HHS' Bold Actions to Tackle Austism Epidemic

"We’re Going Bold to Tackle Autism"

HHS Press Release (Sept 22, 2025)

President Trump, Secretary Kennedy Announce Bold Actions to Tackle Autism Epidemic

WASHINGTON—SEPTEMBER 22, 2025—Speaking from the Roosevelt Room today, President Donald J. Trump and U.S. Health and Human Services (HHS) Secretary Robert F. Kennedy, Jr. announced bold new actions to confront the nation’s autism spectrum disorder (ASD) epidemic, which has surged nearly 400% since 2000 and now affects 1 in 31 American children.  

“For too long, families have been left without answers or options as autism rates have soared,” Secretary Kennedy said. “Today, we are taking bold action—opening the door to the first FDA-recognized treatment pathway, informing doctors and families about potential risks, and investing in groundbreaking research. We will follow the science, restore trust, and deliver hope to millions of American families.”  

First, the U.S. Food and Drug Administration (FDA) will act on a potential treatment for speech-related deficits associated with ASD. The FDA today is publishing a Federal Register notice outlining a label update for leucovorin for cerebral folate deficiency, which has been associated with autism. This action establishes the first FDA-recognized therapeutic for children with cerebral folate deficiency and autistic symptoms.   

The change will authorize treatment for children with ASD, with continued use if children show language, social, or adaptive gains. Following the label update for ASD, state Medicaid programs will be able to cover leucovorin for the indication of ASD, in partnership with the Centers for Medicare & Medicaid Services (CMS). Finally, the National Institutes of Health (NIH) will launch confirmatory trials and new research into the impact of leucovorin including safety studies.  

While promising, it is important to note that leucovorin is not a cure for ASD and may only lead to improvements in speech-related deficits for a subset of children with ASD. Furthermore, this treatment must be administered under close medical supervision and in conjunction with other non-pharmacological approaches for children with ASD (e.g., behavioral therapy). 

“As a physician, I have seen how devastating autism spectrum disorder can be for children and their families,” CMS Administrator Dr. Mehmet Oz said. “Today’s actions represent an unprecedented, comprehensive approach to deepen our understanding of the causes of autism, share what we know and don’t know based on current research, and ensure that every child has a better chance to thrive. By providing access to a drug to treat symptoms associated with autism, we are providing hope to families and providers who have until today had very limited options.” 

Second, HHS will act on acetaminophen. Today, the FDA will issue a physician notice and begin the process to initiate a safety label change for acetaminophen (Tylenol and similar products). HHS will launch a nationwide public service campaign to inform families and protect public health.  

The FDA is responding to prior clinical and laboratory studies that suggest a potential association between acetaminophen use during pregnancy and adverse neurodevelopmental outcomes. FDA also recognizes that there are contrary studies showing no association and that there can be risks for untreated fever in pregnancy, both for the mother and fetus.  

Given the conflicting literature and lack of clear causal evidence, HHS wants to encourage clinicians to exercise their best judgment in use of acetaminophen for fevers and pain in pregnancy by prescribing the lowest effective dose for the shortest duration when treatment is required. Furthermore, FDA recognizes that acetaminophen is often the only tool for fevers and pain in pregnancy, as other alternatives (e.g., NSAIDs) have well documented adverse effects. FDA is partnering with manufacturers to update labeling and drive new research to safeguard mothers, children, and families.

“A growing body of evidence suggests that some children suffering from autism are folate deficient within the brain—a problem that can be treated with leucovorin,” FDA Commissioner Dr. Marty Makary said. “Given the extent of the current autism epidemic, physicians should immediately have this treatment option available for candidate children. We are also sharing new information about the potential risks of acetaminophen so patients can make a more informed decision with their health care provider.” 

Third, NIH today is announcing the recipients of the Autism Data Science Initiative (ADSI), funding 13 projects totaling more than $50 million to transform autism research. ADSI integrates large-scale biological, clinical, and behavioral data with an exposomics approach that examines environmental, nutritional, medical, and social factors alongside genetics.

Projects employ advanced methods such as machine learning and organoid models, address both children and adults across the lifespan, and establish replication hubs to ensure rigor. Each project includes community engagement to align research with the needs of autistic individuals, families, and clinicians.  

“Millions of American families who care for autistic kids need scientists to apply gold standard science, expertise, and open minds to figure out how to help these kids,” NIH Director Dr. Jay Bhattacharya said. “With the Autism Data Science Initiative, NIH is harnessing cutting-edge science to uncover the root causes of autism. We are building knowledge that can improve lives and restore hope for families.”  

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