The Food and Drug Administration (FDA) has taken an extraordinary step, approving an existing generic drug, leucovorin, for autism treatment. This decision has left many experts astonished, as it deviates significantly from the agency’s standard drug review protocols.
Leucovorin, historically prescribed to counter the harsh side effects of chemotherapy, received an unexpected endorsement. President Trump and senior U.S. health officials announced its approval for certain individuals with autism during a White House briefing.
This approval process was a complete reversal of the norm. Usually, pharmaceutical companies conduct extensive research, often collaborating with the FDA on study design, before submitting a comprehensive application for drug approval.
In this instance, however, the FDA stated that it independently reviewed existing medical research and decided to expand the drug’s approved uses on its own initiative.
“Mr. President, you instructed us to prioritize medical righteousness – to be audacious and disregard corporate interests or lobbyists,” declared Dr. Marty Makary, the FDA commissioner, on Monday. “And that’s precisely what we’re accomplishing today.”
Dr. Makary’s unexpected announcement occurred during a presidential briefing on autism. During this event, Mr. Trump repeatedly drew a direct, yet scientifically unproven, link between Tylenol (acetaminophen) and the disorder.
The FDA adopted a more measured tone in its official communication to medical professionals, acknowledging that while some evidence *suggests* a link between Tylenol and autism, this association remains a “subject of ongoing scientific debate.” Health officials committed to funding further research into this potential connection. Kenvue, Tylenol’s manufacturer, maintains that the drug is safe when used as directed and does not cause autism.
This swift decision by the agency has, however, raised numerous questions among independent experts.
Dr. Aaron Kesselheim, a Harvard Medical School professor, described the FDA’s rapid endorsement of leucovorin as “shocking.”
During the briefing, Dr. Makary enthusiastically promoted the drug, suggesting it could benefit “hundreds of thousands” of children with autism. Contradictory to this broad claim, the official FDA notice in the Federal Register specified a much more limited application.
The FDA’s approval for leucovorin tablets is specifically targeted at “cerebral folate deficiency,” a condition marked by insufficient levels of folate (a B vitamin) in the spinal fluid. This deficiency is known to contribute to developmental delays and impaired motor skills in children.
Dr. Kesselheim highlighted a critical issue: before prescribing, doctors would need to diagnose this specific folate deficiency. He questioned how such a diagnosis could be reliably made without a potentially risky and costly spinal tap, a detail not clarified by the agency.
Furthermore, the FDA’s decision lacked clarity on appropriate dosing for children and adults – information typically established through comprehensive clinical studies by drug manufacturers. Dr. Kesselheim pointed out that even the study referenced by the FDA showed only modest improvements in autism symptoms, and only among individuals with specific folate levels in their spinal fluid.
“This approach completely misplaces priorities,” Dr. Kesselheim asserted. “The FDA exists to help patients discern effective treatments from ineffective ones.”
Holly Fernandez Lynch, an associate law professor at the University of Pennsylvania, characterized the FDA’s recommendation as a “science communication disaster.” She argued that the public would likely miss the crucial detail that this heralded autism treatment only applies to a very specific patient group, suggesting the vague wording of the announcement was deliberate.
“It created the impression of ‘problem solved’,” she elaborated, “which is precisely the narrative this administration desired.”
She further criticized the approval, stating that the accompanying description of a minimal study falls significantly short of the agency’s usual standards for evaluating treatments, especially for a prevalent condition like autism. While small studies can be acceptable for exceptionally rare diseases, this context is different.
“This represents a dangerous subversion of the FDA’s fundamental purpose,” she concluded.
The FDA has directed GSK, the original company that secured initial approval for leucovorin decades ago, to update the drug’s label. This procedural action will allow generic manufacturers to incorporate the new usage information. GSK, which ceased selling the drug in the 1980s and 90s, confirmed it would comply with the label update.
Daniel Aaron, an associate professor of law at the University of Utah specializing in FDA studies, warned that this leucovorin decision could establish a problematic precedent. It might encourage other companies to bypass expensive, meticulous, and lengthy clinical trials when seeking drug approvals.
“This approval could very well come back to challenge the FDA significantly in the future,” he remarked.