{"id":174909,"date":"2025-09-28T10:18:08","date_gmt":"2025-09-28T10:18:08","guid":{"rendered":"https:\/\/www.newsbeep.com\/au\/174909\/"},"modified":"2025-09-28T10:18:08","modified_gmt":"2025-09-28T10:18:08","slug":"before-trump-touted-the-drug-leucovorin-for-autism-these-families-had-already-tried-it","status":"publish","type":"post","link":"https:\/\/www.newsbeep.com\/au\/174909\/","title":{"rendered":"Before Trump touted the drug leucovorin for autism, these families had already tried it"},"content":{"rendered":"

Last winter, Brian Noonan read online that some doctors were prescribing an obscure drug<\/a>, 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.<\/p>\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<\/a>. 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<\/a>.<\/p>\n

Last Monday, the drug was thrust into the national spotlight by President Donald Trump in a rambling press conference about autism<\/a> that mainly focused on the president and Health Secretary Robert F. Kennedy\u2019s claim that taking acetaminophen during pregnancy<\/a> 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.<\/p>\n

Noonan\u2019s son Benjamin, who is 4 years old, is one of Frye\u2019s patients.<\/p>\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. <\/p>\n

\"AThe Noonan family. Benjamin, 4, who has autism, has used leucovorin.Courtesy of Brian Noonan<\/p>\n

Other families say the drug conferred larger benefits.<\/p>\n

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.<\/p>\n

\u201cHe\u2019s pretty severe,\u201d Blomgren said. \u201cHe didn\u2019t have any language skills. He was not toilet trained.\u201d <\/p>\n

After starting the medication, Blomgren said Josh\u2019s sleep improved, he\u2019s fully toilet trained and he\u2019s stopped running away from them. <\/p>\n

\u201cIt wasn\u2019t overnight, but we saw major improvement,\u201d he said. <\/p>\n

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.<\/p>\n

Ethan was prescribed the medication off label earlier this month, she said.<\/p>\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

A highly unusual move<\/p>\n

The Food and Drug Administration first approved leucovorin in the 1980s to help reduce the toxic side effects of certain chemotherapy drugs.<\/p>\n

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 <\/a>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<\/a> to include that use.<\/p>\n

But 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<\/a>, 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.) <\/p>\n

It\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<\/p>\n

Alycia Halladay, chief science officer at the nonprofit Autism Science Foundation, was frustrated by the implication that this is a breakthrough in autism research.<\/p>\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

Andrew Nixon, a spokesperson for the Department of Health and Human Services, which oversees the FDA, said in a statement: \u201cAnalysis across 23 publications from 2009-2024 demonstrated the effectiveness for CFD. Overall, 85% of patients experienced some type of clinical benefit including improved speech\/communication capabilities.\u201d Nixon\u2019s statement did not mention autism, and he did not respond to follow-up questions.<\/p>\n

David Mandell, a psychiatry professor at the University of Pennsylvania, said HHS\u2019 claim that 85% of patients experienced a benefit is a \u201cquite a conceptual leap\u201d because it assumes people with cerebral folate deficiency also have autism.<\/p>\n

Cerebral folate deficiency \u201cis an extremely rare event, and it is accompanied by symptoms of epilepsy, really severe neurodevelopmental problems and some of those symptoms can look like autism, but it\u2019s not,\u201d he said.<\/p>\n

\u201cI could not think of a single FDA approval that has such weak evidence in the past 20 years,\u201d Mandell added.<\/p>\n

Frye, the Phoenix neurologist, estimates up to three-quarters of autistic children have antibodies linked to cerebral folate deficiency, based on his research. Other estimates are much lower: Alycia Halladay, chief science officer at the nonprofit Autism Science Foundation, put the rates at roughly 10% to 30%.<\/p>\n

The studies on leucovorin are small but look interesting, said Dr. Scott Gottlieb, a former FDA commissioner.<\/p>\n

\u201cIt certainly merits follow up,\u201d Gottlieb said.<\/p>\n

One of the studies was conducted by Frye: a placebo-controlled trial of more than 40 children with the antibodies, published in Molecular Psychiatry in 2018<\/a>. It found around two-thirds of kids who got the drug saw improvements in verbal communication after 12 weeks. Others saw no changes. The children all had language impairments, as well as a common type of autism that\u2019s not linked to other neurological diseases.<\/p>\n

Mandell, however, said the results shouldn\u2019t be taken as evidence.<\/p>\n

\u201cThey claim that in certain subgroups of kids in their already small sample, they find these very large effects,\u201d he said, adding that larger-scale trials that establish clear outcomes ahead of time are needed to verify the results. <\/p>\n

Kesselheim said it\u2019s important for patients to have access to medications that could have a benefit, especially when there is an unmet medical need. But, he said, there are still a number of unanswered questions.<\/p>\n

\u201cThere is no accurate testing for this,\u201d he said. \u201cWhat are low folate levels? What are normal folate levels? All of that stuff should be worked out.\u201d<\/p>\n

Frye said he currently uses two methods to assess whether a child has a folate deficiency: a spinal tap \u2014 also known as a lumbar puncture \u2014 which involves inserting a needle into the lower back to collect fluid from the spinal cord, and a folate receptor antibody test originally developed for pregnant women that isn\u2019t approved by the FDA for diagnosing folate deficiency in children. <\/p>\n

Edward Quadros, a research professor at SUNY Downstate Health Sciences University who has worked with Frye studying leucovorin for autism, said parents are already trying the drug, including supplement versions, which aren\u2019t regulated and can be dangerous.<\/p>\n

\u201cBy making the FDA approve it, and reputable pharmaceutical companies manufacturing it and selling it, at least it gives you quality dosing,\u201d Quadros said.<\/p>\n","protected":false},"excerpt":{"rendered":"Last winter, Brian Noonan read online that some doctors were prescribing an obscure drug, typically given to cancer…\n","protected":false},"author":2,"featured_media":174910,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[33],"tags":[64,63,137,490],"class_list":{"0":"post-174909","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-medication","8":"tag-au","9":"tag-australia","10":"tag-health","11":"tag-medication"},"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/posts\/174909","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/comments?post=174909"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/posts\/174909\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/media\/174910"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/media?parent=174909"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/categories?post=174909"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/tags?post=174909"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}