Translational Research Award Funding To Test Potential Therapeutics For Rett Syndrome
The program will be carried out by a team of researchers at Case Western Reserve University School of Medicine, led by David M. Katz, Ph.D., Professor of Neurosciences. Building on previous proof-of-concept studies conducted in Dr. Katz's laboratory, the new program will evaluate existing drugs, as well as molecules under development, which may improve breathing problems - one of the more serious complications of the disease - as well as other aspects of Rett syndrome. The researchers will systematically assess several classes of compounds, with a particular focus on improving neuronal signaling mediated by Brain Derived Neurotrophic Factor (BDNF), a key molecule implicated in Rett syndrome.
Dr. David Katz, principal investigator on the study commented "These kinds of translational studies are only possible because of a tremendous amount of basic science research, conducted in many different laboratories, which help us identify potential therapeutic targets. We are deeply grateful to IRSF for their support and encouragement."
IRSF Chief Scientific Officer Dr. Antony Horton stated "This study represents a new opportunity to develop potential treatments for Rett syndrome and demonstrates our continued commitment towards advancing new therapeutic strategies to treat and ultimately reverse this devastating disease."
Part of the study will examine drugs that are currently in development for other neurological disorders including Alzheimer's and Huntington's disease which will be provided by Dr. Frank Longo Professor of Neurology and Neurological Sciences at Stanford University. Dr. Longo founded the biotechnology company PharmatrophiX to further develop and commercialize these drugs. Said Dr. Longo "Studies by Dr. Katz and others indicate that compromised BDNF expression might contribute to Rett syndrome. We have developed the first small molecules that mimic a specific domain of the BDNF protein and activate the BDNF receptor; TrkB. Dr. Katz's studies provide an exciting opportunity to determine the therapeutic potential of these molecules for Rett syndrome."
Research on Rett syndrome in Dr. Katz's laboratory is also supported by the National Institute of Neurological Disease and Stroke, including funds received through the American Recovery and Reinvestment Act of 2009 (ARRA).
About Rett Syndrome
Rett syndrome (RTT), a developmental neurological disorder, occurs almost exclusively in females. RTT results in severe movement and communication problems following apparently normal development for the first six months of life. Characteristic features of the disease include loss of speech and purposeful hand use, repetitive hand movements, abnormal walking, abnormal breathing, slowing in the rate of head growth and increased risk of seizures. Current treatment for girls with RTT includes physical and occupational therapy, speech therapy, and medication for seizures. There is no known cure for RTT. In 2007, researchers heralded a major breakthrough by reversing RTT symptoms in mouse models. RTT is considered a "Rosetta Stone" that is helping scientists understand multiple developmental neurological disorders, and shares genetic links with other conditions such as autism and schizophrenia.
Source: Kathryn Kissam
International Rett Syndrome Foundation
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