Montana’s new “right to try” law can’t come soon enough for some

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.

Kris DeVault, his son Brody, and his wife and young daughter. COURTESY OF THE DEVAULT FAMILY

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.