\n<\/p>\n
If President Donald Trump wanted Americans to take away one message about autism, it was this: Blame Tylenol. During his September press conference on the subject, Trump warned pregnant women more than a dozen times not to take the drug, even though two massive studies had found no meaningful association with the disorder in children. He also spread false rumors that \u201cessentially no autism\u201d can be found in Cuba or among the Amish. The other stated purpose of the event\u2014the announcement of what FDA Commissioner Marty Makary called an \u201cexciting treatment\u201d for autism\u2014was largely overshadowed by the president\u2019s performance.<\/p>\n
But parents of autistic children took note of that promised remedy. How could they not? Officials suggested that the little-known drug, leucovorin, could alleviate the symptoms of profound autism, perhaps allowing children with speech difficulties to find their words. Hundreds of thousands would benefit, according to Makary. No, it wasn\u2019t a cure\u2014officials stopped short of invoking that word\u2014but it sounded like a miracle.<\/p>\n
After the government\u2019s endorsement, many parents rushed to get their hands on leucovorin. They soon discovered that they were effectively taking part in a nationwide experiment with few guidelines\u2014assuming, that is, they could even get a prescription.<\/p>\n
The theory behind treating autism with leucovorin is that the drug gives autistic kids something they\u2019re missing. Children with autism seem to be more likely than other children to produce an antibody that prevents folate, also known as vitamin B9, from reaching their brain. Because folate plays a role in brain development, some researchers\u2014most notable among them Richard Frye, a doctor who has been prescribing and promoting leucovorin for nearly two decades, and who told me he spoke with leaders in the Health and Human Services Department before the press conference\u2014think these antibodies might hamper the growth of a child\u2019s language abilities. Leucovorin is essentially a massive dose of folate, delivered in a form that can bypass those antibodies.<\/p>\n
But that theory, which has long been debated in autism subreddits and Facebook groups, hasn\u2019t gained traction among mainstream autism scientists. They point to a 2018 study that found that autistic children and their non-autistic siblings were equally likely to have these antibodies. The clinical evidence for leucovorin\u2019s effectiveness for autistic kids is limited to a handful of small studies that don\u2019t measure the same outcomes. No large, randomized, placebo-controlled trial has ever been conducted. If you want to get the drug for autism, it has to be prescribed off-label, which many doctors refuse to do.<\/p>\n
Part of the Trump administration\u2019s promise was to make leucovorin more accessible to patients and easier for doctors to prescribe. So far, nothing official has happened. At the press conference, Makary promised that the FDA would change leucovorin\u2019s label so that doctors could start prescribing it to kids with autism. But when the FDA formally laid out its leucovorin plan two days later, it made no mention of autism. The plan instead centered on cerebral folate deficiency, an extremely rare genetic condition. Emily Hilliard, an HHS spokesperson, wrote in an email that \u201cthe FDA\u2019s current action is focused specifically on cerebral folate deficiency,\u201d noting that some of its symptoms overlap with autism. (My other questions about the administration\u2019s leucovorin rollout went unanswered.) A spokesperson for GSK, which originally manufactured the drug and controls the label, told me that the company submitted an application for the label change this week.<\/p>\n
During the press conference, Robert F. Kennedy Jr., the secretary of health and human services, said that HHS would \u201chelp doctors treat children appropriately\u201d with leucovorin. But Alycia Halladay, the chief science officer at the Autism Science Foundation, told me that physicians she\u2019s spoken with have heard nothing from HHS. For her part, Halladay doesn\u2019t believe that enough evidence exists to recommend leucovorin as an autism treatment. The American Academy of Pediatrics has also said that the current data are lacking. In a statement, the organization allowed that if physicians prescribe the medication, they should work with families to monitor side effects; some parents report increased hyperactivity and aggression after their kids take leucovorin.<\/p>\n
Several researchers I spoke with compared the excitement about leucovorin to the enthusiasm for secretin, another drug that was popular as an autism treatment in the 1990s after early promising results. More rigorous clinical trials later showed that it wasn\u2019t effective. Halladay said she\u2019s heard from multiple doctors, including her child\u2019s pediatrician, that they\u2019ve been besieged with calls from parents hoping to get a prescription. One doctor in Chicago told me he had received dozens of inquiries.<\/p>\n
In the absence of official advice on leucovorin, parents\u2014almost always moms\u2014of kids with profound autism have been turning to one another for opinions and encouragement online.<\/p>\n
Some post regular updates on TikTok and Instagram detailing their kid\u2019s progress on leucovorin, saying they\u2019ve noticed more regular eye contact, fewer repetitive movements and sounds, or improvements in language use. In the comments, fellow parents complain about not being able to get the drug (\u201cMy sons pediatrician refuses, wants us to just wait for prescribing guidelines\u201d), ask questions about dosage (\u201cI just gave her 15 mg today is that too much??\u201d), and wonder whether it\u2019s really working (\u201chow do we know we see results, what do we see?\u201d). Others say their child\u2019s behavior didn\u2019t improve\u2014or even grew worse\u2014after taking the medication (\u201cI didn\u2019t see it helping my son more with speech like we hoped\u201d).<\/p>\n
Some insist the drug must be paired with vitamin B12, omega-3, or a certain amino acid; others caution that dairy can reduce its effectiveness. Many want to know about an antibody test that purports to indicate whether a child\u2019s brain is receiving enough folate. But that test isn\u2019t widely accepted as valid, and not all doctors are willing to give it.<\/p>\n
Jessie Carrasco is one of those TikTok moms. She lives with her husband and their three children on the outskirts of the east-Texas city of Tyler. Their oldest son, Ezra, is 11 and has autism. He didn\u2019t talk until he was 4; she credits fish-oil supplements, in part, with helping him speak in short sentences and respond to simple questions. (Some studies suggest that omega-3 fatty acids, found in fish oil, may improve communication in autistic children, though the evidence is mixed.) When Carrasco invited me into their backyard, which was lively with chickens and dogs, Ezra ran up, gave me a quick once-over, and then darted off to play on the swing set with his younger brother.<\/p>\n
Carrasco learned about leucovorin after hearing Trump refer to a new treatment for autism during his speech at Charlie Kirk\u2019s memorial service. She emailed Ezra\u2019s nurse practitioner, who was willing to prescribe it but didn\u2019t know enough about the drug to offer advice on how much to give Ezra or how to deal with potential side effects. Since then, Carrasco has posted more than 200 videos on TikTok, most of them documenting Ezra\u2019s progress on leucovorin. One clip shows him serving himself food from a pot on the stove\u2014the first time he\u2019d ever done that, she told me. Her video of Ezra before and after taking the drug has nearly 6,000 likes.<\/p>\n
\u201cSince we\u2019ve been giving leucovorin, I think everything that we\u2019ve been trying to teach, he\u2019s started to get it more and faster,\u201d she said. Ezra\u2019s behavioral analyst, Margaret Hawkins, told me that before leucovorin, she used to work with Ezra on pronouncing single words; now she\u2019s coaching him on telling three- or four-sentence stories.<\/p>\n
Without Kennedy and Trump, Carrasco might not have learned about leucovorin; she\u2019s grateful to them for giving her more faith in Ezra\u2019s future, and hopes that the progress she\u2019s seen so far won\u2019t turn out to be a mirage. At the same time, she\u2019s frustrated by the lack of information about how to use the drug and the difficulty she and other parents have had getting their hands on it. \u201cI feel like they presented something and then just kind of left it in the dust,\u201d she said. The last time she checked, the pharmacies in Tyler were out of leucovorin; she was planning to drive to a neighboring town to get more. \u201cI got my hopes up and now I\u2019m nervous,\u201d she texted me recently. \u201cI don\u2019t wanna watch him regress.\u201d<\/p>\n
If the posts and comments are any indication, even parents who have managed to get prescriptions, like Carrasco, have been having trouble filling them. Whether the drug is in shortage now is unclear, but it has a history of supply problems going back more than a decade. A drug-shortage database maintained by a pharmacists\u2019 association recently listed leucovorin shortages at five companies. (One of them, Teva, said its 25-milligram pills were in stock, but not its 5-milligram dose; the others either didn\u2019t respond to me or couldn\u2019t confirm whether they had supply issues.)<\/p>\n
Perhaps because of shortages\u2014or difficulties finding a doctor willing to prescribe leucovorin at all\u2014some parents are turning to supplemental forms of folate. Such supplements are far less potent than leucovorin, but are usually cheaper and don\u2019t require a prescription. (Even if leucovorin does turn out to be an effective autism treatment, it\u2019s unknown whether low-dose supplements would have any effect.)<\/p>\n
Offline, parents have been calling Frye, the longtime leucovorin doctor, who believes that as many as 1 million kids could benefit from the drug. He mostly treats children with significant language delays, though he says that the drug can sometimes help higher-functioning kids too. Frye was the lead author of a small 2016 study that found that children with autism who received leucovorin showed improvements in verbal communication, compared with those given a placebo; he also recently published a book called The Folate Fix<\/em>. Ever since the press conference, his office has been inundated with calls and emails from parents who want the drug for their children.<\/p>\n For years, Frye has had a backlog of patients wanting to see him, but the White House announcement, he told me, made an \u201cimpossible situation worse.\u201d He already has about 1,000 patients who are taking leucovorin, and he isn\u2019t planning to accept any new ones until 2028. According to Frye, the FDA has asked him to submit an application to conduct clinical trials on leucovorin as an autism treatment, but last week, he told me that \u201cnot much is going on at this time.\u201d He guesses those trials will take about two years. In the meantime, parents will be left to experiment with leucovorin on their own.<\/p>\n