When Brody DeVault was born in March 2023, his parents expected the usual milestones: first words, first steps, first time standing on a fire ant hill while being eaten alive. But Brody started missing key developmental milestones in speech, movement, and coordination, and a genetic test at age two and a half revealed creatine transporter deficiency (CTD) - a rare condition where the brain and muscles lack the energy they need to develop. There's no cure, but there is a drug in development that might help. It's only been tested in animals and 48 healthy adults, so doctors can't prescribe it. But thanks to a new Montana law, his dad, DeVault, might be able to get it - at least in theory.

Brody is now three, a 'happy, curious, and loving little boy' who struggles to communicate. 'He's got no words, really,' says DeVault. He can't tell his parents if he's hot, cold, hungry, or in pain - recently, he stood on an anthill while fire ants 'went to town on his feet,' and he just looked. He also has muscle weakness, with arms skinnier than his nine-month-old sister's. But DeVault is most worried about Brody's neurological development, because toddlers' brains are exceptionally 'plastic,' and those early years are crucial.

Enter Ceres Brain Therapeutics, a French biotech developing a nasal spray that delivers creatine directly to the brain, bypassing the transporter defect. CEO Thomas Joudinaud says results in mice are promising, and a phase I trial in healthy adults is complete (though unpublished). But the drug hasn't been tested in CTD patients or children, and a phase II trial will be in France - too far for Brody. The FDA's expanded access program is out, because the drug isn't registered with the FDA and isn't manufactured to FDA standards. Even if approved, it's years away from the US market, and Brody might be 'past his plasticity window' by then.

So DeVault is looking at Montana, which has had a 'right to try' law since 2015, allowing terminally ill patients access to unapproved drugs. In 2023, the state expanded it to non-terminally ill patients, provided the drugs have passed phase I. Last weekend, the Department of Health and Human Services finalized rules for clinics, and an Experimental Treatment Review Board (ETRB) will review its first two applications soon. Ceres could apply to sell its treatment via a Montana clinic, but Joudinaud is reluctant, worried about 'getting on the wrong side of the FDA.' DeVault has pleaded with FDA staffers for a written promise that companies won't be penalized later, but no progress.

He's now considering even more exotic options: a clinic in Próspera, a private city in Honduras, that sells unproven stem-cell and gene therapies. Scientists and bioethicists caution against such 'offshore' clinics, and Harvard's Aaron Kesselheim argues that patients 'deserve treatments to be rigorously assessed' so they know what they're getting into. But DeVault pushes back: 'I'm a full-grown human being. I can go to Vegas and blow it all on poker, or buy a silenced 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?' It's a fair point - except science is supposed to be the thing that saves us from our worst Vegas impulses.