We Promised Parents a Miracle Autism Treatment. Then We Took It Away

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<p>On September 22, 2025 FDA Commissioner Marty Makary, alongside HHS Secretary Robert F. Kennedy Jr., <a href="https://www.fda.gov/news-events/press-announcements/fda-takes-action-make-treatment-available-autism-symptoms">announced</a> that the FDA was initiating approval of leucovorin, a decades-old prescription B vitamin also known as folinic acid, as a treatment for cerebral folate deficiency “associated with autism.” Commissioner Makary said the label change could benefit <a href="https://www.youtube.com/watch?v=mrOcRlEDs6s">“hundreds of thousands of kids.”</a> President Trump called it a move that would <a href="https://rollcall.com/factbase/trump/transcript/donald-trump-remarks-health-autism-white-house-september-22-2025/">give “hope to the many parents with autistic children.” </a></p>
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<p>Within weeks, <a href="https://www.brown.edu/news/2026-03-05/autism-briefing-prescriptions">leucovorin prescriptions surged</a> across the country. Parents flooded pediatricians’ offices with questions. Social media exploded with testimonials. The drug became hard to find, creating shortages for the small number of children who had been using it appropriately, under careful medical supervision, for years. </p>
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<p>Then the other shoe dropped.</p>
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<p>In January 2026, the largest randomized controlled trial supporting leucovorin in autism — the <a href="https://pubmed.ncbi.nlm.nih.gov/39243316/">Panda et al. study</a> in the <em>European Journal of Pediatrics</em> — was retracted after independent reviewers found data inconsistencies the journal could not reconcile. The study many had pointed to as the strongest evidence simply disappeared. </p>
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<p>A few months later, on March 10, 2026, the FDA issued leucoviorin’s <a href="https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-patients-cerebral-folate-transport-deficiency">final approval</a>. But it looked nothing like what was promised in September. The drug was approved only for cerebral folate deficiency in patients with a confirmed variant in the FOLR1 gene, an ultra-rare genetic condition affecting <a href="https://www.uclahealth.org/medical-services/clinical-genetics/folinic-acid-cerebral-folate-deficiency-and-autism-faq#:~:text=Fewer%20than%2050%20cases%20have,because%20their%20brain%20lacks%20folate">fewer than one in a million people</a>; Fewer than 50 cases have ever been identified worldwide. In a briefing, an FDA official <a href="https://www.reuters.com/business/healthcare-pharmaceuticals/us-fda-approves-leucovorin-ultra-rare-genetic-disorder-causing-autism-like-2026-03-10/">plainly stated</a>: “We don’t have sufficient data to say that we could establish efficacy for autism more broadly.” </p>
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<p>In just six months, a treatment the FDA claimed would benefit “hundreds of thousands of kids” became an approved therapy for just one in a million.</p>
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<p>The stark contrast between our administration’s September promise and the reality of leucoviorin’s treatment efficacy is not a policy footnote. It deeply impacted families who were falsely reassured by a government agency that help was finally on the way.</p>
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<h2 class="wp-block-heading">The View from My Exam Room </h2>
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<p>I am a child, adolescent, and adult psychiatrist who spends most of my days with children on the autism spectrum and their families. I have prescribed folinic acid. I believe it has a narrow, carefully defined role in treatment for a subset of children with autism, particularly those with documented <a href="https://www.mdpi.com/2075-4426/11/11/1141">folate receptor alpha autoantibodies</a> and evidence of cerebral folate deficiency. I test before I treat, start with a low dose and titrate slowly, all while continuing to prescribe therapies with the strongest evidence: speech, behavioral support, occupational therapy, and educational intervention. </p>
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<p>I have seen leucovorin help some children and do absolutely nothing for others. That is the full, complex picture of this medication, a reality that has been simplified and distorted at the White House podium, at the expense of hopeful American families. </p>
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<p>What I am seeing now in my practice, and hearing from colleagues across the country, is something far more damaging than a policy misstep. </p>
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<p>A mother I work with has a four-year-old son with autism. He has limited verbal language, significant sensory challenges, and had been making slow but real progress with an intensive therapy program — 20 hours a week of speech, OT, and Applied Behavior Analysis. After the September announcement, she read everything she could find online. By October, she had pulled her son out of two therapies to “focus on the leucovorin route,” after finding a provider willing to prescribe without testing. When I saw her in January, her son had lost ground, not because of the leucovorin itself — which at appropriate doses is <a href="https://www.hhs.gov/press-room/autism-announcement-fact-sheet.html">generally safe</a> — but because she had abandoned three months of critical early intervention in favor of a miracle pill. Left with the consequences, she felt betrayed by the system that promised her a breakthrough and then promptly, quietly walked it back. </p>
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<p>She is not the only one. I experience versions of this story regularly now, in the form of parents who act as though I’m gatekeeping life-altering medications when I suggest testing before prescribing, and colleagues whose patients can’t fill legitimate prescriptions because of a shortage driven by a single press conference.</p>
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<h2 class="wp-block-heading">The Institutional Ripple Effects</h2>
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<p>The immediate harms — prescription surges, drug shortages, families abandoning proven therapies — are visible and measurable. But the longer-term repercussions are what keep me up at night as a clinician. </p>
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<p>Trust in institutions is fracturing. When the White House makes a sweeping announcement, and the <a href="https://www.aap.org/en/patient-care/autism/use-of-leucovorin-in-autistic-pediatric-patients/">American Academy of Pediatrics (AAP) responds within weeks</a> stating “[we do not] recommend the routine use of leucovorin (folinic acid) for autistic children,” parents are forced to choose whom to believe. The AAP — the organization that has guided pediatric care for decades — found itself in the position of contradicting the president. </p>
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<p>Clinicians, too, are in a difficult position. Pediatricians and psychiatrists who counsel caution are perceived as obstacles, and while those who prescribe without appropriate evaluation may be rewarded with grateful parents in the short term, they face potential consequences down the line, when the treatment doesn’t deliver what the parent expected, or when it becomes clear that a contraindication was missed. </p>
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<p>Research credibility is also taking a hit. The retraction of the Panda study — cited in the FDA’s own announcement — casts a shadow over the entire field of folate metabolism research in autism. Scientists like Dr. Richard Frye, whose careful <a href="https://pubmed.ncbi.nlm.nih.gov/27752075/">2016 trial</a> demonstrated modest but real benefits in a specific subgroup, now find their work lumped in with a retracted study and a politicized approval process. </p>
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<p>Future funding, future recruitment for clinical trials, and future peer review become harder when the field has been tainted by premature claims. The researchers who have dedicated years to understanding the biology of cerebral folate deficiency deserve better, as do the families of those affected by this deficiency, who are waiting for real answers.</p>
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<p>Importantly, parents bear the emotional toll. This is the part that gets to me. The parents I work with are not naive. They are intelligent, devoted, exhausted people who spend every day fighting for their children. When the government tells them a treatment is coming, they allow themselves to hope. And when the source of that hope is quietly retracted in a press release six months later — without a clarifying press conference, prime-time announcement, or presidential statement — they feel disregarded and abandoned. Parents become angry and cynical, and sometimes stop trusting the therapies that are actually helping their child. </p>
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<h2 class="wp-block-heading">What Should Have Happened </h2>
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<p>There is a small but legitimate <a href="https://pubmed.ncbi.nlm.nih.gov/27752075/">body of research</a> suggesting that folinic acid can improve verbal communication in a carefully selected subgroup of children with autism who have folate receptor alpha autoantibodies. The 2018 Frye trial, while small, was well-designed and demonstrated a meaningful effect, particularly among FRAA-positive children. </p>
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<p>But promising early research in a subgroup is not the same as “FDA-approved treatment for autism,” and families get hurt when the government draws an invisible line between them.</p>
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<p>What should have happened is straightforward: The FDA should have called for a large, multicenter, three phase randomized controlled trial — the kind of rigorous study this question (and families) deserve. The NIH or another research body should have been tasked with funding it. In the meantime, clinicians like me could continue using leucovorin thoughtfully and off-label in selected patients, as we have been doing for years, while the science caught up. </p>
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<p>Instead, we got a chaos-inducing press conference. And families paid the price. </p>
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<h2 class="wp-block-heading">A Path Forward </h2>
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<p>I am not anti-leucovorin. I prescribe it and have seen it help. But I prescribe it the way medicine should work: with testing, with monitoring, with honest conversations about what it can and cannot do, and always alongside the therapies we know make a difference. </p>
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<p>What I am asking for is not radical: Scientific announcements should come from scientific bodies, not political stages. The FDA’s process, imperfect as it is, should never be bypassed. Organizations like the AAP and the CDC should not be forced to contradict a sitting president in order to protect children. And researchers should be allowed to do their work without their findings getting weaponized.</p>
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<p>Most importantly, we need to center the people who actually matter here: the children and the families. They deserve to feel seen, supported, and empowered to thrive. But I can only do that work effectively when the system around me — the regulatory bodies, the research institutions, the government — holds itself to the same standard. </p>
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<p>The leucovorin story is not over. The research will continue. Trials will be funded. We will learn more about which children benefit from leucovorin and why. I look forward to that work. But the lesson of the past six months must not be lost: when we put announcements before evidence, the people who pay the price are the ones who can least afford it.</p>
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