Ceres could also apply to Montana’s ETRB to sell its experimental treatment to Brody’s parents via a clinic in the state. But Joudinaud is reluctant, at least for the time being. While he thinks that Montana’s setup is “very interesting and very pragmatic” and “suitable for our drug,” he’s worried about getting on the wrong side of the FDA.
DeVault has been pleading with FDA staffers for a written statement essentially promising that biotech companies participating in Montana’s program won’t be penalized later on, especially when they eventually try to get their drugs approved in the US. But he hasn’t made any progress.
Now he’s looking beyond Montana. He’s considering accessing treatment in Próspera, a private city and “special economic zone” in Roatán, Honduras, where a clinic sells unproven stem-cell and gene therapies, among others.
Many scientists have cautioned against the use of such “offshore” clinics. Even when it comes to Montana, scientists, bioethicists, and health law experts will caution that phase I clinical trials don’t prove a drug is safe. And they certainly don’t prove a drug’s efficacy, either.
When I spoke to Aaron Kesselheim, a professor of medicine at Harvard Medical School with expertise in health policy and drug regulation, about the Montana law earlier this week, he made his concerns clear. “Patients who want these kinds of treatments deserve them to be rigorously assessed so that [they] can better understand what they’re getting themselves into, and what they’re paying their hard-earned money for.”
But DeVault pushes back on these arguments. “I’m a full-grown human being,” he says. “I’m capable of going to Vegas right now … blowing it all on the poker table, [or] I can go to the gun shop and buy a silenced [semi-automatic rifle] … how come I can’t make a decision to purchase a potential treatment that might change the entire trajectory of my son’s life?”
This article first appeared in The Checkup, MIT Technology Review’s weekly biotech newsletter. To receive it in your inbox every Thursday, and read articles like this first, sign up here.
