A prescription drug traditionally used for cancer treatment and anaemia, leucovorin, has shown potential in helping some non-verbal autistic children develop speech, according to emerging research. Although not officially approved for autism treatment, some families have reported improvements in communication skills after its off-label use. Leucovorin, a form of vitamin B9, supports folate absorption and is believed to aid neurological function in children with autism who may have a deficiency.

Dr Richard Frye, a behavioural child neurologist, has led research into leucovorin’s effects, noting that it may also enhance social interaction, reduce repetitive behaviours, and improve attention. Studies suggest a significant number of autistic children have antibodies that block folate transport to the brain, and leucovorin bypasses this issue by using an alternative pathway. While small-scale studies have shown promising results, experts stress that larger clinical trials are needed to confirm its effectiveness.

Medical professionals warn that while leucovorin has been used safely for decades, it is not suitable for all autistic children. Some experience increased hyperactivity or no benefit at all, making professional medical guidance essential before considering treatment. Researchers are working towards gaining FDA approval, which could lead to standardised dosing, greater medical recognition, and potential insurance coverage.

Experts emphasise that leucovorin is not a standalone solution but could complement behavioural and speech therapies. Autism Speaks’ chief science officer, Dr Andy Shih, cautioned that individual successes cannot yet be applied to the broader autistic community, reinforcing the need for further research. Families are encouraged to consult healthcare professionals before exploring off-label treatments.