The Food and Drug Administration plans to approve a new use for the generic drug leucovorin in the coming weeks to treat kids with “cerebral folate deficiency and autistic symptoms,” according to a POLITICO Magazine opinion piece by federal health leaders published on Monday.
The officials – FDA Commissioner Marty Makary, National Institutes for Health Director Jay Bhattacharya and Centers for Medicare and Medicaid Services Administrator Mehmet Oz – pointed to research they say suggests leucovorin, also known as folinic acid, may help children who are deficient in folate, a vitamin. They said there was evidence leucovorin, which is currently used to treat cancer and anemia patients, can help children with autism improve their verbal communication. But they emphasized in the opinion piece that the drug “is not a cure for autism.”
While scientists say leucovorin, a form of vitamin B, could be promising for a subset of autism patients, they cautioned that the current data is limited and the drug needs more research.
The agency leaders also encouraged pregnant women to use Tylenol and generic acetaminophen “judiciously” in consultation with a health care provider — but acknowledged there is conflicting research about whether use of the medication is correlated with subsequent diagnosis of autism and attention deficit/hyperactivity disorder.
“In the coming weeks, the FDA will approve leucovorin as a treatment for children with cerebral folate deficiency and autistic symptoms. Over half of all American children are insured by Medicaid or CHIP, so upon this FDA label change, states will be required to cover leucovorin around the country,” the agency leaders said, referencing the federal-state health insurance programs for low-income people.
The opinion piece comes as the White House announced the moves on Monday. Health Secretary Robert F. Kennedy Jr. in April pledged to have answers about the causes of autism by September.
Doctors already discourage pregnant women from taking Advil or ibuprofen because of a known risk of birth defects, leaving acetaminophen the only over-the-counter drug approved to treat pain relief and fevers during pregnancy. Fevers, the officials wrote, can also pose a risk to unborn children and cause neural tube defects.
“Peer-reviewed data from large-scale cohort studies, including the Nurses’ Health Study II and the Boston Birth Cohort, find this association,” they wrote, referencing long-term research at Harvard and Johns Hopkins University. “At the same time, we also recognize the literature continues to evolve and evidence from family control studies have failed to find a correlation.”
Both the Society for Maternal Fetal Medicine and the American College of Obstetricians and Gynecologists are retaining their guidance on acetaminophen, according to ACOG President Steven Fleischman.
“These studies are looking for associations, they don’t show causation,” said Dr. Peter Bernstein, a member of American College of Obstetricians and Gynecologists clinical consensus committee. “Not treating a fever may increase the risk of the pregnancy, certainly more than the Tylenol use.”
On Monday, the United Kingdom’s Medicines and Healthcare products Regulatory Agency released a statement from its chief safety officer backing the safety of paracetamol, another name for acetaminophen.
“Patient safety is our top priority. There is no evidence that taking paracetamol during pregnancy causes autism in children,” MHRA Chief Safety Officer Alison Cave said. “Paracetamol remains the recommended pain relief option for pregnant women when used as directed.”
Alice Miranda Ollstein contributed to this report.
