NHS £2 pill eyed for autism treatment as US weighs approval, prompting breakthrough claims

US health officials are weighing whether leucovorin, a folate-boosting drug already used to counter certain chemotherapy toxicities, could be prescribed or more widely adopted as a treatment for autism in children. The development follows statements from the U.S. Food and Drug Administration that the medication could be endorsed for broader use and that its label may be updated to reflect potential new indications. FDA Commissioner Marty Makary emphasized the agency’s duty to inform doctors and the public about emerging options, saying, “We have a duty to let doctors and the public know. We are going to change the label to make it available.” This potential shift comes amid ongoing debate about how autism is diagnosed and treated, and as proponents point to occasional, dramatic improvements in individual patients after folate-related therapy.

Autism diagnoses have risen sharply over the past two decades. Scientists attribute some of the increase to better recognition and earlier detection, though the rise remains controversial in parts of the medical community. In a separate thread, political rhetoric has intertwined with the science: former President Donald Trump used a White House address to warn pregnant women about potential risks of paracetamol, urging caution over its use to relieve fevers. British scientists later cautioned that there is no robust evidence linking the drug to autism and described those claims as fearmongering. Amid these debates, attention has shifted to a potential, low-cost intervention: ensuring adequate folate levels in early development, whether through pregnancy or childhood treatment.

A central figure in the leucovorin narrative is Dr Richard Frye, a pediatric autism specialist at the Rossignol Medical Centre in Arizona, who argues that a large share of autism cases stem from folate deficiencies. Frye has argued that up to roughly three-quarters of children with autism have dangerously low folate levels, and that leucovorin can deliver more folate to the brain than standard folic acid supplements. He describes leucovorin as a simple, safe therapy that has produced notable improvements in dozens of his patients within weeks. Frye’s stance also extends to prenatal care, suggesting that adequate maternal folate could reduce the risk of autism in the first place. He notes that folate is present in many foods and that pregnant women are already advised to take folic acid supplements. In his view, strengthening folate delivery to the developing brain could be a game changer.

The research is not without controversy. Critics within the autism community warn that evidence linking leucovorin to sustained improvements remains mixed, and some argue that observed gains may reflect natural developmental spurts rather than drug effects. Autistica’s Dr James Cusack cautions that outcomes could be influenced by the timing of diagnosis and the broader trajectory of development in early childhood. King’s College London pediatric neurodisability expert Dr Michael Absoud says while there is interest in folate’s role, it is premature to regard leucovorin as a universal solution. “Autism is a spectrum,” Absoud notes, adding that the condition is not simply something to be fixed, but a difference in brain function that requires nuanced supports.

The discussion sits within a broader context of folate in pregnancy. Folate is essential for neural development, and NHS guidance already recommends daily folic acid supplements for pregnant women to reduce certain birth defects. In the United Kingdom, policymakers have moved to strengthen folate intake across the population: starting next year, manufacturers will be legally required to add folic acid to flour, an initiative aimed at reducing folate-related defects. Some observers have called for extending such fortification to other foods, including rice. While experts say early folate exposure during pregnancy shows the strongest potential for impacting autism risk, they emphasize that more research is needed to determine whether folate supplementation can meaningfully reduce autism diagnoses in the population and under what conditions leucovorin would be most effective.

Supporters argue the leucovorin approach addresses a biochemical dimension of autism that could yield meaningful improvements for some children. Frye contends that many patients experience substantial, rapid gains once treated, and he describes leucovorin as a very safe drug with minimal side effects. Critics counter that the data come from small samples and non-randomized settings, urging restraint until robust, large-scale trials confirm the therapy’s efficacy and safety profile across diverse groups. Dr Cusack emphasizes the risk of premature conclusions and urges continued emphasis on evidence-based practice and patient-centered care.

For families touched by autism, personal stories have amplified the discussion. One case highlighted in Frye’s outreach centers on Ryan Baldridge, a Missouri boy diagnosed with autism at age four who was largely non-verbal. After his family sought Frye’s assessment in 2023, Ryan underwent a folate-focused treatment plan involving leucovorin. Within two weeks, Ryan began speaking in full sentences and, for the first time, expressed affection verbally. His father, Kim Baldridge, recalled an unprompted moment when Ryan said, “I love you” to his father, describing it as a breakthrough that felt transformative for the family. The Baldridges caution that such changes are not universal, but they underscore the urgency many families feel for therapies that could improve communication and quality of life for children with autism.

As U.S. regulators weigh the potential for leucovorin to help autistic children, researchers and clinicians alike stress the need for rigorous trials that can clarify who might benefit, at what doses, and at what stages of development. The conversation about leucovorin sits at the intersection of neuroscience, nutrition, pediatrics, and patient advocacy, and it invites careful scrutiny of promising early signals against the long-standing imperative for robust, reproducible science. In the meantime, public health efforts surrounding folate—both in pregnancy and early childhood—continue to be a central theme in autism research and policy discussions, with policymakers and clinicians seeking to translate biochemical insights into practical, proven improvements for affected families.