Kris DeVault’s toddler has a rare genetic disease. He’s desperate to try an experimental drug, even though it has not been through clinical trials.
Kris DeVault is desperate.
His son, Brody, was born in March 2023. It wasn’t long before he started to show signs of developmental delay, says DeVault. As time went on, Brody started missing key milestones in speech, movement, and coordination, he says.
When Brody was around two and a half years old, a genetic test revealed creatine transporter deficiency—a rare condition in which the brain and muscles lack the energy they need to develop.
There are no cures for Brody’s condition. But DeVault has learned of a company developing a drug that might help. That drug is still in the early stages of development and has only been tested in animals and a small number of healthy adults. Doctors can’t prescribe it.
DeVault knows the drug might not work. But he’s doing all he can to access it regardless. And a new law in Montana could make it easier for people in his position to get access to treatments—at least in theory.
Today, Brody is three years old. His dad describes him as a happy, curious, and loving little boy who wants to learn. But Brody struggles to communicate. “He’s got no words, really,” says DeVault. “He wants to communicate more than he’s able to … which then turns into frustration.”
It’s difficult for Brody to tell his parents whether he’s hot, cold, hungry, thirsty, uncomfortable, or even in pain, says DeVault. He recently found Brody standing on an anthill in the backyard, being bitten by red ants. “These fire ants were just going to town on his feet … and he was just looking,” he says.
Brody has muscle weakness too. “He can’t move very fast, he doesn’t have a ton of strength … and it takes a lot of energy for him to walk balanced,” says DeVault. “His arms are skinnier than [those of] his nine-month-old sister.”
It’s concerning, but DeVault is most worried about Brody’s neurological development. Toddlers’ brains are exceptionally “plastic”—the first years of a child’s life are thought to be crucial for long-term brain development.
A biotechnology company in France is working on a drug to help people like Brody. Creatine usually provides brain cells with energy. People with creatine transporter deficiency (CTD) can’t get creatine into the brain.
The team at Ceres Brain Therapeutics is developing a treatment designed to bypass this issue and effectively deliver creatine directly to the brain. So far, the team has seen promising results in mice, says Ceres CEO Thomas Joudinaud.
The company also recently completed a phase I clinical trial that involved testing various doses of the drug, which is delivered as a nasal spray, in 48 healthy adult volunteers. That trial has not yet been published, says Joudinaud. The drug has not been tested in people with CTD, or in children.
“I look at this, and I’m like, that is my one shot for Brody,” says DeVault.
Joudinaud is planning a phase II trial in people with CTD, as well as others with amyotrophic lateral sclerosis. But that trial will take place in France, and it’s unlikely that Brody will be able to take part, says DeVault.
Ceres can’t make the drug available to Brody under an expanded access scheme run by the US Food and Drug Administration either, because the drug has not been registered with the FDA, and because it is currently manufactured in a way that does not comply with FDA regulations, says Joudinaud.
Even if that phase II trial is successful, and if the drug is ultimately approved, it is unlikely to reach the US market for at least a few years. DeVault is worried that will be too late for Brody—he’ll be “past his plasticity window” by then, he says.
Now, with the adoption of a new law in Montana, he theoretically has another option. Montana has had a “right to try” law—which allows terminally ill people to apply for access to unapproved drugs—in place since 2015. In 2023, a new law technically expanded this option to people who were not terminally ill, providing the drugs have been through preliminary phase I clinical trials. A second law aimed to clarify how clinics could sell and administer those treatments to patients. And last weekend, the state’s department of Health and Human Services finalized a set of rules for those clinics.
An experimental treatment review board (ETRB) has been established to review applications for access to experimental, unproven, and unapproved drugs. And it is set to review its first two applications in the coming weeks.
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.
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