Montana right to try law: A desperate dad’s last hope for his rare‑disease son

Montana right to try law opens a narrow path for families like the DeVaults

When Kris DeVault’s three‑year‑old son Brody was diagnosed with creatine transporter deficiency, the family faced a treatment dead‑end. The Montana right to try law, revised in 2023, now offers a legal channel to request unapproved drugs after only a phase‑I trial, a hope that DeVault is desperate to seize.

Brody, who cannot speak and struggles with muscle weakness, needs a therapy that can deliver creatine directly to his brain. French biotech Ceres Brain Therapeutics is testing a nasal‑spray formulation that has cleared a small phase‑I safety study in healthy adults, but it has never been used in children or in patients with the disorder.

“I see my son’s future in a bottle that isn’t even on the market,” DeVault told reporters.

The company plans a phase‑II trial in France, yet U.S. regulators have not cleared the product, and the FDA would block any export that does not meet its manufacturing standards. DeVault has petitioned the agency for a “safe‑harbor” guarantee, to no avail.

Montana’s newly minted experimental treatment review board (ETRB) could, in theory, approve Ceres’s drug for local administration. The board will evaluate the first two applications in the coming weeks, a process Reuters describes as “unprecedented in U.S. health policy.”

Critics warn that phase‑I data do not establish safety, let alone efficacy, and that “off‑shore” clinics in places like Honduras lure desperate families with unproven stem‑cell claims. Harvard’s drug‑policy scholar Aaron Kesselheim cautions, “Patients need rigorous assessment before risking money and health.”

DeVault retorts, “I’m an adult; I can gamble in Vegas or buy a rifle. Why can’t I decide whether to try a therapy that might change my son’s life?” The debate pits parental autonomy against the precautionary principle, a tension that the ↓ 3-year‑old’s fate now embodies.

Reported by: Elena Rostova

Socio-Economic Trends Analyst