406: New hope in treating Huntington's disease and a report card on RFK Jr.'s promises

The episode opens with discussion of a significant development for Huntington's disease patients: Unicure has received FDA approval to submit its gene therapy for U.S. approval after the agency reversed its previous stance. Patient advocate Lauren Holder, who lives with early-stage Huntington's, discusses the emotional impact of this reversal and attributes it to changes in FDA leadership following the departure of Commissioner Marty Makary and Vinay Prasad, who had previously rejected Unicure's data as insufficient. Holder explains that under the previous administration, the FDA required a controversial sham surgery control group in clinical trials, which the Huntington's community viewed as unethical because it could prevent patients from accessing the actual treatment if they progressed too far during the trial period. She describes this as a "death sentence" to the community and credits patient advocacy, including outreach by advocates like Katie Jackson to Senator Ron Johnson's office, with changing the FDA's approach. The new FDA agreement allows for accelerated approval with a confirmatory trial requirement, eliminating the sham surgery requirement and restoring communication between the agency and patient advocates.

The episode then shifts to examining RFK Jr.'s performance as HHS Secretary over his first year-plus in office. STAT reporter Isa Cueto provides an assessment of his progress on the "Make America Healthy Again" (MAHA) agenda. On psychiatric medications, HHS has begun encouraging physicians to wean patients off antidepressants and other psychiatric drugs through a May dear colleague letter, with plans to develop clinical guidelines and host educational webinars. Cueto notes this reflects long-standing MAHA movement beliefs that psychiatric medications do more harm than good. Regarding childhood vaccines, Kennedy has made significant moves including appointing advisors who overhauled the vaccine schedule to reduce recommended shots, though these changes face legal challenges. Despite Trump issuing an executive order supporting the changes and some program rollbacks like reduced MMR coverage for low-income children, healthcare providers are maintaining the original schedule. Declines in childhood vaccine uptake are already being observed. On direct-to-consumer pharmaceutical advertising, Kennedy has long championed a ban, but HHS has taken only limited action, with the FDA focusing on cracking down on deceptive ads on social media rather than pursuing a comprehensive ban. Cueto reports that STAT has been unable to obtain Kennedy's public calendar despite multiple FOIA requests, making it difficult to assess how he spends his time as secretary. Regarding his broader MAHA Commission goals, Cueto's tracking shows only about two dozen of 80 promises have been kept or are in progress. The area where Kennedy appears most successful and where there is broadest support is nutrition and food policy, including updates to dietary guidelines that influence school meals and a focus on reducing ultra-processed foods and added sugars. Cueto suggests these changes may prove more durable since they're broadly popular and aligned with mainstream health consciousness movements.