Scientists hope stem cell research can reverse diseases

LOS ANGELES - Two UCLA scientists were awarded more than $3.6 million in state grants this week to develop innovative tools and technologies that will help overcome the technical hurdles in advancing basic, translational and clinical stem cell research.Dr. Richard Gatti, a professor of pathology and laboratory medicine, will receive a two-year, $1.83 million grant to use Ataxia-Telangiectasia (AT), an inherited neurodegenerative disease found in children, as a model to study the mechanisms that lead to neurodegeneration and to develop a drug that can slow or halt the damage. Dr. Thomas Carmichael, an associate professor of neurology, will receive a two-year, $1.82 million grant to develop tissue bioengineering systems for a stem cell therapy to treat stroke that seeks to circumvent one major treatment bottleneck: the inability of most stem cell therapies to survive and repair the injured brain. The grants were among 19 awarded to researchers at 10 institutions by the California Institute for Regenerative Medicine, the state agency that administers Prop 71 funding for stem cell research. The grants support the development and evaluation of innovative tools and technologies that will help researchers overcome road blocks in stem cell research. The awards were given to scientists to either create new tools and technologies or expand on existing tools and technologies that have shown promise. To date, scientists at the Broad Stem Cell Research Center at UCLA have received 42 grants from the CIRM totaling more than $138.8 million. Gatti's project merges the expertise of two major research cultures - he has longstanding experience in developing a treatment for the progressive childhood disease AT - and William Lowry, an assistant professor of molecular, cellular and development biology, who has had success in converting skin cells into pluripotent stem cells that were then differentiated into cells of the nervous system. Together they hope to develop a disease model for AT. Gatti proposes to start with skin cells grown from biopsies of patients with AT who carry mutations in the ATM gene. He hopes to convert the skin cells into pluripotent stem cells capable of forming the neural cells lacking in AT patients' brains. It's presumed these neural cells need ATM protein to develop normally. Gatti will then test the efficacy of the most promising new compounds being developed over the last six years in his lab on those neural cells. There is currently no other disease model either in animals or a Petri dish evaluating the effects of potentially curative compounds on the nervous system and its development. There is no effective treatment for children with AT or other progressively-deteriorating neural disorders. If successful, Gatti's project may provide insight into at least three new areas for understanding and treating neurodegenerative diseases - providing human neural cells with specific disease-causing mutations; offering a new approach to learning how the human brain develops; and a new class of drugs that may correct the disease-causing mutations and possible reverse neurodegeneration. Stroke currently is the leading cause of adult disability. While most patients survive the initial stroke, they don't fully recover. Up to 1/3 of stroke patients end up in nursing homes or assisted living centers and experience decreased strength or lack of control of the arms and legs. There is no treatment that promotes brain repair and recovery from stroke. While recent studies in animal models show that stem cell transplantation into the brain promotes repair and recovery, no such treatment is available for humans. Three challenges must first be overcome - most of the transplanted cells die, most of the surviving cells don't interact with the surrounding brain, and the process of injecting stem cells into the brain may damage the normal brain tissue near the stroke site. Carmichael will assemble a novel investigative team and research approach to overcome these challenges. Combing the expertise of engineering, stem cell biology and stroke scientists, Carmichael hopes to develop systems to support stem cell therapy in stroke by creating a biopolymer hydrogel to provide a pro-growth and pro-survival environment for stem cells when injected with them into the brain. The hydrogel system uses biological components that mimic the normal brain environment and releases growth factors that enhance survival of the transplanted stem cells. The growth factors hopefully stimulate the normal brain to undergo repair and recovery, providing a dual mechanism for repair. This approach allows targeting of the stroke cavity for a stem cell transplant. The cavity is an ideal target for a stroke stem cell therapy as it can receive a stem cell transplant without displacing normal brain tissue. To learn more about the Broad Stem Cell Research Center at UCLA, visit

********** Published: February 03, 2011 - Volume 9 - Issue 42