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Pluristem unveils portable stem cells thawing device

October 4th, 2012 2:28 pm

Pluristem Therapeutics Ltd. (Nasdaq:PSTI; DAX: PJT: PLTR) today announced that it has developed a portable instrument for thawing its placenta-based stems cell (PLX cells) on-site before they are administered to patients.

Pluristem will ship its off-the-shelf PLX cell therapy product candidates, which will be stored in multiple dose vials that require thawing prior to use. The vial will be placed into the proprietary thawing device and PLX cells will be ready for a convenient intramuscular injection. The company said that if the trials of the instrument are successful, it will be used as the final step in bringing high quality, clinical grade PLX cell products to patients around the globe.

"Pluristem understands the importance of providing a standardized product with every dose of these living drug delivery devices," said Pluristem chairman and CEO Zami Aberman. "If we are successful, we want our PLX cell products, once developed, to be an easy-to-use therapy. This thawing device will give us better control of several variables in our clinical trials and in treating patients after our products have been approved assuming we are successful. This instrument is an additional step in our vision to bring PLX cells as first-line therapies for a variety of indications and to think about the cell delivery process all the way from mass manufacturing to the patient's bedside."

Pluristem's share price rose 4.3% on the TASE today to NIS 16.05, after the announcement, after rising 4.4% on Nasdaq yesterday to $4.07, giving a market cap of $193 million.

Published by Globes [online], Israel business news - http://www.globes-online.com - on October 4, 2012

Copyright of Globes Publisher Itonut (1983) Ltd. 2012

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OncoMed Pharmaceuticals Advances Two Notch Pathway Product Candidates in Clinical Development

October 4th, 2012 2:28 pm

REDWOOD CITY, Calif.--(BUSINESS WIRE)--

OncoMed Pharmaceuticals, Inc., a clinical stage, research and discovery company developing novel therapeutics that target cancer stem cells, today announced clinical progress with two of its Notch pathway product candidates, resulting in $8 million in milestone payments from the companys strategic collaborator GlaxoSmithKline (GSK).

Anti-Notch2/3 (OMP-59R5)

OncoMed has initiated a Phase 1b/2 clinical trial in its anti-Notch2/3 antibody (OMP-59R5) program. In the Phase 1b/2 ALPINE trial (Antibody therapy in first-Line Pancreatic cancer Investigating anti-Notch Efficacy and safety), Anti-Notch2/3 is being tested in combination with gemcitabine in first-line advanced pancreatic cancer patients. Following a Phase 1b safety run-in, a randomized Phase 2 clinical trial will proceed in these patients to compare the efficacy of standard-of-care gemcitabine either with Anti-Notch2/3 or with placebo. The two primary endpoints of the Phase 2 part of the trial will be progression-free survival (PFS) in the Anti-Notch2/3 arm compared to a placebo arm in all patients, as well as in patients who have a particular biomarker. Key secondary and exploratory endpoints include overall survival, response rate, and safety, and these endpoints will be assessed in all patients, as well as in the biomarker positive subset of patients.

Dr. Lon Smith, from the South Texas Accelerated Research Therapeutics (START) Center for Cancer Care, who treated the first patient dosed in the ALPINE study, noted, This is an exciting new clinical trial with a novel anti-cancer treatment that we hope will have a big impact for patients with pancreatic cancer. The fact that the trial also includes a predictive biomarker to potentially identify patients who might gain greater clinical benefit from Anti-Notch2/3 is also a new and exciting direction in the experimental treatment of patients with pancreatic cancer.

Anti-Notch1 (OMP-52M51)

An Investigational New Drug (IND) application filed by OncoMed has been accepted by the FDA, thereby allowing OncoMed to advance its anti-Notch1 antibody (OMP-52M51) to clinical testing. OncoMed plans to initiate a single-agent, dose escalation and expansion Phase 1 clinical trial in hematologic cancers in 2012 and plans to file an additional IND application later in 2012 in solid tumors. The clinical trials will assess safety, pharmacokinetics, pharmacodynamics, and initial evidence of efficacy via a biomarker-based patient selection approach.

The OMP-59R5 Phase 1b/2 clinical program is our first Phase 2 trial and represents a significant advancement in the companys pipeline of anti-cancer stem cell therapies, said Paul Hastings, President and Chief Executive Officer of OncoMed Pharmaceuticals. In addition, the acceptance from the FDA to begin clinical testing for OMP-52M51 represents the fifth product candidate from our R&D platform to be cleared to enter clinical testing. For both programs, we have developed comprehensive biomarker strategies to facilitate efficient development in patients we believe will be most likely to benefit from targeted Notch pathway signaling blockade, added Hastings.

OMP-59R5 and OMP-52M51 are part of OncoMeds collaboration with GlaxoSmithKline. In December 2007, OncoMed and GSK entered into a broad strategic alliance to discover and develop novel product candidates targeting cancer stem cells via Notch pathway signaling modulation. GSK retains an option through the end of certain Phase 2 clinical trials to obtain an exclusive license to OMP-59R5. GSK also retains an option through the end of certain Phase 1 or certain Phase 2 clinical trials to obtain an exclusive license to OMP-52M51.

About Cancer Stem Cells

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StemCells, Inc. Announces First Transplant of Neural Stem Cells Into Patient in Clinical Trial for Dry Age-Related …

October 4th, 2012 2:28 pm

NEWARK, Calif., Oct. 4, 2012 (GLOBE NEWSWIRE) -- StemCells, Inc. (STEM) today announced that the first patient in its Phase I/II clinical trial in dry age-related macular degeneration (AMD) has been enrolled and transplanted. The trial is designed to evaluate the safety and preliminary efficacy of the Company's proprietary HuCNS-SC(R) product candidate (purified human neural stem cells) as a treatment for dry AMD, and the patient was transplanted with the cells yesterday at the Retina Foundation of the Southwest (RFSW) in Dallas, Texas, one of the leading independent vision research centers in the United States. AMD afflicts approximately 30 million people worldwide and is the leading cause of vision loss and blindness in people over 55 years of age.

"This trial signifies an exciting extension of our on-going clinical research with neural stem cells from disorders of the brain and spinal cord to now include the eye," said Stephen Huhn, MD, FACS, FAAP, Vice President and Head of the CNS Program at StemCells, Inc. "Studies in the relevant animal model demonstrate that the Company's neural stem cells preserve vision in animals that would otherwise go blind and support the therapeutic potential of the cells to halt retinal degeneration. Unlike others in the field, we are looking to intervene early in the course of the disease with the goal of preserving visual function before it is lost."

David G. Birch, Ph.D., Chief Scientific and Executive Officer of the RFSW and Director of the Rose-Silverthorne Retinal Degenerations Laboratory and principal investigator of the study, added, "We are excited to be working with StemCells on this ground breaking clinical trial. There currently are no effective treatments for dry AMD, which is the most common form of the disease, and there is a clear need to explore novel therapeutic approaches."

In February 2012, the Company published preclinical data that demonstrated HuCNS-SC cells protect host photoreceptors and preserve vision in the Royal College of Surgeons (RCS) rat, a well-established animal model of retinal disease which has been used extensively to evaluate potential cell therapies. Moreover, the number of cone photoreceptors, which are responsible for central vision, remained constant over an extended period, consistent with the sustained visual acuity and light sensitivity observed in the study. In humans, degeneration of the cone photoreceptors accounts for the unique pattern of vision loss in dry AMD. The data was published in the international peer-reviewed European Journal of Neuroscience.

About Age-Related Macular Degeneration

Age-related macular degeneration refers to a loss of photoreceptors (rods and cones) from the macula, the central part of the retina. AMD is a degenerative retinal disease that typically strikes adults in their 50s or early 60s, and progresses painlessly, gradually destroying central vision. According to the RFSW website, there are approximately 1.75 million Americans age 40 years and older with some form of age-related macular degeneration, and the disease continues to be the number one cause of irreversible vision loss among senior citizens in the United States with more than seven million at risk of developing AMD.

About the Trial

The Phase I/II trial will evaluate the safety and preliminary efficacy of HuCNS-SC cells as a treatment for dry AMD. The trial will be an open-label, dose-escalation study, and is expected to enroll a total of 16 patients. The HuCNS-SC cells will be administered by a single injection into the space beneath the retina in the most affected eye. Patients' vision will be evaluated using both conventional and advanced state-of-the-art methods of ophthalmological assessment. Evaluations will be performed at predetermined intervals over a one-year period to assess safety and signs of visual benefit. Patients will then be followed for an additional four years in a separate observational study. Patients interested in participating in the clinical trial should contact the site at (214) 363-3911.

About HuCNS-SC Cells

StemCells' proprietary product candidate, HuCNS-SC cells, is a highly purified composition of human neural stem cells that are expanded and stored as banks of cells. The Company's preclinical research has shown that HuCNS-SC cells can be directly transplanted in the central nervous system (CNS) with no sign of tumor formation or adverse effects. Because the transplanted HuCNS-SC cells have been shown to engraft and survive long-term, there is the possibility of a durable clinical effect following a single transplantation. StemCells believes that HuCNS-SC cells may have broad therapeutic application for many diseases and disorders of the CNS, and to date has demonstrated human safety data from completed and ongoing clinical studies.

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Bernard Siegel to Deliver Keynote Addresses at Midwest Conference on Stem Cell Biology and Therapy and BioFlorida …

October 4th, 2012 2:23 pm

PALM BEACH, Fla.--(BUSINESS WIRE)--

Genetics Policy Institute (GPI) announced today that Bernard Siegel, Executive Director of GPI, will make two keynote presentations this month at regional conferences: Midwest Conference on Stem Cell Biology and Therapy October 5-7 in Rochester, Michigan and BioFlorida Conference 2012 October 7-9 in Miami, Florida.

Siegel will present a keynote address titled The Power of Advocacy at the Midwest Conference on Stem Cell Biology and Therapy. The Genetics Policy Institute joined with the Oakland University William Beaumont Institute for Stem Cell and Regenerative Medicine (ISCRM) as a collaborating partner for the event. Researchers from hospitals, medical organizations, academic institutions and the business community throughout the Midwest will discuss not only the latest advances in this rapidly expanding field of medical science, but the ethical and moral issues that surround it.

"I am pleased to participate in these important conferences, which showcase the latest scientific developments in their respective regions and beyond. ISCRM and the World Stem Cell Summit have a strong connection, as the Institute was officially launched at our 2010 Summit in Detroit, said Bernard Siegel, GPI's Executive Director and founder of the annual World Stem Cell Summit.

BioFloridas 15th annual Conference is the premier event for Floridas bioscience community. This years meeting will bring together more than 500 professionals from across Florida, the Southeast and the nation to discuss major trends and issues, including topics related to product development, scientific research, business development, financing and public policy.

Siegels keynote address at BioFlorida is titled: The Mandate to Deliver Cures: Aligning Patient Advocacy, Industry and Science. Former Governor Jeb Bush will deliver the second keynote at BioFloridas annual Conference.

The 2012 World Stem Cell Summit is in West Palm Beach, Florida this December, so we have been working closely with the biotechnology community here. I am delighted to partner with BioFlorida as they advance Floridas bioscience industry," said Siegel, who also serves on the Executive Committee of the Alliance for Regenerative Medicine and Board of the Coalition for Advancement of Medical Research. He serves as spokesperson for the Stem Cell Action Coalition.

ABOUT GPI:The Genetics Policy Institute (GPI) supports stem cell research to develop therapeutics and cures. GPI pursues its mission by honoring leadership through the Stem Cell Action Awards, producing the World Stem Cell Summit, publishing theWorld Stem Cell Report, organizing educational initiatives and fostering strategic collaborations. For more information, visitwww.genpol.org.

ABOUT THE WORLD STEM CELL SUMMIT:The 2012 World Stem Cell Summit is presented by GPI and is co-organized by the Interdisciplinary Stem Cell Institute (ISCI) at the University of Miami Miller School of Medicine, Diabetes Research Institute, Beckman Research Institute at City of Hope, Karolinska Institute (home of the Nobel Prize in Physiology and Medicine), International Translational Regenerative Medicine Center (ITRC) and the Institute for Integrated Cell-Material Sciences (iCeMS) at Kyoto University. The Summit is the flagship meeting of the world stem cell community. The 2012 Summit will be held at the Palm Beach County Convention Center in West Palm Beach, Florida, December 3-5, 2012. For more information, visit http://www.worldstemcellsummit.com.

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ORF Genetics to Offer endotoxin- and Animal-free FGFb and mLIF for Stem Cell Research

October 4th, 2012 2:21 pm

REYKJAVIK, Iceland, October 4, 2012 /PRNewswire/ --

ORF Genetics announced today that the company has added endotoxin- and animal-free human Fibroblast Growth Factor Basic (FGF basic) and mouse Leukemia Inhibitory Factor (mouse LIF) to its portfolio of growth factors for stem cell research.

Most growth factors applied in stem cell research today are made in E. coli bacteria, which produce endotoxins that can have adverse effect on stem cell cultures. Other manufacturers of growth factors have various methods to remove these endotoxins, but traces inevitably remain, which can lead to increased death rate of cells and other suboptimal effects in cell cultures. Other growth factors on the market today are made by animal cells. However, most stem cell researchers prefer to use growth factors of non-animal origin to exclude risks of viral contamination and the inclusion of growth factor homologs.

This has led to a market demand for alternative sources of animal-free growth factors, void of endotoxins. ORF Genetics' unique growth factors are produced in the seeds of the barley plant, which does not produce any endotoxins or other substances toxic to mammalian cells.

FGF basic and mouse LIF are key growth factors for the cultivation of their respective stem cells, i.e. FGF basic for human stem cells and mouse LIF for mouse stem cells. Each protein is used to expand the stem cells' populations before researchers make them differentiate into various cell types, such as heart, liver or neural cells.

"ORF Genetics has built a reputation for offering the first plant-made, endotoxin-free and animal-free growth factor portfolio for stem cell researchers. As we are producing these growth factors in our novel plant expression system ORFEUS, we are very happy to be able to offer these high quality growth factors at more efficient prices than market leaders," said Bjrn rvar, CEO of ORF Genetics.

ORF Genetics is a world leader of plant made growth factors and offers a portfolio of endotoxin- and animal-free growth factors for human stem cell research. The company's production takes place in a biorisk-free production system in barley, bypassing conventional bacteria and animal cell production systems. The cultivation of barley takes place in greenhouses in inert volcanic pumice, using renewable geothermal energy.

For more information please contact:

Dr. Hakon Birgisson, Director of Global Market Development Tel: +354-821-1585 email:hakon.birgisson@orfgenetics.com

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'Humanized' mice advance study of rheumatoid arthritis

October 4th, 2012 2:20 pm

Public release date: 3-Oct-2012 [ | E-mail | Share ]

Contact: Marla Paul Marla-Paul@northwestern.edu 312-503-8928 Northwestern University

Researchers at Northwestern University Feinberg School of Medicine have developed the first animal model that duplicates the human response in rheumatoid arthritis (RA), an important step that may enable scientists to discover better medicines to treat the disease.

Corresponding and senior author Harris Perlman, associate professor of rheumatology at Feinberg, introduced his team's new prototype mouse model in a recent online issue of the Journal of Translational Medicine.

"This is the first time human stem cells have been transplanted into mice in order to find RA treatments," Perlman said. "We believe this will improve drug discovery because the reactions we observed were authentic human reactions."

Until now, scientists have relied on the common scientific method of using specially bred mice to find drugs to control RA. However, human and mouse immune systems differ dramatically, so studying RA in these mice does not give an accurate representation of how the disease functions in humans. In some cases, RA drugs that seemed promising based on results in mice failed in human clinical trials.

Mice implanted with human stem cells have been used before mainly to study infectious disease.

The Northwestern team injected day-old mice with human stem cells from umbilical cord blood, including white blood cells, which regulate immunity. Then, RA was introduced in the mice and suppressed with Enbrel, a common first-line drug for joint inflammation in humans. This offered evidence that their immune systems were indeed replicating human defenses.

Scientists seek mouse models that mimic RA in humans in order to learn how the complex disease operates. In the last decade, researchers and physicians have found many subtypes of RA that originate on the molecular level and are each produced by different pathways in the body.

A debilitating disease, rheumatoid arthritis is a chronic autoimmune disorder characterized by persistent inflammation around joint areas, predominantly in the wrist and fingers. The disease causes pain, swelling, stiffness and loss of function and can result in tissue destruction. Approximately 1.3 million people have the disease.

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UCI stem cell center awes students

October 4th, 2012 12:30 am

IRVINE High school sophomore Derek Le said he's always been interested in science. But it wasn't until he saw how researchers could use stem cells to repair tissue damaged by heart attacks that Le thought science could be his future career.

"Wow, this is so amazing," said Le, a student from Westminster High. "They can create cells to fix people's hearts. I think I want to do something like this."

Emily Nordhoff, Junior Research Specialist is harnessed in a body weight support system that is used to measure walking patterns. More than 250 high school students took a tour of UC Irvine's Stem Cell program to learn about stem cell research.

CHRISTINE COTTER, FOR THE REGISTER

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The center was established at UC Irvine in 2010 with help of a $10 million endowment from PIMCO founder and his wife.

Mission

The center's goals include training new researchers, providing state-of-the-art tissue culture labs and equipment, and testing new technologies and products related to stem cells research.

Accomplishments

Recent accomplishments include: securing first federally approved embryonic stem cell based therapy to go to clinical trial; developing of bone marrow stem cell treatment for stroke patients; and development of treatments for retinitis pigmentosa and macular degeneration.

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Verastem to Present at BIO Investor Forum and Participate in Cancer Stem Cell Panel

October 4th, 2012 12:30 am

CAMBRIDGE, Mass.--(BUSINESS WIRE)--

Verastem, Inc., (VSTM) a clinical-stage biopharmaceutical company focused on discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells, announced that members of the management team will present at the BIO Investor Forum on October 9, 2012, at the Palace Hotel in San Francisco, CA.

The company presentation is at 8:30 am PT in the Presidio room and Jonathan Pachter, Ph.D., Verastem Vice President and Head of Research, will participate on a cancer stem cell panel from 10:30-11:25 am PT in the Twin Peaks room.

The panel will bring together scientific experts who are at the forefront of developing novel medicines targeting cancer stem cells. Dr. Pachter will discuss the potential of this approach to create durable clinical responses for patients with cancers that are typically refractory to conventional treatments. Verastem is translating basic science into clinical products based on the seminal publications in Cell and PNAS by Robert Weinberg, Ph.D., Verastem co-founder and chair of the Scientific Advisory Board.

A webcast of the company presentation can be accessed by visiting the investors section of the Companys website at http://www.verastem.com. A replay of the webcast will be archived on the Verastem website for two weeks following the presentation date.

About Verastem, Inc.

Verastem, Inc. (VSTM) is a clinical-stage biopharmaceutical company focused on discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells. Cancer stem cells are an underlying cause of tumor recurrence and metastasis. Verastem is developing small molecule inhibitors of signaling pathways that are critical to cancer stem cell survival and proliferation: focal adhesion kinase (FAK), PI3K/mTOR and Wnt. For more information, please visit http://www.verastem.com.

Forward-looking statements:

Any statements in this press release about future expectations, plans and prospects for the Company constitute forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements. The Company anticipates that subsequent events and developments will cause the Companys views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

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Study Shows Epigenetics Of Adult Stem Cells Influences Organ Creation

October 4th, 2012 12:24 am

October 3, 2012

Alan McStravick for redOrbit.com Your Universe Online

Ask a handful of people about their thoughts and feelings on the use of stem cells for research and therapeutic means and you will find that they each have strong and varying positions on the topic. Outside the scientific community, however, little is known about this highly complex field of research.

The politicization of stem cell research accompanied the 1998 discovery that embryonic stem cells, the building blocks of organ, tissue, bone and brain cells, could be extracted for study and medical use. In 2001, with an order to limit the lines of stem cell research to those already in possession of the scientific community, President George W. Bush largely hampered the development of this field in the United States by limiting government funding for stem cell research. Adult stem cells, or somatic stem cells, were unaffected by this order, but the prevailing wisdom of the genetic community was that adult stem cells were not as dynamic and couldnt be used in the same way as their embryonic cousins.

With a report published Monday in the American Journal of Pathology, that truth no longer seems to be the case. A team led by Manel Esteller, director of the Cancer Epigenetics and Biology Program in the Bellvitge Biomedical Research Institute (IDIBELL), was able to identify epigenetic changes that occur in the somatic stem cells to generate different body tissues.

The use of somatic or adult stem cells had been a regular occurrence since their discovery in the 1950s. It was then that researchers found that bone marrow contains two different kinds of stem cells. The first, called hematopoietic stem cells, form all the types of blood cells in the body. The second, known as bone marrow stromal stem cells, were discovered only a few years later and are effective in the generation of bone, cartilage, fat and fibrous connective tissues.

One thing that has been understood is that the genome of each cell in the human body is identical. This is true regardless of their appearance and function. It is for this reason that certain anomalies, such as cancer, are seemingly incomprehensible as they are unable to be explained by the genome of the host. To better understand such complex genetic deviations, something more is required.

Researchers in this current study offer an explanation via analogy. Epigenetics is defined as the inheritance of DNA activity that does not depend on the strict sequence of it. According to the team, if genetics is the alphabet, spelling would be the epigenetics, referring to chemical changes in our genetic material as well as the proteins that regulate and control their activity.

We now know that somatic stem cells have enormous potential to regenerate damaged organs. By investigating how to use them more effectively in different types of therapies, the research team postulates that it will become easier to steer clear of any sticky ethical complications that might arise from working with embryonic stem cells.

In this study, the team was able to isolate somatic stem cells from body fat, allowing them to transform them into muscle and bone cells. Through their study, they observed the resemblance of the cells created in the laboratory to those of the host individual. They were also able to determine that the cells were biologically secure enough that they might be implanted into waiting patients. Overall, the study was able to show that the epigenome of the cells obtained and maintained in culture closely resembled skeletal and muscle cells that are spontaneously present in nature, though not completely identical.

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New stem cell research could bring choices to heart patients

October 4th, 2012 12:23 am

Contributed photo

Dr. Nabil Dib

They're called "no-option patients."

They've endured angioplasty, stent procedures, bypasses and a long line of medications. None of the treatments has fixed the plaque-plugged coronary arteries that trigger angina, starve the heart of blood and force people to hunch in pain after walking twoblocks.

Adult stem cell research at an Oxnard hospital is aimed at giving themchoices.

"A patient who has no hope will have some hope," said Dr. Nabil Dib, a world-renowned researcher partnering with St. John's Regional Medical Center. "It's a hope for potential therapy that will revise the way we treat cardiovasculardisease."

Stem cells are blank cells that function as the body's building blocks. They are able to grow into many different kinds of cells, including blood, muscle and tissue. Dib's work involves adult stem cells harvested from his patients, as opposed to stem cells that come from embryos and trigger ethicaldebates.

In a clinical trial starting at St. John's and 49 other hospitals across the country, the adult stem cells will be isolated and used to create new blood vessels. It's a way of manipulating the body into building new pathways for blood flow impeded by barricadedarteries.

"We're doing like a bypass a biological bypass," Dibsaid.

The trial is part of a genre of research aimed at using the body's own resources to repair the heart. It could reduce consequences ranging from heart transplants and hospitalizations to heart failure anddeath.

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Study Sheds Light on Bone Marrow Stem Cell Therapy for Pancreatic Recovery

October 4th, 2012 12:21 am

Newswise LOS ANGELES (Oct. 2, 2012) Researchers at Cedars-Sinais Maxine Dunitz Neurosurgical Institute have found that a blood vessel-building gene boosts the ability of human bone marrow stem cells to sustain pancreatic recovery in a laboratory mouse model of insulin-dependent diabetes.

The findings, published in a PLoS ONE article of the Public Library of Science, offer new insights on mechanisms involved in regeneration of insulin-producing cells and provide new evidence that a diabetics own bone marrow one day may be a source of treatment.

Scientists began studying bone marrow-derived stem cells for pancreatic regeneration a decade ago. Recent studies involving several pancreas-related genes and delivery methods transplantation into the organ or injection into the blood have shown that bone marrow stem cell therapy could reverse or improve diabetes in some laboratory mice. But little has been known about how stem cells affect beta cells pancreas cells that produce insulin or how scientists could promote sustained beta cell renewal and insulin production.

When the Cedars-Sinai researchers modified bone marrow stem cells to express a certain gene (vascular endothelial growth factor, or VEGF), pancreatic recovery was sustained as mouse pancreases were able to generate new beta cells. The VEGF-modified stem cells promoted growth of needed blood vessels and supported activation of genes involved in insulin production. Bone marrow stem cells modified with a different gene, PDX1, which is important in the development and maintenance of beta cells, resulted in temporary but not sustained beta cell recovery.

Our study is the first to show that VEGF contributes to revascularization and recovery after pancreatic injury. It demonstrates the possible clinical benefits of using bone marrow-derived stem cells, modified to express that gene, for the treatment of insulin-dependent diabetes, said John S. Yu, MD, professor and vice chair of the Department of Neurosurgery at Cedars-Sinai, senior author of the journal article.

Diabetes was reversed in five of nine mice treated with the injection of VEGF-modified cells, and near-normal blood sugar levels were maintained through the remainder of the six-week study period. The other four mice survived and gained weight, suggesting treatment was beneficial even when it did not prompt complete reversal. Lab studies later confirmed that genetically-modified cells survived and grew in the pancreas and supported the repopulation of blood vessels and beta cells.

Anna Milanesi, MD, PhD, working in Yus lab as an endocrinology fellow, is the articles first author. The researchers cautioned that although this and other related studies help scientists gain a better understanding of the processes and pathways involved in pancreatic regeneration, more research is needed before human clinical trials can begin.

Insulin-dependent diabetes occurs when beta cells of the pancreas fail to produce insulin, a hormone that regulates sugar in the blood. Patients must take insulin injections or consider transplantation of a whole pancreas or parts of the pancreas that make insulin, but transplantation carries the risk of cell rejection.

# # #

PLoS ONE: Beta-cell Regeneration Mediated by Human Bone Marrow Mesenchymal Stem Cells.

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Sigma® Life Science Launches Novel, Affordable Pluripotent Stem Cell Culture Medium

October 3rd, 2012 12:24 pm

ST. LOUIS, Oct. 3, 2012 /PRNewswire/ --Sigma-Aldrich Corporation (SIAL) announced today that Sigma Life Science, its innovative biological products and services research business, has launched Stemline Pluripotent Culture Medium, a novel human pluripotent stem cell culture medium that provides a consistent environment for the long-term maintenance and growth of healthy pluripotent stem cells. The new medium performs equivalently to the industry's leading medium and provides academic and pharmaceutical stem cell research labs with a substantially lower cost alternative to higher priced media. Additional information and sample requests of the Stemline Pluripotent Culture Medium are available at http://www.sigma.com/stemlinepsc.

"The exorbitant cost of media for pluripotent stem cells is a universal complaint from the stem cell research community. Our Stemline Pluripotent Culture Medium performs equivalently to the leading medium for maintaining pluripotency and optimal growth rates, and is produced more efficiently than traditional media, resulting in lower costs. For example, a typical academic lab that consumes three 500 mL bottles of media per week could save at least $12,000 annually using our new Stemline medium. A high-throughput pharmaceutical development team that consumes 20 liters of media weekly could save more than $160,000 annually," said John Listello, Market Segment Manager for Regenerative Medicine at Sigma Life Science.

Culturing pluripotent stem cells can be challenging as many media's undefined, heterogenous mixtures can cause inconsistent growth rates and undesired spontaneous differentiation. The Stemline Pluripotent Stem Cell Culture Medium is serum-free, composed of fully-defined components and has 80% less basic fibroblast growth factor than the leading pluripotent stem cell culture medium. This provides a consistent environment for long-term maintenance of optimal growth rates, viability and pluripotency. Rigorous characterization of the Stemline Pluripotent Stem Cell Culture Medium has demonstrated that cultured pluripotent stem cells display all established pluripotency markers and maintain proper karyotype and the ability to differentiate into each of the three germ layers. The feeder-independent medium also enables culturing with synthetic matricies, thereby eliminating a source of variability that would prohibit later clinical applications.

"Academic and pharmaceutical groups performing toxicology screens, disease-specific stem cell research or studies of the basic mechanisms behind pluripotency and differentiation depend upon a steady supply of consistent, high-performance cell culture medium. This novel Stemline medium extends Sigma's existing position as one of the largest global providers of cell culture media," said Listello.

Existing Stemline stem cell culture media include specialized formulations for expansion of six human adult stem cell and progenitor cell types: hematopoietic, neural, dendritic, mesenchymal, T-cells, and keratinocytes. These six Stemline media are produced under good manufacturing practices (GMP) and have Device Master File certificates from the U.S. Food and Drug Administration.

Sigma Life Science's comprehensive stem cell product portfolio includes custom iPS cell CompoZr ZFN-mediated genetic engineering, Stemgent Reprogramming Lentiviruses, the MISSION shRNA Library with the latest content release from The RNAi Consortium, 3D matrices, growth factors, small molecules, other cell culture media and the industry's most validated antibodies. Sigma Life Science acquired a worldwide license to Kyoto University's iPS cell patent portfolio in February, 2012.

For more information and to request pricing, visit http://www.sigma.com/stemlinepsc.

Cautionary Statement: The foregoing release contains forward-looking statements that can be identified by terminology such as "could," "could expect," "can be," "predictive" or similar expressions, or by expressed or implied discussions regarding potential future revenues from products derived there from. You should not place undue reliance on these statements. Such forward-looking statements reflect the current views of management regarding future events, and involve known and unknown risks, uncertainties and other factors that may cause actual results to be materially different from any future results, performance or achievements expressed or implied by such statements. There can be no guarantee that pluripotent stem cells, pluripotent stem cell media, or related custom services will assist the Company to achieve any particular levels of revenue in the future. In particular, management's expectations regarding products associated with pluripotent stem cells, pluripotent stem cell media, or related custom services could be affected by, among other things, unexpected regulatory actions or delays or government regulation generally; the Company's ability to obtain or maintain patent or other proprietary intellectual property protection; competition in general; government, industry and general public pricing pressures; the impact that the foregoing factors could have on the values attributed to the Company's assets and liabilities as recorded in its consolidated balance sheet, and other risks and factors referred to in Sigma-Aldrich's current Form 10-K on file with the US Securities and Exchange Commission. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those anticipated, believed, estimated or expected. Sigma-Aldrich is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

About Sigma Life Science: Sigma Life Science is a Sigma-Aldrich business that represents the Company's leadership in innovative biological products and services for the global life science market and offers an array of biologically-rich products and reagents that researchers use in scientific investigation. Product areas include biomolecules, genomics and functional genomics, cells and cell-based assays, transgenics, protein assays, stem cell research, epigenetics and custom services/oligonucleotides. Sigma Life Science also provides an extensive range critical bioessentials like biochemicals, antibiotics, buffers, carbohydrates, enzymes, forensic tools, hematology and histology, nucleotides, amino acids and their derivatives, and cell culture media.

About Sigma-Aldrich: Sigma-Aldrich is a leading Life Science and High Technology company whose biochemical, organic chemical products, kits and services are used in scientific research, including genomic and proteomic research, biotechnology, pharmaceutical development, the diagnosis of disease and as key components in pharmaceutical, diagnostics and high technology manufacturing. Sigma-Aldrich customers include more than 1.3 million scientists and technologists in life science companies, university and government institutions, hospitals and industry. The Company operates in 38 countries and has nearly 9,100 employees whose objective is to provide excellent service worldwide. Sigma-Aldrich is committed to accelerating customer success through innovation and leadership in Life Science and High Technology. For more information about Sigma-Aldrich, please visit its website at http://www.sigma-aldrich.com.

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Sigma® Life Science Launches Novel, Affordable Pluripotent Stem Cell Culture Medium

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Patients' own skin cells could restore vision in elderly with macular degeneration

October 3rd, 2012 10:16 am

Washington, October 2 (ANI): A new study has suggested that induced pluripotent stem (iPS) cells - which are derived from adult human skin cells but have embryonic properties - could soon be used to restore vision in people with macular degeneration and other diseases that affect the eye's retina.

In the study conducted by Columbia ophthalmologists and stem cell researchers, adult stem cells developed from a patient's skin cells improved the vision of blind mice.

"With eye diseases, I think we're getting close to a scenario where a patient's own skin cells are used to replace retina cells destroyed by disease or degeneration," said the study's principal investigator, Stephen Tsang, MD, PhD, associate professor of ophthalmology and pathology and cell biology.

"It's often said that iPS transplantation will be important in the practice of medicine in some distant future, but our paper suggests the future is almost here," he stated.

The advent of human iPS cells in 2007 was greeted with excitement from scientists who hailed the development as a way to avoid the ethical complications of embryonic stem cells and create patient-specific stem cells.

Like embryonic stem cells, iPS cells can develop into any type of cell. Thousands of different iPS cell lines from patients and healthy donors have been created in the last few years, but they are almost always used in research or drug screening.

In Tsang's new preclinical iPS study, human iPS cells - derived from the skin cells of a 53-year-old donor - were first transformed with a cocktail of growth factors into cells in the retina that lie underneath the eye's light-sensing cells.

The primary job of the retina cells is to nourish the light-sensing cells and protect the fragile cells from excess light, heat, and cellular debris. If the retina cells die - which happens in macular degeneration and retinitis pigmentosa - the photoreceptor cells degenerate and the patient loses vision.

Macular degeneration is a leading cause of vision loss in the elderly, and it is estimated that 30 percent of people will have some form of macular degeneration by age 75.

In their study, the researchers injected the iPS-derived retina cells into the right eyes of 34 mice that had a genetic mutation that caused their retina cells to degenerate.

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Patients' own skin cells could restore vision in elderly with macular degeneration

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Histogenics Honored as a 2012 “Fierce 15” Company by FierceMedicalDevices

October 3rd, 2012 10:16 am

WALTHAM, Mass.--(BUSINESS WIRE)--

Histogenics, a regenerative medicine company combining cell therapy and tissue engineering technologies to develop highly innovative products for tissue repair and regeneration, announced today that it has been named to the FierceMedicalDevices Fierce 15 list, designating it as one of the leading medical device and diagnostic companies of 2012. FierceMedicalDevicesEditors Mark Hollmer and Damian Garde, in conjunction with Editor-in-Chief John Carroll and Executive Editor Ryan McBride, chose this years winners based on their top management teams, notable financial backing, and promising technologies and market opportunities.

We have worked hard over the past year, securing $49 million in financing and adding key new staff, investors and board members, so that we are now in the position to focus our full attention on continued successful clinical and regulatory execution for NeoCart cartilage regeneration implant, which is currently enrolling patients into the Phase 3 IND clinical study, and the EU regulatory development of our VeriCart cartilage repair scaffold, said Patrick ODonnell, President and Chief Executive Officer of Histogenics. We believe our product candidates have the potential to transform the treatment of cartilage injury with the goal of returning some of the estimated 1.8 million patients each year in the U.S. and E.U. that undergo arthroscopy for knee cartilage defects to their pre-injury level of activity.

Nailing down $49 million in financing in July reinforces the notion that this regenerative medicine company stands out for doing things differently.One example how: The company is well underway enrolling patients in a Phase 3 trial for NeoCart, a cartilage implant that uses a patients own cells to build it before treating cartilage lesions in the knee, said Hollmer.

NeoCart is an autologous neocartilage tissue implant in an ongoing Phase 3 clinical program that utilizes the patients own cells to regenerate cartilage in patients suffering from cartilage lesions in the knee.VeriCart, is a single-step, cell-free collagen scaffold uniquely designed to be used in conjunction with the patients own stem cells to repair small cartilage defects frequently observed in meniscal and anterior cruciate ligament repair procedures. Histogenics is seeking regulatory clearance in the European Union for VeriCart.

An internationally recognized e-newsletter reaching more than 34,000 medical device and diagnostic industry professionals, FierceMedicalDevices provides subscribers with a quick authoritative briefing on the days top stories, with a special focus on clinical studies, FDA/EMEA regulations and post-marketing. Sign up is free at http://www.fiercemedicaldevices.com/signup.

About FierceMarkets

FierceMarkets, a wholly owned subsidiary of Questex Media Group, is a leader in B2B emedia, providing information and marketing services in the telecommunications, life sciences, healthcare, IT, energy, government and finance industries through its portfolio of email newsletters, websites, webinars and live events. Every business day, FierceMarkets wide array of publications reaches more than 1.3 million executives in more than 100 countries.

About Histogenics

Histogenics is a leading regenerative medicine company that combines cell therapy and tissue engineering technologies to develop highly innovative products for tissue repair and regeneration. In May of 2011, Histogenics acquired Israeli cell-therapy company ProChon BioTech. Histogenics flagship products focus on the treatment of active patients suffering from articular cartilage derived pain and immobility. The Company takes an interdisciplinary approach to engineering neocartilage that looks, acts and lasts like hyaline cartilage. It is developing new treatments for sports injuries and other orthopedic conditions, where demand is growing for long-term alternatives to joint replacement. Histogenics has successfully completed Phase 1 and Phase 2 clinical trials in which the NeoCart autologous tissue implants effectiveness is compared to that of standard microfracture surgery. Based in Waltham, Massachusetts, the company is privately held. For more information, visitwww.histogenics.com.

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Histogenics Honored as a 2012 “Fierce 15” Company by FierceMedicalDevices

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Nuvilex Subsidiary Austrianova Singapore to Participate in AusBiotech 2012

October 3rd, 2012 10:13 am

SILVER SPRING, Md. and SINGAPORE, Oct. 2, 2012 (GLOBE NEWSWIRE) -- Nuvilex, Inc. (NVLX), an international biotechnology provider of cell and gene therapy solutions, announced today its wholly-owned subsidiary, Austrianova Singapore Pte Ltd (ASPL) will attend this year's AusBiotech event.

The annual AusBiotech event this year will be held from October 30 - November 2 at the Melbourne Convention and Exhibition Centre, Melbourne, Australia. It has earned a reputation as the industry's premier biotechnology conference for the Asia Pacific region and has successfully expanded its relevance to the Australian and International Biotechnology industries by attracting more than 1100 participants from over 20 countries.

Dr Brian Salmons, CEO of ASPL said, "AusBiotech has grown in stature over the past several years. In prior years, we entered agreements with companies and found it to be one of the most valuable events for networking with new contacts. We anticipate meeting with companies with proprietary therapeutic cells, such as stem cells, that can leverage their technology with our Cell-in-a-Box(R) delivery system. We believe the new contacts we make will expand our customer base and increase the use of cell and gene therapy for making therapeutic products and treating diseases. We will also be promoting our Bac-in-a-Box(R) technology for the first time at this meeting and anticipate generating interest around its potential."

The Chief Executive of Nuvilex, Dr. Robert Ryan, stated "Attendance at this important biotech event in Australia and within easy reach of Southeast Asia will enable us to have increased exposure for our Cell-in-a-Box(R) and Bac-in-a-Box(R) live cell encapsulation technology and to showcase its immense versatility, thus providing our companies greater visibility at a time that such capabilities are becoming more important in the marketplace. It is our goal to bring more projects to fruition from this meeting as more companies today are looking to bring cellular-based therapy and product creation from the drawing board to reality and into regular use."

About Nuvilex

Nuvilex, Inc. (NVLX) is an international biotechnology provider of live therapeutically valuable, encapsulated cells and services for research and medicine. A great deal of work is ongoing to move Nuvilex and its Austrianova Singapore subsidiary forward. This was clearly apparent during Dr. Ryan's trip to Singapore and the advent of new developments in the company as a whole. Our company's own offerings will include cancer, diabetes, other treatments and capabilities using the company's cell and gene therapy expertise and live-cell encapsulation technology.

The Nuvilex, Inc. logo is available at http://www.globenewswire.com/newsroom/prs/?pkgid=13494

Safe Harbor Statement

This press release contains forward-looking statements described within the 1995 Private Securities Litigation Reform Act involving risks and uncertainties including product demand, market competition, and meeting current or future plans which may cause actual results, events, and performances, expressed or implied, to vary and/or differ from those contemplated or predicted. Investors should study and understand all risks before making an investment decision. Readers are recommended not to place undue reliance on forward-looking statements or information. Nuvilex is not obliged to publicly release revisions to any forward-looking statement, reflect events or circumstances afterward, or disclose unanticipated occurrences, except as required under applicable laws.

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Nuvilex Subsidiary Austrianova Singapore to Participate in AusBiotech 2012

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Adult stem cells change their epigenome to generate new organs

October 3rd, 2012 10:12 am

ScienceDaily (Oct. 2, 2012) The team led by Manel Esteller, director of the Cancer Epigenetics and Biology Program in the Bellvitge Biomedical Research Institute (IDIBELL), Professor of Genetics at the University of Barcelona and ICREA researcher, has identified epigenetic changes that occur in adult stem cells to generate different body tissues.

The finding is published this week in The American Journal of Pathology.

The genome of every single cell in the human body is the same, regardless of their appearance and function. Therefore the activity of the tissues and organs and its disorders in complex diseases, such as cancer, cannot be fully explained by the genome. It is necessary something more, and part of the explanation is provided by epigenetics, which is defined as "the inheritance of DNA activity that does not depend on strict sequence of it." That is, if genetics is the alphabet, spelling would be the epigenetics, which refers to chemical changes in our genetic material and their regulatory proteins. The most known epigenetic mark is the addition of a methyl group to DNA. Thus, the epigenome is getting all the epigenetic marks of a living being.

Adult stem cells have an enormous potential to regenerate damaged organs and their use also avoids ethical complications involving embryonic stem cells, as well as technical problems arising from induced stem cells. In this study, researchers have isolated stem cells from body fat and transformed them into muscle and bone cells. Then, it was necessary to know how much resembled are the cells created in the laboratory with those present in one individual and if they were biologically secured enough to be implanted in patients. The study shows that the epigenome of the cells obtained in culture closely resembles that of skeletal muscle cells and they are spontaneously present in nature, although not completely identical.

A key point of the study is that muscle and bone cells produced in the laboratory do not have the tumour epigenome derived from these tumour tissues (rhabdomyosarcoma and osteosarcoma, respectively) so they are safe from a biological perspective. The study coordinator, Manel Esteller, stresses that the research "demonstrates the usefulness of epigenetics in determining the degree of maturity and biosecurity of differentiated tissues used in regenerative medicine against different diseases."

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The above story is reprinted from materials provided by IDIBELL-Bellvitge Biomedical Research Institute.

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

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Adult stem cells change their epigenome to generate new organs

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Blind Mice Get Experimental Stem Cell Treatment For Blindness

October 3rd, 2012 10:11 am

April Flowers for redOrbit.com Your Universe Online

Columbia University ophthalmologists and stem cell researchers have developed an experimental treatment for blindness using the patients skin cells, which has improved the vision of blind mice in testing.

The findings of this research, published online in the journal Molecular Medicine, suggest that induced pluripotent stem cells (iPS) could soon be used to improve vision in people with macular degeneration and other eye retina diseases. iPS cells are derived from adult human skin cells but have embryonic qualities.

With eye diseases, I think were getting close to a scenario where a patients own skin cells are used to replace retina cells destroyed by disease or degeneration, says Stephen Tsang, MD, PhD, associate professor of ophthalmology and pathology & cell biology. Its often said that iPS transplantation will be important in the practice of medicine in some distant future, but our paper suggests the future is almost here.

Scientists were very excited by the advent of human iPS cells when they were discovered in 2007, as they provide a way to avoid the ethical complications of embryonic stem cells. Another advantage is that the iPS cells are created from the patients own skin, eliminating the need for anti-rejection medications. Like the ethically challenged embryonic cells, iPS cells can develop into any type of cell. To-date, no iPS cells have been implanted into people, but many ophthalmologists say that the eye would prove to be ideal testing ground for iPS therapies.

The eye is a transparent and accessible part of the central nervous system, and thats a big advantage. We can put cells into the eye and monitor them every day with routine non-invasive clinical exams, Tsang said. And in the event of serious complications, removing the eye is not a life-threatening event.

Professor Tsang is running a new preclinical iPS study using human iPS cells derived from the skin cells of a 53-year-old donor. The cells were first transformed with a cocktail of growth factors into cells in the retina that lie underneath the eyes light-sensing cells.

Retina cells nourish the light-sensing cells and protect the fragile cells from excess light, heat and cellular debris. In macular degeneration and retinitis pigmentosa, retina cells die, which allows the photoreceptor cells to degenerate causing the patient to lose their vision. It is estimated that 30 percent of people will have some form of macular degeneration by the time they are 75 years old, as it is the leading cause of vision loss in the elderly. Currently, it affects 7 million Americans and that is expected to double by 2020.

The Columbia research team injected the iPS-derived retina cells into the right eyes of 34 mice that had a genetic mutation that caused their retina cells to degenerate. In many of the mice, the iPS cells assimilated into the retina without disruption and functioned as normal retina cells well into the animals old age. Mice in the control group, who received injections of saline or inactive cells, showed no improvement in retina tests.

Our findings provide the first evidence of life-long neuronal recovery in a preclinical model of retinal degeneration, using stem cell transplant, with vision improvement persisting through the lifespan, Tsang says. And importantly, we saw no tumors in any of the mice, which should allay one of the biggest fears people have about stem cell transplants: that they will generate tumors.

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Blind Mice Get Experimental Stem Cell Treatment For Blindness

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Cedars-Sinai study sheds light on bone marrow stem cell therapy for pancreatic recovery

October 3rd, 2012 10:10 am

Public release date: 2-Oct-2012 [ | E-mail | Share ]

Contact: Sandy Van sandy@prpacific.com 808-526-1708 Cedars-Sinai Medical Center

LOS ANGELES (Oct. 2, 2012) Researchers at Cedars-Sinai's Maxine Dunitz Neurosurgical Institute have found that a blood vessel-building gene boosts the ability of human bone marrow stem cells to sustain pancreatic recovery in a laboratory mouse model of insulin-dependent diabetes.

The findings, published in a PLOS ONE article of the Public Library of Science, offer new insights on mechanisms involved in regeneration of insulin-producing cells and provide new evidence that a diabetic's own bone marrow one day may be a source of treatment.

Scientists began studying bone marrow-derived stem cells for pancreatic regeneration a decade ago. Recent studies involving several pancreas-related genes and delivery methods transplantation into the organ or injection into the blood have shown that bone marrow stem cell therapy could reverse or improve diabetes in some laboratory mice. But little has been known about how stem cells affect beta cells pancreas cells that produce insulin or how scientists could promote sustained beta cell renewal and insulin production.

When the Cedars-Sinai researchers modified bone marrow stem cells to express a certain gene (vascular endothelial growth factor, or VEGF), pancreatic recovery was sustained as mouse pancreases were able to generate new beta cells. The VEGF-modified stem cells promoted growth of needed blood vessels and supported activation of genes involved in insulin production. Bone marrow stem cells modified with a different gene, PDX1, which is important in the development and maintenance of beta cells, resulted in temporary but not sustained beta cell recovery.

"Our study is the first to show that VEGF contributes to revascularization and recovery after pancreatic injury. It demonstrates the possible clinical benefits of using bone marrow-derived stem cells, modified to express that gene, for the treatment of insulin-dependent diabetes," said John S. Yu, MD, professor and vice chair of the Department of Neurosurgery at Cedars-Sinai, senior author of the journal article.

Diabetes was reversed in five of nine mice treated with the injection of VEGF-modified cells, and near-normal blood sugar levels were maintained through the remainder of the six-week study period. The other four mice survived and gained weight, suggesting treatment was beneficial even when it did not prompt complete reversal. Lab studies later confirmed that genetically-modified cells survived and grew in the pancreas and supported the repopulation of blood vessels and beta cells.

Anna Milanesi, MD, PhD, working in Yu's lab as an endocrinology fellow, is the article's first author. The researchers cautioned that although this and other related studies help scientists gain a better understanding of the processes and pathways involved in pancreatic regeneration, more research is needed before human clinical trials can begin.

Insulin-dependent diabetes occurs when beta cells of the pancreas fail to produce insulin, a hormone that regulates sugar in the blood. Patients must take insulin injections or consider transplantation of a whole pancreas or parts of the pancreas that make insulin, but transplantation carries the risk of cell rejection.

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Cedars-Sinai study sheds light on bone marrow stem cell therapy for pancreatic recovery

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New study sheds light on bone marrow stem cell therapy for pancreatic recovery

October 3rd, 2012 10:10 am

ScienceDaily (Oct. 2, 2012) Researchers at Cedars-Sinai's Maxine Dunitz Neurosurgical Institute have found that a blood vessel-building gene boosts the ability of human bone marrow stem cells to sustain pancreatic recovery in a laboratory mouse model of insulin-dependent diabetes.

The findings, published in a PLoS ONE article of the Public Library of Science, offer new insights on mechanisms involved in regeneration of insulin-producing cells and provide new evidence that a diabetic's own bone marrow one day may be a source of treatment.

Scientists began studying bone marrow-derived stem cells for pancreatic regeneration a decade ago. Recent studies involving several pancreas-related genes and delivery methods -- transplantation into the organ or injection into the blood -- have shown that bone marrow stem cell therapy could reverse or improve diabetes in some laboratory mice. But little has been known about how stem cells affect beta cells -- pancreas cells that produce insulin -- or how scientists could promote sustained beta cell renewal and insulin production.

When the Cedars-Sinai researchers modified bone marrow stem cells to express a certain gene (vascular endothelial growth factor, or VEGF), pancreatic recovery was sustained as mouse pancreases were able to generate new beta cells. The VEGF-modified stem cells promoted growth of needed blood vessels and supported activation of genes involved in insulin production. Bone marrow stem cells modified with a different gene, PDX1, which is important in the development and maintenance of beta cells, resulted in temporary but not sustained beta cell recovery.

"Our study is the first to show that VEGF contributes to revascularization and recovery after pancreatic injury. It demonstrates the possible clinical benefits of using bone marrow-derived stem cells, modified to express that gene, for the treatment of insulin-dependent diabetes," said John S. Yu, MD, professor and vice chair of the Department of Neurosurgery at Cedars-Sinai, senior author of the journal article.

Diabetes was reversed in five of nine mice treated with the injection of VEGF-modified cells, and near-normal blood sugar levels were maintained through the remainder of the six-week study period. The other four mice survived and gained weight, suggesting treatment was beneficial even when it did not prompt complete reversal. Lab studies later confirmed that genetically-modified cells survived and grew in the pancreas and supported the repopulation of blood vessels and beta cells.

Anna Milanesi, MD, PhD, working in Yu's lab as an endocrinology fellow, is the article's first author. The researchers cautioned that although this and other related studies help scientists gain a better understanding of the processes and pathways involved in pancreatic regeneration, more research is needed before human clinical trials can begin.

Insulin-dependent diabetes occurs when beta cells of the pancreas fail to produce insulin, a hormone that regulates sugar in the blood. Patients must take insulin injections or consider transplantation of a whole pancreas or parts of the pancreas that make insulin, but transplantation carries the risk of cell rejection.

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New study sheds light on bone marrow stem cell therapy for pancreatic recovery

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Blind Mice Get Experimental Stem Cell Treatment For Blindness

October 2nd, 2012 10:21 pm

April Flowers for redOrbit.com Your Universe Online

Columbia University ophthalmologists and stem cell researchers have developed an experimental treatment for blindness using the patients skin cells, which has improved the vision of blind mice in testing.

The findings of this research, published online in the journal Molecular Medicine, suggest that induced pluripotent stem cells (iPS) could soon be used to improve vision in people with macular degeneration and other eye retina diseases. iPS cells are derived from adult human skin cells but have embryonic qualities.

With eye diseases, I think were getting close to a scenario where a patients own skin cells are used to replace retina cells destroyed by disease or degeneration, says Stephen Tsang, MD, PhD, associate professor of ophthalmology and pathology & cell biology. Its often said that iPS transplantation will be important in the practice of medicine in some distant future, but our paper suggests the future is almost here.

Scientists were very excited by the advent of human iPS cells when they were discovered in 2007, as they provide a way to avoid the ethical complications of embryonic stem cells. Another advantage is that the iPS cells are created from the patients own skin, eliminating the need for anti-rejection medications. Like the ethically challenged embryonic cells, iPS cells can develop into any type of cell. To-date, no iPS cells have been implanted into people, but many ophthalmologists say that the eye would prove to be ideal testing ground for iPS therapies.

The eye is a transparent and accessible part of the central nervous system, and thats a big advantage. We can put cells into the eye and monitor them every day with routine non-invasive clinical exams, Tsang said. And in the event of serious complications, removing the eye is not a life-threatening event.

Professor Tsang is running a new preclinical iPS study using human iPS cells derived from the skin cells of a 53-year-old donor. The cells were first transformed with a cocktail of growth factors into cells in the retina that lie underneath the eyes light-sensing cells.

Retina cells nourish the light-sensing cells and protect the fragile cells from excess light, heat and cellular debris. In macular degeneration and retinitis pigmentosa, retina cells die, which allows the photoreceptor cells to degenerate causing the patient to lose their vision. It is estimated that 30 percent of people will have some form of macular degeneration by the time they are 75 years old, as it is the leading cause of vision loss in the elderly. Currently, it affects 7 million Americans and that is expected to double by 2020.

The Columbia research team injected the iPS-derived retina cells into the right eyes of 34 mice that had a genetic mutation that caused their retina cells to degenerate. In many of the mice, the iPS cells assimilated into the retina without disruption and functioned as normal retina cells well into the animals old age. Mice in the control group, who received injections of saline or inactive cells, showed no improvement in retina tests.

Our findings provide the first evidence of life-long neuronal recovery in a preclinical model of retinal degeneration, using stem cell transplant, with vision improvement persisting through the lifespan, Tsang says. And importantly, we saw no tumors in any of the mice, which should allay one of the biggest fears people have about stem cell transplants: that they will generate tumors.

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Blind Mice Get Experimental Stem Cell Treatment For Blindness

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