New $7.7 million grant to propel search for drugs for rare brain disorder

Children born with a damaged gene needed for healthy brain development, SYNGAP1, experience seizures, sensory processing disorders, difficulty speaking, intellectual disability, and autism-like behaviors. It’s a condition without any treatments, one that’s hard both on parents and children, said Gavin Rumbaugh, Ph.D., a neuroscientist at The Herbert Wertheim UF Scripps Institute for Biomedical Innovation & Technology.

Rumbaugh and a team of scientists from the institute have been awarded a five-year grant from the National Institute of Mental Health worth $7.7 million to work toward a treatment. Their goal is to create a pill that restores healthy SYNGAP1 gene production, thereby boosting neuroplasticity, or the ability of the brain to form circuits and connections. The scientists’ hope is that their work will improve the quality of life for both children and adults with the disorder, Rumbaugh said. Collaborators on the grant include Wertheim UF Scripps scientists Courtney Miller, Ph.D., and Ted Kamenecka, Ph.D.

“Seizures can be induced in these children by something as simple as eating the wrong texture of food,” Rumbaugh said. “The benefit of a medication you could take as a pill is that the dose could be adjusted as the children grow.”