Families Explore Leucovorin as Potential Autism Treatment Amid FDA Consideration

Families are increasingly exploring leucovorin, a drug traditionally used to mitigate chemotherapy side effects, as a potential treatment for autism spectrum disorder (ASD). Leucovorin, also known as folinic acid, is a synthetic form of vitamin B9 essential for producing healthy blood cells.

Brian Noonan, whose 4-year-old son Benjamin was diagnosed with autism, reported improvements in Benjamin's speech after starting leucovorin. "He's verbal, but he really struggled to put together sentences," Noonan said. "Since starting on the medicine, the family believes Benjamin's speech has improved, though he still has difficulties with hyperactivity and impulsiveness." Noonan emphasized that they are aware the drug is experimental and continue to pursue other therapies.

Similarly, Ben Blomgren from Minneapolis observed significant progress in his 11-year-old son, Josh, who faced severe language deficits and behavioral challenges. After starting leucovorin, Blomgren noted improvements in sleep, toilet training, and reduced instances of running away. "It wasn't overnight, but we saw major improvement," he said.

Dr. Richard Frye, a behavioral child neurologist in Phoenix, has studied leucovorin's effects on autism for two decades. He clarifies that while leucovorin is not a cure, it may help alleviate certain symptoms in some individuals. Frye prescribes the drug to select patients and underscores the need for further research.

The U.S. Food and Drug Administration (FDA) is considering updating leucovorin's label to include treatment for cerebral folate deficiency, a condition that can affect speech, mood, and behavior, and is sometimes associated with autism. This consideration is based on a limited number of small studies. Dr. Aaron Kesselheim, a professor of medicine at Harvard Medical School, expressed concern over the FDA's approach, stating, "It's incredibly irresponsible... we saw the FDA making an announcement relying on a summary of unclear data and announcing that they had already decided to approve it."

Despite the FDA's deliberations, some families are choosing to try leucovorin off-label, often without insurance coverage. They report varying degrees of improvement in their children's symptoms, particularly in communication and behavior. However, medical experts caution that while initial findings are promising, more comprehensive studies are necessary to fully understand leucovorin's efficacy and safety in treating autism spectrum disorder.