Leucovorin and Autism — Can a Cancer Drug Help Kids With Social Skills?

Leucovorin, also called folinic acid, has quietly stepped into the autism conversation. Once known only as a “rescue drug” for cancer patients, it is now being studied as a possible treatment for autism-related symptoms. But how much of this is science, and how much is just hype?

What is Leucovorin?

Leucovorin is a medical form of vitamin B9 (folate). In oncology, it helps healthy cells survive chemotherapy and boosts the effectiveness of certain cancer medicines. Doctors also use it for anemia and other folate-related conditions.

Now, researchers are curious about its effects on children with autism spectrum disorder (ASD), especially those with cerebral folate deficiency (CFD), a rare metabolic disorder where the brain doesn’t get enough folate.

The Link Between Folate and Autism

Folate is critical for DNA repair, brain development, and neurotransmitter function. In some autistic children, studies have shown low spinal fluid levels of folate. This deficiency overlaps with autism symptoms like speech delays, seizures, and social difficulties.

Giving leucovorin might bypass the problem by delivering folate in an active form the brain can use.

Research So Far

• A 2021 review of 21 studies found improvements in language and social skills in children who took leucovorin.
• A 2024 U.S. trial with about 80 children showed positive changes in communication.
• A 2025 study in China found improvements in social reciprocity with leucovorin treatment.

But experts caution: the studies are small, varied in dosage, and not enough to make leucovorin standard care for autism.

Side Effects and Risks

Common issues include nausea, vomiting, and rare seizures. Since leucovorin is a prescription drug, doctors warn against parents trying it without medical supervision.

Bottomline

Leucovorin may offer hope for specific autistic children, especially those with folate metabolism problems. But until larger trials confirm benefits, families should view it as an experimental option, not a proven cure.

What is leucovorin used for normally?
It’s mainly used in chemotherapy to reduce toxicity and improve drug effectiveness.

Can leucovorin cure autism?
No. It may improve certain symptoms in some children, but it is not a cure.

Is leucovorin the same as folic acid?
No. Folic acid is a supplement, while leucovorin is an activated form of folate used in medicine.

What are the risks of leucovorin?
Possible side effects include allergic reactions, nausea, and seizures.

How quickly does leucovorin work?
Some families report changes in weeks, but results vary widely.

Related Article: Leucovorin vs Folic Acid: The Autism Link Explained

Related Article: Leucovorin and Autism: Cost, Hype and Real Concerns

Read more at: Todaybeat.com