The cancer medication leucovorin shows promise as a therapy for autism spectrum disorder, potentially enhancing communication skills in children, according to federal health officials.
Federal health officials recently highlighted the potential of leucovorin, a cancer medication, to serve as a therapy for autism spectrum disorder (ASD). During a press conference on autism-related topics, FDA Commissioner Marty Makary, National Institutes of Health (NIH) Director Jay Bhattacharya, and Centers for Medicare and Medicaid Services Administrator Mehmet Oz discussed research indicating that leucovorin may improve communication skills in some children with autism.
The U.S. Food and Drug Administration (FDA) is preparing to approve leucovorin as a treatment for speech-related deficits associated with ASD. This approval will involve updating the drug’s label to include treatment for cerebral folate deficiency, a condition linked to autism. Following this label update, state Medicaid programs will be able to cover leucovorin for autism treatment, as stated in a press release from the Department of Health and Human Services (HHS).
The NIH is also set to initiate new research initiatives aimed at examining the safety and effectiveness of leucovorin for children with autism. Preliminary studies suggest that the drug could help some non-verbal children develop the ability to speak.
Leucovorin, a prescription vitamin, is primarily used to treat cancer and anemia. It functions similarly to folic acid, aiding in the restoration of folate levels in patients who struggle to absorb or utilize it effectively. Katy Dubinsky, a New York pharmacist and CEO of Vitalize, a private supplement company, explained that leucovorin is FDA-approved to mitigate the toxic effects of high-dose methotrexate in cancer treatment and to prevent or treat certain types of anemia caused by folate deficiency.
While leucovorin is not currently prescribed for autism, some families have reported significant improvements in their children’s communication skills through its off-label use. Dr. Richard E. Frye, a behavioral child neurologist in Arizona, has conducted research on the drug as part of his exploration of experimental treatments for children with ASD. He noted that leucovorin is a special form of vitamin B9 that is water-soluble and does not accumulate in the body.
Dr. Frye emphasized that folates have been deemed safe for long-term use, which is why they are supplemented in food. He pointed out that the primary benefit of leucovorin for children with autism is its potential to enhance language skills, although it may also improve social functioning, reduce repetitive behaviors, and strengthen attention skills. “We do find that leucovorin helps a substantial number of children with autism,” he said, adding that language improvement is the most measurable outcome in clinical trials.
Dr. Marc Siegel, a clinical professor of medicine at NYU Langone Health and Fox News’ senior medical analyst, also discussed the drug’s positive effects. He explained that central folate deficiency, characterized by a lack of folate in the brain, can disrupt neurological function. Research has shown that a significant percentage of children with ASD have antibodies against folic receptor alpha in the brain. Leucovorin is converted to folate in the brain through a different pathway, which may explain its beneficial effects.
Although small studies have indicated that leucovorin can improve neurological function and communication skills in children with ASD, experts agree that larger studies are necessary to confirm these findings. The long history of leucovorin’s use in cancer treatment helps mitigate associated risks, according to Dr. Frye. “One of the advantages of using leucovorin is that it’s been used for decades to protect the body from the effects of chemotherapy, so we know what dose to use and any side effects it may produce,” he stated.
However, Dr. Frye cautioned that some children may experience hyperactivity or may not respond to leucovorin, necessitating alternative treatments. He stressed the importance of consulting with a medical professional when considering leucovorin for autism treatment. “Leucovorin is given at much higher doses than regular vitamins, so it’s very important to work with a doctor knowledgeable in how to prescribe it,” he advised.
While leucovorin is available at pharmacies, Dr. Frye noted that some children may require a compounded form due to sensitivities to certain additives in commercial products. He expressed hope that the FDA will approve leucovorin for autism, allowing it to be widely prescribed as one of the first treatments following diagnosis.
Dubinsky concurred, stating that physicians may prescribe the “widely used and well-tolerated drug” for autism treatment based on clinical judgment and emerging evidence. She emphasized that FDA approval would lend stronger medical backing, transforming leucovorin from an off-label option into a recognized treatment. This could lead to insurance coverage, standardized dosing guidelines, and greater acceptance within the medical community.
Dr. Siegel added that leucovorin is a “safe drug” and should be considered for broader use based on recent research findings. However, Dr. Frye cautioned that there is “no magic bullet” for autism, noting that many children benefit from a combination of treatments. “It is unlikely that leucovorin will be used alone — it must be used with behavioral and speech therapy,” he said, adding that leucovorin could enhance outcomes for these therapies.
Dr. Andy Shih, chief science officer of Autism Speaks, highlighted the individuality of each child’s experience with autism. He noted that while some small studies have explored leucovorin as a potential treatment, larger controlled clinical trials are essential to determine its efficacy and safety. He urged families to consult with their doctors before considering off-label treatments.
Dubinsky concluded that if larger studies confirm leucovorin’s effectiveness, it could become a more accessible treatment option for children with autism who may benefit from it.
Source: Original article