Leucovorin (Folinic Acid) and Autism: Understanding RFK Jr.’s Announcement

Autism research

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A recent announcement by U.S. health officials has drawn national attention after Health and Human Services Secretary Robert F. Kennedy Jr. (RFK Jr.) revealed new measures to make Leucovorin (folinic acid) more accessible for children with autism who have a rare condition known as Cerebral Folate Deficiency (CFD) (U.S. Food and Drug Administration, 2025).

What Is Cerebral Folate Deficiency (CFD)?

CFD occurs when the body struggles to transport folate (vitamin B9) across the blood–brain barrier, leading to low folate levels in the brain. Some children with autism also have folate receptor-alpha autoantibodies (FRAA) that block folate transport and may respond positively to folinic acid therapy (Rossignol & Frye, 2021). On September 22, 2025, the FDA announced steps to update labeling and improve access to Leucovorin for CFD. Officials emphasized that this treatment may help a subset of children with autism-related folate disorders, but it is not a general autism therapy (FDA, 2025).

What Is Leucovorin?

Leucovorin is a prescription form of folinic acid, an active metabolite of folate approved to reduce chemotherapy side effects and treat certain anemias (The Washington Post, 2025). Interest in its autism use began when studies found that some children with FRAA or CFD improved in speech and communication after high-dose treatment (Frye et al., 2018).

What the Science Shows

• Frye et al. (2018) reported significant improvements in verbal communication among autistic children with FRAA.
• Batebi et al. (2021) found behavioral improvements when folinic acid was combined with risperidone.
• Rossignol & Frye (2021) concluded that folinic acid may help children with CFD, but larger studies are needed.
• Panda et al. (2024) also observed benefits from oral folinic acid supplementation.

The Autism Science Foundation urges caution, noting that while results are encouraging, evidence remains preliminary (Autism Science Foundation, 2025).

Access and Availability

The FDA is working with GSK to accelerate labeling updates for CFD treatment (Reuters, 2025). Prescription Leucovorin is available through pharmacies, but over-the-counter folinic acid supplements are not equivalent in dosage or purity (HHS.gov, 2025).  

The Bottom Line

Leucovorin represents a promising therapy for children with folate-transport issues such as CFD or FRAA. However, it is not a cure for autism.

Families should consult healthcare professionals before starting any supplement or treatment.

* This article is for informational and editorial purposes only.

Sources:

1. U.S. FDA (2025). Actions to Improve Access to Leucovorin for Cerebral Folate Deficiency.
2. Frye R.E. et al. (2018). Molecular Psychiatry.
3. Rossignol D.A. & Frye R.E. (2021). Cells.
4. Batebi N. et al. (2021). PubMed.
5. Panda P.K. et al. (2024). PubMed.
6. The Washington Post (2025). Why Prescription Leucovorin Differs from Over-the-Counter Folate Supplements.
7. Reuters (2025). FDA and GSK to Update Leucovorin Labeling for CFD.
8. HHS.gov (2025). Leucovorin Access and Safety Advisory