Robert F. Kennedy Jr.’s autism remedy is not all it’s cracked up to be, according to his own agency.
Two Food and Drug Administration officials told Politico on Monday that the drug leucovorin, which the Health and Human Services Secretary and the FDA Commissioner Marty Makary had promoted as a helpful treatment for children with autism during a September news conference, lacks sufficient evidence to support its use in that manner.
On Tuesday, the FDA approved leucovorin, also known as folinic acid, for the treatment of adult and pediatric patients with cerebral folate deficiency who have a specific gene variant (CFD-FOLR1), but not for autism.
The FDA press release classified the disease as a “rare genetic condition” that affects the transport of folate, an essential vitamin, to the brain, resulting in “severe developmental delays, movement disorders, seizures, and other serious neurological complications.”
Makary explained at the September conference that he would fast-track the drug’s approval to treat kids with autistic symptoms, despite scientists advising that the drug needed more research.
“This action may benefit some individuals with FOLR1- related cerebral folate transport deficiency who have developmental delays with autistic features,” Makary, 55, said in a statement included in Tuesday’s approval announcement.
Fewer than one in a million people in the U.S. have the rare genetic disorder.
Makary, along with Dr. Mehmet Oz and National Institutes of Health Director Jay Bhattacharya, penned an opinion article for Politico Magazine in September stating that, though leucovorin “is not a cure for autism,” there was evidence suggesting it could help some autistic children improve their verbal communication.
An official told Politico that Makary’s faith in the drug’s efficacy in treating autism stemmed from a trial published in The European Journal of Pediatrics in September 2024. The study was later retracted in January due to errors identified in the reported results.
RFK Jr.’s autism crusade during his tenure as the HHS secretary has included some other questionable claims.
At the same September conference, RFK Jr., 72, and Trump, 79, decried the use of acetaminophen, widely known by brand name Tylenol, by pregnant women, saying that the common painkiller’s use “can be associated with a very increased risk of autism.”
Doctors from trusted health organizations around the world blasted RFK Jr.’s claims, echoing the view that there was no credible evidence to support a connection between the two.
“There is no evidence to link the use of [Tylenol] by pregnant women to autism in their children. None,” said U.K. Health Secretary Wes Streeting in September. “In fact, a major study was done back in 2024 in Sweden, involving 2.4 million children, and it did not uphold those claims.”
Kennedy Jr. also bizarrely tied the disorder to circumcision, saying that “children who are circumcised early have double the rate of autism, and it’s highly likely because they’re given Tylenol.”
When reached for comment, an HHS spokesperson told the Daily Beast: “We were previously referring to cerebral folate deficiency — which can be caused by antibodies blocking folate receptors — rather than cerebral folate transport deficiency, which is caused by a specific genetic mutation. The FDA’s approval of the first treatment for cerebral folate transport deficiency yesterday may benefit some individuals who have developmental delays with autistic symptoms."
The Daily Beast reached out to the FDA for comment.
