Category Archives: Stem Cell Treatment


Provocative Biotech Exposition Reveals Significant Advancements in Stem Cell Research, Immunotherapy, Targeted …

STAMFORD, CT--(Marketwire - Oct 3, 2012) - Noble Financial Capital Markets (Noble) announced today that it has released a full high definition video catalog of its Life Sciences Exposition, BIOX, an investor exposition that was held at the University of Connecticut in Stamford, CT on September 24-25, 2012. In addition to the participation of more than two dozen biotechnology companies, the event featured world renowned key opinion leaders Dr. Mark Noble (University of Rochester), R. Phil Greenberg (Fred Hutchinson Cancer Research Center), Dr. Jeffrey Infante (Sarah Cannon Research Institute), and Dr. Charles Vacanti (Harvard Medical School).

Opening remarks were provided by Dr. Sharon White, Director of the UCONN Stamford campus and Richard Blumenthal, United States Senator for Connecticut. "Washington is dysfunctional and broken," Blumenthal said. "The good news is that scientific advancements and research offer a real opportunity to reach across the aisle; this is an area that should inspire bipartisan cooperation."

Overwhelmingly, the message coming from the Exposition was one of encouragement that we are making great strides in the effective treatment of catastrophic disease, in particular cancers. Determining genetic differences in tumors, which has posed challenges for biomarkers, has improved significantly with advancements in molecular biology. Novel and approved targeted agents is the next rationale step in matching tumor types with appropriate therapies; a developmental step that large pharmaceutical companies have already taken.

Dr. Noble, who led a panel moderated by Noble Financial Senior Analyst, Nathan Cali and consisting of executives from Cytomedix, Cytori, MediStem, Mesoblast and NeoStem, expressed optimism that stem cell therapy will continue to grow exponentially. "Stem cells are a natural cascade offering the potential to treat significant unmet medical needs," he said."The path to better cancer treatment comes from a clearer understanding of cancer stem cell biology."

Rahul Jasuja, Noble Financial's Managing Director of Biotechnology Research moderated the panel which focused on immunotherapy and vaccines. Executives from Argos, Galena, Inovio, NewLink and Prima Biomed joined Dr. Phil Greenberg (referred to as Dr. T-Cell) to debate the challenges and advancements in the understanding of how the immune system is suppressed by cancer cells. While a consensus was not reached in terms of specific methods to harness the immune system, all agreed that the next generation of immunotherapy approaches will aim to "realign" the immune system to destroy cancer.

Jules Msing, Chairman of Noble's Scientific Advisory Board and the former head of Global Drug Licensing for Johnson & Johnson addressed attendees at the opening session and again on day two of the Exposition. "The hope is great that all of the technologies and innovations that will be discussed here today will have a huge impact on healthcare around the world," he said. "This, however, is reliant on the underlying assumption that society's acceptance of healthcare innovation and our willingness to invest in it will continue into the future," he added. Msings' presentation to life science corporate executives centered on the question of why some biotech companies can attract the attention of large pharma while others cannot and why many licensing executives in large pharma are unable to convince their R&D groups or executive committees to make substantial investments in these companies. Mr. Msing negotiated deals with various companies such as Vertex Pharmaceuticals, Genentech, OSI Pharmaceuticals, Bristol Meyers Squibb, CTI Inc., Regeneron, Alkermes, Enzon Pharmaceuticals, GTx Inc, Alliance Pharmaceuticals, Allos Therapeutics, Somatogen, Morphotek, Edison Pharmaceuticals, etc.

Based upon the success of this event, Noble Financial has begun planning its second Life Sciences Exposition for the fall of 2013. Noble's Ninth Annual large-format investor conference that will feature 150 public companies is scheduled for January 22-23, 2013 at the Hard Rock Hotel in Fort Lauderdale.

Complete HDVideo webcast catalog available at: http://www.nobleresearch.com/BioExposition.htm

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Provocative Biotech Exposition Reveals Significant Advancements in Stem Cell Research, Immunotherapy, Targeted ...

New stem cell research could bring choices to heart patients

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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New stem cell research could bring choices to heart patients

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

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

Blind Mice Get Experimental Stem Cell Treatment For Blindness

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

Educational Programs Offered as Part of Cryo-Save´s Cord Blood Awareness Months

ZUTPHEN, the Netherlands, October 2, 2012 /PRNewswire/ --

Cryo-Save Group promotes the awareness of cord blood storage and stem cell therapies at several local educational events held throughout Europe.

Cryo-Save continues its Cord Blood Awareness Months celebration by sponsoring educational events in the Netherlands, Italy and Spain during the months of October and November. It is events like these that Cryo-Save hopes to bring awareness of and research advancements for the stem cell industry.

Stem cells are becoming ever more important in the medical field as a way to treat a broad variety of malignant and non-malignant diseases. The U.S. National Marrow Donor Program sites cord blood as treatment for multiple myeloma, severe aplastic anaemia, beta thalassemia major, SCID and other inherited immune system disorders, and leukaemias and lymphomas. Patients suffering from sickle cell anaemia have been considered cured after being treated with stem cells.[1] Over 4,000 clinical trials using cord blood stem cells are taking place to treat diseases such as cerebral palsy, diabetes and autism with many more potential clinical trials continuing to develop.

"Cryo-Saves efforts to inform the medical professionals and community members about advances in regenerative medicine means that patients suffering from diseases treatable with stem cells can also become better informed, " says Dr. Cherie Daly, Medical Affairs Manager Cryo-Save. "Having a series of events and programs as part of Cord Blood Awareness Months makes an even stronger impact on the meaningfulness of this research and its application." Cryo-Save Group will continue its commitment to promoting the storage of cord blood and stem cells even after Cord Blood Awareness Months by offering several customer related promotions.

Cryo-Save Italy will hold a special meeting for midwives about stem cells, applications and Cryo-Saves storage services on October 2. Dr. Stefano Grossi, Cryo-Save Italy Scientific Director, will be there to present on stem cell applications. During the month of October Cryo-Save Italy will also offer several parent-to-be courses on similar topics in places all around Italy: Brescia, Venice, Bologna, Milan and others.

Midwives in the Netherlands are invited on October 4 to visit the Cryo-Save Laboratory in Niel, Belgium for a unique behind the scenes tour of the ultra-modern facility - an opportunity not available at most other cord blood banks. Attendees will have a chance to be given an update from Dr. Sally Sennitt, Cryo-Save Lab Director about the current applications of stem cells, treatable diseases and new clinical trials. Staff from Cryo-Save, the Netherlands will also report on the position and procedure of stem cell storage in the Netherlands and have a group discussion on the topic. "This event is our chance to give midwives information on the importance of stem cell storage and give them tools for helping parents become better informed of this option, " says Erica Dikkeboom, Cryo-Save Nederland.

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1. University of Illinois at Chicago (2012, June 18). Chicago woman cured of sickle cell disease. ScienceDaily. Retrieved September 12, 2012, from http://www.sciencedaily.com/releases/2012/06/120618194714.htm

Crio-Cord, Cryo-Saves subsidiary in Spain, will hold a stem cell therapy symposium in Madrid on October 16 focusing on cutting edge therapies using umbilical cord stem cells and will also include current investigations using stem cells that are taking place in Madrid. The symposium will be attended by national and international speakers and is the first of its kind to take place in Madrid.

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Educational Programs Offered as Part of Cryo-Save´s Cord Blood Awareness Months

FDA Approves Stemedica Phase II Clinical Trial for Acute Myocardial Infarction With Ischemia Tolerant Mesenchymal Stem …

SAN DIEGO, Calif., Oct. 2, 2012 (GLOBE NEWSWIRE) -- via PRWEB - Stemedica Cell Technologies, Inc., a leader in adult allogeneic stem cell manufacturing, research and development, announced today that the U.S. Food and Drug Administration (FDA) approved its application for an Investigational New Drug (IND) to assess the clinical effects of Stemedyne-MSC (Stemedica's human bone marrow-derived ischemia tolerant mesenchymal cells) in subjects with a myocardial infarct. The Phase IIa double-blinded randomized clinical trial will study approximately forty (40) patients. All patients will initially receive standard care including percutaneous transluminal coronary angioplasty (PTCA) and stenting and, upon completion, will be randomized to receive Stemedyne-MSC intravenously or placebo.

The clinical trial will address the prevalence of cardiovascular disease estimated to carry a global disease burden in excess of $400 billion each year. More than one million patients undergo PTCA and stenting in the Untied States annually; another 800,000 have the procedures each year in Europe.

Nabil Dib, M.D., MSc., F.A.C.C., Director of Cardiovascular Research at Mercy Gilbert and Chandler Regional Medical Centers, and an Associate Professor of Medicine and Director of Clinical Cardiovascular Cell Therapy at the University of California, San Diego, will serve as the principal investigator of the FDA-approved study. Dr. Nib commented, "We've learned from bench top research that not all stem cells are created equally. We believe that the ischemic tolerance of Stemedica's MSCs and the robustness of their protein array will translate into significant patient benefits post myocardial infarction."

Stemedica's interest in this indication was triggered by a successful randomized study in acute myocardial infarction conducted by the National Scientific Medical Center (NSMC) in Astana, Kazakhstan using Stemedyne-MSCs. The study was conducted under clinical protocol and in compliance with the ICH-E6 (Good Clinical Practice) guidelines and local laws. All patients signed an informed consent. Nineteen (19) patients in this study received Stemedyne-MSCs after PTCA and stenting. Administration of Stemedyne-MSC resulted in a statistically-significant decrease in inflammation as judged by the level of C-reactive protein, significant decrease in end-systolic and end-diastolic volume of left ventricle, as well as significant increase in the left ventricular ejection fraction (LVEF) from 38.4% to 54.7% at 6 months post administration, bringing this parameter to a normal range for healthy individuals (50-65%).

Professor Daniyar Jumaniyazov, M.D. Ph.D., principal investigator of the NSMC study commented, "The stem cell transplantation was safe and the procedure was well tolerated. No product-related adverse events were reported. Treatment of patients in this study resulted in improvement of overall and local contractive myocardium functions and also normalization of systolic and diastolic filling of the left ventricle as compared to the control group. Based upon the safety and efficacy results, we will soon conduct a Phase III myocardial infarct clinical trial at the NSMC with Stemedica's ischemia tolerant mesenchymal stem cells."

Lev Verkh, Ph.D., Stemedica Chief Regulatory and Clinical Development Officer commented, "Stemedica's FDA submission included data from the NSMC clinical trial, the results of which were also reported at the annual American College of Cardiology meeting in April, 2012. These results contrasted with reports, at the same conference, of minimal improvement in studies with autologous stem cells. In addition to the United States sites, the study will be duplicated at leading hospitals in Europe, Asia and the Middle East." With regard to the spectrum of stem cell treatment for cardiovascular disease, Dr. Verkh noted that, "Stemedyne-MSC has been approved for the treatment of chronic heart failure at Hospital Angeles, Tijuana, Mexico by COFEPRIS (the Mexican equivalent of the FDA)."

Jackie See, M.D., F.A.C.C., founder of interventional cardiology at the University of California, Irvine, noted, "In the days and weeks following a myocardial infarction we may have the ability to intervene with stem cells to minimize scarring, enhance the amount of functional heart tissue, and restore the microcirculation. Stemedica's ischemia tolerant mesenchymal stem cells are ideal for this purpose. I can foresee the day when all coronary stenting is accompanied by stem cell injection. It is not unreasonable to postulate that the anti-inflammatory and anti-fibrotic effects of the mesenchymal stem cells may have an impact on the incidence of restenosis, a common condition caused by blockage of the stents."

The Stemedyne-MSC product is uniquely manufactured to contain increased amounts of the important growth factors that combat ischemic damage. According to Nikolai Tankovich, M.D., Ph.D., President and Chief Medical Officer of Stemedica, "Our ischemia tolerant MSCs secrete increased amounts of vascular endothelial growth factor (VEGF), which is necessary for new blood vessel development and stromal cell-derived factor (SDF), which is responsible for rescuing dying cells. Stemedyne-MSCs also demonstrate significantly higher migratory abilities. As a company we are unique in our unparalleled scalability, with our master bank at two passages and the cells that go into patients having only been expanded four times. We have the ability to treat more than 500,000 patients with cells created from a single organ donation."

Stemedyne-MSC is one of the three adult allogeneic stem cell products developed by the Company. Other products include Stemedyne-NSC neural human stem cells and Stemedyne-RPE, retinal progenitor epithelial cells available in early 2013. All Stemedica products are unique in their ability to tolerate ischemic conditions.

About Stemedica Cell Technologies, Inc. http://www.stemedica.com

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FDA Approves Stemedica Phase II Clinical Trial for Acute Myocardial Infarction With Ischemia Tolerant Mesenchymal Stem ...

RBCC: Could Stem Cells Hold the Key to Treating Traumatic Brain Injuries?

NOKOMIS, Fla.--(BUSINESS WIRE)--

As part of Rainbow Coral Corp.s mission to deliver effective new cures for traumatic brain injury, the company is investigating promising research on the potential of stem cell therapy to improve the lives of millions suffering from the affliction.

Scientists within the U.S. medical community have begun to see positive results from the treatment of patients with traumatic brain injury (TBI) through the use of stem cells. Significant improvements are seen between three to six months after treatment in brain injury patients.

RBCC is working hard to capitalize on the growing demand for effective treatments for TBI, Parkinsons and other neurological health issues. RBCC is continuing discussions with the license holders for a NASA-developed bioreactor that assists in the expansion of adult stem cells. Such treatments could give RBCC access to markets in excess of $100 billion.

Rainbow BioSciences is dedicated to developing new medical and research technology innovations to compete alongside companies such as Amgen Inc. (NASDAQ:AMGN),Cell Therapeutics, Inc. (CTIC), Abbott Laboratories (NYSE:ABT) andAffymax, Inc.(NASDAQ:AFFY).

For more information on Rainbow BioSciences, please visit http://www.rainbowbiosciences.com/investors.

Follow us on Twitter atwww.twitter.com/RBCCinfo.

About Rainbow BioSciences

Rainbow BioSciences is a division ofRainbow Coral Corp.(OTCBB:RBCC). The company continually seeks out new partnerships with biotechnology developers to deliver profitable new medical technologies and innovations. For more information on our growth-oriented business initiatives, please visit our website at [www.rainbowbiosciences.com]. For investment information and performance data on the company, please visitwww.RainbowBioSciences.com/investors.

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Pediatric Cancer Researcher Recruited to Louisville, Bringing New Treatments for Deadly Childhood Cancers

LOUISVILLE, Ky.--(BUSINESS WIRE)--

Kenneth Lucas, M.D., has joined University of Louisville Department of Pediatrics as division chief of Pediatric Hematology-Oncology and Stem Cell Transplantation, and Kosair Childrens Hospital as Chief, Pediatric Hematology/Oncology. Formerly a Pennsylvania State University researcher and pediatric cancer physician, Lucas brings with him the Phase 1 Trial of a vaccine to prevent recurrence of neuroblastoma and sarcoma, among the most common and deadly of all childhood cancers.

The trial, which began at Penn State a year and one-half ago, is generating referrals from around the world. With Lucass appointment, The Addison Jo Blair Cancer Center at Kosair Childrens Hospital will be the primary site for this trial. Thus far, he has recruited about half the patients allowed for this study.

Having Dr. Lucas join our faculty and take over leadership of our pediatric cancer program is a step forward for the children with cancer in our community and a tribute to the acclaim our cancer specialists have already earned, said Gerard P. Rabalais, M.D., chairman, University of Louisville Department of Pediatrics. His innovative childhood cancer treatment will significantly advance our mission to bring new clinical pediatric knowledge to the bedside and provide excellent healthcare to the regions children.

Kosair Childrens Hospitals cancer center is rated among the best in the nation by U.S. News and World Report. Dr. Lucass new therapies could propel us to the very top of that list. Currently, there are only three or four centers nationwide doing pediatric cancer vaccine studies, so we are very excited to be able to add this treatment, said Thomas D. Kmetz, division president, Womens and Childrens Services, president, Kosair Childrens Hospital.

Lucas replaces Salvatore Bertolone, M.D., who has been named University of Louisville Department of Pediatrics Chief Clinical Operations Officer. Bertolone will continue to see pediatric cancer patients in addition to overseeing the operations of the departments 13 subspecialty practices.

With the addition of Dr. Lucass vaccine studies, we can offer children in Kentucky and beyond a new way to treat cancer, potentially boosting a childs immune response to attack cancer cells. Now children with cancer from Kentucky can receive the latest state-of-the-art care in their own community, where they can rely on family members and friends at a very turbulent time, Bertolone noted.

Phase 1 Clinical Trial: Decitabine and Vaccine Therapy for Relapsed Neuroblastoma and Sarcoma

This leading edge therapy combines two techniques. First, the patients blood is collected and separated so that a vaccine against specific cancer proteins can be grown. It takes about a month to grow the vaccine.

The child is then given a low dose of chemotherapywhich causes the body to produce more of the targeted cancer proteinsand vaccinated with the protein-specific serum. The vaccine prompts the childs own immune system to attack and kill the cancer proteins and tumor cells. The treatment typically takes four months to complete.

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Pediatric Cancer Researcher Recruited to Louisville, Bringing New Treatments for Deadly Childhood Cancers

Quebec lowers maximum age for stem-cell donor registration

Hma-Qubec has lowered the maximum age of those who can sign up to be stem-cell donors from 50 to 35, raising concerns among some people in minority ethnic communities that the policy will limit their chances of finding a match if they get certain forms of cancer.

The organization that runs Quebec's blood and tissue bank made the change quietly last fall, only mentioning it in a newsletter it published in February.

The director of Hma-Qubec's stem cell donor registry, Diane Roy, said transplant doctors generally prefer younger donors because their stem cells have an increased chance of survival for recipients. Plus, it costs nearly $500 to test each potential donor, which there's no point in spending on older donors if the doctors who are using the stem cells don't want them.

Hma-Qubec also maintains that while in the rest of Canada, people can still register to be stem cell donors up to age 50, many European countries cut off registration at age 35.

However, in France and Belgium, the maximum age is still 50, while in Britain it's 40.

Hma-Qubec's new policy is already turning away potential donors of stem cells.

Steve Bonspiel, a 36-year-old Mohawk man, attended a special stem cell donor drive a few weeks ago to help find a match for a cancer patient from the Kahnawake First Nation. Bonspiel said he was surprised when Hma-Qubec told him he couldn't be a donor, but he pushed and eventually was allowed to provide a sample though other, older Mohawks were turned away.

The new policy will make it especially harder for people from smaller ethnic communities to find a donor match, he said.

"For Mohawks and for native people, our pool all of a sudden is a lot smaller," Bonspiel said. "For anybody who is suffering from leukemia who needs a bone marrow transplant, all of a sudden their primary pool has been reduced.

"There's only so many Mohawk people, there's only so many native people. So we have a special DNA makeup, so now they have to go to other places, elsewhere across the country, across the province. There's not a lot of people who want to be donors."

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Quebec lowers maximum age for stem-cell donor registration

Immune system harnessed to improve stem cell transplant outcomes

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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Immune system harnessed to improve stem cell transplant outcomes