Families Blindsided as Feds Pull Back on Leucovorin Claims

CNN and USA Today contributed to this report.

The government’s messaging on leucovorin went from megaphone to whisper — and a lot of parents say they were left hanging. The drug was hyped last fall as a possible help for many kids with autism, but the Food and Drug Administration has now narrowed its thumbs-up to a vanishingly rare genetic condition. Here’s what that means for families, prescribers and anyone who’s been swept up in the frenzy.

“I kind of felt vindicated with what I was doing. But now they’re saying, ‘Oh, nope, we’re out, and we were wrong,'” says Keith Joyce, who started giving the drug to his 5-year-old after the White House attention.

Meagan Meagan Johnson, another mom who spent weeks hunting the drug, says she’s “upset” and “hurt” after hopes pinned on leucovorin collapsed into a much narrower FDA approval.

Last September, the White House — with figures such as Donald Trump and Robert F. Kennedy Jr. front and center — loudly suggested leucovorin could help thousands of kids with autism. That public push sent prescriptions and demand soaring. But after a systematic review, FDA officials and Commissioner Marty Makary concluded the evidence doesn’t support broad use for autism. Instead the agency approved leucovorin only for cerebral folate deficiency linked to FOLR1 variants, a genetic disorder with fewer than about 50 documented cases worldwide.

Mixed, thin, and narrow. Small studies and case reports hinted at benefits for children who truly have cerebral folate transport problems. But the big, world-changing randomized trial that proponents cited was later retracted — the journal behind it identified data errors. The major pediatric groups, including the American Academy of Pediatrics and the Child Neurology Society, have warned that proof of benefit for the broader autism population is limited and more research is needed.

When officials started talking up the drug, demand spiked. A recent paper in The Lancet and other reports showed big increases in prescriptions. Companies that do the folate-receptor antibody test — notably ReligenDX — saw test volumes multiply (the firm said it processed roughly seven times more tests at the peak and is now doing about 1,600 a month). The test isn’t cheap — roughly $300 out of pocket — and it isn’t universally endorsed by the research community.

Many clinicians applaud the FDA for sticking to the data. Autism Speaks’s chief science officer called the agency’s move consistent with the evidence. Local neurologists also urge caution: doses being used off-label can be far higher than normal folate needs — sometimes orders of magnitude higher — and long-term safety at those doses isn’t well charted. Dr. Audrey Brumback says parents should work closely with a specialist if they’re considering the drug; Dr. Helen Tager-Flusberg hopes other HHS agencies reinforce the FDA’s careful stance.

Practical advice for families right now

• Talk to a specialist before making any change. Don’t stop or start leucovorin without a clinician who knows your child’s history and can track labs and side effects.
• Ask about genetic testing. The FDA’s approval targets a specific FOLR1 genetic defect — if your child doesn’t have that, the case for leucovorin is far weaker.
• Beware the diagnostics shopping rush. Some tests being sold are unproven; ask whether the test is validated and whether insurance will cover confirmatory testing (spinal-fluid testing is more invasive but sometimes more accurate).
• Watch for price-gouging and snake-oil offers. When demand skyrockets, opportunists appear. If a practitioner asks for large cash payments or promises cures, get a second opinion.
• Keep doing therapies that we know help. Behavioral therapies, speech, occupational therapy and educational supports remain the backbone of care for many kids. Leucovorin, where it helps, is one piece of a larger plan.
• Document and communicate: if you’re seeing improvement, write down timelines and other changes — sometimes gains are natural developmental steps, and clinicians need good records to sort that out.

This episode shows how powerful a podium can be: headlines pushed families into action before the science caught up. Some parents report real improvements and don’t want to lose access; others worry that the hype drew attention (and money) away from tested interventions. The middle path — sober, transparent research — is what most experts say they want next.

If you’re confused, frustrated or angry — that’s understandable. As Dr. Brumback put it, clinicians aren’t the enemy; they’re the ones who can help you make the safest, most informed choices. Science may not give easy answers today, but it can protect families from harm tomorrow.