Vitamin Drug Shows Promise in Boosting Speech for Autistic Children

An existing prescription drug, leucovorin, is offering new hope for non-verbal children with autism by potentially unlocking their ability to speak. This drug, a prescription vitamin similar to folic acid, aids in restoring folate levels in those who struggle with absorption, according to Katy Dubinsky, a New York pharmacist and CEO of Vitalize.

Dubinsky explained that leucovorin is already FDA-approved for reducing the toxic effects of high-dose methotrexate in cancer treatment and for preventing anemia caused by folate deficiency.

While not specifically intended for autism, some families have reported significant communication improvements in their autistic children through off-label use, experts note. Dr. Richard E. Frye, a behavioral child neurologist in Arizona, has studied leucovorin as part of his research into experimental treatments for autism spectrum disorder.

Frye describes leucovorin as a special, water-soluble form of vitamin B9 that doesn't accumulate in the body. He also notes the long-term safety of folates, which is why they're used to supplement our food.

The primary benefit observed in children with autism is improved language skills, Frye stated, with potential advantages including enhanced social function, reduced repetitive behaviors, and improved attention. "We do find that leucovorin helps a substantial number of children with autism," he said. "Language is one of the most obvious things, so that is what we measured in our clinical trial."

Dr. Marc Siegel, a clinical professor of medicine at NYU Langone Health, explained that central folate deficiency—a lack of folate in the brain—can disrupt neurological function. He noted that many children with autism spectrum disorder have antibodies against folic receptor alpha in the brain.

Leucovorin converts to folate through a different pathway in the brain, according to Siegel. "Small studies are showing that it improves neurological function and communication skills in kids with ASD, but larger studies need to be done."

Doctors highlight that leucovorin's established history of use helps mitigate potential risks. Frye mentioned its decades-long use in protecting the body from chemotherapy effects, providing knowledge about appropriate dosages and potential side effects.

However, some children may experience hyperactivity or show no response to leucovorin, requiring alternative treatments. Experts emphasize the importance of consulting with a medical professional when considering leucovorin for autism.

"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," Frye advised. "It is important to test the patient for abnormalities in the folate pathway … so we know that they are a candidate for this medication." He also added that some children might need a compounded form of leucovorin due to sensitivities to additives in commercial products.

Frye's ultimate goal is FDA approval for leucovorin as an autism treatment, making it widely accessible upon diagnosis. Dubinsky supports this, stating that FDA approval would provide stronger medical backing, potentially leading to insurance coverage, standardized guidelines, and broader medical acceptance.

Siegel considers leucovorin a "safe drug" worthy of large-scale consideration based on recent research.

Frye cautioned that there's "no magic bullet" for autism, and many children benefit from combined treatments. He stated, "It is unlikely that leucovorin will be used alone — it must be used with behavioral and speech therapy. We believe that leucovorin can improve outcomes for these therapies."

Andy Shih, chief science officer of Autism Speaks, stressed the uniqueness of each child's experience with autism. He urged families to consult with doctors before considering off-label treatments, emphasizing the need for larger controlled clinical trials to determine leucovorin's efficacy and safety.

Dubinsky concluded that if larger studies confirm leucovorin's effectiveness, it could become a more accessible treatment for children with autism who may benefit from it.