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Bone marrow donor meets recipient

By JoanneRUSSELL25

DENVER - Can you imagine having a family member who's diagnosed with a disease and no one in your family, including yourself, is a match to donate.

That's the reality for 70 percent of patients needing bone marrow or stem cells. They have no other choice but to go through the bone marrow registry.

Those families rely on complete strangers who are willing to donate whatever they can in hope of saving someone's life.

One of those donors is Aurora resident Denise Camacho. She joined the bone marrow registry never thinking that anything would ever come of it.

"I have a family friend that works with Bonfils," Camacho said. "She emailed me and my family and said there's a huge need for minorities to join the registry. So we went down not knowing anyone of us would ever be called."

But just two years later, she was called to make a donation.

"I got a phone call that I'm a match, but I need to go in for further testing. All they told me was that there was a 13-year-old boy in Cleveland who has leukemia." Camacho said. "How do you say 'no' when there's a family out there that you can help and possibly save a life. I was going to do what I could."

That 13-year-old boy was Enrique Linares. He was diagnosed with acute lymphoblastic leukemia.

His entire family, 38 people in all, were tested to be a donor but none of them were a match.

After nearly two years, spent mostly in the hospital, there was a match. It was a match no one expected. Camacho is unrelated and has a different blood type.

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Stem Cells Improve Visual Function in Blind Mice

By raymumme

Newswise An experimental treatment for blindness, developed from a patients skin cells, improved the vision of blind mice in a study conducted by Columbia ophthalmologists and stem cell researchers.

The findings suggest 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 eyes retina.

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 the studys principal investigator, 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.

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.

No iPS cells have been transplanted into people, but many ophthalmologists say the eye is the 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 says. And in the event of serious complications, removing the eye is not a life-threatening event.

In Tsangs 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 eyes 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. Macular degeneration currently affects 7 million Americans and its incidence is expected to double by 2020.

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.

In many animals, the human cells assimilated into mouse retina without disruption and functioned as normal retina cells well into the animals old age. Control mice that got injections of saline or inactive cells showed no improvement in retina tests.

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Medistem and Superview Biotechnology Co. Ltd. Initiate Collaboration on Therapeutics Development Using Antibody and …

By raymumme

SAN DIEGO--(BUSINESS WIRE)--

Medistem Inc (Pink Sheets:MEDS) announced today the initiation of a collaboration with Superview Biotechnology Co. Ltd, a subsidiary of Yinhuan Holding Co from Yixing, China. The joint work will be aimed at using proprietary stem cell lines developed by Medistem for screening of monoclonal antibodies for therapeutic activity in the area of regenerative medicine. As part of the collaboration, the two companies will evaluate various candidates jointly, as well as apply for grants and share research data.

To date, the majority of stem cell companies are focusing on the stem cell itself being a product. By collaborating with Superview Biotechnology, we aim to assess the feasibility of developing antibodies that can modulate the activity of stem cells that already exist in the body, said Thomas Ichim, CEO of Medistem. This approach not only provides methods of activating stem cells but also allows for the development of stem cell adjuvant therapies that could be used to resurrect stem cell candidates that failed in clinical trials.

Superview Biotechnology has developed proprietary methods of rapidly generating monoclonal antibodies to esoteric protein targets. Medistem has a history of success in the area of stem cells, being the only company to take a stem cell product from discovery to FDA clearance in the short span of 4 years.

One of the significant driving forces behind our company is to develop innovative targets for our monoclonal antibodies. Although monoclonal antibodies have generated sales of billions of dollars in areas ranging from rheumatoid arthritis, to cancer, to preventing blindness, we feel that the potential of this therapeutic tool is only beginning to be recognized, said Jiong Wu, CEO of Superview Biotechnology. Our opinion is that the barriers to entry for monoclonal antibody-based therapies modulating endogenous stem cells is lower than stem cell based therapies. We are eager to work with the Medistem team at exploring this hypothesis.

A joint grant is expected to be filed with the National Natural Science Foundation of China to support part of the proposed collaboration by end of October, 2012.

Cautionary Statement

This press release does not constitute an offer to sell or a solicitation of an offer to buy any of our securities. This press release may contain certain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Forward-looking statements are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified. Future events and actual results could differ materially from those set forth in, contemplated by, or underlying the forward-looking information. Factors which may cause actual results to differ from our forward-looking statements are discussed in our Form 10-K for the year ended December 31, 2007 as filed with the Securities and Exchange Commission.

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Translational Regenerative Medicine: Market Prospects 2012-2022

By Dr. Matthew Watson

NEW YORK, Oct. 1, 2012 /PRNewswire/ -- Reportlinker.com announces that a new market research report is available in its catalogue:

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Houston Stem Cell Summit Announces Extraordinary Lineup of Keynote Speakers

By daniellenierenberg

HOUSTON, Oct. 1, 2012 /PRNewswire/ --The Houston Stem Cell Summit will host an extraordinary lineup of keynote speakers who represent the most accomplished stem cell scientists, clinicians and entrepreneurs in the United States. Joining these distinguished speakers will be Governor of Texas, Rick Perry, consistent champion of adult stem cell therapies.

(Logo: http://photos.prnewswire.com/prnh/20120831/NY66463LOGO )

The Houston Stem Cell Summit will be held October 26 27 in its namesake city and will highlight the latest therapeutic research regarding the use of adult stem and progenitor cell therapies. The Summit will also provide a forum for entrepreneurs to discuss their latest efforts to commercialize stem cell therapies, and to debate and discuss FDA and other legal and regulatory issues impacting stem cell research and commercialization.

Opening Keynote Address October 26, 2012 Arnold I. Caplan, PhD, Professor of Biology and Professor of General Medical Sciences (Oncology) Case Western Reserve University

Dr. Caplan has helped shape the direction and focus of adult stem cell research and commercialization. Virtually every adult stem cell company and literally tens of thousands of research papers are based on Dr. Caplan's original and ground breaking research. Professor Caplan is considered to be the "father" of the mesenchymal stem cell and first described this progenitor cell in his landmark paper; "Mesenchymal stem cells", Journal of Orthopaedic Research 1991;9(5):641-650. Since that foundational study, Dr. Caplan has published over 360 manuscripts and articles in peer reviewed journals. Dr. Caplan has been Chief Scientific Officer at OrthoCyte Corporation since 2010. In addition, Dr. Caplan co-founded Cell Targeting Inc. and has served as President of Skeletech, Inc. as its founder. He is the recipient of several honors and awards from the orthopedic research community. Dr. Caplan holds a Ph. D. from Johns Hopkins University Medical School and a B.S. in chemistry from the Illinois Institute of Technology.

Summit Keynote Address October 26, 2012 Texas Governor Rick Perry

Governor Perry is the 47th and current Governor of Texas. Governor Perry has long championed the role of medical technologies in building the future of not only Texas, but also the United States. In many ways, his strong advocacy on behalf of research and advanced medical technologies is one of his strongest and as yet underappreciated legacies. In addition to his service to the state of Texas, Governor Perry has also served as Chairman of the Republican Governors Association in 2008 and again in 2011. Despite a rigorous schedule, particularly in the teeth of this election season, Governor Perry has graciously made time to speak and encourage the researchers, patients, companies and physicians who form the fabric and future of the stem cell therapy community.

Texas Medical Center Keynote Address, October 27, 2012 James T. Willerson, MD

Over the course of his career, Dr. James T. Willerson has served as a medical, scientific and administrative leader for each of the major institutions that are the foundation of the Texas Medical Center. Dr. Willerson is currently President and Medical Director, Director of Cardiology Research, and Co-Director of the Cullen Cardiovascular Research Laboratories at Texas Heart Institute (THI). Dr. Willerson was appointed President-Elect of THI in 2004 and became President and Medical Director in 2008. He is also an adjunct professor of Medicine at Baylor College of Medicine and at The University of Texas MD Anderson Cancer Center. He is the former chief of Cardiology at St. Luke's Episcopal Hospital and the former chief of Medical Services at Memorial Hermann Hospital.

Dr. Willerson has served as a visiting professor and invited lecturer at more than 170 institutions.

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Immune system harnessed to improve stem cell transplant outcomes

By daniellenierenberg

ScienceDaily (Oct. 1, 2012) A novel therapy in the early stages of development at Virginia Commonwealth University Massey Cancer Center shows promise in providing lasting protection against the progression of multiple myeloma following a stem cell transplant by making the cancer cells easier targets for the immune system.

Outlined in the British Journal of Hematology, the Phase II clinical trial was led by Amir Toor, M.D., hematologist-oncologist in the Bone Marrow Transplant Program and research member of the Developmental Therapeutics program at VCU Massey Cancer Center. The multi-phased therapy first treats patients with a combination of the drugs azacitidine and lenalidomide. Azacitidine forces the cancer cells to express proteins called cancer testis antigens (CTA) that immune system cells called T-cell lymphocytes recognize as foreign. The lenalidomide then boosts the production of T-cell lymphocytes. Using a process called autologous lymphocyte infusion (ALI), the T-cell lymphocytes are then extracted from the patient and given back to them after they undergo a stem cell transplant to restore the stem cells' normal function. Now able to recognize the cancer cells as foreign, the T-cell lymphocytes can potentially protect against a recurrence of multiple myeloma following the stem cell transplant.

"Every cell in the body expresses proteins on their surface that immune system cells scan like a barcode in order to determine whether the cells are normal or if they are foreign. Because multiple myeloma cells are spawned from bone marrow, immune system cells cannot distinguish them from normal healthy cells," says Toor. "Azacitidine essentially changes the barcode on the multiple myeloma cells, causing the immune system cells to attack them," says Toor.

The goal of the trial was to determine whether it was safe, and even possible, to administer the two drugs in combination with an ALI. In total, 14 patients successfully completed the investigational drug therapy. Thirteen of the participants successfully completed the investigational therapy and underwent a stem cell transplant. Four patients had a complete response, meaning no trace of multiple myeloma was detected, and five patients had a very good partial response in which the level of abnormal proteins in their blood decreased by 90 percent.

In order to determine whether the azacitidine caused an increased expression of CTA in the multiple myeloma cells, Toor collaborated with Masoud Manjili, D.V.M., Ph.D., assistant professor of microbiology and immunology at VCU Massey, to conduct laboratory analyses on bone marrow biopsies taken from trial participants before and after treatments. Each patient tested showed an over-expression of multiple CTA, indicating the treatment was successful at forcing the cancer cells to produce these "targets" for the immune system.

"We designed this therapy in a way that could be replicated, fairly inexpensively, at any facility equipped to perform a stem cell transplant," says Toor. "We plan to continue to explore the possibilities of immunotherapies in multiple myeloma patients in search for more effective therapies for this very hard-to-treat disease."

In addition to Manjili, Toor collaborated with John McCarty, M.D., director of the Bone Marrow Transplant Program at VCU Massey, and Harold Chung, M.D., William Clark, M.D., Catherine Roberts, Ph.D., and Allison Hazlett, also all from Massey's Bone Marrow Transplant Program; Kyle Payne, Maciej Kmieciak, Ph.D., from Massey and the Department of Microbiology and Immunology at VCU School of Medicine; Roy Sabo, Ph.D., from VCU Department of Biostatistics and the Developmental Therapeutics program at Massey; and David Williams, M.D., Ph.D., from the Department of Pathology at VCU School of Medicine, co-director of the Tissue and Data Acquisition and Analysis Core and research member of the Developmental Therapeutics program at Massey.

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Experimental Stem Cell Therapy May Help Burn Victims

By raymumme

For more than 40 years, Lesley Kelly of Glasgow, Scotland, lived with third-degree burns that stretched over 60 percent of her body.

Kelly was 2 years old when she fell into a bathtub filled with hot water that scorched most of the right side of her body. She lost full range of motion around many of her joints.

"When you have bad scarring, the buildup is very thick and has no elasticity," said Kelly, 45, whose right elbow was most affected by the buildup of scar tissue. "The problem with thermal burn scarring [is that] it's hard to get the range of motion."

Kelly underwent numerous reparative surgeries through the years, but the scar tissue continued to grow back. The procedures did not lessen the look of her scars.

In 2011, Kelly underwent a new, experimental procedure that used stem cells from her own fat tissue to repair the buildup around her right elbow.

Surgeons cleaned the scar buildup around the elbow and used liposuction to pull fat from off Kelly's waist. They separated the fat cells from the stem and regenerative cells, which were then injected into the wound on Kelly's arm. The procedure took less than two hours.

Within months, Kelly was able to regain 40 degrees of motion that she had lost more than 40 years ago.

"If this technology was available earlier in my life, my scars would not have been as bad," said Kelly.

There are an estimated 50,000 to 70,000 burn cases each year in the U.S., according to the American Burn Association.

The stem cell therapy, approved in the U.K. to treat soft tissue wounds, is now gaining traction in the U.S.

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New Therapy May Help Burn Victims

By NEVAGiles23

Lesley Kelly, 45, underwent stem cell therapy to repair scar tissue buildup in her right arm. (Cytori Therapeutics, Inc.)

By Lara Salahi, ABC News For more than 40 years, Lesley Kelly of Glasgow, Scotland, lived with third-degree burns that stretched over 60 percent of her body.

Kelly was 2 years old when she fell into a bathtub filled with hot water that scorched most of the right side of her body. She lost full range of motion around many of her joints.

"When you have bad scarring, the buildup is very thick and has no elasticity," said Kelly, 45, whose right elbow was most affected by the buildup of scar tissue. "The problem with thermal burn scarring [is that] it's hard to get the range of motion."

Kelly underwent numerous reparative surgeries through the years, but the scar tissue continued to grow back. The procedures did not lessen the look of her scars.

In 2011, Kelly underwent a new, experimental procedure that used stem cells from her own fat tissue to repair the buildup around her right elbow.

Surgeons cleaned the scar buildup around the elbow and used liposuction to pull fat from off Kelly's waist. They separated the fat cells from the stem and regenerative cells, which were then injected into the wound on Kelly's arm. The procedure took less than two hours.

Within months, Kelly was able to regain 40 degrees of motion that she had lost more than 40 years ago.

"If this technology was available earlier in my life, my scars would not have been as bad," said Kelly.

There are an estimated 50,000 to 70,000 burn cases each year in the U.S., according to the American Burn Association.

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StemCells, Inc. Achieves Spinal Cord Injury Milestone With First Neural Stem Cell Transplant Into Patient With Sensory …

By LizaAVILA

NEWARK, Calif., Sept. 27, 2012 (GLOBE NEWSWIRE) -- StemCells, Inc. (STEM) today announced that the first patient with an incomplete spinal cord injury has been enrolled in the Company's Phase I/II clinical trial in chronic spinal cord injury and transplanted with the Company's proprietary HuCNS-SC(R) neural stem cells. The patient, a Canadian man who suffered a thoracic spinal cord injury from a sports-related accident, was administered the cells yesterday at Balgrist University Hospital, University of Zurich, a world leading medical center for spinal cord injury and rehabilitation. This is the first patient in the second cohort of the trial, which will be comprised of four patients who retain some sensory function below the level of trauma and are therefore considered to have an incomplete injury.

"This is an important milestone for StemCells and the spinal cord injury community as it is the first time anyone has ever transplanted neural stem cells into a patient with an incomplete injury," said Stephen Huhn, MD, FACS, FAAP, Vice President and Head of the CNS Program at StemCells, Inc. "Given the encouraging interim data from the most severely injured patient cohort that we reported earlier this month, testing patients with less severe injury should afford us an even better opportunity to continue to test safety and to detect and assess clinical changes. Unlike the patients in the first cohort, patients with incomplete injuries have retained a degree of spinal cord function that might be even further augmented by transplantation with neural stem cells."

Earlier this month, the Company reported that interim six-month data from the first patient cohort in the Phase I/II clinical trial continued to demonstrate a favorable safety profile, and showed considerable gains in sensory function in two of the three patients compared to pre-transplant baselines. Patients in the first cohort all suffered a complete injury to their spinal cord, leaving them with no neurological function below the level of injury. Following transplantation with HuCNS-SC cells, there were no abnormal clinical, electrophysiological or radiological responses to the cells, and all the patients were neurologically stable through the first six months after transplantation. Changes in sensitivity to touch, heat and electrical stimuli were observed in well-defined and consistent areas below the level of injury in two of the patients, while the third patient remained stable. Importantly, the changes in sensory function were confirmed objectively by measures of electrical impulse transmission across the site of injury, each of which correlated with the clinical examination.

About the Spinal Cord Injury Clinical Trial

The Phase I/II clinical trial of StemCells, Inc.'s HuCNS-SC(R) purified human adult neural stem cells is designed to assess both safety and preliminary efficacy. Twelve patients with thoracic (chest-level) neurological injuries at the T2-T11 level are planned for enrollment, and their injuries must have occurred within three to twelve months prior to transplantation of the cells. In addition to assessing safety, the trial will assess preliminary efficacy based on defined clinical endpoints, such as changes in sensation, motor function and bowel/bladder function. The Company has dosed the first patient cohort, all of whom have injuries classified as AIS A according to the American Spinal Injury Association Impairment Scale (AIS). In AIS A injuries, there is no neurological function below the injury level. The second cohort will be patients classified as AIS B, in which there is some preservation of sensory or motor function below the injury level. The third cohort will be patients classified as AIS C, in which there is some preservation of both sensory and motor function.

All patients will receive HuCNS-SC cells through direct transplantation into the spinal cord and will be temporarily immunosuppressed. Patients will be evaluated regularly in the post-transplant period in order to monitor and assess the safety of the HuCNS-SC cells, the surgery and the immunosuppression, as well as to measure any recovery of neurological function below the injury site. The Company intends to follow the effects of this therapy long-term, and each of the patients will be invited to enroll into a separate four year observational study after completing the Phase I/II study.

The trial is being conducted at Balgrist University Hospital, University of Zurich, a world leading medical center for spinal cord injury and rehabilitation, and is open for enrollment to patients in Europe, Canada and the United States. Enrollment for the second cohort is now underway. If you believe you may qualify and are interested in participating in the study, please contact the study nurse either by phone at +41 44 386 39 01 or by email at stemcells.pz@balgrist.ch.

Additional information about the Company's spinal cord injury program can be found on the StemCells, Inc. website at http://www.stemcellsinc.com/Therapeutic-Programs/Clinical-Trials.htm and at http://www.stemcellsinc.com/Therapeutic-Programs/Spinal-Cord-Injury.htm, including video interviews with Company executives and independent collaborators.

About Balgrist University Hospital

Balgrist University Hospital, University of Zurich is recognized worldwide as a highly specialized center of excellence providing examination, treatment and rehabilitation opportunities to patients with serious musculoskeletal conditions. The clinic owes its leading international reputation to its unique combination of specialized medical services. The hospital's carefully-balanced, interdisciplinary network brings together under one roof medical specialties including orthopedics, paraplegiology, radiology, anesthesiology, rheumatology, and physical medicine. More information about Balgrist University Hospital is available at http://www.balgrist.ch.

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Grants Pass man meets donor who saved his life

By NEVAGiles23

GRANTS PASS This month, Jerry Condit met the only man in the world who could and did save his life.

Condit needed a stem cell transplant to replace bone marrow lost to leukemia, a blood-attacking cancer diagnosed in January 2008 on his 69th birthday.

Doctors estimated he had two years to live if it went untreated. But finding a suitable donor was difficult. In fact, the National Marrow Donor Program says only about half of the people in need of transplants ever find a donor.

After searching national and international registries of millions of potential donors, doctors found only one match: Marco Rixen, a 34-year-old resident of Germany.

He matched 11 of the 12 markers they were looking for, said Condits wife, Jan. It was enough to consider Rixen a match.

The transplant was performed in May 2008 at Oregon Health & Science University in Portland. Rixen made his donation at a medical center in Germany, where a courier rushed the stem cells to a plane bound for Portland. Less than 36 hours later, Condits transfusion was under way.

For the first two years after the transplant, Condit and Rixen could communicate only through the bone marrow donation agency. After that, names and e-mail addresses were released.

The two kept in touch, and one day the Condits got a message from Rixen that said he was planning to visit the United States.

I didnt know if I would ever get the chance to meet him, said Condit, who cant travel because of his vulnerable immune system. He wrote us and said he was coming here, and we just about fell over.

Rixen and his wife, Anja, spent Sept. 19 with the Condits in Grants Pass before heading to Las Vegas to renew their wedding vows in front of an Elvis impersonator and then visit the Grand Canyon.

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Stem cell transplantation for boy with thalassaemia

By NEVAGiles23

A 10-year-old boy has been infused with stem cells harvested from the bone marrow of his brother to treat him for thalassaemia a disorder caused by destruction of red blood cells. Called allogeneic transplantation of stem cells, this was done at Kovai Medical Center and Hospital.

D. Dhanush may not have to undergo expensive and excruciating blood transfusion anymore if his body accepts the donor cells. But his condition will have to be evaluated very minutely for the next two years to confirm that the cells donated by his brother have been received well and adapted him.

Presenting the boy before media persons, Clinical Haematologist and Head of the Bone Marrow Transplant Unit T. Rajasekar explained that transplantation was of two types autologous and allogeneic.

The autologous procedure involves harvesting of stem cells from the patients themselves (those suffering from thalassaemia or leukaemia). The extracted cells are frozen and stored for high dose treatment.

After being treated, these are infused into the patient through a vein. This procedure was done for one person suffering from myeloma (cancer of plasma cells or white blood cells that produce anti-bodies that help fight infections/diseases) and another with a relapsed lymphoma (cancer of the lymphocytes cells that are part of immune system).

Under the allogeneic procedure, matching stem cells from a donor are used. Mostly, these cells are from siblings or a close relative as they need to pass the human leukocyte antigen (HLA) matching test. HLA matching is required, or the cells will be rejected by the recipient. Ideally, it is sibling whose cells will match because he or she will have the HLA from both parents. It is the combination of HLAs from both parents that are found in the children.

The cells can be harvested from the marrow or from the blood. In the case presented on Tuesday, Dr. Rajasekar said the cells were brought out of the bone marrow in Dhanushs brother and into his blood, from where these were harvested.

Chairman of the hospital Nalla G. Palaniswami said the tough procedure was performed by the new Comprehensive Cancer Centre, which was gradually bringing in specialists of all sub-specialities of cancer care. Only then can this be called a comprehensive centre, he said.

The hospital would form a KMCH Foundation, which would use funds from donors to treat poor children suffering from cancer and some other disorders that required expensive treatment.

The stem cell transplantation that Dhanush, the son of a police head constable, underwent cost Rs.12 lakh. Of this, Rs.9 lakh was provided by a donor, Dr. Palaniswami said. Dean of the hospital V. Kumaran and Head of Department of Interventional Radiology Mathew Cherian spoke on how the cancer centre was established and how developments were being made.

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International Stem Cell Corp Granted Key Patent for Liver Disease Program

By NEVAGiles23

CARLSBAD, CA--(Marketwire - Sep 25, 2012) - International Stem Cell Corporation ( OTCQB : ISCO ) (www.internationalstemcell.com) ("ISCO" or "the Company") a California-based biotechnology company, today announced that the United States Patent and Trademark Office (USPTO) has granted the Company a patent for a method of creating pure populations of definitive endoderm, precursor cells to liver and pancreas cells, from human pluripotent stem cells.This patent is a key element of ISCO's metabolic liver disease program and allows the Company to produce the necessary quantities of precursor cells in a more efficient and cost effective manner.

The patent, 8,268,621, adds to the Company's growing portfolio of proprietary technologies relating to the development of potential treatments for incurable diseases using human parthenogenetic Stem Cells (hpSC).Human parthenogenetic stem cells are unique pluripotent stem cells that offer the possibility to reduce the cost of health care while avoiding the ethical issues that surround the use of fertilized human embryos.Aside from the Company's current liver disease program, this new patented method can be used as a route to create pancreatic and endocrine cells that could be used in future studies of diabetes and other metabolic disorders.

ISCO currently has the largest collection of hpSC including cell lines which immune match the donor, as is the case with induced pluripotent stem cells (iPS), and cell lines which immune-match millions of individuals and potentially reduce tissue rejection issues.The Company is focusing its therapeutic development efforts on three clinical applications where cell and tissue therapy is already proven but where there currently is an insufficient supply of safe and efficacious cells: Parkinson's disease, inherited/metabolic liver diseases and corneal blindness.

About International Stem Cell Corporation

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

To receive ongoing corporate communications via email, visit: http://www.b2i.us/irpass.asp?BzID=1468&to=ea&s=0

To like our Facebook page or follow us on Twitter for company updates and industry related news, visit: http://www.facebook.com/InternationalStemCellCorporation and http://www.twitter.com/intlstemcell

Safe harbor statement

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

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International Stem Cell Corp Granted Key Patent for Liver Disease Program

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Celprogen Obtained US Patent (US8,236,297B2) Method of Treating Lactose Intolerance Utilizing Genetically Engineered …

By Dr. Matthew Watson

LOS ANGELES--(BUSINESS WIRE)--

Celprogen Inc., a leader in the Stem Cell Research and Therapeutics industry for the development of stem cell technologies for regenerative medicine, today announced that they obtained a Patent for Treating Lactose Intolerance Utilizing Genetically Engineered Bacteria US8,236,297B2. Acquired lactase deficiency is the most common disorder of complex carbohydrate absorption throughout the world, affecting 75% of world population. In the United States 15% of Caucasians, over 50% of Hispanics and over 80% of African-Americans suffer from lactose intolerance.

The present invention relates to genetically engineered bacteria that are able to colonize the mammalian intestine and actively produce mammalian lactase. This lactose-digesting enzyme is stable and active under the conditions normally found in the mammalian small intestine. Experimental subjects colonized with the genetically engineered bacteria show improved ability to digest lactose in dairy foods.

About Celprogen Inc.

Celprogen Inc. is a global Stem Cell Research & Therapeutics company which is developing a proprietary portfolio of unique therapeutics products and life science research tools that includes genetic engineering technologies, stem cell technologies for regenerative medicine, as well as bio-engineering products for tissue & organ transplants. Headquartered in San Pedro, California, Celprogen is committed to the research, development, and manufacture of quality Stem Cell, Cancer Stem Cell and Primary Cell Culture products to serve our global community. Additional information about Celprogen is available at http://www.celprogen.com.

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Celprogen Obtained US Patent (US8,236,297B2) Method of Treating Lactose Intolerance Utilizing Genetically Engineered ...

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Stem Cell Therapy—Breakthrough in Health Paradigm

By daniellenierenberg

By Sharmistha Banerjee - September 25, 2012 | Tickers: NBS, OSIR, PSTI | 0 Comments

Sharmistha is a member of The Motley Fool Blog Network -- entries represent the personal opinions of our bloggers and are not formally edited.

Far-reaching accomplishments in the biotechnology sector meet its most ambitious expectations, stem cell therapy. The birth of this new industry has boosted the enthusiasm and energy of investors and has brought unprecedented capability and optimistic predictions. New developments in regenerative medicine are bringing about exciting, novel approaches to create therapies for hard to treat diseases. The biotechnology industry has been soaring in 2012 as companies both large and small have shown impressive growth.

The cell therapy space has seen relatively small companies making strides in the right direction with increased government support. Osiris Therapeutics (NASDAQ: OSIR) a leading stem cell company is currently the only company with an approved cell therapy. The approval is more of a first step in a long walk for Osiris. Reuters reported that shares of Osiris Therapeutics rose 15% on May 30, 2012, after U.S. health regulators said the stem cell technology company's wound treatment was eligible for reimbursement when used in hospitals in out-patient settings or in ambulances. The company carries over a $300 million market capitalization and trades at $9.50 per share, primarily on the strength of a recent Canadian approval for its stem cell drug for graft-versus-host disease. Osiris Therapeutics has a 1-year low of $4.12 and a 1-year high of $14.46. The company has a market cap of $311.3 million and a price-to-earnings ratio of 90.98. Investors are impressed and optimistic with Osiris progress in cell-based therapies. They currently have a $9.75 target price on the stock. Despite having to negotiate a more challenging regulation process the company has continued to show investors strong gains in 2012.With a current ratio of 8.51 and debt equity of (0.00%) the company boasts of a financially secure position in the market.

Pluristem Therapeutics (NASDAQ: PSTI) a small firm with a market cap of less than $180 million has been concentrating on its placenta-based cell therapies, is considered one of the more advanced in the cell therapy arena, and unlike OSIR, its lead candidates treat diseases that could potentially return significant revenue. The upside for PSTI is lower costs, quicker healing time, ease of administration, and most importantly, it can grow vessels and provide the possibility of a cure, which has led to optimism surrounding the stock. Shares of Pluristem Therapeutics are up over 3.98% and most likely headed higher in the days ahead. It has traded higher by 85% during the last three months and is now valued at $200 million. Pluristem may actually beat OSIR in the race to become the first U.S. approved cell therapy with its bone marrow therapy, in which it has recently applied for approval. Pluristem is a company that I think is showing great promise. From the stock's action in the last several months, it is clear investors recognize that Pluristem's unique platform technology has the potential for tremendous value in a lucrative range of medical markets both the very large and the very small. The company wins both ways. Its clinical segment is creating candidates with large revenue potential, with analysts projecting peak sales of $700 million for AMR-001, which treats patients following acute myocardial infarction. The company is reasonably well funded with around $42 million in cash and cash equivalents.

NeoStem (NYSEMKT: NBS) is by far the leader in regards to the manufacturing business, and no other company comes close. In addition, its stock has returned the most over in the last three months, with a 100% gain. NeoStem stocks looks promising as a biotechnology investment. First, the company is focusing on several promising areas of new stem cell treatment development. Second, its contract manufacturing business brings in revenues to offset some of its drug development expenditures. Third, the contract manufacturing business could earn substantial royalties if any of the products on which it works with customers proves to be a commercial success. NeoStem's manufacturing segment which is also known as PCT, is well positioned to return larger gains over the next 24 months with several late stage candidates under development. a $110 million company that has increased in value by 70% during the last three months, In addition to the PCT business, NeoStem's most promising therapy is aimed at preventing major cardiac problems following acute myocardial infarction (AMI), an area that is potentially a multibillion-dollar business. NeoStem's therapy is meeting endpoints never before reached,

The three companies discussed above are showing much potential for growth and each present a significant upward shift in the current stock prices while contributing greatly to the advances of cell therapy.

Osiris is the closest to generating substantial revenue by already having two approvals, and is currently testing its therapy on other diseases, thereby leaving open the possibility of future gains. Pluristem has candidates to treat diseases in potentially large markets, and is expanding with its manufacturing facility. Although Stem Cells is in the early phases of development, it still has a very innovating therapy that, if proven effective, could advance the space even further. NeoStem possesses all the benefits of an innovating technology, a diversified pipeline, and is a candidate with significant revenue potential.

At this point, it appears that the entire space is moving forward and has lifted observers' expectations by making rapid progress. It makes sense that these three stocks would trade with such considerable gains, as investors can now identify the benefits of cell therapies. And as more approvals occur, it could be a space that trades considerably higher regardless of the market's indecisiveness. With the sector growing and maturing, investing in biotech stocks seems a promising choice in future.

SharmisthaB has no positions in the stocks mentioned above. The Motley Fool has no positions in the stocks mentioned above. Try any of our Foolish newsletter services free for 30 days. We Fools may not all hold the same opinions, but we all believe that considering a diverse range of insights makes us better investors. The Motley Fool has a disclosure policy.If you have questions about this post or the Fools blog network, click here for information.

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Stem Cell Therapy—Breakthrough in Health Paradigm

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‘Enrolments for biotechnology will see a rise in three years’ – The Hindu

By Dr. Matthew Watson


The Hindu
'Enrolments for biotechnology will see a rise in three years'
The Hindu
Biotechnology education in the State is undergoing course correction and will get its shine back in a couple of years, chairperson, Karnataka Vision Group on Biotechnology, Kiran Mazumdar-Shaw, said in Bangalore on Tuesday. There was a temporary dip in ...
Biotech courses in Bangalore not industry ready: Kiran Mazumdar-ShawDaily News & Analysis

all 3 news articles »

Source:
http://news.google.com/news?q=biotechnology&output=rss

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The 48th Nobel Conference: Our Global Ocean – Gustavus Adolphus College News (blog)

By Dr. Matthew Watson


Gustavus Adolphus College News (blog)
The 48th Nobel Conference: Our Global Ocean
Gustavus Adolphus College News (blog)
Next week, distinguished scholars and researchers in the fields of biogeochemistry, oceanography, deep-sea biology, molecular genetics, and coral ecology will travel to Gustavus Adolphus College to take part in the 48th annual Nobel Conference, titled ...

and more »

Source:
http://news.google.com/news?q=molecular-genetics&output=rss

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Anticipated short-term cell therapy industry clinical milestones

By Dr. Matthew Watson


What follows is an interesting but not exhaustive list of cell therapy industry clinical milestones we anticipate in the next 3-9 months as selected from the list of cell therapy products we are tracking in late-stage or post-commercial development.  


There are other commercial milestones we are monitoring as well as other clinical milestones we expect to see related to cell therapy products in earlier stages of the development pipeline that are not included below.


CellCoTec (http://www.cellcotec.com)
  • Having completed a trial in Europe of their device to enable POC production of an autologous chondrocyte cellular product in/with a biodegradable, load-bearing scaffold for the treatment of articular cartilage defects, they have now submitted their CE market application.  The CE mark application is under review and they anticipate a response in October.  
  • This device and the potential emergence of Sanofi's MACI in the European market next year may have an impact on Tigenix (EBR:TIG) most directly.



ERYtech Parma (http://www.erytech.com)

  • Their 'pivotal' phase 2/3 trial in Europe of lead product, GRASPA, for the treatment of Acute Lymphoblastic Leukemia (ALL) is scheduled for completion 2H 2012. 


GamidaCell (http://www.gamidacell.com)

  • Their 'pivotal' phase 2/3 trial in the US, Israel, and Europe of lead product, StemEx, for the treatment of leukemia and lymphoma, in joint development with Teva, completed enrollment in February and is scheduled for completion 2H 2012.  They have not been shy about the fact they expect to be in the market in 2013.


Innovacell (http://www.innovacell.com)

  • They raised over 8m Euro in April for a phase 3 trial in Europe for their lead product, ICES13, for the treatment of stress-urinary incontinence which was scheduled for a preliminary clinical data readout in Q4 2012 and be ready for market authorization in 2013. Since announcing the capital raise the company has been stone silent and no clinical trial registry has been filed.  Status unknown.


Miltenyi Biotec (www.miltenyibiotec.com)

  • Their phase 3 trial in Germany of CD133+ cells as an adjunct to CABG surgery for myocardial ischemia or coronary artery disease is scheduled for completion in January.


NovaRx (http://www.novarx.com)

  • Their phase 3 trial in US, Europe, and India of their lead product, Lucanix, for the treatment of advanced Non-small Cell Lung Cancer (NSCLC) following front-line chemotherapy is scheduled in clnicaltrials.gov for completion in October but we have learned they expect their next 'interim analysis' in February.


NuVasive (http://www.nuvasive.com)

  • They have a series of trials scheduled to complete 2H 2012 intended to provide additional clinical data to support its marketing of Osteocel Plus for the treatment of a growing number of orthopedic applications.


Sanofi's Genzyme (http://www.genzyme.com)

  • Having completed their phase 3 trial in Europe of MACI for knee repair (symptomatic articular cartilage defects of the femoral condyle including the trochlea), they expect to file their market authorization application (MAA) in 1H 2013.


Hope that's helpful and gives you a sense some of the late-stage things to watch for in the coming weeks and months.  



--Lee

Source:
http://feeds.feedburner.com/CellTherapyBlog

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The cost of clinical trial data bias/loss, FDA’s new job and the need for bold leadership.

By Dr. Matthew Watson


The scandal of clinical trial data loss is eroding the fundamentals of evidence-based research and clinical medicine.


Before you right this post off as the stuff of conspiracy theories, fear-mongering, and 'alternative world views' consider that this view is shared by the likes of the FDA, the International Committee of Medical Journal Editors, the Cochrane Collaboration, and researchers at institutions like Johns Hopkins School of Medicine.


Here's the underlying premise as succinctly described by author Ben Goldacre:

"Drugs are tested by the people who manufacture them, in poorly designed trials, on hopelessly small numbers of weird, unrepresentative patients, and analysed using techniques that are flawed by design, in such a way that they exaggerate the benefits of treatments. Unsurprisingly, these trials tend to produce results that favour the manufacturer.

When trials throw up results that companies don't like, they are perfectly entitled to hide them from doctors and patients, so we only ever see a distorted picture of any drug's true effects. Regulators see most of the trial data, but only from early on in a drug's life, and even then they don't give this data to doctors or patients, or even to other parts of government. This distorted evidence is then communicated and applied in a distorted fashion."

Authors M. Todwin and J. Abramson summarize it thusly:

"Trials with positive results generally are published more frequently than studies that conclude that a new drug poses greater risks or is no more effective than standard therapy or a placebo. Furthermore, some articles may distort trial findings by omitting important data or by modifying prespecified outcome measures. Lack of access to detailed information about clinical trials can undermine the integrity of medical knowledge."

Here is a great list of very recent resources that may convince you of the merits of this concern:

Yesterday, the US Secretary of Health and Human Services announced (in an FR notice) that the FDA was now charged with ensuring all organizations comply with the heretofore enacted but relatively unenforced  requirement to submit all relevant clinical trial data to http://www.clinicaltrials.gov

For further commentary on this move see the following reports from:
What is abundantly clear to me is that the FDA is left almost powerless - and if not powerless than certainly without sufficient resources - to successfully enforce its new power.  This requires collective industry leadership.  Bold, industry-initiated standards, infrastructure and old-fashioned peer pressure.

Here's what I wish.  

I wish that as a cell therapy industry we - through organizations like ISSCR, ARM, ISCT, etc and leading publishers of some of our leading journals like Regenerative Medicine, Cytotherapy, Cell Stem Cell, Stem Cells, etc - would take a leadership position on an issue like this.

I believe that as a relatively small and nascent sector of the biopharma industry we are more likely capable of collaborating on something important like this than larger, more established [entrenched] and diverse sectors.  Of course it requires the political will and cajones.

The payoff from our sector in taking a leadership role on this issue could potentially be enormous in terms of providing our sector with truly transparent and useful data.  Perhaps even more important would be the public profile such leadership would provide the sector.  Such a move requires bold leadership, pain, and cost but this is the kind of stuff that moves the needle and goes down as critical pivot points in history. 

Just my thought for the day...

--Lee

Source:
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Fortune Magazine on California Stem Cell Agency: Warm, Personal and Favorable

By Dr. Matthew Watson


California's $3 billion stem cell
research effort today garnered a handsome dollop of favorable
national news coverage– a lengthy piece in Fortune magazine that
spoke of looming stem cell cures and the leading role of the state
stem cell agency.

The article led the Fortune web page online at one point this morning and
likely will be read by tens of thousands of persons, although it was not the cover story on the print product. 
Written by a former senior editor of
the magazine, Jeffrey O'Brien of Mill Valley, Ca., the piece was warm
and personal. He began with the story of his 95-year-old
grandmother and her health issues, ranging from arthritis to macular
degeneration. And he wrote,

“The citizens of California have
spoken. If my grandmother and I had the power to get the rest of the
country to follow, we would.”

O'Brien also discussed the science and
finances of the stem cell business. He said,

“To be clear, the earliest stem cell
therapies are almost certainly years from distribution. But so much
progress has been made at venerable research institutions that it now
seems possible to honestly discuss the possibility of a new medical
paradigm emerging within a generation. Working primarily with rodents
in preclinical trials, MDs and Ph.D.s are making the paralyzed walk
and the impotent virile. A stem cell therapy for two types of macular
degeneration recently restored the vision of two women. Once they
were blind. Now they see!

“Some experts assert that AMD could
be eradicated within a decade. Other scientists are heralding a
drug-free fix for HIV/AIDS. Various forms of cancer, Parkinson's,
diabetes, heart disease, stroke, and ALS have already been eradicated
in mice. If such work translates to humans, it will represent the
type of platform advancement that comes along in medicine only once
in a lifetime or two. The effect on the economy would be substantial.
Champions of stem cell research say it would be on the order of the
Internet or even the transistor.”

O'Brien continued,

“The obstacles along the road from
lab rat to human patients are many, of course, but the biggest by far
is money. With the dramatic events in the lab, you might think that a
gold rush would be under way. That's far from true. Long time
horizons, regulatory hurdles, huge R&D costs, public sentiment,
and political headwinds have all scared financiers. Wall Street isn't
interested in financing this particular dream. Most stem cell
companies that have dared go public are trading down 90% or more from
their IPOs. Sand Hill Road is AWOL. The National Venture Capital
Association doesn't even have a category to track stem cell
investments.”

As for the California stem cell agency
itself, the article contained remarks from its Chairman J.T.Thomas,
President Alan Trounson and former chairman Robert Klein about the origins and progress of the California Institute for Regenerative Medicine (CIRM).
O'Brien wrote, 

“The $1.7 billion awarded so far has made one obvious mark on the state: a dozen gleaming research institutions. CIRM has proved adept at getting billionaires to donate funds to the cause.”

O'Brien interviewed a several
prominent businessmen who have contributed tens of millions of
dollars to stem cell research “about the prospects of a legitimate industry emerging.” One was “bond genius” Bill Gross, who has
contributed to UC Irvine. Gross replied.

“Goodness, you're talking to the
wrong guy. Our donation had nothing to do with business.”

Eli Broad, another big stem cell donor,
said pretty much the same thing. And Andy Grove, the former chairman
of Intel, was “surprisingly full of doom and gloom.” O'Brien
wrote,

“For close to two hours, Grove argues
passionately about how the FDA is enabling predatory offshore
industries by impeding progress and the many reasons financiers want
no part of stem cells. "VCs aren't interested because it's a
shitty business," he says. Big Pharma? Forget it. CIRM? "There
are gleaming fucking buildings everywhere. That wasn't necessary."
When I press him to be constructive, he wearily offers one possible
solution. Rather than courting billionaires to put their names on
buildings, we need a system of targeted philanthropy in which the 99%
can sponsor the individual stem cell lines that matter to them.”

O'Brien said, however,

“It was clear during our talk that
Grove wants an economic model for stem cell research and development
to emerge, even if he's not willing to bet money on its happening.
And that puts him in good company.”

While the Fortune article has its
negative points about stem cell research, it is about as laudatory as
it is going to get at this point for the California stem cell agency.
The piece recognizes and even celebrates much of the work of the
agency. The article clearly details the void in financing
for commercialization of stem cell research, bolstering support for
efforts like those in California. Importantly, it also helps to push
the activities of the stem cell agency more fully into the national
discussion of stem cell research and its future. That should pay off
again and again in future news coverage and also benefit the stem
cell agency as it explores the possibility of additional funding –
either private or public – after the cash for new awards runs out
in 2017.

(The story is in the Oct. 8, 2012, edition of Fortune.)

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

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$700,000 Blue-ribbon Study of CIRM All But Finished

By Dr. Matthew Watson


The $700,000 study of the $3 billion
California stem cell agency is nearly concluded and is expected to be
released sometime in November.

A draft of the report has been sent out
for “peer review” and no additional public meetings are
scheduled, according to a spokeswoman for the Institute of
Medicine(IOM)
, which is conducting the study. The IOM did not respond
to questions from the California Stem Cell Report about the number of peer reviewers or how they were selected.
The study began last year under a contract with the stem cell agency, which commissioned the effort, in
part, because agency directors hoped the findings by the blue-ribbon
panel would bolster efforts to win voter approval of another multi-billion dollar state bond issue. More recently the agency has
explored the possibility of private financing to continue operations.
The agency is expected to run out of
funds for new awards in 2017. It currently has something in the
neighborhood of $700 million for awards that is not already committed
in one fashion or another.
Christine Stencel, senior media
relations officer for the IOM, said in an email,

There will be no
further information-gathering meetings. The committee members have
finished drafting their report and it is now undergoing peer review.
Reviewers are anonymous to study staff and committee members; they
will be listed in the front matter of the report when it’s finished
and released.”

She said the stem
cell agency will not be given an opportunity to comment further.
Stencel said,

Sponsors are not
treated as peer reviewers; that is, they’re not afforded an
opportunity to comment on IOM draft reports prior to public release.
IOM is aiming for a public release in November (the exact time frame
will hinge on the duration of the peer review, which is influenced by
people’s schedules and adherence to deadlines). IOM is looking at
options for how best to hold this release, whether there will be an
event of some sort. Once plans are set, they’ll be noted on the
project web pages and IOM will alert the various stakeholders and
interested parties of the plans. The study is moving along and we’re
looking forward to the report’s debut in the not too distant
future.”

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

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