San Diego Doctor Weighs in on Leucovorin Treatment for Autism
Leucovorin as a potential treatment for autism is in the national spotlight, but a San Diego doctor has actually been using the medication to treat symptoms for years.
Dr. Lisa Loegering is a developmental-behavioral pediatrician based in Del Mar. She has been working with people on the autism spectrum for decades, and has been working with leucovorin as a potential treatment for the neurodivergence for a few years.
Here’s what she had to say about the medication and its potential positive effects on children diagnosed with autism.
What is Leucovorin?
Leucovorin, also known as folinic acid, is a prescription medication derived from the active form of folic acid. It’s been widely used for years in oncology, infectious disease management, and treatment of folate deficiency–related conditions, but not officially for the treatment of autism symptoms.
Currently, leucovorin is most commonly used to safeguard healthy cells from the toxic effects of methotrexate, a chemotherapy drug used in cancer treatment. According to the Federal Drug Administration (FDA), it’s been approved for that use since 1983.
Leucovorin is essentially a form of B9 vitamin, or folate. Folate is essential for a number of bodily functions, including brain development and function. There’s some research suggesting that some children with autism show a deficiency in the vitamin.
As such, President Donald Trump and his administration has directed the FDA to change the product’s label to include language indicating that leucovorin can be used to “improve certain symptoms in adults and pediatric patients with cerebral folate deficiency.”
At a press conference on Monday, Health and Human Services Secretary Robert F. Kennedy Jr. called leucovorin an “exciting therapy that may benefit large numbers of children who have suffered from autism,” while President Trump added the drug “gives hope to the many parents with autistic children.”
How Does Leucovorin Treat Autism?
As mentioned, some studies have shown that there appears to be a link between a deficiency of vitamin B9 or folate in the brain and some of the symptoms of autism. According to Dr. Lisa of Integrative Pediatrics of Del Mar, this research led her to the use of leucovorin to treat symptoms of autism.
“What was discovered is that around 70% of kids with autism make antibodies that bind to the folate transport system,” Dr. Lisa said. “So they become deficient in folate in their brain, even if it’s normal in their blood, and this is called cerebral folate deficiency.”
Leucovorin, essentially, works to bypass the mechanisms causing cerebral folate deficiency, or CFD.
“In some double-blind studies, groups of children with autism who were given leucovorin showed improvements in language and behavior,” Dr. Lisa said. A second group who received a placebo did not show improvements, she added.
Dr. Lisa added that a patient needs to take leucovorin for an extended period of time, such as a few years. In her experience, however, long-term patients can stop taking the treatment after some years and still keep the benefits to language and behavior.
Some Caveats to Leucovorin Treatment
There are a couple of important caveats to keep in mind, Dr. Lisa said. For one, she said that the vast majority of medical treatments don’t work in 100% of patients. In the studies of leucovorin, for example, about two-thirds of those given the treatment showed improvements. Dr. Lisa said that’s still very promising.
Secondly, autism is a complex and multi-factorial diagnosis. While leucovorin could address symptoms of some forms of autism, it’s certainly not a cure for every form of autism spectrum disorder.
“There are genetics that make you more likely to have autism, but it’s not generally one gene. It’s a combination of genes,” Dr. Lisa said. “And then environmental factors come in and interplay with these many genes.”
The best way to know whether a child with autism will respond well to leucovorin is to take a folate antibody test, Dr. Lisa said.
“People who have these antibodies will find potential improvements in this treatment,” she said. “There’s a lower chance for people who don’t test positive for those antibodies.”
Leucovorin Autism Treatment in San Diego
Using Leucovorin to treat some forms of autism has been an off-label use of the medication for a while. While more research needs to be done to solidify the link between CFD and some forms of autism, organizations like the Medical Academy of Pediatrics and Special Needs (MAPS) — of which Dr. Lisa is a member — still believes this is a cutting-edge treatment.
According to Dr. Lisa, she hopes that the FDA’s move to create new label language will help streamline treatment and insurance coverage, and spur more research into the drug’s effects on patients with autism.
“By making it approved, hopefully more kids will have access to it,” she said.
You can learn more about leucovorin treatment for autism in San Diego by reaching out to Dr. Lisa and her clinical staff through her website.
Media credit: Banner image and White House photo licensed from Adobe
