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What is Autism? Will Leucovorin become a new treatment? Here's what experts say

Posted By: Aditya Gogoi Posted On: Sep 23, 2025Share Article
The U.S. Food and Drug Administration (FDA) approved the drug leucovorin on Monday for use in treating autism. President Donald Trump has recently made a controversial announcement of promoting an unproven connection between autism and the painkiller Tylenol.

What is Autism? Will Leucovorin become a new treatment as Donald Trump links Tylenol to Autism | Explained

The U.S. Food and Drug Administration (FDA) approved the drug leucovorin on Monday for use in treating autism. President Donald Trump has recently made a controversial announcement of promoting an unproven connection between autism and the painkiller Tylenol.

Some smaller studies have connected these issues with more severe autism symptoms and indicate that treatment with leucovorin might help improve verbal and social skills, as well as reduce irritability in these children.

Recent studies have indicated that leucovorin may help alleviate certain autism symptoms in specific children. However, experts in the field warned that larger and more rigorous clinical trials are needed before drawing firm conclusions.

Also Read: Trump claims Tylenol use during pregnancy linked to autism, suggests Leucovorin as treatment

Trump took a more aggressive stance than his own Food and Drug Administration, which had issued cautious guidance advising doctors to “consider minimising” the use of acetaminophen during pregnancy due to inconclusive evidence about a potential link to autism.

Trump's remarks coincided with his administration's push to expand access to a potential, though unproven, treatment for autism, as well as a broader commitment to increase research into the disorder.

Health Secretary Robert F. Kennedy Jr. promised earlier this year to determine the cause of autism by September. That baffles brain experts, who say there is no single cause and that the rhetoric appears to ignore decades of scientific research into the genetic and environmental factors that can play a role.

What is autism?

Autism isn't a disease.

Until the 1990s, autism diagnoses were mostly limited to children with the most severe symptoms. According to the World Health Organisation (WHO), autism spectrum disorders (ASD) are a range of conditions marked by varying levels of difficulty with social interaction and communication.

“They are also often associated with unusual behaviours and activity patterns, such as challenges in shifting between tasks, intense focus on specific details, and unique responses to sensory experiences,” said the WHO.

The increase isn't among the profound cases; it's an increase in the milder cases that weren't historically considered autism, said expert Helen Tager-Flusberg of Boston University, as reported by AP.

Autism rates have been going up for decades

WHO noted that in 2021, it was estimated that around 1 in 127 people globally were affected by autism. This figure is an average, and prevalence rates can vary widely depending on the study. Some well-designed studies have reported significantly higher rates.

However, in many low- and middle-income countries, the true prevalence of autism remains unknown due to limited data.

Genetics plays a major role in Autism, says expert

Research has shown that genetics plays a major role in autism, with scientists identifying hundreds of genes linked to the condition. These genes can be inherited, even if the parent doesn't show any signs of autism, or they can arise from spontaneous mutations during early brain development, when rapidly dividing cells may make errors.

Also Read: Virtually no autism as… Donald Trump uses Cuba, Amish examples to justify Tylenol warning

Experts believe that various combinations of genetic and environmental factors can influence how the fetal brain develops. These may include the father's age, preterm birth, and certain maternal health conditions during pregnancy — such as fever, infections, or diabetes, AP report.

What about Tylenol?

Some research has suggested a potential link between acetaminophen (a common over-the-counter pain reliever) taken during pregnancy and a higher risk of autism. However, many other studies have found no such connection.

The Coalition of Autism Scientists noted on Monday that acetaminophen use in pregnancy has not increased in recent decades, while autism rates have continued to rise, casting further doubt on any direct link.

At the same time, the Society for Maternal-Fetal Medicine warns that untreated fevers during pregnancy—especially in the first trimester—can raise the risk of complications such as miscarriage, preterm birth, and other health issues.

Also Read: Trump administration plans to link autism in children to Tylenol, warn pregnant women against use: Report

On Monday, the FDA issued guidance to doctors recommending they limit the use of acetaminophen during pregnancy, while also acknowledging the ongoing uncertainty. The agency stated, “To be clear, while many studies have reported an association between acetaminophen and autism, a causal relationship has not been established, and there are also studies that contradict this finding.”

Tylenol's existing label already advises pregnant women to consult their doctor before using the medication.

Meanwhile, the Society for Maternal-Fetal Medicine maintains that acetaminophen remains a safe and appropriate option for use during pregnancy.

What is the possible new autism treatment?

According to a notice in the Federal Register, the FDA authorised leucovorin, a concentrated form of folic acid, which is typically recommended to pregnant women to support healthy fetal development.

What is Leucovorin?

Leucovorin, also called folinic acid, is a type of folate, which is an essential B vitamin (B9). The U.S. Food and Drug Administration has approved leucovorin to counteract the toxic effects of certain chemotherapy drugs, such as methotrexate, which interfere with the body's ability to use folate, Reuters reported.

Also Read: How autistic creators are using art to reclaim their personal narratives

Leucovorin is also used to boost the effectiveness of other chemotherapy treatments and to manage certain kinds of anaemia. It can be administered either orally or through intravenous injection.

According to the FDA website, more than six companies manufacture leucovorin.

How is Leucovorin different?

Folate helps cells grow and divide. It also supports the immune system and helps produce healthy red blood cells. In developing fetuses, inadequate folate leads to a type of serious birth defect called neural tube defects.

Research from SUNY Downstate Medical Center in Brooklyn, New York, and other institutions suggests that up to 75% of children with autism may have genetic variations that affect their ability to process folate or have autoimmune disorders that hinder folate transport to the brain, Reuters reported.

Also Read: How Emirates is making air travel easier for people with Autism

“Women already are told to take folic acid before conception and during pregnancy because it reduces the chances of certain birth defects, advice that possibly could help lower autism risk as well,” Tager-Flusberg said.

Leucovorin is sold for other health conditions and is already used by some families in hopes of helping autism. But Tager-Flusberg cautioned that only a few small, first-step studies have been done so far.

“Is this something worth pursuing? Yes, it is potentially in a subset of individuals,” she said. But there needs to be a large, very rigorous study to prove if it really works.

Scientists and leading autism advocacy groups have firmly debunked any concerns linking vaccines to autism.

Childhood vaccines undergo extensive testing to ensure safety, including studies on how and when multiple vaccines are given together, and their safety is continuously monitored for years after approval.

“No doubt children will suffer” from Monday's claims, said Dr. Paul Offit, a pediatrician and vaccine expert at the Children's Hospital of Philadelphia.

Trump administration under pressure

The Trump administration is facing significant pressure from Health Secretary Robert F. Kennedy Jr.'s broad Make America Healthy Again movement to address the rising number of autism diagnoses in the U.S. in recent years.

According to experts, this increase is largely due to an expanded definition of autism—which now includes milder, spectrum-based cases—and improvements in diagnostic practices, AP reported.

Also Read: Trump says $10 drug to cost $20 after ‘1,000% price cut': Bizarre claim baffles netizens — ‘Is that the right math?'

They emphasise that autism has no single cause and warn that the current rhetoric overlooks decades of scientific research into the genetic and environmental factors involved.

In addition to budget cuts at federal health agencies, the CDC has been internally divided over Kennedy's controversial vaccine stance. Just last week, a key immunisation panel—now including several vaccine sceptics appointed by Kennedy—revised its guidance on COVID-19 and other vaccinations.

(With inputs from Associated Press, Reuters)

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