From cancer to care: Leucovorin’s journey as a repurposed autism treatment

Leucovorin, a folate derivative once used in chemotherapy, is now being repurposed for autism after evidence of benefits in cerebral folate deficiency. This development illustrates the intersection of biomedical innovation, regulatory flexibility and public health policy.

What is leucovorin?

Leucovorin, or folinic acid, is a modified form of folate (Vitamin B9). Unlike regular folate, it can be directly absorbed and utilised by the body. Traditionally, it was used in cancer therapy, especially to counteract the toxic effects of methotrexate. It also finds use in certain types of anaemia and as a supportive cancer drug.

Why is it in the news?

In September 2025, the US FDA began the approval process to expand leucovorin’s use for cerebral folate deficiency (CFD), a rare condition marked by impaired folate transport into the brain. Symptoms of CFD overlap with those of autism spectrum disorder (ASD): developmental delays, seizures and communication deficits.

The turning point came from clinical studies (2009-2024) showing that leucovorin improved speech, social behaviour and overall autism severity in children with folate receptor autoantibodies (FRAA). These children cannot transport folate properly to the brain. By bypassing this pathway, leucovorin helps restore folate balance in the nervous system.

Why consider leucovorin for autism?

Clinical results: Randomised trials in the US, France and India show significant improvement in communication and adaptive behaviour.

Symptom relief: Reduced autism severity scores (ADOS, CARS) in FRAA children.

Safety: No serious adverse effects reported.

Scientific rationale: Folate is critical for neurotransmitters, DNA methylation and neurodevelopment. Disruptions can derail brain growth; leucovorin helps plug this gap.

Policy and social relevance for India

1. Public health challenge: Autism prevalence is rising, with heavy socio-economic costs for families and the state.
2. Drug repurposing as policy innovation: A decades-old chemotherapy rescue drug may now transform autism care.
3. Regulatory foresight: FDA’s move underlines the importance of flexible, evidence-based policymaking. India too must be adaptive.
4. Screening and diagnostics: Wider testing for folate receptor antibodies and CFD could become essential.
5. Equity and ethics: Affordability, access and responsible use need policy safeguards to avoid exploitation.
6. Research collaboration: Government, pharma (e.g., GSK’s Wellcovorin) and academia must work together.

Scientific and ethical concerns

• Evidence strongest in specific autism subgroups (FRAA-positive); generalising prematurely could mislead
• Long-term safety and efficacy remain under study
• Ethical questions on pricing, patent extensions and equitable access
• Overmedicalisation of autism must be avoided

Big picture

Leucovorin is a classic case of how old drugs can find new lives through science and policy. For civil service aspirants, it’s a reminder that health policy is not static. It evolves at the intersection of research, regulation and social need. Autism, once considered beyond medical intervention, may soon see therapeutic hope, raising both opportunities and challenges for governance.

Possible Civil Service (Main) questions

Analytical questions

1. Discuss how drug repurposing, using leucovorin as a case study, highlights the role of innovation in public health policy.
2. Examine socio-economic impact of autism in India and assess whether pharmacological interventions like leucovorin should be prioritised over social support mechanisms.
3. How can regulatory bodies in India balance fast-tracking new drug indications with ensuring safety, affordability and equitable access?

Critical questions

1. “Leucovorin’s promise lies as much in policy foresight as in medical research.” Discuss.
2. Critically analyse the ethical challenges of using repurposed drugs in vulnerable populations such as children with autism.
3. To what extent should India invest in screening technologies (like FRAA testing) before mainstreaming leucovorin for autism care?