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).
The FDA officials also noted that among the few studies supporting the use of leucovorin for autism treatment, one of the largest was retracted last month over errors in its data and statistical analysis. Reanalysis of the data was unable to reproduce the original positive findings.
Leucovorin, or folinic acid, is a reduced form of folic acid that’s used to treat impaired folate metabolism. Such an impairment can occur as a side effect of certain chemotherapy drugs, such as methotrexate.
CFD is a neurological condition marked by abnormally low amounts of folate metabolites in the brain and includes symptoms such as developmental delays, seizures, and movement abnormalities, as well as others that can resemble autism. It affects fewer than 1 in a million in the US.
While some small, preliminary studies have suggested leucovorin could help ease autism symptoms in some patients, the findings are far from conclusive. Some researchers have hypothesized that some patients with autism may have low cerebral folate levels due to antibodies that block the vitamin from the brain. But, as the Autism Science Foundation noted in September, non-autistic family members of autistic individuals can also have these antibodies, suggesting they are not a cause of the condition.
