Clinicians are divided amid a growing demand for an unproven autism drug and wanting to preserve their patient-physician relationships, NPR reported March 16.
Last fall, a federal press conference touted a new, unproven, treatment for autism: leucovorin. This derivative of vitamin B9, also called folinic acid, is commonly used during cancer treatment. Federal health officials said it could help treat cerebral folate deficiency, a rare brain condition, as well as benefit thousands of kids with autism. The announcement led to a surge of parents seeking the medication, the creation of social media groups sharing physician referrals and notes, and pushback from major medical groups.
In the middle, clinicians are struggling between the drug risks and patient satisfaction. Here’s what to know:
1. The conference led many parents to have the impression that leucovorin is a proven treatment for autism or that many children with autism have cerebral folate deficiency, NPR found.
2. Using leucovorin to treat patients with cerebral folate deficiency has been done for decades and is not controversial, experts told NPR. The medication is generally considered safe and will most likely not cause harm if used briefly, but there is no safety profile established in children with autism, and no data on long-term use for longer than six months in that patient population. Current formulations of the drug persist in the bloodstream for long periods, which could have unintended effects. Dosage for the medication typically follows protocols created in 2013 and can be up to 100 times higher than the minimum recommended daily intake of folate.
3. Major medical associations like the American Academy of Pediatrics and the Coalition of Autism Scientists said they do not recommend the drug for autism due to insufficient evidence to support its use.
4. Many major health systems, such as Kaiser Permanente and Oregon Health and Science University, have issued similar guidance and stated their physicians will not prescribe the drug until more research on its safety and effectiveness is released.
5. Leucovorin prescriptions increased by 71% in the last quarter of 2025.
6. Some have raised concerns that the increased demand for the drug could cause a shortage, which could negatively impact cancer patients who regularly use the drug. Some parents who have been prescribed the drug for their children said they have to bounce between pharmacies to get their prescription filled.
7. Although most physicians at major health centers will not prescribe the drug, some private practices will, and show a high percentage of up to 80% of patients with autism who have been given prescriptions.
8. The fee for leucovorin is also substantial for patients. One patient told NPR that a physician willing to prescribe the drug was going to charge $1,896 plus a nonrefundable $299 deposit, with follow-ups priced at $948. Fees for the initial physician visit ranged between $150 to $1,500. The drug itself is covered by some insurance plans, and is not considered to be expensive.
9. The administration walked back the claim that the drug could treat autism in March 2026, and expanded approval only for a specific subset of cerebral folate deficiency that has fewer than 50 cases worldwide.
“Right now, we don’t have sufficient data to say that we could establish efficacy for autism more broadly,” a senior FDA official said in a briefing. “It’ll be up to patients to talk with their physicians to see if that might be right for them.”
10. The FDA has approved a new use for Wellcovorin, or leucovorin calcium tablets, to treat cerebral folate deficiency, after initiating efforts to expand approval for autism-related symptoms.
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