FDA Approves Old Drug as Autism Treatment, Causing Some Experts to Raise Concerns

President Trump and top health officials endorsed the approval of an old generic drug, leucovorin, as a treatment for autism during a White House briefing on Monday. The agency's decision to allow the expanded use of leucovorin, which has been used to treat chemotherapy's toxic effects, has sent some customers scrambling to buy over-the-counter supplements with the same ingredients, particularly folinic acid. The FDA's typical standard for reviewing drugs was departed from in this case, as the agency made the approval decision on its own without a formal application from a pharmaceutical company.

Key Takeaways:

• The FDA approved tablets of leucovorin specifically to treat "cerebral folate deficiency," a condition characterized by low levels of folate in spinal fluid associated with delays in development and poor motor skills.
• Dr. Marty Makary, the FDA commissioner, announced that about 20 to 50 percent of children with autism have this deficiency, and the agency believes the prescription drug can improve speech and cognitive abilities in people with autism.
• However, some outside experts, like Dr. Aaron Kesselheim, a professor of medicine at Harvard Medical School, have raised concerns over the lack of clear diagnosis and the risk of overprescription without a confirmed diagnosis.
• The F.D.A.'s action also failed to clarify the appropriate dose of leucovorin for children and adults, which is typically determined by each drugmaker from clinical studies.
• The approval of leucovorin has sparked concerns about the scientific evidence supporting the treatment, with some experts warning that the agency's approval could set a precedent for other companies to skip costly clinical trials.
• Some customers are already buying over-the-counter supplements containing folinic acid, which may not be regulated as stringently as prescription drugs, raising concerns about the quality and efficacy of these products.
• Edward V. Quadros, a research professor at SUNY Downstate Health Sciences University, has studied the relationship between folate and autism and warns against the use of online folinic acid supplements due to their manufacturing regulations.

Statistics:

• About 20 to 50 percent of children with autism have cerebral folate deficiency, according to Dr. Makary.
• Leucovorin has been used to treat chemotherapy's toxic effects.
• The FDA has approved tablets of leucovorin specifically to treat cerebral folate deficiency.
• The agency believes the prescription drug can improve speech and cognitive abilities in people with autism.
• The F.D.A.'s approval of leucovorin falls below the standard for reviewing a treatment for a condition as common as autism.

Sources:

• "FDA approves drug to treat autism" by Tierney L. Cross, The New York Times
• "FDA commissioner, Dr. Marty Makary, announces approval of leucovorin" by The New York Times
• "Statement from the Health and Human Services Department" by The New York Times
• "GSK, the original company that applied for approval of leucovorin, will update the label on the product" by GSK
• "The Wellness Company, a Florida supplement seller, advertised a vitamin B product that contains folate" by The Wellness Company