A low-cost drug long used in cancer care is showing promise in helping some children with autism improve their speech and behavior.
The medication, called leucovorin, is a form of folate, a B vitamin important for brain development. It costs only a few dollars per pill and is already approved by the FDA for other medical uses. Some doctors are now prescribing it “off-label” for children with autism.
Research suggests many autistic children may have trouble getting enough folate into the brain. In some cases, antibodies block the transport of folate where it is needed. Leucovorin appears to bypass this blockage and improve communication skills.
In small studies, children who took leucovorin for several months showed gains in verbal communication and understanding language. Some previously nonverbal children began speaking words and short sentences.
However, experts caution that the treatment is still considered experimental for autism. Larger clinical trials are needed before it can receive full FDA approval for this use. Doctors also stress that leucovorin is not a cure and does not replace therapies such as speech or behavioral treatment.
Researchers are continuing studies to confirm who may benefit most. While early results are encouraging, specialists advise parents to see this as a hopeful development, not a guaranteed solution.