One family’s breakthrough may provide hope for thousands of parents who’ve been told their children will never talk.
When their son, Mason, now 5, was diagnosed with nonverbal autism at two-and-a-half years old, the Connor family was determined to find something to help him speak.
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.
Frye suggested trying leucovorin, an inexpensive generic drug that is commonly prescribed to cancer patients to allay the effects of chemotherapy.
Just three days later, Mason said his first words.
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.
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 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.
“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. Of 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, given either leucovorin or a placebo. After 12 weeks, those who took the drug showed “improvement in measures of verbal communication” as compared with those on the placebo.
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.
“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.”
“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,” he explained. “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.
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.
