A student attends a 'Many Pieces, One Community' autism awareness event in Phoenix on April 26, 2023. © Joel Angel Juarez/The Republic / USA TODAY NETWORK
(New York, NY) – A recent announcement by U.S. health officials has captured national attention after Health and Human Services Secretary Robert F. Kennedy Jr. revealed new steps to make leucovorin (also known as folinic acid) more accessible for children with autism who have a rare condition called cerebral folate deficiency (CFD).
What Is Cerebral Folate Deficiency (CFD)?
CFD occurs when the body has trouble moving folate (vitamin B9) across the blood–brain barrier, resulting in low folate levels in the brain. This condition can cause neurological symptoms and developmental delays. Some children on the autism spectrum have also been found to have folate receptor-alpha autoantibodies (FRAA) antibodies that block folate transport and may respond to folinic acid therapy.
On September 22, 2025, the US Food and Drug Administration (FDA) announced actions to update labeling and improve access to leucovorin for CFD. While the treatment may help some children with autism-related symptoms linked to CFD, experts stress that this is not a general autism therapy.
(Source: U.S. Food and Drug Administration, 2025)
What Is Leucovorin (Folinic Acid)?
Leucovorin is a prescription form of folinic acid, a biologically active version of folate. It has long been approved by the FDA for other uses, such as reducing side effects from chemotherapy (methotrexate) and treating certain types of anemia. Interest in its use for autism began after studies found that some autistic children with FRAA or CFD improved in areas such as speech and communication when treated with
leucovorin.
It’s important to note that prescription leucovorin differs from over-the-counter folinic acid supplements in both dosage and manufacturing quality. Physicians caution that self-treating with store-bought supplements or taking high doses without guidance can be unsafe. (Source: The Washington Post, 2025)
What the Science Shows
Several clinical trials have explored leucovorin’s potential benefits:
- A double-blind, placebo-controlled trial by Frye et al. (2018, Molecular Psychiatry) found significant improvements in verbal communication among autistic children with FRAA who received high-dose folinic acid.
- Other small studies, such as Batebi et al. (2021, PubMed), noted behavioral improvements when folinic acid was added to risperidone therapy.
- A 2021 systematic review (Rossignol & Frye, Cells) concluded that folinic acid may help children with autism and CFD but emphasized the need for larger, long-term studies.
- The Autism Science Foundation and other expert groups urge caution, saying that while early results are encouraging for a subset of children, the evidence is still preliminary.
Access and Availability
Prescription leucovorin can be obtained through a doctor and filled at most pharmacies. The FDA is currently working with GSK, which holds the drug’s US application, to expedite label changes for CFD treatment. Over-the-counter folinic acid supplements are widely available online and in health stores, but medical professionals warn that these are not equivalent to prescription leucovorin.
(Sources: Reuters, 2025; HHS.gov, 2025)
The Bottom Line
Leucovorin shows promising potential for a subset of autistic children who have folate-transport issues, such as CFD or FRAA. However, it is not a cure for autism, and more research is needed. Families should consult a qualified healthcare provider before beginning any supplement or treatment.
