Dr. Marty Makary, the commissioner of the Food and Drug Administration, announced on Monday that the agency would be modifying the label of a relatively obscure medicine so that “it can be available for children with autism.”
He was referring to leucovorin, or folinic acid, a modified version of vitamin B9, also known as folate — which is naturally found in beans, leafy greens, eggs, beets and citrus.
Folate helps the body make red blood cells and is important for cell growth. It’s especially crucial during early pregnancy to lower the risk of major birth defects in a baby’s brain or spine. Studies suggest that folate levels can affect our health in various ways, and scientists are researching what role folate plays in depression, dementia, heart disease and autism.
Some people have antibodies that interfere with how folate is transported within the body, and small studies suggest that a number of people with autism — in some cases up to 75 percent — may have these antibodies.
In a Federal Register notice filed on Monday, the F.D.A. said it was approving leucovorin tablets for people with “cerebral folate deficiency,” based on a review of studies from 2009 to 2024 that found that they “improve certain symptoms.” The agency, noting that more studies were needed, cited one study that compared 40 people on the medication and 40 on a placebo; those who took the medication showed “substantial improvement” of the deficiency symptoms.
The medicine has been used off-label to treat people diagnosed with cerebral folate deficiency for about two decades.
Symptoms of cerebral folate deficiency usually begin to show up around the age of 2 when children start to experience speech difficulties, intellectual disabilities and, in some cases, seizures. They may also have tremors and difficulty controlling their muscle movements.
Leucovorin is formulated to bypass some of the problems that can block folate from entering the brain, said Edward V. Quadros, a research professor at SUNY Downstate Health Sciences University and an expert on folate metabolism.
In studies, some children with autism who received folinic acid have seen big improvements in their receptive language, said Dr. Richard E. Frye, who researches autism. He has found that switch in the ability to understand spoken or written words, as well as in their expressive language, and the ability to communicate thoughts and feelings, particularly among the children who had antibodies that block folate transport to the brain.
But for some of the children who participated in Dr. Frye’s studies, the leucovorin had no effect at all.
“It is not a cure, but improves the function of many children substantially,” he said.
Larger studies that use placebos would be needed to pinpoint exactly who would benefit from the medicine and at what specific dose, said Dr. Alycia Halladay, the chief science officer of the Autism Science Foundation.
She and other experts warned that it was too early to view the drug as a potential cure or a means of prevention for those with autism.
“Where there’s limited amount of funding, then there are much more higher priority items than this,” Dr. Halladay said. “The data just are not there.”
Leucovorin is currently approved by the F.D.A. to treat the harmful side effects of chemotherapy drugs like methotrexate and to help treat colorectal cancer, when used in combination with the cancer drug fluorouracil. Leucovorin is also used to treat megaloblastic anemia, which commonly occurs when the body doesn’t get enough folic acid.
The drug can be administered via a pill, injection or intravenously — and can also be compounded into a liquid medicine, a process that is not typically covered by insurance.
The side effects associated with taking leucovorin are minimal, Dr. Quadros said, because the medicine is derived from a water-soluble vitamin. Within 24 hours most of it is expelled in your urine.
One analysis found that adverse effects included aggression, agitation, headache, insomnia and increased tantrums.
Christina Jewett contributed reporting.
Christina Caron is a Times reporter covering mental health.
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