.png)
President Donald Trump and HHS Secretary Robert F. Kennedy Jr. challenged us to break down the walls between our agencies so we can rapidly address the health crises facing the American people. Today, we announce an approach to provide relief for children on the autism spectrum.
This administration’s bold action — opening the door to the first FDA-recognized treatment for autism, confronting environmental and medical risk factors, and investing in groundbreaking research — follows the science, restores trust and will deliver hope to millions of families. The nearly five-fold increased prevalence of autism in recent decades demands a rapid response — with prompt research and by acting on information as it becomes available.
Despite the sharp rise in autism prevalence, there is no scientific consensus on what is causing rates to grow, and families have few effective tools to prevent or treat it. Today, the Trump administration launched a trio of initiatives representing unprecedented cooperation across the National Institutes of Health (NIH), the U.S. Food & Drug Administration (FDA), and the Center for Medicare & Medicaid Services (CMS), the three agencies we lead, to provide help for families.
The NIH has launched the Autism Data Science Initiative to accelerate autism research, devoting an additional $50 million to the cause. Nearly 250 research teams applied, and NIH peer reviewers chose the 13 best projects focused on root causes and therapies, including replication and validation studies to guarantee gold standard science.
The studies feature a new kind of science called exposomics, where scientists consider environmental and medical factors, nutrition and events during pregnancy in combination with biology and genetics to answer vital questions. Holistic approaches to science like this — conducted in partnership with families and physicians — are the best way to avoid blind spots in our approach.
Already, from our intense research efforts over the last five months, scientists at the Department of Health and Human Services identified multiple potential hypotheses including some involving mitochondrial dysfunction and microbiome-related physiology. However, two signals have emerged that could help parents today, which is why we are acting now.
Cerebral Folate Deficiency
While the pathophysiology of autism is still being understood, one potential area of interest is the folate transport system in the central nervous system. There are both genetic and autoimmune etiologies that can impact the ability of children to effectively absorb folate in the brain due to impaired or damaged folate transport receptors. This can lead to a phenotype of developmental delay consistent with autism spectrum disorder and can occur even if folate levels in the body are otherwise normal.
A relatively inexpensive, generic prescription drug called leucovorin can help some children to heal by bypassing the transport blockage. Peer-reviewed publications and randomized clinical trials have documented that up to 60 percent of folate-deficient children with ASD can have improved verbal communication if given leucovorin. Some clinicians have reported that children with a folate receptor antibody, which blocks the receptor, may be more likely to benefit from leucovorin. Leucovorin is a drug that has been on the market for decades and has a favorable safety profile. It is important to note that leucovorin is not a cure for autism but has demonstrated an improvement in speech-related deficits for autism.
Parents are understandably impatient when they learn that clinical trials will take years to complete. Americans are tired of waiting, so we are cutting regulatory red tape to enable families to pursue treatment now, as directed by their physician.
Our agencies will continue to work together in an unprecedented lockstep fashion to meet the needs of Americans. In the coming weeks, the FDA will approve prescription leucovorin as a treatment for children with cerebral folate deficiency and autistic symptoms. Over half of all American children are insured by Medicaid or CHIP, so upon this FDA label change, states will be required to cover leucovorin around the country. States will also have tools to monitor its use and ensure appropriate utilization. CMS will also issue guidance to convene states and existing research networks to support the FDA and NIH in their research efforts of leucovorin, while also maintaining the privacy of health information of beneficiaries.
Acetaminophen
Over the past few years, observational evidence has suggested that when moms take acetaminophen during pregnancy, especially close to delivery, it is correlated with subsequent diagnosis of conditions like autism and ADHD in their children. Peer-reviewed data from large-scale cohort studies, including the Nurses’ Health Study II and the Boston Birth Cohort, find this association.
At the same time, we also recognize the literature continues to evolve and evidence from family control studies have failed to find a correlation. Furthermore, acetaminophen is the only over-the-counter medication approved to treat fevers during pregnancy, and high fevers in pregnant moms can pose a risk to their unborn child as well, such as neural tube defects.
What should parents do? In light of this evidence and lack of clear alternatives, we believe acetaminophen should be used judiciously in pregnancy, and under medical supervision of an obstetrician, as a practical, prudent medical approach that balances risks and benefits. This caution could be extended to infants and toddlers.
By updating medication labels, paying for a therapeutic solution, and funding new research, we are putting the health of children first in line with our radical transparency mission. This is how we rebuild trust in public health — by giving parents the information they need to make the best choices for their children.
English (US)