Could an inexpensive generic drug help improve autism symptoms? The parents of a nonverbal autistic boy in Arizona believe so. They say their son, then three years old, spoke his first words within days of taking a medication derived from folic acid, given to chemo patients to relieve side effects.

Mason Connor, now 5, showed signs of autism since the age of one and experienced a speech delay. By 2 ½ years, doctors diagnosed him with nonverbal autism, but his parents were determined to find a way to help him speak.

"We just started researching on our own. And that's when my husband Joe came across Dr. Frye in a research study he was doing," Mason's mother, Caroline Connor, recollected.

Dr. Richard Frye is a pediatric neurologist who conducts experimental research on treatments for autism. He is investigating if Leucovorin, used as an antidote to the harmful effects of cancer medication methotrexate, helps improve speech in autistic children. Leucovorin, a derivative of folic acid is also used for treating certain types of anemia.

The idea behind using this drug for autism is that some children may have trouble transporting folic acid into the brain, which could contribute to neurological issues. Leucovorin helps bypass this blockage, potentially improving speech.

Mason's first words were at the age of three, just three days after he started Leucovorin, the parents said.

The drug is currently prescribed off-label for autism since the FDA has not approved it for this use. Given its potential for repurposing, Frye hopes to secure approval to make the treatment more widely available.

"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," Frye said.

Studies have shown that women's intake of folic acid supplements during pregnancy was linked to reduced risk of autism in children. Researchers have also noted that mothers of children with autism had a lower dietary intake of folic acid during pregnancy.

A 2020 study involving 48 children with autism found that Leucovorin significantly improved verbal communication in those with ASD. Although the drug has been used safely for decades, studies need to examine its long-term effects, optimal dosing, and treatment duration for autism.

Read more