(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."
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.
About the International Rett Syndrome Foundation
IRSF is the world's leading private funder of basic, translational and clinical Rett syndrome research, funding over $21M in high-quality, peer-reviewed research grants and programs to date. Annually, IRSF hosts the world's largest gathering of global Rett researchers and clinicians to establish research direction and priorities while exchanging ideas and the most recent information. IRSF is the most comprehensive non-profit organization dedicated to providing thorough and accurate information about Rett syndrome, offering informational and emotional family support, and stimulating research aimed at accelerating treatments and a cure for Rett syndrome and related disorders. IRSF has earned Charity Navigator's most prestigious 4 star rating. To learn more about IRSF and Rett syndrome, visit www.rettsyndrome.org or call IRSF at 1-800-818-RETT (7388).
About Case Western Reserve University School of Medicine
Founded in 1843, Case Western Reserve University School of Medicine is the largest medical research institution in Ohio and is among the nation's top medical schools for research funding from the National Institutes of Health. The School of Medicine is recognized throughout the international medical community for outstanding achievements in teaching. The School's innovative and pioneering Western Reserve 2 curriculum interweaves four themes--research and scholarship, clinical mastery, leadership, and civic professionalism--to prepare students for the practice of evidence-based medicine in the rapidly changing health care environment of the 21st century. Eleven Nobel Laureates have been affiliated with the school. Annually, the School of Medicine trains more than 800 M.D. and M.D./Ph.D. students and ranks in the top 25 among U.S. research-oriented medical schools as designated by U.S. News & World Report "Guide to Graduate Education." The School of Medicine's primary affiliate is University Hospitals Case Medical Center and is additionally affiliated with MetroHealth Medical Center, the Louis Stokes Cleveland Department of Veterans Affairs Medical Center, and the Cleveland Clinic, with which it established the Cleveland Clinic Lerner College of Medicine of Case Western Reserve University in 2002. http://casemed.case.edu.