IRSF announces first 2010 ANGEL Grant for testing potential Rett Syndrome therapies

ANGEL Award for novel therapies to improve breathing and other symptoms

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."

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).

Source: International Rett Syndrome Foundation

Comments

The opinions expressed here are the views of the writer and do not necessarily reflect the views and opinions of News Medical.
Post a new comment
Post

While we only use edited and approved content for Azthena answers, it may on occasions provide incorrect responses. Please confirm any data provided with the related suppliers or authors. We do not provide medical advice, if you search for medical information you must always consult a medical professional before acting on any information provided.

Your questions, but not your email details will be shared with OpenAI and retained for 30 days in accordance with their privacy principles.

Please do not ask questions that use sensitive or confidential information.

Read the full Terms & Conditions.

You might also like...
Research shows gut bacteria's role in mental resilience and reduced anxiety