Kris DeVault, a Montana resident, is searching for a way to access an experimental drug for his three-year-old son, Brody, who suffers from creatine transporter deficiency (CTD). The rare genetic condition prevents the brain and muscles from properly utilizing energy, leading to significant developmental delays and physical weakness. While a French biotechnology firm, Ceres Brain Therapeutics, is developing a potential nasal spray treatment, the drug has not yet been tested in children or patients with CTD.

Montana recently updated its "right to try" laws, which were originally established in 2015 for terminally ill patients. A 2023 expansion allows individuals who are not terminally ill to seek access to unapproved drugs, provided those treatments have completed preliminary phase I clinical trials. The state has established an Experimental Treatment Review Board (ETRB) to oversee these applications, with the first reviews expected shortly.

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Despite the legislative change, practical access remains difficult. Ceres CEO Thomas Joudinaud expressed interest in the Montana framework but remains hesitant due to potential conflicts with US Food and Drug Administration (FDA) regulations. Because the drug is not registered with the FDA and is manufactured outside of its current regulatory standards, the company risks penalties that could jeopardize future US approval. DeVault has unsuccessfully sought assurances from the FDA that companies participating in the Montana program would not face repercussions.

The situation has left families navigating a complex landscape of medical ethics and regulation. While some experts, including Harvard Medical School professor Aaron Kesselheim, emphasize that phase I trials do not guarantee safety or efficacy, advocates like DeVault argue that parents should have the autonomy to pursue potential life-altering treatments for their children. Faced with the limitations of the Montana program, DeVault is now considering options in offshore clinics, such as those located in the special economic zone of Próspera, Honduras.

Source: MIT Technology Review