Introduction to Leucovorin for Autism
Last winter, Brian Noonan read online that some doctors were prescribing an obscure drug, typically given to cancer patients, for autism. Curious, he looked into it for his son Benjamin, who had just been diagnosed with autism in October.
“We jumped on it,” Noonan said. “It felt right and it made sense.”
What is Leucovorin?
The medication was leucovorin, also called folinic acid. It’s a synthetic form of vitamin B9 or folate, which the body needs to make healthy blood cells. During pregnancy, folate is important to reduce the risk of birth defects.
Last Monday, the drug was thrust into the national spotlight by President Donald Trump in a rambling press conference about autism that mainly focused on the president and Health Secretary Robert F. Kennedy’s claim that taking acetaminophen during pregnancy could cause autism. Trump briefly mentioned that an “existing drug” — referring to leucovorin — may help with certain symptoms of autism.
Leucovorin in the Autism Community
For those in the autism community, leucovorin is not new. Dr. Richard Frye, a behavioral child neurologist in Phoenix has studied leucovorin and autism for two decades. He made clear that it is not a cure for autism and that more research is needed. He does, however, prescribe it to some people with autism.
Noonan’s son Benjamin, who is 4 years old, is one of Frye’s patients.
“He’s higher functioning,” Noonan said. “He’s verbal, but he really struggled to put together sentences.” Since starting on the medicine, the family believes Benjamin’s speech has improved, though he still has difficulties with hyperactivity and impulsiveness.
Families’ Experiences with Leucovorin
Still, Noonan added, he’s under no impression that the drug is a miracle pill. Benjamin also attends a behavioral program preparing him for kindergarten, Noonan said, and he plans to enroll him in speech and occupational therapy. “We very, very much understand we’re still taking an experimental medication,” he said.
Other families say the drug conferred larger benefits.
Ben Blomgren, of Minneapolis, said his 11-year-old son, Josh, had been prescribed leucovorin off-label in February after he was at risk of being kicked out of school, even as they tried behavioral modification methods.
“He’s pretty severe,” Blomgren said. “He didn’t have any language skills. He was not toilet trained.”
After starting the medication, Blomgren said Josh’s sleep improved, he’s fully toilet trained and he’s stopped running away from them.
“It wasn’t overnight, but we saw major improvement,” he said.
Yomarie Miranda, of Florida, said she saw improvements in her 7-year-old son Ethan after he started the medication, including following instructions in class.
Ethan was prescribed the medication off label earlier this month, she said.
“He’s now looking at me when I speak with him. He’s talking more than before with complete sentences,” she said.
A Highly Unusual Move
The Food and Drug Administration first approved leucovorin in the 1980s to help reduce the toxic side effects of certain chemotherapy drugs.
In the decades since, researchers have also studied whether it might treat cerebral folate deficiency (CFD), a neurological condition that makes it harder for folate to reach the brain. Some children with autism also have cerebral folate deficiency, which neurologists say can affect speech, mood and behavior. Last Monday, the FDA said it planned to update the drug’s label to include that use.
But the agency’s decision, experts say, rests on just a handful of small studies. And despite Trump’s endorsement, the maker of the brand-name version, GSK, has yet to submit an application to the agency to change the label. In a statement, GSK said it will be submitting the request to expand the approval to include cerebral folate deficiency, though the statement does not mention autism. (Because leucovorin is already an FDA-approved drug, doctors have been able to prescribe it “off label” for other uses, though insurance may not cover it.)
It’s a highly unusual move for the FDA: pushing a drug for chemotherapy side effects as a therapy for autism without a formal submission and limited evidence.
Expert Reactions
“It’s incredibly irresponsible,” said Dr. Aaron Kesselheim, a professor of medicine at Harvard Medical School. “For 60-plus years, we’ve counted on the FDA to help patients distinguish between products that work and products that don’t work. And here we saw the FDA making an announcement relying on a summary of unclear data and announcing that they had already decided to approve it.”
Alycia Halladay, chief science officer at the nonprofit Autism Science Foundation, was frustrated by the implication that this is a breakthrough in autism research.
“It’s not like scientists have just been staring at their belly buttons for 20 years, not looking at autism treatments,” Halladay said in an interview earlier this week. “They have, but the standards have been very high to get [treatments] approved.”
Conclusion
The use of leucovorin for autism is a complex issue, with some families reporting improvements in their children’s symptoms, while experts express concerns about the limited evidence and unusual FDA decision. More research is needed to fully understand the potential benefits and risks of leucovorin for autism.
FAQs
Q: What is leucovorin?
A: Leucovorin, also called folinic acid, is a synthetic form of vitamin B9 or folate.
Q: How is leucovorin used for autism?
A: Leucovorin is being used off-label to treat cerebral folate deficiency, a neurological condition that can affect speech, mood, and behavior in some children with autism.
Q: What are the potential benefits of leucovorin for autism?
A: Some families have reported improvements in their children’s symptoms, including improved speech and behavior.
Q: What are the potential risks of leucovorin for autism?
A: The limited evidence and unusual FDA decision have raised concerns among experts, who emphasize the need for more research to fully understand the potential benefits and risks of leucovorin for autism.
Q: Is leucovorin a cure for autism?
A: No, leucovorin is not a cure for autism, and more research is needed to fully understand its potential benefits and risks.
