The Trump administration is drawing attention to old generics as a potential new treatment for autism, though there’s limited evidence to back this up. This has raised concerns among doctors and scientists, who argue that further studies are needed on the drug in question.
During a recent press conference, President Trump and key health officials spoke highly of leucovorin, a synthetic version of vitamin B9 that requires a prescription. Although the Food and Drug Administration (FDA) has approved it to address side effects from certain chemotherapy drugs, it is also used off-label for individuals diagnosed with brain folate deficiency (CFD).
CFD is an extremely rare neurological condition marked by low vitamin B9 levels in the brain. Importantly, not everyone with CFD has autism, nor do all those with autism have CFD.
Leucovorin is believed to assist folic acid in reaching the brain, which leads to a theory that it could enhance language and cognitive skills in people on the autism spectrum as long as there is a folic acid deficiency.
During the White House address, Trump made a controversial connection between Tylenol, acetaminophen, and autism; FDA Commissioner Marty McCurry joined him to announce the agency’s approval for leucovorin. He claimed it could potentially benefit “hundreds of thousands” of children diagnosed with autism.
“President, you instructed us to take bold actions, focusing on medical correctness rather than business concerns,” McCurry stated.
In a separate interview with TMZ, Mehmet Oz, who oversees Medicare and Medicaid services, suggested that leucovorin might effectively serve “the reverse of autism,” but his claims were based on conversations with a physician conducting trials on autistic children. Oz expressed urgency for results, questioning whether one should wait while a child struggles.
Critics, however, contend that this administration is endorsing drugs with unproven efficacy and questionable safety records. They worry that offering hope without substantial backing could mislead countless families.
Even if leucovorin becomes more accessible, there’s a significant shortage of autism specialists to prescribe it, and not all children on the spectrum would qualify. The possibility of meaningful results remains uncertain.
Alycia Halladay, Chief Science Officer at the Autism Science Foundation, highlighted that recommendations for this drug as a treatment are premature. She noted that the limited clinical trials available included only a handful of participants, and results have been inconsistent. While the drug is deemed safe for children with cancer, its safety for those with autism remains unexamined.
“If the administration aims to fund larger trials, that would be beneficial,” Halladay stated, emphasizing that leucovorin should not be considered for FDA approval based on the current evidence.
While the FDA has adjusted leucovorin’s approved uses, it only sanctions it for adults and pediatric patients with brain folate deficiency, not autism specifically. The agency found that orally administered leucovorin calcium tablets “are addressing certain symptoms,” based on studies conducted between 2009 and 2024.
“It was misleading to assert during the press conference that this would be made available to those with autism. There’s no supporting evidence for its effectiveness across the autism spectrum,” Halladay remarked.
Audrey Brambach, a child neurology expert from the University of Texas in Austin, pointed out the necessity for large-scale control studies to discern who may experience benefits from the drug and in what doses. Current CFD treatments involve regular lumbar punctures to monitor folic acid levels, which is impractical for all children on the spectrum.
“We’re certainly not going to impose that on every child with autism to ensure correct dosing,” Brambach stated. She also indicated that autism encompasses various symptoms beyond cognitive issues and, since vitamin B9 has multiple roles, it’s challenging to determine if any improvement relates directly to autism itself.
“If its effects alleviate some related issues, it might appear to improve core autism behaviors, but it could just mean the person is functioning better overall,” Brambach explained, adding that leucovorin might really serve as a disease-modifying therapy rather than merely addressing symptoms, though there seems to be some ambiguity on this point.
In a conversation with Brian Kilmeade from Fox News Radio, Oz claimed that the necessary data would eventually emerge. He stated, “We give it, Marty is changing labels and ensuring the medication is covered. Doctors intend to prescribe this and gather data on its efficacy.”
