An analysis of DNA from thousands of patients might offer new targets for developing drugs for autism and other neurological disorders, including attention-deficit/hyperactivity disorder and schizophrenia.
The study, published in Nature Communications, compared more than 6,700 patients with autism to 12,500 control subjects and identified three gene families that play functional roles in the disorder -- the CALM1 network, the MXD-MYC-MAX gene network, and the GRM gene family.
The research team is planning a clinical trial in selected ADHD patients of a drug that activates the glutamate receptor metabotropic (GRM) pathway.
"If drugs affecting this pathway prove successful in this subset of patients with ADHD, we may then test these drugs in autism patients with similar gene variants," said study researcher Hakon Hakonarson, MD, PhD, director of the Center for Applied Genomics at the Children's Hospital of Philadelphia.
A large new analysis of DNA from thousands of patients has uncovered several underlying gene networks with potentially important roles in autism, attention-deficit/hyperactivity disorder and schizophrenia.
“Neurodevelopmental disorders are extremely heterogeneous, both clinically and genetically,” said study leader Hakon Hakonarson, MD, PhD, director of the Center for Applied Genomics at The Children’s Hospital of Philadelphia (CHOP).