Potential Speech Gains in Autistic Children Linked to Off-Label Use of Leucovorin

An off-label prescription drug has shown promise in helping some non-verbal children with autism communicate more effectively. Evidence suggests that leucovorin, a prescription form of vitamin B9, may improve speech in some kids on the autism spectrum.

Leucovorin acts similarly to folic acid, aiding in restoring folate levels in children who may struggle with absorption. According to Katy Dubinsky, a New York pharmacist and co-founder of Vitalize, a private supplement company, this vitamin is primarily known for its FDA-approved use in lessening the toxic side effects of high-dose methotrexate during cancer treatment as well as preventing or treating certain anemia related to folate deficiency.

Though leucovorin is not officially prescribed for autism treatment, numerous families assert that off-label use has led to noticeable enhancements in their children’s communication skills.

Research Insights from Experts

Dr. Richard E. Frye, a behavioral child neurologist based in Arizona, dives deeper into the research surrounding leucovorin. His studies focus on experimental treatments for autism spectrum disorder, and he emphasizes the significant benefits of leucovorin.

“Leucovorin is a unique form of vitamin B9, and importantly, it is water-soluble. It does not accumulate within the body, which is a safety feature,” Frye shared. “Folates have been established as safe for long-term use, which is why they are commonly added to our foods.”

Types of Improvements Observed

According to Frye, the primary advantage of leucovorin for autistic children is its potential to enhance language skills. Other reported benefits include improved social interactions, a decrease in repetitive behaviors, and heightened attention spans.

“In our clinical trials, we have noted that leucovorin positively impacts a substantial number of children on the spectrum, with language development standing out as a primary measure,” he conveyed.

Dr. Marc Siegel, a clinical professor of medicine at NYU Langone Health and senior medical analyst at Fox News, discussed the underlying mechanisms for this positive effect. He noted that a significant number of children with autism experience a deficiency in folate within the brain, which can hinder neurological function.

“A high percentage of children with autism spectrum disorder have been found to possess antibodies against folic receptor alpha in the brain,” Siegel stated. “Leucovorin, however, is converted to folate in the brain through a different pathway, which may explain its effectiveness.”

Emerging small-scale studies hint that leucovorin enhances neurological function and communication skills in affected children, but Siegel stresses the necessity of larger-scale studies for confirmation.

Safety Profile and Administration

One of the notable advantages is leucovorin’s well-documented safety record from its long-term use, primarily in oncology. Frye assured, “Because leucovorin has been utilized for decades to mitigate chemotherapy effects, we are confident in the established dosing guidelines and known side effects.”

Nevertheless, it is important to note that some children may experience hyperactivity or may not respond to leucovorin, potentially requiring alternative treatment strategies.

Families exploring leucovorin for autism should ideally collaborate closely with healthcare professionals. Frye recommends this collaboration, highlighting the importance of administering the correct dosages, which significantly exceed typical vitamin levels. “Testing for abnormalities in the folate pathway is crucial to identifying suitable candidates for this medication,” he advised.

Compounded Forms and Access Challenges

While leucovorin is accessible at pharmacies, Frye points out that some children might need a compounded version due to sensitivities to additives in commercial formulations.

“Our ambition is to achieve FDA approval for leucovorin for autism treatment, enabling it to be a standard prescription for newly diagnosed children,” he stated. Such approval could bolster the legitimacy of this treatment option.

The Role of Clinical Trials and Future Perspectives

Dubinsky shared her perspective on the prescription landscape for leucovorin, agreeing that physicians may prescribe this well-tolerated drug for autism based on clinical judgment and emerging evidence. However, she underscores the essential role of clinical trials.