Montana's right to try law has become a possible, and still highly uncertain, route for Kris DeVault as he tries to obtain an experimental drug for his 3-year-old son, Brody. DeVault told MIT Technology Review that Brody has creatine transporter deficiency, a rare genetic condition that leaves the brain and muscles short of the energy source they need to develop.
Brody was born in March 2023. DeVault said his son later began missing developmental milestones involving speech, movement and coordination. A genetic test, when Brody was about two and a half, identified creatine transporter deficiency.
There is no cure for the condition. DeVault described Brody as curious and affectionate, but said he has very limited speech and struggles to express basic needs such as hunger, thirst, discomfort or pain. He also said Brody has muscle weakness and tires easily when walking.
DeVault's main fear is timing. Early childhood is a period of rapid brain development, and he worries that waiting years for a drug approval process could leave Brody beyond a useful developmental window.
What is Montana's right to try law?
Montana has allowed terminally ill patients to seek access to unapproved drugs under a right-to-try law since 2015. A 2023 expansion extended that path to some patients who are not terminally ill, if the experimental treatment has completed an initial phase 1 clinical trial. A later law clarified how clinics could sell and administer those treatments, and Montana's health department has finalized rules for those clinics, according to MIT Technology Review.
The state has also created an experimental treatment review board to evaluate requests for access to unapproved therapies. That board is expected to consider its first two applications in the coming weeks.
For families such as the DeVaults, the statute offers a legal opening. It does not force a drug company to participate, make an unapproved drug available, or settle what federal regulators might do later.
The drug Brody's father wants is still early
Ceres Brain Therapeutics, a French biotech company, is developing a nasal-spray treatment intended for people with creatine transporter deficiency. Creatine normally helps supply energy to brain cells, but people with the disorder cannot move creatine into the brain properly. Ceres CEO Thomas Joudinaud told MIT Technology Review that the company's drug is designed to bypass that transport problem and deliver creatine to the brain.
Joudinaud said the company has seen encouraging results in mice. Ceres has also completed an unpublished phase 1 trial that tested different doses in 48 healthy adult volunteers. The drug has not been tested in children or in people with creatine transporter deficiency.
Joudinaud is planning a phase 2 trial in France for people with creatine transporter deficiency and people with amyotrophic lateral sclerosis. DeVault said Brody is unlikely to be able to join that trial.
Ceres also cannot provide the treatment to Brody through the US Food and Drug Administration's expanded-access process, Joudinaud said, because the drug has not been registered with the FDA and its current manufacturing process does not meet FDA rules.
Why the Montana path is not straightforward
Ceres could seek permission from Montana's review board to sell the treatment to Brody's parents through a clinic in the state. Joudinaud called Montana's approach pragmatic and potentially suited to the drug, but said he is wary of provoking problems with the FDA before any future US approval effort.
DeVault has asked FDA staff for written assurance that companies using Montana's program would not be punished later in the federal approval process. He said he has not obtained that assurance.
He is also considering Próspera, a private city and special economic zone in Roatán, Honduras, where a clinic sells unproven stem-cell and gene therapies, according to MIT Technology Review. Many scientists have warned against offshore clinics offering unproven treatments.
Aaron Kesselheim, a Harvard Medical School professor who studies health policy and drug regulation, told MIT Technology Review that early trials do not establish whether a drug works, and phase 1 testing does not prove a treatment is safe for the patients who may later seek it. His view is that patients deserve rigorous assessment so they know what risks they are taking and what they are paying for.
DeVault rejects the idea that regulators should block him from taking that risk for his son. His argument is blunt: adults can spend money on dangerous or irrational choices in plenty of legal ways, and he wants the same freedom to pay for a treatment he believes might change Brody's life.
This story draws on original reporting from MIT Technology Review.
