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.
\n\u201cWe jumped on it,\u201d Noonan said. \u201cIt felt right and it made sense.\u201d<\/p>\n
The medication was leucovorin, also called folinic acid. It\u2019s 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.
\n
\nLast 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\u2019s claim that taking acetaminophen during pregnancy could cause autism. Trump briefly mentioned that an \u201cexisting drug\u201d \u2014 referring to leucovorin \u2014 may help with certain symptoms of autism.<\/p>\n
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.
\nNoonan\u2019s son Benjamin, who is 4 years old, is one of Frye\u2019s patients.
\n\u201cHe\u2019s higher functioning,\u201d Noonan said. \u201cHe\u2019s verbal, but he really struggled to put together sentences.\u201d Since starting on the medicine, the family believes Benjamin\u2019s speech has improved, though he still has difficulties with hyperactivity and impulsiveness.<\/p>\n
Still, Noonan added, he\u2019s 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. \u201cWe very, very much understand we\u2019re still taking an experimental medication,\u201d he said.
\nOther families say the drug conferred larger benefits.
\nBen 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.
\n\u201cHe\u2019s pretty severe,\u201d Blomgren said. \u201cHe didn\u2019t have any language skills. He was not toilet trained.\u201d
\nAfter starting the medication, Blomgren said Josh\u2019s sleep improved, he\u2019s fully toilet trained and he\u2019s stopped running away from them.
\n\u201cIt wasn\u2019t overnight, but we saw major improvement,\u201d he said.
\nYomarie Miranda, of Florida, said she saw improvements in her 7-year-old son Ethan after he started the medication, including following instructions in class.
\nEthan was prescribed the medication off label earlier this month, she said.
\n\u201cHe\u2019s now looking at me when I speak with him. He\u2019s talking more than before with complete sentences,\u201d she said.<\/p>\n
The Food and Drug Administration first approved leucovorin in the 1980s to help reduce the toxic side effects of certain chemotherapy drugs.
\nIn 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\u2019s label to include that use.
\nBut the agency\u2019s decision, experts say, rests on just a handful of small studies. And despite Trump\u2019s 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 \u201coff label\u201d for other uses, though insurance may not cover it.)
\nIt\u2019s 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.<\/p>\n
\u201cIt\u2019s incredibly irresponsible,\u201d said Dr. Aaron Kesselheim, a professor of medicine at Harvard Medical School. \u201cFor 60-plus years, we\u2019ve counted on the FDA to help patients distinguish between products that work and products that don\u2019t 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.\u201d
\nAlycia Halladay, chief science officer at the nonprofit Autism Science Foundation, was frustrated by the implication that this is a breakthrough in autism research.
\n\u201cIt\u2019s not like scientists have just been staring at their belly buttons for 20 years, not looking at autism treatments,\u201d Halladay said in an interview earlier this week. \u201cThey have, but the standards have been very high to get [treatments] approved.\u201d<\/p>\n
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.<\/p>\n
Q: What is leucovorin?
\nA: Leucovorin, also called folinic acid, is a synthetic form of vitamin B9 or folate.
\nQ: How is leucovorin used for autism?
\nA: 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.
\nQ: What are the potential benefits of leucovorin for autism?
\nA: Some families have reported improvements in their children’s symptoms, including improved speech and behavior.
\nQ: What are the potential risks of leucovorin for autism?
\nA: 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.
\nQ: Is leucovorin a cure for autism?
\nA: No, leucovorin is not a cure for autism, and more research is needed to fully understand its potential benefits and risks.<\/p>\n","protected":false},"excerpt":{"rendered":"
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. \u201cWe jumped on it,\u201d Noonan said. \u201cIt felt right and it […]<\/p>\n","protected":false},"author":2,"featured_media":29263,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"rank_math_lock_modified_date":false,"footnotes":""},"categories":[26],"tags":[],"class_list":{"0":"post-29262","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-health"},"jetpack_featured_media_url":"https:\/\/i3.wp.com\/media.nbcchicago.com\/2025\/09\/GettyImages-2236335489-e1759097285131.jpg?quality=85&strip=all&resize=1200%2C675&w=1200&resize=1200,675&ssl=1","_links":{"self":[{"href":"https:\/\/citystuff.com\/chicago\/wp-json\/wp\/v2\/posts\/29262"}],"collection":[{"href":"https:\/\/citystuff.com\/chicago\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/citystuff.com\/chicago\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/citystuff.com\/chicago\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/citystuff.com\/chicago\/wp-json\/wp\/v2\/comments?post=29262"}],"version-history":[{"count":1,"href":"https:\/\/citystuff.com\/chicago\/wp-json\/wp\/v2\/posts\/29262\/revisions"}],"predecessor-version":[{"id":29264,"href":"https:\/\/citystuff.com\/chicago\/wp-json\/wp\/v2\/posts\/29262\/revisions\/29264"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/citystuff.com\/chicago\/wp-json\/wp\/v2\/media\/29263"}],"wp:attachment":[{"href":"https:\/\/citystuff.com\/chicago\/wp-json\/wp\/v2\/media?parent=29262"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/citystuff.com\/chicago\/wp-json\/wp\/v2\/categories?post=29262"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/citystuff.com\/chicago\/wp-json\/wp\/v2\/tags?post=29262"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}