One family’s breakthrough may provide hope for thousands of parents who’ve been told their children will never talk.

For many parents of children with nonverbal autism, finding an effective treatment can feel like an uphill battle. Therapies often take years to show progress, and in many cases, children remain unable to communicate verbally. However, for the Connor family, a surprising discovery changed everything.

When their son, Mason, now 5, was diagnosed with nonverbal autism at 2 1/2 years old, the Connor family was determined to find something to help him speak. They tried multiple therapies, dietary changes, and even alternative treatments, but nothing seemed to work. Each attempt ended in disappointment, leaving them feeling increasingly desperate for a solution.

After multiple failed treatments, Mason’s father, Joe, came across a study by Dr. Richard Frye, a pediatric neurologist who was researching experimental treatments for children with autism. His work explored the link between folate levels and brain function, particularly in children with autism.

Frye suggested trying leucovorin, an inexpensive generic drug that is commonly prescribed to cancer patients to allay the effects of chemotherapy.

The Connors had little to lose and everything to gain, so they decided to try it.

Just three days later, Mason said his first words.

It was an emotional moment for the family—one they had long hoped for but never expected to come so soon. Mason’s mother, Emily, described the experience as nothing short of miraculous. “Hearing his voice for the first time was something I’ll never forget,” she said.

Leucovorin is made from folic acid, also called vitamin B9, which occurs in the form of folate in foods such as leafy greens, Brussels sprouts, and avocados. This vitamin plays a crucial role in brain function, particularly in early development.

Some research has shown that many children with autism have low folate levels in their brains, a condition known as cerebral folate deficiency (CFD). Other studies indicate that a significant number of children with autism possess antibodies that can interfere with folate transport into the brain. This impairment may contribute to difficulties in communication, learning, and overall cognitive function.

This might help explain why the drug may help children with autism with their communication issues.

“It could really have a substantial impact on a very good percentage of children with autism,” Frye told CBS.

But despite the promising results, more research is needed.

“We’ve done the science, and the next step is that we want to get more funding so we can actually get it FDA approved,” he added.

That science includes his 2020 study, published in Seminars in Pediatric Neurology. It found that of the 44 children with both autism and folate receptor alpha autoantibodies, two-thirds treated with the drug showed language improvement.

Another study he published in 2018 involved 48 children with autism and language impairment who were given either leucovorin or a placebo. After 12 weeks, those who took the medication showed “improvement in measures of verbal communication” as compared with those on the placebo.

The results were promising, but the challenge remains in bringing leucovorin into mainstream autism treatment.

Mason’s not the only child whose parents have spoken out about success with leucovorin.

One little girl named Meghan, who underwent a clinical trial at the Barrow Neurological Institute at Phoenix Children’s, saw improvement in language, communication, behavior, and social skills. Her transformation was remarkable.

“We were almost convinced she was taking [leucovorin calcium in the first phase] because we saw significant changes within two weeks of her starting the study,” her mother Marie said. “It was pretty dramatic.”

The improvement wasn’t just noticeable in therapy sessions—it impacted their daily lives.

“It’s improved our family life tremendously,” she added.

Sadly, there’s a catch.

“Leucovorin’s an old drug and you can get it for a very low price,” Frye said. “So nobody is going to make a lot of money on it. So there’s no reason for them to invest.”

That means that, for now, the medication can only be prescribed for autism “off-label”—which is when a drug is prescribed for a condition that differs from its FDA-approved purpose.

For families like the Connors, this creates an added challenge. While some doctors are willing to prescribe leucovorin based on existing research, others hesitate without official FDA approval. This leaves many parents searching for physicians who understand the potential benefits of the drug.

Despite these hurdles, researchers and advocacy groups continue to push for further studies and funding. The goal is to secure FDA approval so that leucovorin can become a widely available treatment option for children with autism.

As awareness grows, more families are learning about this potential breakthrough and discussing it with their doctors. Many parents have turned to online forums and support groups to share their experiences after taking generic drug leucovorin and to advocate for further research.

According to the CDC, around 1 in 36 children have autism spectrum disorder in the United States, an increase from the 1 in 44 estimate in 2018. This rising prevalence highlights the urgent need for more treatment options that can help improve communication and quality of life for children with autism.

For parents like Joe and Emily Connor, the hope is that one day, all families will have access to treatments that can make a real difference—just as leucovorin did for Mason.

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