Federal regulators are distancing themselves from earlier optimism about a cheap, decades-old drug touted as a potential breakthrough for children with autism. Per Politico, two senior FDA officials said on Monday that the agency doesn't have enough solid evidence to support leucovorin (aka folinic acid) as an autism treatment—a sharp shift from September, when Health Secretary Robert F. Kennedy Jr. and FDA Commissioner Marty Makary publicly highlighted the drug as a promising option that could help "hundreds of thousands" of children, citing a now-retracted study.
On Tuesday, the FDA instead approved leucovorin for a different, extremely rare condition: FOLR1-related cerebral folate transport deficiency, a genetic disorder that disrupts folate's entry into the brain and can cause severe developmental delays, movement problems, and seizures. The Washington Post reports that fewer than 50 cases of FOLR1-CFTD in total have been logged. The drug, already used in cancer and anemia care, is now the only FDA-approved therapy for that disorder in both children and adults, per Politico. One official stressed that while doctors may still prescribe leucovorin off-label for autism on an individual basis, the agency can't say it works for that broader population.
The FDA's literature review found the evidence strongest for the ultra-rare folate deficiency condition, not autism; the key randomized trial supporting autism use was pulled by the European Journal of Pediatrics over data errors. The agency says it remains open to companies formally studying leucovorin for autism and is monitoring demand after earlier publicity triggered supply concerns. A recent paper in the Lancet noted that prescriptions for leucovorin for kids ages 5 to 17 surged 71% higher than usual in the three months after the September presser praising the drug, per the AP.
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