Montana right-to-try rules raise hopes for rare disease treatment

Kris DeVault is seeking access to an experimental treatment for his son, Brody, who was born in March 2023 and later diagnosed with creatine transporter deficiency, a rare condition that deprives the brain and muscles of energy needed for development. Brody struggles with speech, communication, movement, and muscle strength, and DeVault worries that waiting years for a conventional approval path could miss a critical window for neurological development.

Ceres Brain Therapeutics in France is developing a nasal-spray treatment designed to deliver creatine to the brain. CEO Thomas Joudinaud says mouse studies have been promising, and the company recently completed an unpublished phase I trial testing doses in 48 healthy adult volunteers. The drug has not been tested in children or in people with CTD, and Ceres says it cannot use the FDA’s expanded access pathway because the drug is not registered with the agency and its current manufacturing does not meet FDA rules.

Montana’s right-to-try framework, first adopted in 2015 and expanded in 2023, could theoretically offer another route for patients who are not terminally ill if a treatment has been through preliminary phase I clinical trials. State rules now create an experimental treatment review board to evaluate applications, but Ceres remains wary of possible FDA repercussions. Experts warn that early trials do not establish safety or efficacy, while DeVault argues families should be allowed to accept the risk when no approved treatment exists.