FDA files notice to update leucovorin label for cerebral folate deficiency; officials tout autism speech benefits
The FDA has filed a Federal Register notice to update the prescription leucovorin label to add cerebral folate deficiency (CFD) and language about "speech‑related deficits associated with ASD," a move announced at a White House press briefing where officials said Medicaid coverage and further NIH study of safety and effectiveness are expected. Scientists and autism groups warned the evidence is limited—existing studies are small, short and methodologically weak—White House materials stressed the change targets CFD not autism broadly, and some experts urged that leucovorin remain in controlled trials even as some clinicians begin off‑label use.
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📰 Sources (4)
Leucovorin for autism? Many scientists — and parents — are skeptical
New information:
- Provides on‑the‑record reactions from scientists and parents expressing skepticism about leucovorin as an autism treatment.
- Details White House promotion linking leucovorin to autism and notes administration statements blaming acetaminophen (Tylenol), adding political context.
- Quotes named researchers (e.g., Jonathan Sebat) and parents (e.g., Caitee Donovan, Jax Bayne) challenging the evidence and noting CFD is rare and its connection to autism unclear.
What to know about leucovorin, unproven autism drug touted by Trump
New information:
- Dr. Richard Frye—an advocate/researcher tied to the treatment—says he was surprised the administration moved to repurpose the drug without additional studies.
- Leading autism researchers and advocacy groups publicly distanced themselves, with David Mandell saying evidence is 'very weak' and there is 'nothing resembling even moderate evidence' that leucovorin treats autism symptoms.
- Reporting that some clinicians are already prescribing leucovorin off‑label—reworking chemotherapy formulations or ordering new versions from compounding pharmacies.
- Expert warnings (Dr. Lawrence Gray) urging that leucovorin should remain confined to controlled clinical trials until stronger evidence emerges.
Trump’s autism initiative embraces little-tested vitamin as a treatment
New information:
- FDA has already filed a Federal Register notice to change the prescription leucovorin label.
- A White House fact sheet clarifies the label update is for cerebral folate deficiency (CFD), not autism in general, and acknowledges more studies are needed for autoimmune CFD.
- FDA Commissioner Martin Makary did not note that caveat at the podium and asserted 'hundreds of thousands of kids' would benefit.
- The administration named recipients of $50 million in autism-related research funding; additional leucovorin trials were not among the listed projects.
- Experts highlighted that existing leucovorin studies in autism are small (dozens of participants), short (≤6 months), and have methodological limitations.
Cancer drug could double as autism therapy, and is poised for FDA approval
New information:
- HHS says FDA will approve leucovorin labeling for 'speech-related deficits associated with ASD' and add cerebral folate deficiency
- Officials: FDA’s Marty Makary, NIH’s Jay Bhattacharya, and CMS’s Mehmet Oz announced the steps at a Monday presser
- Medicaid coverage is expected to follow the label update; NIH will initiate additional research on safety and effectiveness