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Children's Neurobiological Solutions, Inc. (CNS) is a national, non-profit, 501(c)(3) organization, whose mission is to orchestrate cutting-edge, collaborative research with the goal of expediting the creation of effective treatments and therapies for children with neurodevelopmental abnormalities, birth injuries to the nervous system, and related neurological problems.In addition, CNS strives to provide families and health care providers with user-friendly access to state-of-the-art information and education supporting their decision-making processes.
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Facts:Observations:
- Approximately 15 million children in the United States, between the ages of 0-19 years, are afflicted with neurological conditions that severely limit their quality of life and lifespan.
- Special education alone for these children costs society approximately 36 billion dollars annually. These costs include more personnel for learning disabled classes, transportation to out of district placements, out of district schools for more involved children, equipment, aids, etc.
- There are no known cures and limited biomedical therapeutics. The majority of present and past research and fundraising dollars focus on saving lives and supportive services such as physical therapy, special education and care giving.
- Recent advances in biomedicine, particularly in the fields of developmental neurobiology, stem cell research and genetics, has opened the gateway towards the discovery of brain repair therapies which can enhance mobility and cognition, giving quality of life and health to these children.
Resolutions:
- Almost every disease in adulthood has a pediatric correlate. Or, to put it another way, most pediatric diseases have an adult version. Many direct parallels exist between pediatric diseases and their adult counterparts. Often the pediatric version of the disease, however, has a better defined etiology ¥ for example, an identified gene or cell-type abnormality ¥ that makes it more amenable to a focused investigation.
- Information that might be gained from the pediatric diseases will undoubtedly contribute to our knowledge and treatment of the adult counterpart whose etiology might be more heterogeneous, complex, less well-understood and categorized, or vexed by many more confounding variables attributable to lifestyle and environment. This is particularly true for neurodegenerative and demyelinating diseases, spinal cord degenerations, movement disorders and even stroke.
- Taking advantage of these exciting new fields, Children's Neurobiological Solutions has developed a world-renowned, cross-institutional Scientific Advisory Board of neuroscientists and clinicians, collaborating to achieve aggressive research goals. CNS research goals are focused on the discovery and development of therapeutics that will improve the functional abilities and health of these children, enhancing their quality of life and reducing the burdens of their caretakers and society.
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- Genetic / Metabolic Causes Of Neurological Disease:
A significant number of neurological handicaps in children may be ascribed to defects in genes that then allow waste products to build up in the brain or that preclude useful proteins from being made. Other gene defects prevent newborn cells from finding their right locations in the brain or making proper connections.
Other genetic defects cause cells to die prematurely. Although some of these diseases have names and their genes are known (e.g., Tay-Sachs disease), other diseases do not yet have their genes identified (e.g. certain forms of autism). In some, defective genes have been identified but it is uncertain why that mutation should cause a neurological problem because the role of the gene is not yet well-understood (e.g., Rett Syndrome).
To know how to use genetic information therapeutically, one must know what that gene does and how to compensate for its absence or dysfunction. Sometimes, even when we think we understand a disease and we know the genetic defect involved and we believe we understand why that gene's defect should cause a neurological problem, we realize that we have an oversimplified view of the brain because simply replacing that gene or missing gene product (known as a "protein") does not result in a cure (e.g., Krabbe's disease).
In those cases we need to understand how the genes and the brain interact in order to design more effective therapies.
- Basic Cellular Developmental Neurobiology:
This category includes:
- Studying neuronal and glial differentiation, i.e., how an immature cells knows what kind of brain cell it needs to become and how the wide variety of cell types in the nervous system come to exist.
- Studying potential causes for subtle dysgenesis (i.e. how a cell of a given type knows where it is supposed to reside, what connections it is supposed to make, where it is supposed to make those connections and with whom and, importantly, why a cell might fail to do any of these things correctly.
- Precursor cell biology and potential future cellular therapeutics (i.e., how one can study the most immature cells in the nervous system, understand the signals to which they respond, and mobilize such cells (either in the brain or grown in a dish for later reimplantation).
- Acquired Neurological Dysfunction:
Some neurological handicaps result from an injury or problem that happens to a fundamentally normal nervous system. These may include infections, trauma, inflammation, autoimmune attack, lack of blood flow and/or oxygen (called "hypoxia-ischemia" ¥ a stroke-like injury).
Some forms of cerebral palsy (including periventricular leukomalacia) may fit into this category and may be amenable to cellular/molecular interventions. Interestingly, some problems may look to be "acquired" but really occur because the nervous system is not really normal but is somehow more susceptible to an "insult" that it should have normally been able to ward off or sustain without a problem.
- Transitional Therapeutics:
This category would include attempts to devise therapeutic interventions that might be regarded as "stop-gap" measures; i.e., treatments that might diminish the degree of a handicap, improve the quality of day-to-day living, or even "buy time" and prolong survival until a more definitive but longer-to-develop treatment has been devised.
These "transitional" therapeutics might include bioengineering and/or combined biological-electronic approaches that might enhance communication or motor performance. Novel pharmacological therapies might also fit in this category.
- Discretionary:
This category affords discretion to the board of directors and scientific advisory board to support and encourage novel approaches to elucidating etiology, prevention, amelioration, or treatment of neurological dysfunction. Diseases whose etiology is not yet well enough defined to be able to be placed cleanly in any of the above categories might be funded here.
Such complex neurological entities as "autism", "dyslexia", "attention deficit disorder" might be addressed in this category if the applications are scientifically rigorous and the experimental plans meritorious and likely to yield useful data. Pre-application letters of intent would be absolutely necessary for this category and only modest amounts of "seed" money would be allocated initially.
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Children's Neurobiological Solutions Scientific Advisory Board:
- Steven A. Goldman, M.D., Ph.D.
Dr. Goldman holds a B.A. degree from University of Pennsylvania, a Ph.D. in Neurobiology from The Rockefeller University and an M.D. from Cornell University Medical College. He is presently a professor of Neurology and Neuroscience at Cornell University Medical College in New York City where his research focuses on neuronal plasticity and regeneration. He was elected in 2001 to the American Society of Clinical Investigation.
- Harley Kornblum, M.D., Ph.D.
Dr. Kornblum is currently an Associate Professor of Molecular and Medical Pharmacology and Pediatrics at the UCLA School of Medicine. He is a Pediatric Neurologist, a member of the Crump Institute for Molecular Imaging, The Brain Research Institute and a Principal Investigator of the UCLA/Department of Energy Laboratory of Structural Biology and Nuclear Medicine. His degrees include a B.A. in Biology from Washington University and a Ph.D., and M.D. from UC Irvine. His research interests include the biology and use of CNS stem cells, brain development, brain repair and epilepsy.
- Jeffrey Macklis, M.D., D. HST
Research in Dr. Macklis' laboratory is directed toward the cellular repair of complex cerebral cortex circuitry, focusing on neuronal differentiation, development of connectivity, precursor transplantation, and molecular manipulation of endogenous neural precursors within murine neocortex. He is an Associate Professor of Neurology [Neuroscience] at Harvard Medical School, in the Division of Neuroscience, Children's Hospital, and Program in Neuroscience, Harvard Medical School. He is a core faculty member of the Harvard University Graduate Program in Neuroscience and M.D.-Ph.D. Program, and he is a faculty member of the Harvard-M.I.T Division of Health Sciences and Technology. He is also a staff neurologist and Co-Director of the Parkinson's Disease and Related Disorders Program at Brigham and Women's Hospital.
- Ronald D.G. McKay, Ph.D.
Dr. McKay holds a B.S. and Ph.D. from the University of Edinburgh where his doctoral work examined DNA organization and chromosome structure. He has conducted research at Cold Spring Harbor Laboratory and in 1984 joined the faculty at MIT where he continued to examine neuronal organization. He accepted his current position in 1993 as chief of the Laboratory of Molecular Biology, at the National Institute of Neurological Disorders and Stroke (NINDS). His laboratory is focused on understanding stem cell differentiation.
- Mark Noble, Ph.D.
Dr. Noble is currently a Professor of Genetics in the Center for Cancer Biology at the University of Rochester School of Medicine and Dentistry. His research is focused on stem cell biology, regeneration in the central nervous system, redox modulation of precursor cell function, developmental disorders, brain tumor biology and adverse effects of chemotherapy on neural stem cell populations. He received his Ph.D. from Stanford University in 1977.
- Mahendra Rao, M.D., Ph.D.
Dr. Rao received his M.D. in 1983 from Grant Medical College in India and his Ph.D. from Cal Tech in 1990. He is currently the Section Chief for the Stem Cell Program at the National Institutes of Aging Laboratory of Neuroscience at the National Institutes of Health (NIH). His laboratory is interested in defining the molecular and cellular interactions that instruct a pluripotent cell to differentiate into cells restricted to a particular phenotype.
- Jeffrey D. Rothstein, M.D. Ph.D.
Dr. Rothstein is a Professor of Neurology and Neuroscience and a faculty member of the Graduate Program in Cellular and Molecular Medicine. His research is specialized in neuromuscular disease with particular interest in Amyotrophic Lateral Sclerosis (ALS), idiopathic stupor, epilepsy and motor neuron degeneration. He received his M.D. from University of Illinois College of Medicine and his Ph.D. from University of Illinois Health Sciences Center.
- Evan Snyder, M.D., Ph.D.
Dr. Snyder received his Ph.D. in neurobiology and M.D. from the University of Pennsylvania concurrently. He is presently Assistant Professor of Neurology at Harvard Medical School and Assistant in Neurology, Medicine and Neurosurgery at Children's Hospital, Boston. His clinical interests are in neonatal neurology and neurointensive care. His laboratory research is concerned with the study of factors determining the differentiation of neural stem cells and progenitors with a view towards understanding plasticity in mammalian neural development. This work also focuses on the transplantation of neural stem cells as a tool for gene therapy and repair in various models of CNS dysfunction and neurodegeneration.
- Clive Svendsen, Ph.D.
Dr. Svendsen is currently the Director of the Stem Cell Research Program at the University of Wisconsin-Madison as well as a Professor of Anatomy and Neurology. He received his Ph.D. from Cambridge University in 1991. His research interests include: central nervous system development with particular interest in factors affecting proliferation and differentiation of human neural stem cells and translating this knowledge to treat neurological trauma and disease. By producing stem cells which make therapeutic proteins, he also hopes to provide novel ways of delivering such factors directly to brain cells. His laboratory is presently studying genes and proteins which control the differentiation of human neural stem cells. In parallel, human neural stem cells have been transplanted into animal models of Parkinson's and Huntington's disease, Multiple Sclerosis and stroke to assess functional outcome.
- James Thomson, Ph.D., VDM
James A. Thomson is a University of Wisconsin-Madison developmental biologist in the Department of Anatomy in the School of Medicine who also serves as the chief pathologist at the Wisconsin Regional Primate Research Center on the UW-Madison campus. He received his doctorate in veterinary medicine in 1985 and his doctorate in molecular biology in 1988, both at the University of Pennsylvania. Since joining the Wisconsin Regional Primate Research Center, he has conducted pioneering work in the isolation and culture of non-human primate and human embryonic stem cells, undifferentiated cells that have the ability to become any of the cells that make up the tissues of the body. Dr. Thomson directed the group that reported the first isolation of embryonic stem cell lines from a non-human primate in 1995, work that led his group to the first successful isolation of human embryonic stem cell lines in 1998. The major focus of his lab is on understanding how primate embryonic stem (ES) cells choose between self-renewal, death, and differentiation to specific lineages.
- Angelo Vescovi, Ph.D.
Dr. Vescovi currently serves as Co-Director of Research for the Institute for Stem Cell Research in Milan, Italy. He received his Ph.D. in Biological Sciences from the University of Milan in 1987. Dr. Vescovi spent two years as a post-doctoral fellow at the Department of Pathology of the University of Calgary, Canada working at the isolation of neural stem cells. From 1993 through 1999 he was the supervisor of two strategic projects on "Neural Stem Cell Transplantation For Neurodegenerative Disorders" funded the Italian Ministry of Health while also acting as vice-director of research at Neurospheres Ltd, Calgary, Canada from 1995 through 1998. From 1995 through 2001 he has been an adjunct professor at the Course of Physiology at the University of Milan.
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Children's Neurobiological Solutions (CNS) Board of Directors:
- Fia Richmond, Founder
Fia is a determined mother of Palmer Clay Richmond, who remains undiagnosed with a brain injury. Palmer's condition, and the struggles of other children like him, has motivated Fia to start Children's Neurobiological Solutions. After an exhaustive search and inquiry into the traditional medical community that offered few answers, her relentless search led her to the emerging field of biomedical research and Drs. Evan Snyder and Jeffrey Macklis. Fia holds a M.A. in clinical psychology from Pacifica Graduate Institute and currently resides in Santa Barbara, California.
- Phillip H. Richmond, Co-Founder
Phil, Fia's husband and father of Palmer Clay is a director of The Richmond Organization, Inc. and the Essex Music Group London. Phil's constant support of his wife Fia, his desire to help Palmer, along with his business experience, provides essential energy and guidance in moving CNS forward in its mission.
- Jeffrey Macklis, M.D., Ph.D.
Dr. Macklis is an Associate Professor of Neurology [Neuroscience] at Harvard Medical School, in the Division of Neuroscience, Children's Hospital, and Program in Neuroscience, Harvard Medical School. He is a core faculty member of the Harvard University Graduate Program in Neuroscience and M.D.-Ph.D. Program, and he is a faculty member of the Harvard-M.I.T Division of Health Sciences and Technology. He is also a staff neurologist and Co-Director of the Parkinson's Disease and Related Disorders Program at Brigham and Women's Hospital. Research in Dr. Macklis' laboratory is directed toward the cellular repair of complex cerebral cortex circuitry, focusing on neuronal differentiation, development of connectivity, precursor transplantation, and molecular manipulation of endogenous neural precursors within murine neocortex.
- M. Morton Mitchell, Treasurer
Mitch is grandfather to Molly, a beautiful child with an undiagnosed genetic disorder. Mitch's love for his granddaughter and family ignite him with a special interest in the research supported by Children's Neurobiological Solutions. He is President and CEO of First Components International, which produces and markets semiconductor components and silicon materials. His is also President of VLS Associates, who provides consulting services to the semiconductor industry. Mitch's strong corporate background, along with his commitment to family, is a key asset to CNS. Mitch presently resides in Fountain Hills, Arizona.
- Evan Snyder, M.D., Ph.D.
Dr. Snyder received his Ph.D. in neurobiology and M.D. from the University of Pennsylvania concurrently. He is presently Assistant Professor of Neurology at Harvard Medical School and Assistant in Neurology, Medicine and Neurosurgery at Children's Hospital, Boston. His clinical interests are in neonatal neurology and neurointensive care. His laboratory research is concerned with the study of factors determining the differentiation of neural stem cells and progenitors with a view towards understanding plasticity in mammalian neural development. Dr. Snyder's work also focuses on the transplantation of neural stem cells as a tool for gene therapy and repair in various models of central nervous system dysfunction and neurodegeneration. Dr. Snyder's love of children, his devoted interest and extensive clinical experience with the pediatric diseases, combined with his in-depth knowledge of developmental neurobiology, is an invaluable asset to achieving CNS's goals.
- Sue Ellen Strong, B.S., M.B.A., Secretary
Sue Ellen is the mother of Katherine, who sustained significant brain damage after contracting viral encephalitis at 4 weeks of age. Sue Ellen received an M.B.A. from Rutgers University and holds a B.S. in General Science from Villanova University. Prior to Katherine's birth, Sue Ellen worked as an account manager for General Electric Company. She currently resides in Gillette, New Jersey with husband Carl, Katherine, and son Christopher.
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- Children's Neurobiological Solutions
1726 Franceschi Road
Santa Barbara, CA 93103
Phone: 866-267-5580 or 805-965-8838
Fax: 805-963-6633
Email: cnsfoundation@home.com
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