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American Focus > Blog > Health and Wellness > Leucovorin has a place in autism treatment, researcher says, but he tamps down parents’ expectations
Health and Wellness

Leucovorin has a place in autism treatment, researcher says, but he tamps down parents’ expectations

Last updated: October 1, 2025 1:32 am
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Leucovorin has a place in autism treatment, researcher says, but he tamps down parents’ expectations
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Retired autism researcher Robert Hendren, who conducted a study of leucovorin.Courtesy Nicholas Roznovsky

In the realm of autism treatment, the conversation surrounding leucovorin has gained traction primarily through the enthusiastic advocacy of parents and small studies suggesting benefits. This chemotherapy drug, known scientifically as folinic acid, has become a focal point for some families seeking alternative therapies for their autistic children.

Recently, the controversy surrounding leucovorin has escalated with President Trump’s announcement that the federal government plans to revise the drug’s labeling to facilitate its wider distribution. The FDA’s approval is specifically aimed at treating a subset of individuals with autism who also suffer from cerebral folate deficiency, a neurological disorder that hinders vital brain nutrient transport.

Research indicates a notable overlap between autism and cerebral folate deficiency, with some studies, like one report, estimating that around 38% of those on the autism spectrum may experience this deficiency.

However, evidence supporting the efficacy of leucovorin remains sparse and largely derived from smaller trials. Retired researcher Robert Hendren, who contributed to this growing body of work, expressed caution about over-inflated expectations of the treatment. “While I have not witnessed remarkable results from leucovorin or folinic acid, I do believe it can play a supportive role in treating autism spectrum disorder (ASD),” he stated.

Hendren’s own study, involving twelve children, yielded minimal fluctuations in their conditions after a twelve-week leucovorin regimen. Recalling his time at UCSF and previous roles, Hendren shared that local parents of autistic children had encouraged him to explore every avenue for assistance, which included considering leucovorin alongside injectable vitamin B12.

Q&A with Robert Hendren: Insights on Leucovorin and Autism Research

What piqued your interest in leucovorin towards the end of your research career?

My initial interest stemmed from a suggestion by Richard Frye, who proposed applying for funding to investigate the effects of B12 and leucovorin. The grant was unsuccessful, but it opened doors for further contemplation. In our metabolomics studies, we focused on how various bodily pathways manage oxidative stress and inflammation, factors relevant to autism. Thus, we decided to conduct exploratory research into leucovorin’s effects.

Your study had a small participant group. What were your findings?

While the overall results did not indicate significant progress in speech or language skills among most participants, we did observe a small subgroup that demonstrated slight improvements. Earlier investigations by Frye had indicated a possibility of enhanced communication in specific individuals.

What kind of additional research is necessary to back Frye’s observations?

Future studies should start with assessing cerebral folate deficiency levels before administering leucovorin to evaluate its impact comprehensively. Most evidence suggests that even those with cerebral folate deficiency may only experience modest benefits from leucovorin or folinic acid. It’s essential to acknowledge that while leucovorin might aid in alleviating some symptoms, it is not a cure-all. Autism is complex, involving numerous unique genetic and environmental factors.

How did your findings influence parent perspectives on leucovorin use?

Post-study, very few families continued administering leucovorin. Out of approximately 60 families at Oak Hill School, only a handful still pursued the treatment, often voicing uncertainty about its effectiveness yet expressing a desire to try anything that might assist their child.

What drove your exploration into non-traditional autism treatments?

Parents at the MIND Institute highlighted the DAN (Defeat Autism Now) movement and urged me to learn from them. Although DAN had numerous anecdotal testimonials, the lack of rigorous scientific grounding made it challenging to validate their claims. Funding for studies on dietary supplements poses challenges, given pharmaceutical companies cannot patent these substances.

How did early interactions with the DAN community shape your views?

I was particularly impressed by Bernie Rimland, who emphasized that autism was not tied to maternal behavior, contrary to the prevailing beliefs of prior decades. I believe that understanding autism as a neurodevelopmental rather than purely cerebral disorder has spurred innovations in research and treatment.

As an expert in autism research, what are your thoughts on President Trump’s sweeping claims regarding Tylenol and autism?

While I appreciate their advocacy for autism awareness, I find President Trump’s statements regarding Tylenol’s role in autism misleading and not evidence-based. He inaccurately asserts that mothers should feel guilty for their choices during pregnancy when scientific evidence does not support a direct correlation. Addressing mothers’ health concerns, particularly during pregnancy, must prioritize scientific accuracy and empathy.

What is your stance on Robert F. Kennedy Jr.’s persistent claims linking vaccines to autism?

Despite finding him engaging and intelligent, he lacks a scientific foundation in his claims regarding vaccines. Such beliefs undermine the importance of sound research and risk reversing the progress we have made in understanding autism. The notion that one treatment, like leucovorin, could be a definitive remedy for autism is unrealistic, especially given the recent assurances that a comprehensive understanding of autism would be achieved within a specific timeline, which most experts deem implausible.

This rewritten article maintains the original structure, content, and references involved in discussions regarding leucovorin and its interactions with autism research. It is suited for seamless integration into a WordPress platform, preserving HTML formatting while ensuring the information is dynamically presented for readers.

See also  NIH Director Tries To Calm Health Researchers’ Nerves After Trump Directives
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