The U.S. government has announced that leucovorin, a prescription medication typically used for cancer and anemia, will now be available for treating autism in some children. On Monday, the Trump administration revealed that the U.S. Food and Drug Administration (FDA) would modify the drug's label to expand its use. This change allows leucovorin to be prescribed for cerebral folate deficiency, a rare condition linked to autism.

Cerebral folate deficiency occurs when folate levels in the blood are low, despite normal levels elsewhere in the body. Symptoms often appear around age two and can include developmental delays and movement issues, which are also common in autism. FDA officials stated that research indicates leucovorin may help improve verbal communication in children with autism who are folate deficient. "Children are suffering and deserve access to potential treatments that have shown promise," said FDA Commissioner Marty Makary.

The FDA's approval allows for continued use of leucovorin in children with autism if they show improvements in language, social skills, or adaptive behavior. However, experts in Australia have cautioned that the research supporting leucovorin's effectiveness for autism is still in its early stages and should not lead to widespread use.

Leucovorin, also known as calcium folinate, is a form of vitamin B9. It is commonly used to mitigate side effects from certain chemotherapy drugs and to treat anemia caused by folate deficiency. The drug can be administered in pill form or through injections. Side effects may include fever, nausea, rashes, and sleep disturbances.

Research into leucovorin's potential benefits for autism has produced mixed results. Some studies suggest that it may lead to improvements in behavioral and language outcomes. For instance, a recent study in India found that children taking leucovorin scored better on autism rating scales compared to those on a placebo. Similar findings have emerged from smaller studies in China, Iran, and France.

Despite these findings, experts like Andrew Whitehouse, a professor of autism research, emphasize that the evidence remains weak. He stated, "Right now, the science does not come close to the standard needed to recommend leucovorin in the clinical management of autism." He called for larger, more rigorous studies to determine the drug's true efficacy.

Clinical psychologist Adam Guastella echoed these concerns, noting that while some studies show promise, they are not of sufficient quality for widespread application. The FDA has also acknowledged that leucovorin is not a cure for autism and may only benefit a subset of children. The agency stressed that treatment should be closely monitored and combined with non-pharmacological approaches, such as behavioral therapy.

The causes of autism are complex and not fully understood. Genetics play a significant role, with numerous genes linked to the disorder. Environmental factors, such as maternal age, have also been associated with autism. While folate is crucial during pregnancy, the relationship between maternal folate levels and autism risk remains inconsistent.

Current autism treatments focus on alleviating symptoms that affect daily functioning and quality of life. Experts emphasize the importance of supportive environments, including inclusive education and community understanding, rather than framing autism as a condition that needs to be fixed. Many researchers advocate for evidence-based practices to support the development of autistic children.