On Monday, September 23, 2024, NPR featured a report about an experimental drug, zatolmilast, that is showing life-changing potential for at least one young man with Fragile X syndrome.
A Year of Transformation for Jason
“I have a different child in my house... He gets himself to work, he walks downtown, gets his haircut, gets lunch. He wouldn't have done any of that before.” - Lizzie Mazzola
Lizzie Mazzola's son Jason completed a double-blind, placebo-controlled clinical trial of zatolmilast last year. He now takes the drug as part of an open label extension, which is offered to everyone who completes the trial.
Before starting the medicine, Jason, now 24, faced severe challenges with anxiety and communication, needing constant supervision. Since taking zatolmilast, he has flourished. “It helps me focus a lot, helps me get more confident, more educated,” says Jason.
Zatolmilast: New Hope Even for Adults
Other parents of individuals with Fragile X are also reporting big changes with zatolmilast. These personal stories are backed by data from an earlier FRAXA-funded phase 2 clinical trial of zatolmilast in 30 adult males with Fragile X which demonstrated improvements in memory, language, and an iPad-based measure of overall intelligence.
“Many people assume that intellectual impairment is permanent,” said Dr. Michael Tranfaglia, FRAXA’s chief scientific officer. “These families’ experiences with zatolmilast suggests otherwise. They dispel any lingering doubts that Fragile X or intellectual disabilities in general are potentially treatable.”
Promising Treatments Pack the Pipeline
If the current pivotal trials succeed, zatolmilast has the potential to be the first drug approved by the FDA for Fragile X syndrome. FRAXA is proud to have moved this work forward alongside dedicated scientists like Dr. Elizabeth Berry-Kravis and the teams at Tetra Therapeutics and Shionogi.
Zatolmilast is just one of many drugs in the pipeline for Fragile X. It appears to be a very good drug, and we are optimistic that it will be approved soon, but there is much more to come. Because no single drug is right for everyone, FRAXA and our academic researchers and pharmaceutical partners are developing more medicines to improve the lives of individuals with Fragile X.
Zatolmilast Clinical Trials Are Enrolling Individuals with Fragile X
These clinical trials are enrolling males ages 9-45 with Fragile X at clinics across the US.
Thank you for your continued support of FRAXA Research Foundation!
