In September, the Trump administration took what it called “bold actions” on autism that included touting the generic drug leucovorin as a promising treatment. In a news release, Marty Makary, commissioner of the Food and Drug Administration, claimed a “growing body of evidence suggests” the drug could be helpful. And at a White House press event, Makary suggested it might help “20, 40, 50 percent of kids with autism.”
“Hundreds of thousands of kids, in my opinion, will benefit,” he said at another point in the event.
The bold claims were apparently persuasive. A study published in The Lancet last week found that new outpatient prescriptions of leucovorin for children ages 5 to 17 shot up 71 percent in the three months after the Trump administration’s actions.
But it became clear today that the rest of the FDA did not share Makary’s and the other administration officials’ view. In an announcement, the regulatory agency said it had approved leucovorin for a rare genetic condition—but not for autism.
In comments to the Associated Press, senior FDA officials said they found little evidence for expanding the drug’s use to autism and, thus, narrowed its review to the treatment of the rare genetic condition, which is cerebral folate deficiency (CFD) in adults caused by a genetic mutation in the folate receptor 1 gene (CFD-FOLR1).
