Can leucovorin cure autism? Meet the moms determined to find out

A humble, decades-old folate compound — used not to fight cancer but to ease the side effects of chemotherapy — has become the latest flashpoint in America’s health wars.

On September 10, the Trump administration announced that the FDA would move toward approving leucovorin for children with cerebral folate deficiency, a rare metabolic disorder linked to autism in some cases. Supporters hailed it as long-overdue recognition of promising small studies; critics called it another example of the MAHA agenda politicizing science.

While bureaucrats and scientists bicker, families with real skin in the game tirelessly run their own experiments and share their results, hoping the science will eventually catch up.

The debate since has been fierce, with professional groups such as the American Academy of Pediatrics advising against the off-label use of leucovorin for autism, warning that the evidence remains preliminary — while prominent physicians call for larger, biomarker-guided trials to confirm what early studies suggest.

A parent’s love

All parties insist their motives are pure, but this latest skirmish is a reminder of how tangled those motives can be. What drives the people and institutions pushing medical science forward is often a sincere desire to help people, yes — mixed in with ambition, rivalry, financial interest, and the unspoken urge to be the one who’s right.

But there’s another force at work here, deeper and simpler, and it tends to override all the rest: a parent’s love for a child.

This is the same love that kept the parents of children with cystic fibrosis pushing to understand a condition doctors considered hopeless, or that led a Hollywood father to resurrect a forgotten epilepsy therapy to help his son. And now it’s the force animating hundreds of parents who believe a decades-old folate compound has literally given their autistic children a voice.

While bureaucrats and scientists bicker, families with real skin in the game tirelessly run their own experiments and share their results, hoping the science will eventually catch up.

Even before the FDA signaled approval of leucovorin for cerebral folate deficiency — a rare metabolic disorder with links to autism — parents have been sharing reports of progress with the drug on Reddit forums and in Facebook groups to share anecdotal reports of progress. A few families have also told their stories in clinic-produced or news-segment videos.

A treatment’s hope

Leucovorin, also called folinic acid, is a bioactive form of folate. It’s been used for decades to “rescue” patients from high-dose chemotherapy. In autism, it’s being repurposed to bypass what some researchers call a “folate transport blockade.”

Up to 70% of autistic children in certain studies test positive for folate receptor alpha autoantibodies — immune proteins that prevent folate from reaching the brain. The result: cerebral folate deficiency. High-dose folinic acid appears to restore that supply, sometimes with striking behavioral effects.

Dr. Richard Frye, a pediatric neurologist at Phoenix Children’s Hospital, led one of the first controlled trials in 2016. His team found improved verbal communication in FRAA-positive children treated with leucovorin. Later case studies described language bursts, better eye contact, and calmer affect.

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Photo by ISSAM AHMED/AFP via Getty Images

From ‘no words’ to the Pledge of Allegiance

The parents themselves provide more affecting testimony. Carolyn Connor’s son Mason was 1 when she realized something was amiss: “He wasn’t talking. No language. No words.”

When their pediatrician downplayed this lag in development as typical in boys, she and her husband began doing their own research, which led them to Frye. Three days after starting leucovorin, Mason spoke his first words.

Now 6, he continues to take the medication, and continues to thrive.

Beth Ann Kersse’s daughter was diagnosed with autism at age 3. “In her vocabulary she had about three or four words,” Kersse said in a video uploaded by Washington, D.C.-based Potomac Psychiatry.

“But she didn’t call me ‘Mom.’ She kind of would point at me,” she added.

That’s when Kersse and her husband began exploring leucovorin. Two years later, Kersse describes her almost 5-year-old daughter’s transformation as “incredible.”

“The other day she stood up and put her hand over her heart, and she recited the Pledge of Allegiance, and we were just like, OK … I didn’t know we knew that. … She’s able to have a full conversation; she can tell us how she’s feeling.”

Late last month, Nebraska pediatrician Dr. Phil Boucher posted a case study detailing how a 3.5-year-old autistic girl responded to leucovin treatment, citing texts from her mother reporting that she was “blown away” by the changes she observed:

She is starting to consistently look at people when they call her name. … She’s becoming more interested in her little sister. … She also has started taking some of the baby dolls that we have and has been covering them up with a blanket, giving them a kiss, and saying, “Night night.”

As Boucher is careful to point out, anecdotal success stories like these don’t prove the drug works. But to those experiencing the improvement firsthand, they’re a promising sign that a simple, inexpensive vitamin derivative can do what years of therapy can’t.

And if this promise does indeed bear fruit, leucovorin treatment will be the latest of many homegrown revolutions in medical care spearheaded by determined mothers and fathers unwilling to wait for consensus.