A generic chemotherapy drug enables a nonverbal autistic child to speak, offering hope for affordable autism treatments.
In a groundbreaking development, a low-cost medication traditionally used in chemotherapy has enabled a nonverbal autistic boy to speak his first words. This unexpected discovery offers new hope for affordable and effective autism interventions.

When Mason Connor was diagnosed with nonverbal autism at two and a half years old, his parents embarked on an exhaustive quest for treatments. After numerous unsuccessful attempts, they consulted Dr. Richard Frye, a pediatric neurologist exploring innovative therapies for autism.
Dr. Frye recommended leucovorin, an inexpensive generic drug commonly used to mitigate chemotherapy side effects. Remarkably, within just three days of starting the medication, three-year-old Mason began to speak. Leucovorin, derived from folic acid (vitamin B9), is believed to address cerebral folate deficiency, a condition observed in many autistic children that may impede communication.

Studies led by Dr. Frye have demonstrated leucovorin’s potential to improve communication for children with autism. In one study, two-thirds of participants showed language improvements after treatment. Despite these promising results, leucovorin remains unapproved by the FDA for autism treatment, limiting its widespread adoption.

The affordability of leucovorin presents a paradox; its low cost offers accessibility but diminishes financial incentives for pharmaceutical companies to pursue FDA approval for autism treatment. Consequently, the drug is currently prescribed off-label, a common practice for conditions lacking approved treatments.
Mason’s progress exemplifies the potential impact of repurposing existing medications for autism. As he prepares to enter mainstream kindergarten, his journey inspires hope for other families seeking effective and affordable autism therapies.