Listen to this articleThe basics:
- HHS unveils autism initiatives, including FDA-recognized leucovorin treatment
- Tylenol pregnancy warning prompts strong opposition from doctors, Kenvue, BioNJ and more
- NIH launches $50M Autism Data Science Initiative to advance research
- Critics say guidance risks confusing patients and undermining public health
The Trump administration announced new actions Sept. 22 aimed at tackling autism, including one pertaining to the use of Tylenol during pregnancy that has elicited strong pushback.
“For too long, families have been left without answers or options as autism rates have soared,” said Health and Human Services Secretary Robert Kennedy Jr. “Today, we are taking bold action.”
The actions include:
- Establishing leucovorin as the first Food and Drug Administration-recognized therapeutic for children with cerebral folate deficiency (which has been associated with autism) and autistic symptoms
- FDA will publish a Federal Register notice outlining a label update for leucovorin. That change will authorize treatment for children with autism spectrum disorder (ASD) as well as continued use if children show language, social or adaptive gains
- Following that label update, state Medicaid programs can cover leucovorin for the indication of ASD
- Officials note that while promising, leucovorin is not a cure and may only lead to improvements in speech-related deficits for a subset of children with ASD
- National Institutes of Health will launch confirmatory trials and new research into the impact of leucovorin, including safety studies
- FDA says it is responding to prior clinical and laboratory studies that suggest a potential association between acetaminophen (Tylenol and similar products) use during pregnancy and adverse neurodevelopment outcomes
- The agency also says it recognizes that there are contrary studies showing no association; and that there are risks for untreated fever in pregnancy, both for the mother and fetus
- Given the lack of clear casual evidence, the Department of Health and Human Services says it encourages clinicians to exercise their best judgment
- Issuing a physician notice on acetaminophen that includes beginning the process of initiating a safety label change
NIH also announced the recipients of the Autism Data Science Initiative, funding 13 projects totaling more than $50 million to transform autism research.
Consult your care provider
Kennedy said the actions open 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,” said Kennedy.
The administration said the actions come amid a surge in cases of ASD, for which diagnoses have jumped nearly 400% since 2000 and now affect 1-in-31 American children.
“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,” said FDA Commissioner Dr. Marty Makary. “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.”
The president boiled down the acetaminophen piece to this.
“Taking Tylenol is not good,” said President Donald Trump. “I’ll say it. It’s not good.”
The issue of autism links and root causes has been a hot-button one. It also played a major role in the 2024 election, with Kennedy leading his Make American Healthy Again movement. MAHA eventually aligned with the Trump campaign, resulting in Kennedy’s appointment to the top health leadership role.
He has since made finding autism links and root causes a cornerstone of HHS under his leadership. Kennedy has been a longtime skeptic of vaccines and critic of the pharmaceutical industry.
Facing facts
The announcements drew sharp rebukes from a number of doctors, scientists, advocacy groups and more, as well as from Summit-headquartered Kenvue — a J&J spinoff that manufactures Tylenol.
“We believe independent, sound science clearly shows that taking acetaminophen does not cause autism,” Kenvue said in a statement. “We strongly disagree with any suggestion otherwise and are deeply concerned about the health risks and confusion this poses for expecting mothers and patients.
“Acetaminophen is the safest pain reliever option for pregnant women as needed throughout their entire pregnancy. Without it, women face dangerous choices: suffer through conditions like fever that are potentially harmful to both mom and baby or use riskier alternatives. High fevers and pain are widely recognized as potential risks to a pregnancy if left untreated.”
“The facts are that over a decade of rigorous research … confirms there is no credible evidence linking acetaminophen to autism. We stand with the many public health and medical professionals who have reviewed this science and agree.
— Tylenol maker Kenvue
Kenvue also noted that acetaminophen is one of the most widely studied pain relieves and fever reducers in infants and children. The company pointed to numerous randomized, controlled clinical trials that support its safety in infants and children, when used as directed.
“The facts are that over a decade of rigorous research, endorsed by leading medical professionals and global health regulators, confirms there is no credible evidence linking acetaminophen to autism,” the statement continued. “We stand with the many public health and medical professionals who have reviewed this science and agree.
“We will continue to reinforce that expecting mothers speak to their health professionals before taking any over-the-counter medication and will explore all options to protect the health interests of American women and children.”
Safety first
Leading New Jersey life sciences and health care organizations quickly responded, as well.
“The life sciences sector is driven by using the scientific method to discover new and more effective ways to improve the lives of patients and individuals worldwide,” said Debbie Hart, president and CEO, BioNJ, in a statement. “As a community, every decision made at a company or research institution must be based on the best evidence available. Any guidance regarding health care decisions that is not backed by a robust body of peer-reviewed scientific evidence undermines this legacy and contradicts the foundation of reliability in the life sciences.
“BioNJ is hopeful that any health guidance leverages the availability of considerable scientific expertise to ensure that Patients and clinicians have the best evidence-based information available.”
“From rigorous R&D and clinical trials to comprehensive approval and regulatory processes, life sciences companies have long partnered with safety and efficacy agencies in the U.S. and globally to ensure that patient safety remains the cornerstone of advancing human health,” said Chrissy Buteas, president and CEO of the HealthCare Institute of New Jersey (HINJ), in a statement.
“They remain committed to upholding the highest standards of scientific integrity and medical safety as they pursue their mission: to improve and save lives worldwide.”
Public health risks
Steven Fleischman is president of the American College of Obstetricians and Gynecologists (ACOG). In a statement, he said that suggestions that acetaminophen use in pregnancy causes autism rates are not only “highly concerning” to clinicians – but also irresponsible.
He said it creates a harmful and confusing message for pregnant patients, including those who rely on this “beneficial medicine” during pregnancy.
“Today’s announcement by HHS is not backed by the full body of scientific evidence and dangerously simplifies the many and complex causes of neurologic challenges in children,” said Fleischman. “It is highly unsettling that our federal health agencies are willing to make an announcement that will affect the health and well-being of millions of people without the backing of reliable data.”
Today’s announcement by HHS is not backed by the full body of scientific evidence and dangerously simplifies the many and complex causes of neurologic challenges in children.
– Steven Fleischman, president, American College of Obstetricians and Gynecologists
“Any association between acetaminophen and autism is based on limited, conflicting, and inconsistent science and is premature,” said Autism Science Foundation Chief Science Officer Dr. Alycia Halladay. “This claim risks undermining public health while also misleading families who deserve clear, factual information.
“For many years, RFK and President Trump have shared their belief that vaccines cause autism, but this is also not supported by the science, which has shown no relationship between vaccines and autism.”
Democratic U.S. Sen. Andy Kim wrote on X, “Show us the receipts because this isn’t backed by science. This is what happens when you fire experts, defund research, manipulate data, and place political appointees in positions of power critical to the health of our nation.”
Show us the receipts because this isn’t backed by science. ⁰⁰This is what happens when you fire experts, defund research, manipulate data, and place political appointees in positions of power critical to the health of our nation. https://t.co/JG7M1csNh8
— Senator Andy Kim (@SenatorAndyKim) September 22, 2025
