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March 11, 2026<\/p>\n
2 min read<\/p>\n
Add Us On Google<\/span>Add SciAm<\/span><\/span><\/p>\n FDA declines to approve drug touted by Trump as a treatment for autism<\/p>\n The FDA on Tuesday approved leucovorin as a treatment for a rare genetic condition, not for autism, as the Trump administration had suggested<\/p>\n By Claire Cameron edited by Clara Moskowitz<\/span><\/p>\n Sarah Silbiger\/Getty Images<\/p>\n The U.S. Food and Drug Administration on Tuesday approved leucovorin, a synthetic form of vitamin B9, as a treatment for a rare genetic condition that causes folate deficiencies in the brain. The decision comes just months after U.S. president Donald Trump, Secretary of Health and Human Services Robert F. Kennedy, Jr., and FDA chief Marty Makary lauded the drug as a treatment for autism. After a scientific review, the FDA, which falls under Kennedy\u2019s oversight, determined that there weren\u2019t enough data to support that use of the drug.<\/p>\n \u201cAutism is not caused by a folate deficiency,\u201d says David Mandell, a professor of psychiatry at the University of Pennsylvania, who studies autism. \u201cThe data suggesting so are outdated and weak.\u201d<\/p>\n Leucovorin is typically used to manage cancer patients\u2019 side effects from chemotherapy. But last September Makary said the FDA would move to make the drug \u201cavailable to children with autism.\u201d Now the agency has approved its use for cerebral folate deficiency\u2014a rare genetic condition that may affect fewer than one in a million people, though its true prevalence is unknown. It is caused by a mutation on the FOLR1 gene and can produce some similar symptoms to autism, such as communication issues. But autism is a separate condition, and a broad diagnosis. Although it can sometimes be tied to specific genetic factors, many researchers believe autism has no single cause.<\/p>\n If you’re enjoying this article, consider supporting our award-winning journalism by subscribing. By purchasing a subscription you are helping to ensure the future of impactful stories about the discoveries and ideas shaping our world today.<\/p>\n “There is a strong rationale for prescribing leucovorin for this specific condition,” says Helen Tager-Flusberg, a psychologist and autism researcher at Boston University, adding that she was glad the FDA had been clear that there was no evidence to suggest it could treat autism. “[Cerebral folate deficiency] is a very different neurological disorder, and importantly the FOLR1 gene has never been association with autism,” she says.<\/p>\n The FDA\u2019s decision is welcome, Mandell says, but it won\u2019t undo the interest the Trump administration has raised in using the drug for treating autism. \u201cPandora\u2019s box is already open,\u201d he adds. Prescriptions for off-label leucovorin\u2014that is, for using the drug to treat a condition it isn\u2019t approved for\u2014have skyrocketed among children by 71 percent after the officials touted its potential benefits for autism. Tager-Flusberg says that it remains to be seen how the FDA\u2019s decision will affect off-label use.<\/p>\n \u201cRFK, Jr.\u2019s premature and ill-informed announcement that leucovorin can cure autism led many families to pay out of pocket for this drug when they could have been using that money for better purposes,\u201d Mandell says. \u201cNow these families are experiencing the whiplash that happens when politics and untested theories get ahead of science.\u201d<\/p>\n If you enjoyed this article, I\u2019d like to ask for your support. Scientific American<\/span> has served as an advocate for science and industry for 180 years, and right now may be the most critical moment in that two-century history.<\/p>\n I\u2019ve been a Scientific American<\/span> subscriber since I was 12 years old, and it helped shape the way I look at the world. SciAm <\/span>always educates and delights me, and inspires a sense of awe for our vast, beautiful universe. I hope it does that for you, too.<\/p>\n If you subscribe to Scientific American<\/span>, you help ensure that our coverage is centered on meaningful research and discovery; that we have the resources to report on the decisions that threaten labs across the U.S.; and that we support both budding and working scientists at a time when the value of science itself too often goes unrecognized.<\/p>\n In return, you get essential news, captivating podcasts, brilliant infographics, can’t-miss newsletters, must-watch videos, challenging games, and the science world’s best writing and reporting. You can even gift someone a subscription.<\/p>\n There has never been a more important time for us to stand up and show why science matters. I hope you\u2019ll support us in that mission.<\/p>\n","protected":false},"excerpt":{"rendered":" March 11, 2026 2 min read Add Us On GoogleAdd SciAm FDA declines to approve drug touted by Trump as a treatment for autism The FDA on Tuesday approved leucovorin as a treatment for a rare genetic condition, not for autism, as the Trump administration had suggested By Claire Cameron edited by Clara Moskowitz Sarah<\/p>\n","protected":false},"author":1,"featured_media":46500,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[50],"tags":[915,5814,3149,249,5284,14381,485],"class_list":{"0":"post-46499","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-environment","8":"tag-approves","9":"tag-autism","10":"tag-condition","11":"tag-fda","12":"tag-genetic","13":"tag-leucovorin","14":"tag-rare"},"_links":{"self":[{"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=\/wp\/v2\/posts\/46499","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=%2Fwp%2Fv2%2Fcomments&post=46499"}],"version-history":[{"count":0,"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=\/wp\/v2\/posts\/46499\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=\/wp\/v2\/media\/46500"}],"wp:attachment":[{"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=46499"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories&post=46499"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=%2Fwp%2Fv2%2Ftags&post=46499"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}On supporting science journalism<\/h2>\n
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