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Stem cells improve visual function in blind mice

ScienceDaily (Oct. 1, 2012) An experimental treatment for blindness, developed from a patient's 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 eye's retina.

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

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

The primary job of the retina cells is to nourish the light-sensing cells and protect the fragile cells from excess light, heat, and cellular debris. If the retina cells die -- which happens in macular degeneration and retinitis pigmentosa -- the photoreceptor cells degenerate and the patient loses vision. Macular degeneration is a leading cause of vision loss in the elderly, and it is estimated that 30 percent of people will have some form of macular degeneration by age 75. 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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Stem cells improve visual function in blind mice

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Houston Stem Cell Summit Announces Extraordinary Lineup of Keynote Speakers

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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Houston Stem Cell Summit Announces Extraordinary Lineup of Keynote Speakers

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World Renowned Scientists and Advocates to Celebrate and Shine Light on Stem Cell Breakthroughs

IRVINE, CA--(Marketwire - Oct 1, 2012) - Oct. 3 marks International Stem Cell Awareness Day, a global celebration where leading scientists, researchers and supporters will acknowledge the scientific advances of stem cell research and its ability to potentially treat a variety of diseases and injuries in the 21st century. This dedicated community is committed to unlocking the potential of stem cells and has made significant strides since the discovery of a method to grow human stem cells less than 15 years ago.

"This is a critical and historic time for stem cell research," said Peter Donovan, Ph.D., director, Sue & Bill Gross Stem Cell Research Center, UC Irvine. "We're literally on the brink of developing new treatments for some of the world's most devastating diseases and injuries. The act of simply raising awareness about this research is one of the best things people can do to help accelerate the process. This event is a great opportunity for everyone to help spread the word and build momentum through a timely mass effort."

Scientists at UC Irvine and other research facilities around the globe continue to work diligently to develop therapies to treat life threatening and debilitating conditions such as Alzheimer's disease, multiple sclerosis, macular degeneration, cancer, Huntington's disease, Parkinson's disease, brain disorders and paralysis caused by spinal cord injuries. These efforts continue to give hope to millions who suffer from these devastating conditions by offering revolutionary treatments and potential cures.

There are several research programs taking place at the Sue & Bill Gross Stem Cell Research Center at UC Irvine that continue to break down barriers and open doors to new treatments for major diseases and injuries:

Spinal Cord and Traumatic Brain Injuries: Neurobiologist Hans Keirstead, Ph.D., as well as husband and wife scientists Aileen Anderson, Ph.D., and Brian Cummings, Ph.D., are conducting stem cell studies to develop treatments for the more than 1.3 million Americans who suffer from spinal cord injuries. Their advancements have led to the world's first clinical trial of human neural stem cell-based therapy for chronic spinal cord injuries (Anderson/Cummings) and the first FDA approved clinical trials using embryonic stem cells (Keirstead). Their research is significant because no drug or other forms of treatment have been able to restore function for those suffering from paralysis. In addition, Cummings and Anderson are applying their stem research to traumatic brain injury, a leading cause of death and disability worldwide, especially in children and young adults.

Alzheimer's Disease: An estimated 35 million people worldwide suffer from Alzheimer's disease, five million of whom live in the U.S. Frank LaFerla, Ph.D., director of UC Irvine's Institute for Memory Impairments and Neurological Disorders, and Matthew Blurton-Jones, Ph.D., of the Sue & Bill Gross Stem Cell Research Center, UC Irvine, have shown for the first time that neural stem cells can rescue memory in mice with advanced Alzheimer's disease, raising hope for a potential treatment in humans. Their work is expected to move to clinical trials in less than five years.

Huntington's Disease: Huntington's disease is a degenerative and ultimately fatal brain disorder that takes away a person's ability to walk, talk and reason. It affects about 30,000 people in the U.S. with another 200,000 or more likely to inherit the disorder. Leslie Thompson, Ph.D., and her team of researchers are currently investigating new stem cell lines and techniques to support the area of the brain that is susceptible to the disease with the hope of developing a cure for future generations.

Macular Degeneration, Retinitis Pigmentosa and Inherited Blindness: Henry Klassen, M.D., Ph.D. has focused his stem cell research on regenerating damaged retinal tissue to restore sight to people suffering from retinitis pigmentosa (an inherited form of degenerative eye disease) and macular degeneration which usually affects older people and leads to loss of vision. Macular degeneration affects millions of Americans. His work hopes to find cures and treatments for corneal and retinal eye disease.

New Website Helps Spread the Word Online To commemorate International Stem Cell Awareness Day and encourage support of stem cell research, an interactive website has been created. Advocates are asked to visit http://www.StemCellsOfferHope.com and share online a wide range of key facts, downloadable images and links to other valuable resources within their social networks.

International Stem Cell Awareness Day Events at UC Irvine The Sue & Bill Gross Stem Cell Research Center at UC Irvine will celebrate International Stem Cell Awareness Day by hosting three special events. An open house will take place on Oct. 1 for high school students. A UC Irvine student, faculty and staff open house will take place on Oct. 2. Finally, an all-day science symposium on Oct. 3 will feature a "Meet the Scientist" interactive forum. The forum and symposium are open to all UC Irvine scientists, clinicians, graduate students, post-docs and members of the community. To RSVP for any these events or for more information, include the name of the event in the subject line and email stemcell@research.uci.edu.

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World Renowned Scientists and Advocates to Celebrate and Shine Light on Stem Cell Breakthroughs

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Researchers find multiple similarities between cancer cells and induced pluripotent stem cells

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

Contact: Charles Casey charles.casey@ucdmc.ucdavis.edu 916-734-9048 University of California - Davis Health System

(SACRAMENTO, Calif.) UC Davis investigators have found new evidence that a promising type of stem cell now being considered for a variety of disease therapies is very similar to the type of cells that give rise to cancer. The findings suggest that although the cells -- known as induced pluripotent stem cells (iPSCs) -- show substantial promise as a source of replacement cells and tissues to treat injuries, disease and chronic conditions, scientists and physicians must move cautiously with any clinical use because iPSCs could also cause malignant cancer.

The article, "Induced pluripotency and oncogenic transformation are related processes," is now online in the journal, Stem Cells and Development.

"This is the first study that describes the specific molecular pathways that iPSCs and cancer cells share from a direct comparison" said Paul Knoepfler, associate professor of cell biology and human anatomy, and principal investigator of the study. "It means that much more study is required before iPSCs can be used clinically. However, our study adds to a growing knowledge base that not only will help make stem cell therapies safer, but also provide us with new understandings about the cancer-causing process and more effective ways to fight the disease."

Since 2007, cell biologists have been able to induce specialized, differentiated cells (such as those obtained from the skin or muscle of a human adult) to become iPSCs. Like embryonic stem cells, iPSCs are a type of stem cell that is able to become any cell type. This "pluripotent" capability means that iPSCs have the potential of being used in treatments for a variety of human diseases, a fundamentally new type of clinical care known as regenerative medicine.

iPSCs are considered particularly important because their production avoids the controversy that surrounds embryonic stem cells. In addition, iPSCs can be taken from a patient's own skin and induced to produce other needed tissues, thereby evading the possibility of immunologic rejection that arises when transplanting cells from a donor to a recipient. In contrast to therapies based on ES cells, iPSCs would eliminate the need for patients to take immunosuppressive drugs.

Earlier research indicated that both ES cells and iPSCs pose some health risks. Increasing evidence suggests that pluripotency may be related to rapid cellular growth, a characteristic of cancer. iPSCs, as well as embryonic stem cells, are well known by scientists to have the propensity to cause teratomas, an unusual type of benign tumor that consists of many different cell types. The new UC Davis study demonstrates for the first time that iPSCs -- as well as ES cells -- share significant similarities to malignant cancer cells.

The investigators compared iPSCs to a form of malignant cancer known as oncogenic foci that are also produced in laboratories; these cell types are used by medical researchers to create models of cancer, particularly sarcoma. Specifically, the scientists contrasted the different cells' transcriptomes, comprised of the RNA molecules or "transcripts." Unlike DNA analysis, which reflects a cell's entire genetic code whether or not the genes are active, transcriptomes reflect only the genes that are actively expressed at a given time and therefore provide a picture of actual cellular activity.

From this transcriptome analysis, the investigators found that the iPSCs and malignant sarcoma cancer cells are unexpectedly similar in several respects. Genes that were not expressed in iPSCs were also not expressed in the cancer-generating cells, including many that have properties that guide a cell to normally differentiate in certain directions. Both cell types also exhibited evidence of similar metabolic activities, another indication that they are related cell types.

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Researchers find multiple similarities between cancer cells and induced pluripotent stem cells

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Multiple similarities discovered between cancer cells and induced pluripotent stem cells

ScienceDaily (Sep. 28, 2012) UC Davis investigators have found new evidence that a promising type of stem cell now being considered for a variety of disease therapies is very similar to the type of cells that give rise to cancer. The findings suggest that although the cells -- known as induced pluripotent stem cells (iPSCs) -- show substantial promise as a source of replacement cells and tissues to treat injuries, disease and chronic conditions, scientists and physicians must move cautiously with any clinical use because iPSCs could also cause malignant cancer.

The article, "Induced pluripotency and oncogenic transformation are related processes," is now online in the journal, Stem Cells and Development.

"This is the first study that describes the specific molecular pathways that iPSCs and cancer cells share from a direct comparison" said Paul Knoepfler, associate professor of cell biology and human anatomy, and principal investigator of the study. "It means that much more study is required before iPSCs can be used clinically. However, our study adds to a growing knowledge base that not only will help make stem cell therapies safer, but also provide us with new understandings about the cancer-causing process and more effective ways to fight the disease."

Since 2007, cell biologists have been able to induce specialized, differentiated cells (such as those obtained from the skin or muscle of a human adult) to become iPSCs. Like embryonic stem cells, iPSCs are a type of stem cell that is able to become any cell type. This "pluripotent" capability means that iPSCs have the potential of being used in treatments for a variety of human diseases, a fundamentally new type of clinical care known as regenerative medicine.

iPSCs are considered particularly important because their production avoids the controversy that surrounds embryonic stem cells. In addition, iPSCs can be taken from a patient's own skin and induced to produce other needed tissues, thereby evading the possibility of immunologic rejection that arises when transplanting cells from a donor to a recipient. In contrast to therapies based on ES cells, iPSCs would eliminate the need for patients to take immunosuppressive drugs.

Earlier research indicated that both ES cells and iPSCs pose some health risks. Increasing evidence suggests that pluripotency may be related to rapid cellular growth, a characteristic of cancer. iPSCs, as well as embryonic stem cells, are well known by scientists to have the propensity to cause teratomas, an unusual type of benign tumor that consists of many different cell types. The new UC Davis study demonstrates for the first time that iPSCs -- as well as ES cells -- share significant similarities to malignant cancer cells.

The investigators compared iPSCs to a form of malignant cancer known as oncogenic foci that are also produced in laboratories; these cell types are used by medical researchers to create models of cancer, particularly sarcoma. Specifically, the scientists contrasted the different cells' transcriptomes, composed of the RNA molecules or "transcripts." Unlike DNA analysis, which reflects a cell's entire genetic code whether or not the genes are active, transcriptomes reflect only the genes that are actively expressed at a given time and therefore provide a picture of actual cellular activity.

From this transcriptome analysis, the investigators found that the iPSCs and malignant sarcoma cancer cells are unexpectedly similar in several respects. Genes that were not expressed in iPSCs were also not expressed in the cancer-generating cells, including many that have properties that guide a cell to normally differentiate in certain directions. Both cell types also exhibited evidence of similar metabolic activities, another indication that they are related cell types.

"We were surprised how similar iPSCS were to cancer-generating cells," said Knoepfler. "Our findings indicate that the search for therapeutic applications of iPSCs must proceed with considerable caution if we are to do our best to promote patient safety."

Knoepfler noted, for example, that future experimental therapies using iPSCs for human transplants would most often not involve implanting iPSCs directly into a patient. Instead, iPSCs would be used to create differentiated cells -- or tissues -- in the laboratory, which could then be transplanted into a patient. This approach avoids implanting the actual undifferentiated iPSCS, and reduces the risk of tumor development as a side effect. However, Knoepfler noted that even trace amounts of residual iPSCs could cause cancer in patients, a possibility supported by his team's latest research.

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Multiple similarities discovered between cancer cells and induced pluripotent stem cells

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The great stem cell dilemma

By Jeffrey M. O'Brien, contributor

Stem cells stored in liquid nitrogen at Advanced Cell Technology in Marlborough, Mass.

FORTUNE -- Imagine yourself the proud but rueful owner of an ancient Jaguar. Every day you dread the uncertainty that comes with trying to get from here to there -- there, more often than not, being the shop. No sooner does one ailment find repair than another appears. At best, it's a slow, uncomfortable ride. Lonely too. There's really no one around who fully understands your plight.

That is how Patricia Riley describes life in a 95-year-old body. Riley, who reached that milestone birthday last St. Patrick's Day, lives alone in the same 1,100-square-foot house in Plainfield, Conn., that she's called home for 64 years, having survived her husband (heart disease), a daughter (breast cancer), and every friend she ever had. "All the people I knew have all gone, Jeffrey," she says in a quivering voice laced with melancholy. "They've all died. I go to church and I never see people my age." Her remaining family includes two daughters, five grandchildren, and eight great-grandchildren, including my two young sons. In a nod to her French-Canadian heritage, we call her Mme.

Mme attributes her longevity to good genes, but she clearly owes a debt to modern medicine. Over the years she's had a cholecystectomy, a hysterectomy, esophageal surgery, a stroke, and ulcerative colitis. Lately she relies on a cane and a walker, and her daily regimen includes pain pills for arthritis, two inhalers for asthma, high-blood-pressure meds, a statin, vitamins, digestion aids, and an anti-anxiety drug that she calls "my nerve pill." Her vision also comes courtesy of medical science. Three years ago Mme was diagnosed with a form of age-related macular degeneration, or AMD, a disease of the back of the retina that is the leading cause of vision loss in the developed world. The ophthalmologist gave her a choice: a needle into her eyeballs every six weeks, or blindness. Mme opted for the injections and now receives shots of an off-label cancer drug called Avastin, which has demonstrated efficacy in halting the progress of her type of AMD. Holding the ailment at bay is all she can hope for. "I'll have to go for as long as I live," she says. "It's just a treatment -- it's not a cure."

Treatments, not cures. This, in a nutshell, is the MO of our health care system, and it's precisely the reason that regenerative medicine -- and stem cell therapy in particular -- has been the subject of so much hope and hype over the past decade or so. Stem cell therapies promise to empower a body to fight ailments by enabling it to build new parts. Think about growing new neurons or heart tissue. Think about the difference between perpetually slathering that old Jag with Bondo and having it heal itself overnight in the garage.

MORE:Stem cell dollars: California leads the way

While stem cells have ignited plenty of religious outrage and political grandstanding, behind the headlines the underlying science has been advancing the way science often does -- by turns slowly and dramatically. 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.

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.

Big Pharma would seem to be the most obvious benefactor. The drug companies understand the complexities (and billion-dollar outlays) involved in bringing therapies to market. A few drug companies have kicked the tires on stem cells over the years, but waiting for them to undo the current model is akin to banking on Big Oil to rethink energy. They may do it, but it's unlikely to be by choice. Which leaves stem cell researchers begging for state and federal grants at a time scientific funding is under siege.

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The great stem cell dilemma

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