FDA Approves Old Drug as Autism Treatment, Leaving Experts Concerned

The US Food and Drug Administration (FDA) has approved an old drug, leucovorin, as a treatment for some people with autism, departing from its typical standard for reviewing drugs. The decision was sparked by a White House briefing, where President Trump and top health officials endorsed the drug as a potential therapy for autism. However, experts have raised concerns about the FDA's decision, citing unanswered questions about the drug's effectiveness and potential misuse.

Key Takeaways:

• The FDA approved leucovorin, a generic drug used to treat chemotherapy's toxic effects, as a treatment for cerebral folate deficiency, a condition associated with delays in child development and poor motor skills.
• The approval was made by the FDA on its own, without a formal application from a pharmaceutical company, which is a rare departure from the agency's standard process.
• Leucovorin is estimated to help "hundreds of thousands" of children with autism, according to FDA Commissioner Dr. Marty Makary.
• However, experts have questioned the FDA's decision, citing concerns about the drug's effectiveness, its narrow application, and the lack of clarity on its dosage and diagnosis.
• Dr. Aaron Kesselheim, a professor of medicine at Harvard Medical School, called the FDA's endorsement of leucovorin "shocking" and noted that the agency's action left many questions unanswered.
• The approval process also failed to clarify the appropriate dose of leucovorin for children and adults, which is typically determined by each drugmaker from clinical studies.
• Some experts have warned that the FDA's decision could set a precedent for other companies to skip costly clinical trials before asking the agency for approval.
• The FDA's action has already led to a run on over-the-counter supplements containing the active ingredient in leucovorin, folinic acid.
• Parents of autistic children have turned to folinic acid supplements as a way of overcoming difficulties accessing the drug in the past.

Statistics:

• 20-50% of children with autism have cerebral folate deficiency, according to Dr. Makary.
• Leucovorin is estimated to help "hundreds of thousands" of children with autism.
• Folinic acid supplements can be purchased online without a prescription, creating a more convenient, and also more lightly regulated, avenue for parents to buy the product.
• The supplement industry makes an estimated $140 billion in annual sales (note: this figure is not explicitly mentioned in the article, but is a common estimate).

Sources:

• The New York Times article (no date mentioned in the original text)
• The Food and Drug Administration (FDA)
• The White House
• Harvard Medical School
• University of Pennsylvania
• University of Utah
• iHerb
• The Wellness Company
• Centers for Medicare and Medicaid Services
• Health and Human Services Department
• GSK (GlaxoSmithKline)
• Daniel Aaron, associate professor of law at the University of Utah
• Dr. Aaron Kesselheim, professor of medicine at Harvard Medical School
• Holly Fernandez Lynch, associate professor of law at the University of Pennsylvania
• Edward V. Quadros, research professor at SUNY Downstate Health Sciences University
• Daniel Fabricant, president and chief executive of the Natural Products Association