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

Brody DeVault, a three-year-old boy, has been battling a rare condition called creatine transporter deficiency (CTD) since he was born in March 2023. This condition leaves his brain and muscles lacking essential energy for proper development, and there are currently no cures available. A genetic test confirmed Brody's condition, which has left his parents desperate for a potential treatment.

A biotechnology company in France, Ceres Brain Therapeutics, is developing a drug that aims to deliver creatine directly to the brain, bypassing the transporter deficiency. The drug is still in early development stages and has only been tested in animals and a limited number of healthy adults. Doctors cannot prescribe the drug, but Brody's father, Kris DeVault, is determined to find a way to access it.

With the implementation of a new "right to try" law in Montana, Brody and his family now have a theoretical option to seek access to the experimental drug. The law, which has been in place since 2015, allows terminally ill patients to apply for unapproved treatments. In 2023, the law was expanded to include patients who are not terminally ill, provided the drugs have undergone preliminary phase I clinical trials.

However, several obstacles stand in the way of Brody's access to the drug. The drug has not been registered with the US Food and Drug Administration (FDA), and it is not manufactured in compliance with FDA regulations. Therefore, the FDA will not allow the drug to be made available to Brody through an expanded access scheme. Even if Brody's case is successful and the drug is ultimately approved, it is unlikely to reach the US market for several years.

Kris DeVault is understandably worried about the tight timeline, as Brody's brain is still in a crucial "plastic" stage for development during his early years. He is now considering reaching out to Montana's Experimental Treatment Review Board (ETRB) to review his application for access to the experimental drug. However, DeVault is hesitant to proceed until he receives a written assurance from the FDA that participating biotech companies will not face penalties later on.

In the meantime, Brody continues to struggle with communication, muscle weakness, and neurological development. His parents are left to cope with the challenges of caring for a child with limited verbal abilities and uncontrolled physical limitations. The future of Brody's treatment remains uncertain, as he waits for potential access to the experimental drug in a state that may not be able to provide it in a timely manner.