Scientists in Cambridge, Massachusetts are beginning a clinical trial of a potential medication designed to correct a central neurochemical defect underlying Fragile X syndrome.
The study will evaluate safety, tolerability, and optimal dosage in healthy volunteers. The new trial tests Seaside Therapeutics' novel compound, STX107, that selectively and potently targets the synaptic connections in the brain. It will focus on a compound, designated STX107, that selectively inhibits one type of mGluR receptor, mGluR5. Evidence in mice with Fragile X-like symptoms suggests that reducing levels of mGluR5 can restore normal synaptic protein synthesis and improve function.
The initial phase 1 study of STX107 will involve healthy volunteers. If results suggest that the medication is safe and tolerable, the study will progress to a phase 2 test of dosage and efficacy in adults with Fragile X syndrome. If STX107 shows promise in adults, the compound will be assessed for pediatric safety (with funding from the Best Pharmaceuticals for Children Act through NICHD) prior to initiating clinical trials in children.
Read the full press release here and read the background to the current series of drug trials on the FRAXA website here