STAT+: Inside FDA, career staffers describe how political pressure is influencing their work

WASHINGTON — The inquiry came in August, and struck scientists at the Food and Drug Administration as highly unusual. 

The leader of the center that regulates prescription medicines wanted to know what they thought about leucovorin, a generic drug that’s mainly used to alleviate side effects of cancer therapies. He’d seen some promising studies and thought the agency could find a way to approve it as an autism treatment. 

Autism, with its broad spectrum of symptoms, is one of the most challenging conditions to treat with medication. For the FDA itself to push for such a significant change to a drug’s label, and based on a handful of small studies — it would be unheard of, officials told STAT.

The request would have seemed completely random if not for health secretary Robert F. Kennedy Jr.’s longstanding interest in autism. In April, Kennedy announced his intention to identify autism’s root cause by September. President Trump zeroed in on the condition, too, and the political pressure to announce something big and splashy was ramping up. 

Even given the president’s interest in the subject, the request was so extraordinary that FDA staff pushed back, STAT has learned. The drug center director who made the request, George Tidmarsh, eventually came to a compromise with his staff: He agreed to ask GSK, the original manufacturer of the drug, to instead submit an application for cerebral folate deficiency, a rare neurological disorder that can have overlapping symptoms with autism. This episode was described by two people familiar with the situation.

But at the Trump administration’s press conference on autism in September, the career staff’s efforts to ground the move in science didn’t seem to matter. When FDA Commissioner Marty Makary reintroduced the public to leucovorin, he touted the drug as an autism treatment.

“Today, the FDA is filing a federal register notice to change the label on an exciting treatment called prescription leucovorin so that it can be available to children with autism,” he said. “Leucovorin holds promise for hundreds of thousands of kids with autism,” he later tweeted.  

Reshma Ramachandran, a health services researcher and clinician at Yale School of Medicine, said the sequence of events is the antithesis of how the FDA is supposed to function.