As the U.S. Food and Drug Administration (FDA) moves closer to approving folinic acid as an official treatment for a brain-related condition linked to autism, many parents are eager to know how it might help children with autism spectrum disorder.
Folinic acid, also known as leucovorin, is a lab-made version of folate (vitamin B9). Folate is an important nutrient found in foods like leafy greens, beans, and citrus fruits.
It supports brain development, helps with cell growth, and is needed to make DNA. Folic acid, often taken during pregnancy to prevent birth defects, is another form of this vitamin but is used in much smaller doses.
Doctors have been prescribing folinic acid for decades to reduce side effects from chemotherapy, especially when patients are treated with a strong drug called methotrexate.
In the past 20 years, doctors also began using it to treat a rare condition called cerebral folate deficiency, or CFD. In CFD, the brain doesn’t get enough folate, which can lead to serious neurological symptoms.
What’s new now is that the FDA is preparing to officially approve folinic acid as a treatment for CFD. This means it will no longer be an “off-label” use but an approved one.
CFD has long been connected with autism spectrum disorder, as both conditions can involve speech delays and motor problems. Many children with CFD have something called folate receptor autoantibodies (FRAAs).
These are antibodies that block folate from entering the brain. Interestingly, children with autism are about 19 times more likely to have these antibodies compared to children who are developing typically.
Studies suggest that folinic acid may help improve verbal skills and behavior in autistic children—especially those who test positive for FRAAs. While the early results are encouraging, experts caution that this treatment is not a cure for autism. It’s being approved to treat CFD, not autism itself.
Folinic acid works by giving the brain the folate it needs in a form that can bypass the usual transport system, which doesn’t work well in people with CFD. According to experts, it’s a useful treatment, but there are still many questions, such as how long someone should take the drug and what the ideal dose is.
The FDA is reviewing studies and medical reports published between 2009 and 2024 instead of requiring new large-scale clinical trials. This faster approach may help bring the treatment to children who need it sooner. FDA Commissioner Dr. Marty Makary said that children deserve access to treatments that show promise.
While folinic acid is generally safe and well-tolerated, doctors must still be careful. It can reduce the effectiveness of seizure medications, which are sometimes prescribed to children with autism or CFD. So, doctors need to check for drug interactions.
Another concern is making sure there is enough folinic acid for everyone. Since leucovorin is used in cancer treatment, a sudden increase in demand could affect cancer patients who depend on it.
In summary, folinic acid may help children with CFD and possibly some children with autism who have folate transport issues. But more research is needed. Doctors and families should stay informed and weigh the benefits and risks before starting treatment.
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