Kris De Vault’s three‑year‑old son Brody has a rare creatine transporter deficiency that stunts brain and muscle growth. No cure exists, but a nascent drug from Ceres Brain Therapeutics—still in animal and small human trials—may help. The drug, delivered as a nasal spray, has shown promise in mice and a phase‑I study of 48 healthy adults, yet it cannot be prescribed outside a formal trial.

Montana’s expanded right‑to‑try law now lets patients who are not terminally ill apply for unapproved treatments that have passed phase I. An experimental treatment review board will evaluate requests, potentially allowing Ceres to supply Brody through a state clinic. The company is cautious, fearing FDA penalties, while De Vault seeks assurances that participation won’t jeopardize future approval.

De Vault also looks abroad to a clinic in Honduras, where unproven therapies are sold, though experts warn such offshore options lack rigorous safety data. The debate highlights the tension between desperate families and the need for thorough drug evaluation.

This story first appeared in MIT Technology Review’s The Checkup newsletter.