Leucovorin, a medication that’s derived from folic acid, is in the spotlight after federal health officials approved the drug as a potential treatment for autistic, non-verbal children. According to experts, this move by health officials is unexpected, considering there haven’t been enough research studies to produce scientific evidence that the drug is effective. According to families with autistic youth, the move is an exciting, welcomed change.

Leucovorin is not a new drug. It’s a generic medication that’s been used for a long time to treat patients with cancer who are undergoing chemotherapy. According to reports, families within autism communities began attempting to give their children Leucovorin, and they were surprised by the results. Some families claimed that their nonverbal, autistic children started to show improvements in being able to speak after starting to take the drug regularly. These reports, while taken very seriously by health experts, were not scientific studies, so researchers got to work.

So far, there have only been small, randomized trials to test out Leucovorin’s effectiveness on treating nonverbal autism. One of those studies looked at 48 children over the course of 12 weeks. This study showed that 65% of children on leucovorin had notable improvements in language compared to only 24% of the children on the placebo.

Another great finding from these small trials is that the drug does generally appear to be safe for nonverbal autistic youth, but it does include side effects like temporary hyperactivity and irritability.

One interesting finding from these smaller trials is that the drug appears to be most effective for children who have specific metabolic differences. While it’s unclear exactly how it works, yet it seems that these metabolic differences that prevent proper folate transportation to the brain are eased through the use of Leucovorin, which is derived from folic acid.

All these findings are exciting and promising, but it’s also clear that more large-scale studies are needed to fully understand how Leucovorin helps nonverbal youth. Clinicians have also warned families not to consider Leucovorin a cure, especially since studies still show mixed results when it comes to the drug’s effectiveness. These great benefits may only apply to patients who are experiencing metabolic differences.

Another important thing for families to consider is that Leucovorin is a generic type of drug, so unfortunately, consistency and availability may vary based on where patients live.

Finding Ways to Cope With an Autism Diagnosis

If you’re currently in Las Vegas or a nearby area and your family is looking for ways to help improve the life of your autistic loved one, then you may want to consider the services offered here at the Nevada Autism Center. Our facility seeks to help empower autistic youth to become as successful and independent as possible by offering consistent ABA therapy and community support.

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Last Updated: January 16th, 2025