MIT Technology Review is reporting that a new law in Montana aims to expand access to experimental medical treatments, offering a potential avenue for families seeking unapproved drugs for rare conditions. The Review highlights the case of Kris DeVault, whose three-year-old son, Brody, suffers from creatine transporter deficiency (CTD), a rare genetic condition that impairs brain and muscle development.
DeVault is seeking an experimental drug from Ceres Brain Therapeutics, a French biotechnology company. The drug, delivered as a nasal spray, is designed to bypass the brain's inability to absorb creatine in CTD patients. Ceres has seen promising results in mice and completed a phase I clinical trial in healthy adults, but the drug has not yet been tested in children or individuals with CTD. The company plans a phase II trial in France, but Brody is unlikely to participate, and the drug is not registered with the U.S. Food and Drug Administration, making it inaccessible through standard expanded access programs.
Montana's new legislation, which builds on a 2015 "right to try" law, now allows people who are not terminally ill to access unapproved drugs that have completed preliminary phase I trials. The state has established an Experimental Treatment Review Board (ETRB) to review applications, with its first two reviews expected soon. While Ceres CEO Thomas Joudinaud finds Montana's setup "very interesting," he is reluctant to participate due to concerns about potential FDA repercussions. DeVault has been pressing FDA staffers for assurances that companies participating in Montana's program will not be penalized later.
Scientists, bioethicists and health law experts caution that phase I trials do not prove a drug's safety or efficacy. Aaron Kesselheim, a Harvard Medical School professor, emphasized the importance of rigorous assessment for patients considering such treatments. DeVault, however, argues for the right to make decisions about potential treatments that could alter his son's life trajectory, even considering options in offshore clinics like Próspera in Honduras.
Full Article: Montana’s new “right to try” law can’t come soon enough for some