FDA finds little evidence that a drug touted by Trump can help people with autism

The Food and Drug Administration on Tuesday approved a generic medication for a rare brain disorder, significantly narrowing the scope from earlier claims made by Trump administration officials that the drug could benefit a large portion of people with autism.

The agency approved leucovorin for children and adults with a genetic condition that limits delivery of folate, a form of vitamin B, to the brain. FDA officials estimate this ultrarare condition affects fewer than 1 in a million Americans.

This approval represents a substantial retreat from statements made during a White House news conference in September, when President Donald Trump and FDA Commissioner Marty Makary suggested the drug could help autism patients more broadly.

“It might be 20, 40, 50% of kids with autism,” Makary had claimed at the September event, suggesting a potential breakthrough for millions of Americans on the autism spectrum.

However, senior FDA officials clarified on Monday that their review was ultimately focused on the strongest scientific evidence, which only supported the drug’s use for patients with the specific rare mutation affecting brain folate levels. Officials also noted that one study supporting leucovorin’s use for autism was retracted earlier this year, undermining previous claims.

Autism researchers were quick to caution against viewing the drug as a treatment for the broader autism community.

“There is no evidence to say that leucovorin will help most people with autism, and there’s certainly no evidence to say it’s safe,” said Dr. Alycia Halladay of the Autism Science Foundation in an interview.

Halladay emphasized that there is no established consensus on how many people with autism have the folate brain disorder. Some physicians diagnose the condition using specialty laboratory tests that have not received FDA approval, further complicating the picture.

The administration’s promotion of the drug last year aligned with broader promises from Health Secretary Robert F. Kennedy Jr. to determine the cause of autism by September – a timeline many experts in the field considered unrealistic given the complex nature of the condition.

Leucovorin is a synthetic form of folate, a nutrient essential for healthy pregnancies. Prior to Tuesday’s approval, the drug was primarily used to reduce side effects of certain chemotherapy drugs and to treat a rare blood disorder.

While patients with the specific condition targeted by the FDA’s approval experience movement disorders, seizures, and other neurological problems that may resemble autism symptoms, major medical organizations have not endorsed leucovorin for autism treatment.

The American Academy of Pediatrics does not recommend routine use of leucovorin for autistic children, including those with cerebral folate deficiency. Despite this, prescriptions for the drug surged following the White House announcement.

A paper published in The Lancet last week found that leucovorin prescriptions for children aged 5 to 17 increased by 71% in the three months immediately following Trump’s September news conference, highlighting how political pronouncements can rapidly influence medical practice before scientific consensus is reached.

“We’ve seen huge increases in leucovorin prescriptions for autism because of the initial premature and ill-informed announcement that it can treat autism symptoms,” said David Mandell, an autism expert at the University of Pennsylvania. “Now families are experiencing whiplash about what constitutes best practice for their children.”

The abrupt rise in prescriptions has also led to supply issues, with some families of children with autism reporting difficulties obtaining the medication in recent weeks. FDA officials told reporters they are allowing imports of the drug by foreign manufacturers to address shortages, as the original manufacturer, GSK, has no plans to resume production.

Halladay warned parents against seeking out the drug, citing reports of adverse effects including irritability, aggression, and hyperactivity when used in people with autism.

“If parents are insistent on trying this, they should know that it may cause harm and it may do no good,” she cautioned.

The FDA’s review reportedly began after administration officials spoke with an Arizona-based neurologist who prescribes the drug for autism patients and operates an online education business focused on the experimental treatment.

While proponents suggest that some people with autism have specific antibodies blocking folate from entering the brain, the Autism Science Foundation and other research organizations note that non-autistic relatives often have these same antibodies, suggesting they may not be a significant factor in autism.

Most researchers agree that autism likely results from a complex interplay of genetic and environmental factors, with no single cause explaining the full spectrum of conditions.