When Mason Conner, a young boy from Arizona, was diagnosed with autism at just two and a half years old, his parents faced the difficult challenge of seeing him remain nonverbal despite trying various therapies.
However, a surprising breakthrough occurred when Mason started taking a simple and affordable medication initially developed for cancer patients — and within a few days, he spoke his first words. This unexpected progress has generated new optimism in the search for effective autism treatments.
Mason Conner was diagnosed with autism at age two and a half after his mother noticed that he wasn’t speaking.
After several years of therapies that showed little improvement, Mason’s parents sought advice from a doctor researching novel treatments for autism. Dr. Richard Frye, a pediatric neurologist at Rossignol Medical Center in Arizona, suggested they try Leucovorin, a generic drug related to folic acid.
Only three days after beginning the $2.50 medication, Mason began to speak for the first time at age three.
Folic acid (vitamin B9) is vital for DNA synthesis and repair and is naturally found in foods such as leafy vegetables, peas, lentils, beans, eggs, yeast, and liver.
Leucovorin is commonly administered to cancer patients to protect their red blood cells from the toxic effects of chemotherapy.
However, studies indicate that some children with autism may have a disruption that prevents them from properly absorbing folate, which could be linked to difficulties with communication.
Leucovorin can bypass this disruption and deliver folate directly where it is needed.
Dr. Frye told CBS News, “[Leucovorin] may have a major impact on a significant number of children with autism.”
Recent CDC statistics show that nearly 2 million children in the U.S.—about one in every 36—are diagnosed with autism.
While some children receive a diagnosis as early as age two, most are diagnosed closer to age five.
The National Institutes of Health estimates that nearly 70% of children with autism have folate receptor autoantibodies—immune proteins that attack the body’s own tissues.
These autoantibodies can prevent folate from entering the brain, potentially causing delays in language development.
Folic acid is also a standard component of prenatal vitamins, as it helps prevent neural tube defects during fetal development.
In one of Dr. Frye’s studies, 44 autistic children with these autoantibodies took 50 mg of leucovorin daily for four months.
All participants showed improvements in language skills, behavior, hyperactivity, mood, attention, and irritability.
A 2018 study by Dr. Frye’s team reported that leucovorin treatment led to significant improvements in language abilities and reductions in irritability, hyperactivity, and lethargy.
Leucovorin can be administered in several forms, including infusions, injections, or oral tablets.
Dr. Frye stated, “We have the scientific data; now we need funding to pursue FDA approval.”
Earlier research also showed that some autistic children have antibodies that block folate (vitamin B9) from reaching the brain, causing a deficiency.
Leucovorin, a form of folate that is easily absorbed, can overcome this barrier and enter the brain.
Although doctors can prescribe leucovorin off-label for autism, it is currently FDA-approved only for chemotherapy patients and certain anemia conditions.
The number of physicians currently prescribing leucovorin for autism is unknown.
Dr. Frye expressed concern that because leucovorin is inexpensive, there is limited financial motivation for further research.
Other studies have suggested that women who take folic acid during pregnancy might reduce their child’s risk of autism by about 40%.
Now five years old, Mason continues on leucovorin and his parents hope he will attend mainstream kindergarten this fall after his earlier nonverbal phase.
This extraordinary case underscores leucovorin’s potential—a low-cost, widely accessible medication—to improve communication and other symptoms in some children with autism. While additional research and FDA approval are necessary, early results indicate that targeting folate metabolism may provide a promising new treatment approach.
Both families and healthcare providers should stay updated on emerging therapies like this, as they could open new possibilities for children living with autism. Mason’s progress offers hope that with timely intervention, many autistic children can experience meaningful developmental gains and a better quality of life.