The Food and Drug Administration (FDA) announced this Tuesday the approval of the drug leucovorina synthetic form of vitamin B9 that the Trump administration has promoted as a treatment for autism.
But the FDA did not approve the drug for autism, but for brain folate deficiencya rare neurological condition characterized by low levels of vitamin B9 in the brain.
Contrary to messages that the president and FDA Commissioner Marty Makary issued in September when they announced a plan to relabel the drug, a top federal agency official said Monday that There is insufficient data to support the use of leucovorin as a treatment for autism..
“We don’t have enough data to say that we can establish efficacy for autism more broadly,” the official said. “It will be up to patients to talk to their doctors to see if that might be right for them.”
Leucovorin is primarily used to help mitigate the side effects of chemotherapy or improve its effectiveness in cancer patients. However, at a press conference on September 22, Makary stated that the FDA was taking steps to change the drug’s label “so that it can be made available to children with autism,” and opined that “hundreds of thousands of children will benefit from it.”
Trump assured at the time that the drug’s updated label would “reflect potential benefits in reducing some symptoms of autism.”
“This gives hope to many parents with autistic children that it is possible to improve their lives,” he said.
Many experts who research or treat autism disputed that rhetoric, saying the drug needed more study before being given to patients with autism.
Alycia Halladay, chief scientific officer of the Autism Science Foundation, added that Tuesday’s FDA announcement “1000% contradicts” the government’s rhetoric.
The actual change that the FDA proposed almost six months ago did not say that the drug would be approved to treat autism. Rather, the agency explained that it was initiating approval of leucovorin tablets for patients with brain folate deficiency and described an overlap between symptoms of that condition and autism, such as difficulties in social communication, sensory processing and repetitive behaviors.
An estimated one in every million people suffers from brain folate deficiency, and although some researchers suspect the condition could be linked to autism, it is thought to only affect a small minority of patients.
Although leucovorin is not approved to treat autism, doctors may prescribe it off-label. Some did so before Trump’s comments in September, based on the results of a few small trials conducted mostly outside the United States. One of those trials, published in the European Journal of Pediatrics, was withdrawn in January after the authors identified several errors in their data.
In the two and a half months after Trump’s announcement in September, leucovorin prescriptions for children increased 71%, according to data published last week in the medical journal The Lancet.
“The alarm has been raised and we have already seen in the data that leucovorin prescriptions have skyrocketed,” Halladay added. “I don’t think that will change with this announcement that it’s only approved for brain folate deficiency.”
David Mandell, a professor of psychiatry at the University of Pennsylvania, said this week’s FDA announcement amounts to a mixed message, given the Trump administration’s description of leucovorin in September.
“This is terrible for families: this push and pull over what works to treat autism, what doesn’t treat it, what causes it and what doesn’t cause it,” he said. “Families deserve better than that. They deserve more careful science. They deserve more accurate information.”
Still, Mandell said he was “relieved” that the FDA did not approve leucovorin for patients with autism, “given the very weak data and my understanding that the current trials being conducted are not promising for autism.”
