FDA finds little evidence that a drug touted by Trump can help people
with autism
[March 11, 2026]
By MATTHEW PERRONE
WASHINGTON (AP) — The Food and Drug Administration on Tuesday approved a
generic medication for a rare brain disorder, while walking back
statements by President Donald Trump and other administration officials
that the drug showed great promise for people with autism.
The agency said it 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 the ultrarare condition impacts
fewer than 1 in a million people in the U.S.
It's a major step back from comments made at a White House news
conference in September, when Trump and FDA commissioner Marty Makary
announced the drug was under review to benefit patients with autism,
some of whom have a form of the vitamin brain deficiency.
“It might be 20, 40, 50% of kids with autism,” Makary said at the news
conference.
But senior FDA officials told reporters Monday that their review was
narrowed to focus on the strongest evidence, which only supported the
drug’s use by patients with the rare mutation that impacts folate levels
in the brain.
The FDA officials also pointed out that one study supporting the drug’s
use for autism was retracted earlier this year.
Autism researchers on Tuesday reiterated that the drug has not been
shown safe or effective for the vast majority of people with the brain
disorder.
“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 noted there is also no established figure for how many people
with autism have a form of the folate brain disorder. Some doctors
diagnose the folate condition using a specialty laboratory test that
isn't FDA-approved.
The administration's White House event touting the drug last year
followed promises from Health Secretary Robert F. Kennedy Jr. to
determine the cause of autism by September.
Leucovorin is a synthetic metabolite of folate, which is essential for
healthy pregnancies and is recommended for women before conception and
during pregnancy. The drug's current FDA label covers leucovorin's use
in reducing side effects of certain chemotherapy drugs and treating a
rare blood disorder.
Patients affected by the condition targeted by Tuesday's approval
experience movement disorders, seizures and other neurological problems
that can resemble symptoms of autism.
But professional medical societies say it’s far from clear whether the
drug helps people with autism.
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 The American Academy of Pediatrics
doesn’t recommend routine use of leucovorin for autistic children,
including those with the brain condition known as cerebral folate
deficiency.
Unresolved questions about the drug haven’t stopped U.S. doctors
from prescribing it.
A paper published in The Lancet last week found that leucovorin
prescriptions for children aged 5 to 17 were 71% higher than normal
in the three months immediately following Trump’s late September
news conference.
“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."
Some families of children with autism have reported trouble getting
prescriptions filled in recent weeks.
FDA officials told reporters the agency is allowing imports of the
drug by foreign drugmakers to help boost supply. The drug's original
manufacturer, GSK, does not plan to relaunch its version of the
drug.
Halladay, of the Autism Science Foundation, cautioned parents
against seeking out the drug, noting reports of side 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 said.
Trump officials originally decided to review the drug after speaking
with an Arizona-based neurologist who prescribes the drug for autism
patients and runs an online education business focused on the
experimental treatment.
The theory behind the drug's use is that some people with autism
have specific antibodies that block folate from entering the brain.
But the Autism Science Foundation and other groups note that
non-autistic relatives of people with the disorder often have the
same antibodies, suggesting they're not a factor in the condition.
While there is no single cause behind autism, most researchers say
science points to genetic and environmental factors as playing a
role.
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