IRSF announces translational research award funding to test potential therapeutics for Rett syndrome
ANGEL Award for novel therapies to improve breathing and other symptoms
The International Rett Syndrome Foundation (IRSF) announced that it will commit $446,000 in 2010 to fund a large translational research program to test potential therapeutics for Rett Syndrome in mouse models of the disease. The funds will be awarded through the IRSF's new Advanced Neurotherapeutic Grant of Excellence (ANGEL) mechanism which seeks to provide funds for translational research.
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.
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."
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