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IRSF announces translational research award funding to test potential therapeutics for Rett syndrome

Wed, 01/27/2010 - 11:23am
EurekAlert

(Cincinnati, OH) - Today, 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. IRSF is the world's largest private source of Rett syndrome research funding, supporting nearly $21 million in research programs to date. This is the first ANGEL Grant supported by IRSF in 2010.

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."

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