By: Dr. J. Thomas Megerian, pediatric neurologist and clinical director of the Thompson Autism and Neurodevelopmental Center at Rady Children’s Health
Many families have questions following recent federal announcements and media reports about vaccine schedules, Tylenol use during pregnancy, and possible new treatments for autism. We know these headlines can create uncertainty, questions and concern.
As a pediatric health care organization, Rady Children’s Health is committed to delivering evidence-based care rooted in science and delivered with compassion. Our recommendations remain aligned with leading medical organizations, which currently provide the following guidance:
Vaccines
The American Academy of Pediatrics (AAP), the Advisory Committee on Immunization Practices (ACIP) for the Centers for Disease Control and Prevention (CDC), and the California Department of Public Health (CDPH) continue to recommend the current childhood vaccine schedule as the best way to protect children against serious illness. Consistent with our commitment to science-based evidence, we know that no current study has found any link between vaccines and autism.
Tylenol (Acetaminophen)
Some recent reports have suggested a connection between acetaminophen use in pregnancy and autism. Current evidence does not establish a causal link. Moreover, studies that control for family genetics and maternal health show that earlier associations disappear. Decades of research show that autism has complex causes involving both genetics and environmental influences working together. Importantly, autism is not caused by anything parents did or did not do. The American College of Obstetricians and Gynecologists has not changed its guidance and continue to consider acetaminophen an appropriate option for managing pain or fever during pregnancy when used as directed.
Leucovorin Research
Leucovorin is an FDA-approved drug used to treat certain cancers and types of anemia. Research on its use in autism comes from a limited number of studies, and the evidence remains limited. At this time, Leucovorin is not part of standard care for autism. Larger, well-controlled clinical trials are still needed to confirm its efficacy and safety as a therapeutic treatment before any recommendations can be made.
Scientific guidance is essential, and so too is the way we support people with autism, which is a lifelong neurodevelopmental condition that is part of the broader spectrum of neurodiversity. Children and adults on the autism spectrum are valued members of our community, bringing unique strengths, perspectives and potential. We also recognize that for many families and individuals with autism, the effect on function and quality of life is profoundly impairing and life-altering, affecting not only the lives of the individual but those who care for them.
Recognizing and embracing neurodiversity means respecting the different experiences and severity of symptoms that are unique to each individual and their families as well as their unique ways of thinking, processing, and experiencing the world. People with autism and their families deserve care, respect, and support free from stigma. Our role is not to speculate about unproven claims but to walk alongside individuals and families with empathy, accurate information, and high-quality, evidence-based care.
FAQs
Folinic Acid (Leucovorin) and Autism
Did the FDA approve folinic acid for autism? No. The FDA has not approved folinic acid (also called leucovorin) for autism. The FDA recently updated the label of an older medicine to include a condition called cerebral folate deficiency (CFD). CFD can sometimes look like autism in some children, but this is not the same as approval for autism.
So, can folinic acid treat autism? We do not know. Studies are still ongoing. Some children with autism may also have folate problems, like CFD or certain antibodies, and may respond better to folinic acid. But to be clear: folinic acid is not approved as an autism treatment for all children.
What is folinic acid? Is it the same as folic acid? Folinic acid is a special form of folate (a type of vitamin B). It is different from folic acid (the common vitamin in foods and supplements) and from L-methylfolate. Folinic acid canreach the brain even when usual folate pathways are blocked.
Who might benefit? Children who have CFD or certain antibodies (called folate receptor antibodies) may beneft from folinic acid. Some very early research in a few small studies shows these children may see small improvements in language and some autism-related behaviors. At this time, it is too early to know who might benefit.
What does research show? A few small studies found some children improved in language, social interaction and behavior when taking folinic acid. Some studies have found that treatment with folinic acid, a form of folate that bypasses the blocked receptors, can improve symptoms in children with autism who test positive for people who make antibodies against their own folate receptors, preventing the brain from absorbing folate which is needed for brain function and health.Results are hopeful but not conclusive. Larger studies are needed before any broad recommendations can be made.
Is folinic acid safe? In short-term use, it is usually well tolerated. Long-term safety is not known. Side effects can include upset stomach, diarrhea, nausea and vomiting, fatigue, trouble sleeping, loss of appetite, irritability, hair loss, and skin inflammation. As with any drug or food, fatal allergic reactions can occur. Always talk to your doctor before starting any medicine.
Do we need special tests first? Sometimes. Spinal fluid tests or checking for folate receptor autoantibodies in the fluid that surrounds the brain can help decide if a child has CFD and might response to folinic acid. Most insurance will not pay for tests to check for blood antibodies against the folate receptor. Your doctor will guide you.
Can my child try folinic acid now? Possibly. Doctors can prescribe it “off label” (not officially for autism). If tried, it should be used along with – not instead of – standard autism therapies.
Will insurance cover it? Insurance may only cover it if CFD or a medical need is clearly shown.
What’s the Thompson Autism and Neurodevelomental Center’s approach? We may try folinic acid for a few months in some children, watch carefully for real improvements, and continue only if it clearly helps.
Bottom line for folinic acid: Folinic acid is not approved for autism. Some children may benefit, especially if they have folate problems, but more research is needed. Any use should be supervised by your doctor.
Bottom line for acetaminophen: There is no proof that acetaminophen causes autism. The small link in some studies may come from other factors. Always use it carefully and talk with your pregnancy care team.
Talk with us
If you are thinking about folinic acid, testing for folate issues, or safe medicines in pregnancy, please ask your care team. We are here to help you weigh possible benefits, uncertainties, and safer choices for your family.
For more information, please contact the Thompson Autism and Neurodevelopmental Center at Rady Children’s Health Orange County :(714) 288-7651
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