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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During a Senate hearing yesterday, Kennedy noted that a CDC study found a 260% higher rate of autism in boys who got the MMR vaccine.
A Department of Health and Human Services (HHS) report commissioned by Robert F. Kennedy Jr. indicates Tylenol use during pregnancy is one factor contributing to the U.S. autism epidemic, according to media reports.
Sources close to the matter said low levels of folate as well as the use of the acetaminophen-based drug will be named in the forthcoming HHS autism report as among potential causal factors behind autism.
Kennedy, secretary of the HHS, told Fox and Friends last week that his agency was about to reveal causes of autism and make government regulation recommendations accordingly. His remarks suggest that mothers’ Tylenol use and folate deficiency will be among a multitude of factors cited in the HHS report.
“There is not a single cause, there are many, many — there’s an aggregation of causes,” said Kennedy. “We are now developing sufficient evidence to ask for regulatory action on some of those, or recommendations.”
He noted the documented explosion in autism rates, which have gone from less than one in 10,000 in 1970 to one case for every 31 Americans, according to the Centers for Disease Control and Prevention (CDC). Even if autism is over-diagnosed, a significant portion of these children are severely impaired, indicating a real, notable increase in the disorder.
“Most cases now are severe,” Kennedy said in April while discussing the results of a CDC autism survey. He explained that “25% of the kids who are diagnosed with autism are non-verbal, non-toilet trained,” and have other dysfunctional behaviors typical of severe autism like head-banging.
During a Senate hearing on Thursday, Kennedy also pointed to a link between the Measles, Mumps, and Rubella (MMR) vaccine and autism.
“In 2002, CDC did an internal study of Fulton County, Georgia, children, and looked at children who got the MMR vaccine on time and compared those to kids who got them later. The data from that study showed that black boys who got the vaccine on time had a 260% greater chance of getting an autism diagnosis than children who waited,” Kennedy explained.
“The chief scientist on that, Dr. William Thompson, the senior vaccine safety scientist at CDC, was ordered to come into a room with four other co-authors by his boss, Frank DeStefano, who’s the head of the Immunization Safety Branch, in order to destroy that data,” said Kennedy.
This story was divulged by CDC whistleblower Dr. William Thompson, one of the co-authors who intentionally omitted the 2004 study data showing the MMR vaccine-autism connection in black children.
While an evolving, broadening definition of autism may contribute to increased diagnoses, Kennedy believes “environmental toxins” have contributed to a full-blown epidemic of autism.
“We’re going to look at vaccines, but we’re going to look at everything. Everything is on the table, our food system, our water, our air, different ways of parenting, all the kind of changes that may have triggered this epidemic,” the HHS head previously told Fox News.
“It is an epidemic,” Kennedy insisted. “Epidemics are not caused by genes. Genes can provide a vulnerability, but you need an environmental toxin.”
“We know that it is an environmental toxin that is causing this cataclysm,” said Kennedy, “and we are going to identify it.”
Officials at the the National Institutes of Health and the Centers for Medicare & Medicaid Services announced a partnership Wednesday to research “root causes of autism spectrum disorder.”
As part of the project, NIH will build a real-world data platform enabling advanced research across claims data, electronic medical records and consumer wearables, according to the agencies.
“We’re using this partnership to uncover the root causes of autism and other chronic diseases,” said HHS Secretary Robert F. Kennedy Jr. “We’re pulling back the curtain – with full transparency and accountability – to deliver the honest answers families have waited far too long to hear.”
CMS and NIH will start this partnership by establishing a data use agreement under CMS’ Research Data Disclosure Program focused on Medicare and Medicaid enrollees with a diagnosis of autism spectrum disorder or ASD.
“This partnership is an important step in our commitment to unlocking the power of real-world data to inform public health decisions and improve lives,” NIH Director Dr. Jay Bhattacharya said. “Linking CMS claims data with a secure real-world NIH data platform, fully compliant with privacy and security laws, will unlock landmark research into the complex factors that drive autism and chronic disease – ultimately delivering superior health outcomes to the Americans we serve.”
Researchers will focus on autism diagnosis trends over time, health outcomes from specific medical and behavioral interventions, access to care and disparities by demographics and geography and the economic burden on families and healthcare systems, according to a news release.
