CHICAGO >> President Donald Trump\u2019s embrace of an old generic drug called leucovorin for use against a rare disorder that causes autism-like symptoms has triggered a surge in demand from parents who believe it could unlock speech and social connection in their autistic children.<\/p>\n
That has become a challenge for pediatricians and specialists who caution that the science on leucovorin in people with autism is limited and does not support widespread use.<\/p>\n
In the month since Food and Drug Administration Commissioner Marty Makary promoted the decades-old drug from GSK, saying it could help \u201chundreds of thousands\u201d of children with autism, doctors and researchers say they have been inundated by parents seeking information.<\/p>\n
\u201cMy Facebook feed is flooded with parents swearing that leucovorin works,\u201d said Dr. David Mandell, a professor of psychiatry and autism researcher at the University of Pennsylvania.<\/p>\n
Tens of thousands of people have joined a Facebook group called Leucovorin for Autism, started in May by Keith Joyce, legal guardian to four-year-old Jose, who is taking the drug.<\/p>\n
Joyce credits Jose\u2019s improvements in verbal communication and social awareness to leucovorin. The site gained 5,000 members on the day of Trump and Makary\u2019s White House announcement and now has 84,000.<\/p>\n
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Mandell and other scientists and doctors say FDA\u2019s endorsement without requiring large, randomized clinical trials leaves practitioners facing emotional pleas from families while lacking data, guidance or confidence to prescribe the drug responsibly.<\/p>\n
Leucovorin is approved to treat chemotherapy side effects but can be prescribed off-label for autism symptoms.<\/p>\n
\u201cIt puts physicians in a very tough position because they\u2019re being asked to prescribe something that is not evidence-based,\u201d said Dr. Shafali Jeste, an autism expert and head of pediatrics at UCLA, who does not prescribe leucovorin despite repeated requests.<\/p>\n
The American Academy of Pediatrics today said it does not recommend leucovorin for routine use in children with autism.<\/p>\n
Autism rates have risen to 1 in 31 8-year-olds, the U.S. Centers for Disease Control and Prevention reported in April. Finding the condition\u2019s root cause and potential treatments is something Health Secretary Robert F. Kennedy Jr. and Trump have championed.<\/p>\n
Despite the FDA commissioner\u2019s broad comments, his agency proposed a narrower expanded approval tied to cerebral folate deficiency (CFD), a genetic condition that can cause autism-like symptoms. It affects roughly one in 1 million children worldwide.<\/p>\n
The argument for wider use is built on small studies suggesting many children with autism have autoantibodies that block folate \u2014 a vitamin important for brain signaling \u2014 from entering the brain, causing a deficiency similar to CFD.<\/p>\n
An estimated 75% of autistic children are believed to have these autoantibodies, but their significance is unproven, said Dr. Karam Radwan, director of the Neurodevelopmental Disorders Program at the University of Chicago.<\/p>\n
A Health and Human Services spokesperson said the FDA based its plan to update the leucovorin label to include CFD on an analysis of over 40 case studies published from 2009 to 2024. Overall, 85% of patients experienced some benefit, including improved speech\/communication capabilities.<\/p>\n
HHS said leucovorin could be useful in children who have autoantibodies to folate, but conceded \u201cthe data are limited and need to be replicated.\u201d<\/p>\n
A spokesperson said the National Institutes of Health will support follow-up research into leucovorin\u2019s impact on CFD, as well as any potential benefit to individuals with autism. Post-market surveillance and safety studies are part of the plan.<\/p>\n
Parents have interpreted the FDA announcement as a green light for leucovorin\u2019s use in autism, sharing treatment tips that led Facebook to take down Joyce\u2019s site. It was reinstated with rules banning dosing discussions, only to be removed again last week for other violations.<\/p>\n
Joyce started researching the drug after watching a news program featuring a boy with autism on the treatment who showed marked speech improvements.<\/p>\n
He found just three studies, all by the same author, that he deemed credible. No large trials in autistic children comparing leucovorin to a placebo have been conducted.<\/p>\n
Joyce reviewed the drug\u2019s track record in cancer patients, where leucovorin has been linked with insomnia, agitation and depression. In children, doctors and parents say it can lead to hyperactivity, aggression and feeling overwhelmed.<\/p>\n
Jose\u2019s neurologist balked at prescribing leucovorin, but the boy\u2019s behavioral pediatrician, who heard about the drug at a conference, was willing to.<\/p>\n
The child\u2019s care team measured language skills before treatment and four months later, Joyce said.<\/p>\n
He is more aware of the world around him, and more responsive, Joyce said. \u201cIt\u2019s not curing his autism, but it improved his quality of life. I\u2019m convinced it\u2019s real.\u201d<\/p>\n
Radwan, who offers the drug in his practice, said doctors don\u2019t fully understand who may benefit, by how much and whether it is sustainable. So far, the benefit is \u201cpretty modest,\u201d he said.<\/p>\n
Parents in some online communities are calling for caution.<\/p>\n
Sophia Urwin, 33, a single parent from Wellington, New Zealand, whose four-year-old, non-verbal son was diagnosed with autism in 2022, is concerned about desperate parents turning to over-the-counter versions of folinic acid.<\/p>\n
\u201cIt\u2019s really easy to get swept up in thinking something is a miracle \u2018cure\u2019 when you\u2019ve been skating on thin ice for so long,\u201d she said.<\/p>\n","protected":false},"excerpt":{"rendered":"CHICAGO >> President Donald Trump\u2019s embrace of an old generic drug called leucovorin for use against a rare…\n","protected":false},"author":2,"featured_media":109092,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[33],"tags":[163,85,46,482],"class_list":{"0":"post-109091","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-medication","8":"tag-health","9":"tag-il","10":"tag-israel","11":"tag-medication"},"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/posts\/109091","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/comments?post=109091"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/posts\/109091\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/media\/109092"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/media?parent=109091"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/categories?post=109091"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/tags?post=109091"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}