What is leucovorin? Inside the drug giving new hope to autism patients

Can a generic drug designed to make chemotherapy work better benefit people with autism?

President Trump is touting an “amazing” drug — leucovorin — as “an answer to autism,” a broad range of conditions that affect how people learn, behave, communicate and interact with others.

A form of vitamin B9, also known as folate, leucovorin has been prescribed to protect healthy cells from the toxic side effects of chemotherapy, to enhance chemotherapy and to treat cerebral folate deficiency.

Leucovorin is a generic drug designed to make chemotherapy work better.McGuff

Folate is needed for brain development. Research has found that some children with neurodevelopmental disorders show signs of cerebral folate deficiency, a shortage of B9 in the brain.

“If you’re going to the doctor and looking for an autism pill, it doesn’t exist,” Dr. Richard Frye, a pediatric neurologist researching leucovorin as a potential autism treatment, previously told The Post. “But leucovorin has helped a lot of children.”

What is autism?

Around 1 in 36 US children is believed to have some form of autism.

Evidence suggests that genetic and environmental factors may contribute to the risk of developing it. Researchers have spent decades trying to pinpoint those exact factors.

Autism is a disorder that affects how people learn, behave, communicate and interact with others.Alexis Scholtz/peopleimages.com – stock.adobe.com

Trump said his administration will reveal how autism “happens, so we won’t let it happen anymore.”

Diagnoses in the US have skyrocketed in recent years, increasing by 175% between 2011 and 2022.

Experts have blamed the spike largely on greater awareness of autism, improved screening and updated diagnostic criteria.

How could leucovorin help?

month without insurance and even less with coverage.

Supporters are hopeful that the medication delivers folate effectively and efficiently to the brain to improve communication and language skills.

Scientists have found that some children with neurodevelopmental disorders carry folate receptor alpha autoantibodies, which block the proper transport of folate into the brain.

One study found that more than 75% of children with autism had FR⍺ autoantibodies, compared to just 10% to 15% of children without autism.

In a 2012 clinical trial, leucovorin was tested on 44 children with autism and FR⍺ autoantibodies. One-third of those treated with the drug showed significant language improvement.

In one recent, exciting case, a toddler with nonverbal autism — whose parents had been told their child would never speak — uttered his first words just three days after starting the drug.

What are the downsides?

The process of obtaining leucovorin begins with a blood test that detects autoantibodies blocking a receptor essential for transporting folate into the brain.

Around 1 in 36 US children are believed to have some form of autism.Photographee.eu – stock.adobe.com

Those who test positive are significantly more likely to respond to treatment.

Despite promising findings, scientists and doctors caution that leucovorin is still an experimental treatment, suitable only for individuals with a specific genetic or metabolic profile.

“It doesn’t address the root cause of autism and what is happening in the brain,” developmental-behavioral functional neurologist Robert Melillo, author of “Disconnected Kids,” told The Post.

“Every few years, there is one thing like this that people latch onto, but there is no one magic bullet.”

 

The long-term effects of the drug are unknown.

Frye noted that leucovorin on its own isn’t a cure-all. While his patients were taking the medication, they also continued other therapeutic interventions, such as applied behavior analysis and speech therapy.

“You have to treat a lot of things to make the body well,” Frye said. “What [leucovorin] does, we think, is accelerate the effectiveness of all these therapies.”

In addition to language comprehension, Frye is interested in exploring how leucovorin can mitigate other symptoms associated with autism, such as difficulty socializing.

What do you think? Post a comment.

Meanwhile, Health and Human Services Secretary Robert F. Kennedy Jr. may fast-track US Food and Drug Administration approval for leucovorin as an autism treatment.

The Trump administration is also expected to announce that using acetaminophen while pregnant can raise the odds that the baby will develop the disorder.

Major health organizations have said that acetaminophen (Tylenol is one brand name) is safe for use in pregnancy.

By vpngoc

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