely used. However, none of these tests specifically measure stem cells and none determine the stem cell biological activity required for a potency assay. The only cell functionality test presently used in this field, especially for umbilical cord blood transplantation, is the colony-forming unit (CFU) assay, which is subjective, non-validated and has been used since the early 1970s. HALO-96 PQR changes this paradigm. It is particularly needed in the umbilical cord blood stem cell transplantation field by providing an application-specific test incorporating all of the compliance characteristics required not only by regulatory agencies(3) and standards organizations, but also the cord blood community(4).

Stem cell potency is one of the most important parameters necessary for any therapeutic product, especially stem cells. Without it, the dose cannot be defined and the transplantation physician has no indication as to whether the product will engraft in the patient. The number of cord blood units collected and stored and the number of cord blood stem cell transplantations have increased exponentially over the last 12 years. During this time, significant advancements have been made in pre- and post stem cell transplantation procedures. Yet the tests used during the preparation and processing of the cells have remained unchanged and do not even measure the biological functionality of the stem cells being transplanted. Indeed, the standards organizations responsible for applying regulatory guidance to the community have so far failed to allow any new and alternative assays to be used during cord blood processing. HALO-96 PQR is the first test that actually quantitatively characterizes and defines the stem cells in cord blood, mobilized peripheral blood or bone marrow as high quality and potent active ingredients for release prior to transplantation. Presently, approximately 20% engraftment failure is encountered in cord blood transplantation. HALO-96 PQR could help reduce the risk of engraftment failure by providing valuable and time-sensitive information on the stem cells prior to use. HALO-96 PQR complies with the guidelines not only with the cord blood community, but also with regulatory agencies thereby providing a benefit to both the stem cell transplantation center and the patient, said Ivan Rich, Founder and CEO of HemoGenix (www.hemogenix.com).

About HemoGenix, Inc.

HemoGenix is a privately held Contract Research Service and Assay Development Laboratory based in Colorado Springs, Colorado. Specializing in predictive in vitro stem cell toxicity testing, HemoGenix provides its services to small, medium and many of the largest biopharmaceutical companies. HemoGenix has developed several assays for stem cell therapy and regenerative medicine applications. These and other patented and proprietary assays are manufactured and produced in Colorado Springs and sold worldwide. HemoGenix has been responsible for changing the paradigm and bringing in vitro stem cell hemotoxicity testing into the 21st century. With HALO-96 PQR the company is now also changing the paradigm to become a leader in stem cell therapy assays. To this end, HemoGenix is a member of the Alliance for Regenerative Medicine and working with other companies to decrease risk and improve safety for the patient.

Literature Cited

Original post:
HemoGenix® FDA Master File to Measure Blood Stem Cell Potency for Cellular Therapy Products:

Editor's Choice Main Category: Pancreatic Cancer Also Included In: Cancer / Oncology;Stem Cell Research Article Date: 27 Jun 2012 - 10:00 PDT

Current ratings for: Metformin Shows Promise For Pancreatic Cancer Patients

3 (2 votes)

In combination with the standard chemotherapy for pancreatic cancer, metformin was observed to efficiently eradicate both cancer stem cells and more differentiated cancer cells that form the bulk of the tumor. The study was presented at the American Association for Cancer Research's Pancreatic Cancer: Progress and Challenges conference in Lake Tahoe, Nev., from June 18-21, 2012 by Christopher Heeschen, M.D., Ph.D., a professor for experimental medicine at the Spanish National Cancer Research Centre in Madrid.

Heeschen said that the majority of clinical trials of pancreatic cancer during the last 15 years failed to demonstrate a notable improvement in the average survival, which indicates for various reasons the methods used in these trials were insufficient. However, within the last few years, scientists have discovered cancer stem cells, which contrary to the cancer cells that make up the bulk of the tumor, consist of a small subset of cells that are resistant to conventional therapy.

He continued:

The team discovered that metformin-pretreated cancer stem cells proved especially sensitive to changes to their metabolism through the activation of AMPK, as metformin killed the cancer stem cells, but only stopped the cell's growth in more differentiated cancer cells.

Heeschen explained:

Their findings were supported in an experiment with mice, in which they treated immunocompromised mice that were implanted with various sets of patient-derived tumors with a combination of metformin and the standard chemotherapeutic treatment for pancreatic cancer, gemcitabine. The results were reduced tumors and a prevention of relapse in contrast to mice treated only with metformin or with gemcitabine.

Heeschen remarked: "Intriguingly, in all tumors treated with metformin to date, relapse of disease was efficiently prevented and there were no noticeable adverse effects."

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Metformin Shows Promise For Pancreatic Cancer Patients

ScienceDaily (June 27, 2012) Scientists at the Max Planck Institute of Immunobiology and Epigenetics in Freiburg have gained important insights for stem cell research which are also applicable to human tumours and could lead to the development of new treatments. As Rolf Kemler's research group discovered, a molecular link exists between the telomerase that determines the length of the telomeres and a signalling pathway known as the Wnt/-signalling pathway.

Telomeres are the end caps of chromosomes that play a very important role in the stability of the genome. Telomeres in stem cells are long and become shorter during differentiation or with age, but lengthen again in tumour cells.

The Wnt/-catenin signalling pathway controls numerous processes in embryonic development, such as the formation of the body axis and of organ primordia, and is particularly active in embryonic and adult stem cells. The -catenin protein plays a key role in this signalling pathway. The incorrect regulation or mutation of -catenin leads to the development of tumours.

Rolf Kemler's research group has now shown that -catenin regulates the telomerase gene directly, and has explained the molecular mechanism at work here. Embryonic stem cells with mutated -catenin generate more telomerase and have extended telomeres, while cells without -catenin have low levels of telomerase and have shortened telomeres.

This regulation mechanism can also be found in human cancer cells. These discoveries could lead to the development of a new approach to the treatment of human tumours.

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The above story is reprinted from materials provided by Max-Planck-Gesellschaft.

Note: Materials may be edited for content and length. For further information, please contact the source cited above.

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Regulation of telomerase in stem cells and cancer cells

Public release date: 27-Jun-2012 [ | E-mail | Share ]

Contact: Clare Ryan clare.ryan@ucl.ac.uk 44-203-108-3846 University College London

Stem cells from patients with a rare form of muscular dystrophy have been successfully transplanted into mice affected by the same form of dystrophy, according to a new study published today in Science Translational Medicine.

For the first time, scientists have turned muscular dystrophy patients' fibroblast cells (common cells found in connective tissue) into stem cells and then differentiated them into muscle precursor cells. The muscle cells were then genetically modified and transplanted into mice.

The new technique, which was initially developed at the San Raffaele Scientific Institute of Milan and completed at UCL, could be used in the future for treating patients with limb-girdle muscular dystrophy (a rare form in which the shoulders and hips are primarily affected) and, possibly, other forms of muscular dystrophies.

Muscular dystrophies are genetic disorders primarily affecting skeletal muscle that result in greatly impaired mobility and, in severe cases, respiratory and cardiac dysfunction. There is no effective treatment, although several new approaches are entering clinical testing including cell therapy.

In this study, scientists focused on genetically modifying a type of cell called a mesoangioblast, which is derived from blood vessels and has been shown in previous studies to have potential in treating muscular dystrophy. However, the authors found that they could not get a sufficient number of mesoangioblasts from patients with limb-girdle muscular dystrophy because the muscles of the patients were depleted of these cells.

Instead, scientists in this study "reprogrammed" adult cells from patients with limb-girdle muscular dystrophy into stem cells and were able to induce them to differentiate into mesoangioblast-like cells. After these 'progenitor' cells were genetically corrected using a viral vector, they were injected into mice with muscular dystrophy, where they homed-in on damaged muscle fibres.

The researchers also showed that when the same muscle progenitor cells were derived from mice the transplanted cells strengthened damaged muscle and enabled the dystrophic mice to run for longer on a treadmill than dystrophic mice that did not receive the cells.

Dr Francesco Saverio Tedesco, UCL Cell & Developmental Biology, who led the study, said: "This is a major proof of concept study. We have shown that we can bypass the limited amount of patients' muscle stem cells using induced pluripotent stem cells and then produce unlimited numbers of genetically corrected progenitor cells.

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Successful transplant of patient-derived stem cells into mice with muscular dystrophy

A white earpiece connects to a cellphone atop a piece of Plexiglas surrounding her wheelchair. Her limp yet functional fingertips push the screen to accept a call one of many throughout the day from an ever-growing client base.

Novatos Abelina Magana, a mother of three teenagers, is an optimistic woman, a quality she says was essential in her survival, her recovery and her journey back to work in May.

Magana, 39, smiled as her caregiver wheeled her into her new home office, which is filled with paperwork, computers and a large whiteboard on the wall that lists potential clients. With seven escrows in the works, she focuses on four home listings, just weeks after restarting her career.

Five years ago Maganas husband gunned her down in an attempted murder-suicide, leaving her a quadriplegic. Shes amazed doctors by not only returning to her real estate career at Novato-based Frank Howard Allen Realtors but gaining mobility in her arms through controversial stem cell treatments.

She is so successful because she is a great agent, said Miguel Paredes, Maganas business partner. Its because people respect her and shes extremely intelligent.

Paredes assisted Magana on her first lease and has since eagerly committed to working with this woman who inspires him with her ambitious attitude.

She was rookie of the year and sold over 40 homes during her first year in 2002, Paredes said. Shes a fighter. Shes always had that reputation in the community.

Magana keeps motivated by biking eight miles a day with the help of an electric stimulator pad that forces her muscles to respond. Its part of a four-hour daily regimen of physical therapy. Shes also gained the use of both arms after undergoing two stem cell treatments in Panama.

Were at the beginning stages, but I know for a fact that it helps people, she said. It helped me.

The expensive treatments were made possible by local fundraising efforts. The first treatment cost $30,000, the second $21,000, and the final will cost $15,000. Travel and hotel expenses are not included.

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Eternal optimist