The NIH is granting $35 million in funding over the next five years to support the Centers for Collaborative Research in Fragile X program, which is dedicated to investigating Fragile X syndrome-associated disorders and developing novel treatments for them. The Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), the National Institute of Mental Health, and the National Institute of Neurological Disorders and Stroke are all contributing funding and expertise.
Research teams led by the investigators below will be receiving grants:
- Kimberly M. Huber, Ph.D., at the University of Texas Southwestern Medical Center in Dallas, whose team will study brain circuits in both mouse models and people to try to determine the causes of the heightened sensitivity to sound experienced by many people with Fragile X.
- Joel D. Richter, Ph.D., at the University of Massachusetts Medical School in Worcester, in collaboration with Gary J. Bassell, Ph.D., at Emory University in Atlanta, GA, and Eric Klann, Ph.D., at New York University. Dr. Richter’s team plans to study three molecules that appear to play important underlying roles in Fragile X syndrome and could be potential targets for future drug development.
- Stephen T. Warren, Ph.D., at Emory University, whose team plans to sequence the genomes of patients with FMR1 gene mutations to identify whether additional genes may affect an individual’s likelihood of developing certain health problems associated with FMR1 mutations. Their focus will be on epilepsy in boys with Fragile X syndrome and on Fragile X-associated tremor/ataxia syndrome (which they say tends to be seen in older men) and Fragile X-associated primary ovarian insufficiency.
Since its founding in 2000, the Centers for Collaborative Research in Fragile X have reportedly advanced the field of Fragile X research with several findings, including shedding light on the basic functions of the FMR1 gene (Fragile X and its associated disorders are linked to mutations in FMR1) and on the risk of transmitting FMR1 gene mutations across generations. “Each of these centers is focused on a specific research challenge and has the promise to make a significant impact on the field in the next five years,” Tiina Urv, Ph.D., chair of the NIH Fragile X Syndrome Research Coordinating Group and a program director at the NICHD, said in a statement.
This isn't the first time the NIH has financed research into treating conditions related to Fragile X syndrome: Last year the Institutes gave SAGE Therapeutics an award said to be worth up to $10 million to support the company’s development of a drug—a Positive Allosteric Modulator targeting GABAA—that could be used to treat anxiety and social deficits in Fragile X patients.