Montana’s Expanded ‘Right to Try’ Law Faces Hurdles in Providing Access to Experimental Drugs

Kris DeVault, a Montana father, is seeking experimental treatment for his three-year-old son, Brody, who suffers from a rare genetic condition known as creatine transporter deficiency. The disorder prevents the brain and muscles from effectively utilizing energy, leading to significant developmental delays and physical challenges. While a French biotechnology firm, Ceres Brain Therapeutics, is developing a nasal spray designed to bypass this deficiency, the drug remains in early-stage development and has not been tested in children or patients with the condition.

Montana recently finalized regulations for an expanded "right to try" law, which allows non-terminally ill patients to access experimental drugs that have completed phase I clinical trials. The state has established an experimental treatment review board to oversee these applications. However, despite the legal framework, access remains difficult. Ceres CEO Thomas Joudinaud expressed interest in the program but remains hesitant, citing concerns that participating could negatively impact the company's future relationship with the US Food and Drug Administration (FDA).

The FDA currently does not recognize the program, and the drug in question is not manufactured in compliance with federal regulations. DeVault has unsuccessfully sought assurances from the FDA that companies participating in the Montana initiative would not face future penalties. Consequently, he is exploring alternative options, including seeking treatment at offshore clinics in Honduras, a practice that many medical experts and bioethicists warn against due to the lack of proven safety and efficacy.

Critics, including Harvard Medical School professor Aaron Kesselheim, emphasize that phase I trials do not establish the safety or effectiveness of a treatment. They argue that patients require rigorous assessment to understand the risks and costs involved. DeVault, however, maintains that families should have the autonomy to make medical decisions for their children, stating, "I’m capable of going to Vegas right now... blowing it all on the poker table... how come I can’t make a decision to purchase a potential treatment that might change the entire trajectory of my son’s life?"

Source: MIT Technology Review