September 24, 2025 – Washington, D.C.
The U.S. Food and Drug Administration (FDA) has taken the unusual step of pushing for the approval of leucovorin (Wellcovorin) to treat cerebral folate deficiency (CFD), a rare condition seen in some children with autism. The initiative, announced at a White House event on September 22, has triggered heated debate among doctors, researchers, and advocacy groups.
Normally reserved for chemotherapy support, leucovorin could gain a new label for autism-related use after the FDA urged former manufacturer GlaxoSmithKline (GSK) to submit an application. Unlike typical approvals, which rely on large randomized trials, the FDA cited data from published studies and case reports involving just over 40 patients.
Experts Question the Evidence
Autism specialists and medical groups, including the Autism Science Foundation, slammed the move as premature, warning the evidence is weak and inconclusive. “This risks creating false hope,” critics said, stressing that leucovorin is not a cure and may benefit only a small subset of children with confirmed CFD.
Concerns also extend to safety and supply. Advocates warn parents against buying unregulated folinic acid supplements online, while the American Cancer Society fears new demand could cause shortages for cancer patients.
Political Spotlight Intensifies Controversy
The announcement, led by U.S. Health and Human Services Secretary Robert F. Kennedy, Jr., gained praise from former President Donald Trump, who repeated debunked claims linking autism to Tylenol and vaccines. Medical experts condemned the remarks, saying they risk fueling misinformation.
Federal officials confirmed Medicaid will cover prescriptions if approved, with private insurers expected to follow. The FDA and National Institutes of Health (NIH) pledged to fund additional research, but final approval depends on GSK’s pending application.
For now, the FDA’s bold move has left the medical community divided—between those calling it a breakthrough for families and those warning it could undermine scientific standards.
