FDA neapstipina Leukovorinu par autizmu, piešķir to retai asinsūdeņu slimībai

on PinterestMore research is still needed before leucovorin should be approved as an autism treatment.

on PinterestMore research is still needed before leucovorin should be approved as an autism treatment. Westend61/Getty Images

• Federal regulators have approved leucovorin for the treatment of cerebral folate deficiency, a rare neurological disorder.
• The FDA approval, however, did not include the use of leucovorin as a treatment for autism.
• The Trump administration has touted leucovorin as an autism treatment, but experts agree that more research is needed before leucovorin should be approved for this purpose.

A medication used primarily to help relieve the side effects of chemotherapy has been given the green light to be used as a treatment for a rare neurological disorder.

Officials at the Food and Drug Administration (FDA) announced on March 10 the approval of the drug leucovorin to help treat adults and children with cerebral folate deficiency. This rare condition is characterized by low levels of vitamin B9 in the brain.

However, the FDA announcement did not mention the use of leucovorin as a potential treatment for autism.

In September 2025, President Donald Trump and other administration officials suggested that the FDA had started a process to approve leucovorin as a treatment to manage symptoms in autistic people. However, there’s no word yet on a timetable or process for such an approval.

Leucovorin is the first approved treatment for cerebral folate deficiency. The FDA decision is being praised as long overdue relief for individuals with that condition.

“Today’s approval represents a significant milestone for patients living with cerebral folate transport deficiency due to the FOLR1 variant, a rare genetic condition that has had no FDA-approved treatment options until today,” said FDA commissioner Marty Makary, MD, in the agency’s announcement.

Leucovorin and cerebral folate deficiency

Leucovorin is a prescription medication used mainly to reduce the toxic side effects of chemotherapy agents such as methotrexate.

The medication works by helping to restore folic acid to healthy cells that have had that substance depleted by the effects of chemotherapy.

Leucovorin is considered to be effective as a “rescue agent” for people undergoing certain types of chemotherapy.

Cerebral folate deficiency is a condition that usually manifests in young children. It can cause:

• developmental delays
• seizures
• movement abnormalities

It’s estimated that 1 in 1 million people worldwide have cerebral folate deficiency, although the true prevalence is unknown. It’s also been estimated that 38–70% of autistic children may have cerebral folate deficiency.

However, experts say those percentages may be inflated because much of the data has been drawn from FRAT tests, a blood exam that can be inaccurate.

While individuals with cerebral folate deficiency may have a higher risk of autism, but autistic people don’t necessarily have a higher risk of cerebral folate deficiency.

Alycia Halladay, MD, the chief science officer for the Autism Science Foundation, said she’s glad that people with cerebral folate deficiency finally have a treatment.

“This will probably help them. The mechanism is there,” she told Healthline. “I feel any relief would be helpful.”

Antonio Hardan, MD, a professor of psychiatry and behavioral sciences at Stanford University, said that early intervention with leucovorin for cerebral folate deficiency is key.

“Case reports [support] complete clinical and radiological recovery when treatment is

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