Montana’s “right to try” law, expanded in 2023, could give families like the DeVaults a pathway to access experimental drugs that have not yet received U.S. approval.
Family’s fight against a rare disorder
Kris DeVault’s son Brody was born in March 2023 and soon showed signs of developmental delay. By the time Brody was two and a half, genetic testing identified creatine transporter deficiency (CTD), a condition that blocks the brain and muscles from receiving the energy they need. There is no cure, and standard treatments cannot address the underlying deficiency.
Brody’s symptoms include missing speech milestones, muscle weakness, and difficulty communicating basic needs. “He’s got no words, really,” DeVault says, noting that the child’s frustration often escalates when he cannot express discomfort. The family also worries about the narrow window of brain plasticity that early childhood provides for long‑term development.
A French biotechnology firm, Ceres Brain Therapeutics, is developing a nasal‑spray drug designed to bypass the transporter defect and deliver creatine directly to the brain. In mouse models the approach has shown promising results, and a recent phase I trial in 48 healthy adults evaluated several dose levels. The trial has not been published, and the drug has not yet been tested in patients with CTD or in children.
According to Ceres CEO Thomas Joudinaud, a phase II study targeting CTD patients and individuals with amyotrophic lateral sclerosis is planned in France. The trial, however, would likely be inaccessible to Brody, and the company cannot provide the drug through the U.S. FDA’s expanded‑access program because it has not been registered with the agency and is produced in a manner that does not meet FDA standards.
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Montana’s regulatory experiment
Montana first adopted a right to try statute in 2015, allowing terminally ill patients to request unapproved therapies after a phase I trial.
In 2023 the law was broadened to include non‑terminal conditions, provided the drug has completed preliminary phase I testing.
While the Montana law aims to balance patient autonomy with regulatory safeguards, the reality for families like the DeVaults remains uncertain. The experimental drug’s timeline suggests that even if the phase II trial succeeds, FDA approval could take several years—potentially beyond the critical developmental window for Brody.
For now, Brody’s parents continue to monitor developments and weigh the risks of pursuing treatment abroad. The outcome of Montana’s first applications could set a precedent for how other states handle similar requests, and it may influence whether biotech firms decide to participate in state‑run pathways without compromising future FDA submissions.
