On October 6, 2025, the Roosevelt Room of the White House buzzed with anticipation as President Donald Trump and Health and Human Services Secretary Robert F. Kennedy Jr. addressed the nation on a topic that has stirred both hope and controversy: autism. The event, which drew a crowd of reporters, medical professionals, and advocates, was intended as a major policy announcement on autism treatments and prevention. Instead, it quickly became a lightning rod for debate, with claims and counterclaims ricocheting across the medical community and the public sphere.
At the heart of the event was the Trump administration's push to fast-track approval of leucovorin, a decades-old drug traditionally used to mitigate the toxic effects of certain cancer treatments, for a new indication: cerebral folate deficiency, a metabolic disorder that can manifest with neurological symptoms, some of which overlap with those seen in autism. According to Reuters, this move was orchestrated by the Food and Drug Administration (FDA), which enlisted pharmaceutical giant GSK to expedite a new use application for leucovorin. The aim? To provide doctors with additional justification for prescribing the drug to patients with cerebral folate deficiency and, by extension, some children with autism.
"My nurses have been saying the phone is ringing off the hook," Dr. Larry Gray, an expert in developmental and behavioral pediatrics at Lurie Children's Hospital of Chicago, told Reuters, highlighting the surge in demand following both a CBS news segment and President Trump's endorsement of the drug. While leucovorin is already FDA-approved and can be prescribed off-label, the label change would likely prompt more insurance plans to cover it for this new use, making it more accessible to families seeking answers.
But the FDA's decision to bypass the usual lengthy label update process and new clinical trials has raised eyebrows. Typically, such updates—especially for generic drugs—require extensive consultation and can take up to a year and a half, noted Skadden lawyer Rachel Turow in her comments to Reuters. This time, the agency invoked an obscure rule to reinstate GSK's original approval application, basing the label change on its own review of 40 patient cases from literature published between 2009 and 2024. GSK, which sold the drug as Wellcovorin until 1997, now moves to complete the application "as quickly as possible," with the FDA signaling it could process the request in as little as four to six months—or even faster.
Critics, however, are not convinced. "The evidence for leucovorin's use was limited and potentially suggestive of benefit for a small subgroup of autistic children. Larger trials are needed," Dr. Andy Shih, chief science officer at Autism Speaks, told Reuters. Dr. Karam Radwan, who directs the Neurodevelopmental Disorders Program at the University of Chicago, echoed this caution: "You want to replicate that with a different lab, in a different setting, to make sure we have enough support for the change." Current evidence is based on just four studies involving 50 to 60 patients each, three of which were conducted by the same author. Three new mid-stage trials are underway, testing a liquid formulation of leucovorin for early language impairment in autistic children, but results are not expected until December at the earliest.
Despite these reservations, the Trump administration has framed the move as a fulfillment of campaign promises to the "Make America Healthy Again" movement and to families seeking solutions to what Kennedy has called an "epidemic." Autism rates in the U.S. have soared, now estimated at 1 in 31 children by age eight. At a White House event on September 22, 2025, Kennedy, Trump, and other officials not only backed leucovorin as a potential treatment but also issued a controversial warning against the use of Tylenol (acetaminophen) by pregnant women, citing studies that suggest a possible link to autism.
This latter claim, in particular, has drawn fierce criticism from health experts. According to the Casper Star-Tribune, Trump exhorted pregnant women to avoid Tylenol, despite the American College of Obstetricians and Gynecologists' longstanding recommendation that acetaminophen is safe during pregnancy. Trump went further, suggesting delays in key childhood immunizations or separating combination vaccines—positions that fly in the face of overwhelming scientific evidence debunking any link between vaccines and autism. "Don't let them pump your baby up with the largest pile of stuff you've ever seen in your life," Trump said, using vivid and exaggerated language that left many in the medical establishment aghast.
Health experts and medical groups were quick to respond. Arthur Caplan of the New York University School of Medicine called the event "the saddest display of a lack of evidence, rumors, recycling old myths, lousy advice, outright lies, and dangerous advice I have ever witnessed by anyone in authority in the world claiming to know anything about science." The Tylenol manufacturer, Kenvue, also issued a statement disputing any link between their product and autism, warning that avoiding Tylenol could force pregnant women to choose between enduring potentially dangerous fevers or turning to riskier alternatives.
For his part, President Trump acknowledged the lack of consensus, saying, "I'm just making these statements from me. I'm not making them from these doctors." He stressed that his advice was based on "common sense" and the "information that we have," though he conceded, "when they, uh, talk about, you know, different results, different studies, I talk about a lot of common sense. And they have that, too. They have that too, a lot."
While the administration's actions have energized some parent advocates and those desperate for new treatment options, many in the scientific and medical communities remain deeply skeptical. Dr. Ameet Sarpatwari, a pharmaceutical policy researcher at Harvard Medical School, described the FDA's label change as at most a "hollow bureaucratic victory" without robust supporting evidence. Aaron Kesselheim, also at Harvard, called the process "very atypical," pointing out that without the FDA sharing its data or trials, it's hard to know if the agency is following normal standards of evidence.
In the broader conversation about autism, learning disabilities, and neurodevelopmental disorders, experts stress the importance of distinguishing between autism and learning disabilities. As reported by Stacker, while autism is not a learning disability, the two can co-occur, and certain learning disabilities—such as dyscalculia, dyslexia, and dysgraphia—are more common among autistic individuals. Diagnosis and treatment require careful observation and tailored interventions, from speech and language therapy to occupational and sensory integration therapies.
As the debate rages on, one thing is clear: the search for answers about autism—its causes, treatments, and best practices for support—remains as urgent and contentious as ever. The Trump administration's latest moves may have changed the bureaucratic landscape, but for families, doctors, and researchers, the quest for real solutions continues—one study, one conversation, and one child at a time.
