A Montana law allows access to unapproved drugs for non-terminally ill patients, but developers remain hesitant to participate due to potential FDA regulatory concerns.
Key facts
- •Brody DeVault, 3, has creatine transporter deficiency, a rare condition that limits brain and muscle energy.
- •Ceres Brain Therapeutics is developing a nasal spray that has completed a phase I trial in 48 healthy adults.
- •Montana's 2023 law allows non-terminally ill patients to access drugs that have passed phase I clinical trials.
- •The FDA has not provided assurances that companies participating in Montana's program will be exempt from future regulatory penalties.
- •Kris DeVault is considering seeking treatment at an offshore clinic in Próspera, Honduras, due to the lack of domestic options.
Kris DeVault is seeking an experimental nasal spray for his three-year-old son, Brody, who suffers from creatine transporter deficiency. While a new Montana law theoretically allows access to drugs that have cleared phase I trials, the French developer of the treatment remains reluctant to participate. The company fears potential backlash from the U.S. Food and Drug Administration, leaving the family to consider other options as they race against the child's critical brain development window.
By the numbers
The Search for Treatment
Brody DeVault was diagnosed with creatine transporter deficiency after missing developmental milestones in speech and movement. The condition prevents the brain and muscles from receiving necessary energy. Ceres Brain Therapeutics is developing a nasal spray to deliver creatine directly to the brain, which has shown promise in mice and completed a phase I trial with 48 healthy adult volunteers. However, the drug has not been tested in children or patients with the condition.
Montana's Legislative Path
Montana expanded its 'right to try' laws in 2023 to include patients who are not terminally ill, provided the drugs have passed phase I trials. The state has established an experimental treatment review board to oversee applications, with the first reviews expected in the coming weeks. Despite this, Ceres CEO Thomas Joudinaud remains cautious, citing concerns about how participating in the program might affect future FDA approval processes for the drug.
Regulatory and Ethical Concerns
Experts, including Harvard Medical School professor Aaron Kesselheim, warn that phase I trials do not confirm a drug's safety or efficacy. DeVault has sought written assurances from the FDA that companies participating in the Montana program will not be penalized, but he has received no such commitment. Consequently, the family is exploring other options, including clinics in the special economic zone of Próspera in Honduras.
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This article was independently rewritten by ManyPress editorial AI from reporting originally published by MIT Technology Review.
