How Families Discovered the Benefits of Leucovorin for Autism Before Trump’s Endorsement

Last winter, Brian Noonan read online that some doctors were using a drug usually for cancer to help children with autism. His son, Benjamin, had just been diagnosed, and Brian felt curious. “We jumped on it,” he said. “It felt right and it made sense.”

The drug is called leucovorin, or folinic acid, a form of vitamin B9 that helps our bodies create healthy blood cells. It’s particularly important for pregnant women to reduce the risk of birth defects.

Recently, leucovorin gained attention after President Trump mentioned it during a press conference about autism. He claimed that it might help with some symptoms, alongside discussing other studies connecting acetaminophen use during pregnancy to autism.

In the autism community, leucovorin isn’t a new concept. Dr. Richard Frye, a child neurologist in Phoenix, has studied it for over twenty years. He emphasizes that leucovorin isn’t a cure and more research is essential. However, he does prescribe it for certain patients. Benjamin is one of them.

Noonan shares that since starting the drug, Benjamin’s speech has improved. “He’s verbal, but he struggled with sentences,” he noted. Despite these advancements, Benjamin still faces challenges with hyperactivity. The family is aware that they are trying an experimental medication, along with other therapies like speech and occupational support.

Other families report more significant gains. Ben Blomgren from Minneapolis said his son, Josh, who is 11, was prescribed leucovorin earlier this year when he faced expulsion from school. Josh had difficulty with language skills and was not yet toilet trained. After beginning the medication, his sleep improved, and he became fully toilet trained. “We saw major improvements,” Ben said.

Yomarie Miranda from Florida observed positive changes in her 7-year-old son, Ethan. Since starting leucovorin, Ethan follows classroom instructions better and communicates more effectively. “He looks at me when I speak and talks in complete sentences now,” she remarked.

Leucovorin received FDA approval in the 1980s to reduce chemotherapy side effects. Recently, attention has focused on its potential effects on children with cerebral folate deficiency (CFD), a condition that can limit folate from reaching the brain. This deficiency can affect speech and behavior, and some children with autism also have CFD. The FDA has announced plans to update the drug’s label to include its use for this condition.

Despite this, experts express concern over the insufficient evidence supporting leucovorin as a treatment for autism. Dr. Aaron Kesselheim, a professor at Harvard, called the FDA’s recent endorsement “irresponsible.” He noted that the agency should differentiate between effective treatments and those without proven results. Similarly, Alycia Halladay from the Autism Science Foundation criticized the notion that leucovorin represents a breakthrough in autism treatment.

Experts cite a 2023 analysis that showed about 85% of CFD patients experienced some clinical benefits. However, there is debate regarding how many children with autism also have CFD. Estimates vary widely, with Dr. Frye suggesting around 75% while other sources argue it’s between 10% to 30%.

One notable study published in Molecular Psychiatry in 2018 showed that about two-thirds of children on leucovorin improved in verbal communication. However, Dr. David Mandell cautioned that the study’s small sample size limits its applicability. He stressed the need for larger, clearer trials to confirm any benefits.

Dr. Kesselheim emphasizes that access to potential treatments is vital for patients with unmet medical needs, but questions still linger over the validity of testing for folate deficiencies. Currently, methods include lumbar punctures and tests not yet FDA-approved for children.

As discussions continue, many parents are already trying supplements and unregulated versions of leucovorin, which can pose risks. Edward Quadros, a researcher studying leucovorin, believes that FDA approval could ensure quality dosing, providing a safer option for families seeking answers.

While opinions vary, it’s clear that the conversation around leucovorin and autism is only just beginning. Ongoing research is essential to provide more clarity and direction for families navigating these complex challenges.

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