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.