By Julie Steenhuysen
CHICAGO (Reuters) – President Donald Trump is using an old generic drug called leacovorin to treat mild anxiety disorders caused by parents’ cravings for their children.
That has been a challenge for pediatricians and experts who caution that the science on leucovorin in people with autism is limited and does not support widespread use.
In the month since Food and Drug Administration Commissioner Marty Mazaks promoted the decades-old drug from GSK, saying it could help “hundreds of thousands” of children with autism, doctors and researchers say they have been bombarded by parents seeking information.
“My Facebook feed is full of parents swearing that leucovorin works,” said Dr. David Mandell, a professor of psychology and autism researcher at the University of Pennsylvania.
Lack of information
Tens of thousands of people have joined a Facebook group called Leucovorin for Autism started in May by Keith Joyce, legal guardian to four-year-old Jose, who is taking the drug.
Joyce attributes Jose’s improvements in verbal communication and social awareness to Leucovorin. The site gained 5,000 members on the day of Trump and Mary’s White House announcement and now has 84,000.
Mandell and other doctors say that the FDA’s production without requiring large clinical trials leaves the application coaches dealing with emotional requests that are not in families while there is a lack of information.
Leucovorin is approved to treat the side effects of chemothethepy but can be off-labeled for autism symptoms.
“It puts doctors in a very difficult position because they are being asked to provide something that has no evidence,” said Dr. Shafali Jeste, an autism specialist at UCLA, who refuses to grant repeated requests despite repeated requests.
On Friday, the American Academy of Pediatrics said it does not recommend leucovorin for routine use in children with autism.
Autism rates have risen to 1 in 31, the US Centers for Disease Control and Prevention reported in April. Finding the root causes of conditions and possible treatments is what Secretary of Health Robert F. Kennedy Jr. and Trump have been proud of it.
A common neurological condition
Despite the broad comments of the FDA, his agency proposes that several extended approvals are combined with cerebral palsy (CFD), a genetic condition), a genetic condition that can cause autism-like symptoms. It affects about one in 1 million children worldwide.
The argument for widespread use is based on small studies that suggest many children with autism have autoantibodies that block folate – an important vitamin for entering the brain, causing brain deficits, causing cognitive deficits, causing cognitive deficits, causing cognitive deficits, causing cognitive deficits, causing cognitive deficits, causing CFD deficits.
It is estimated that 75% of Autistic children are believed to have these autoantibodies, but their importance is not clear, said Dr. Karam Radwan, director of the Neurodevelopmental Program at the University of Chicago.
A spokesperson for Health and Human Services said the FDA based its plan to review the leucovorin label to include CFD in an analysis of 40 studies published from 2009-2024. Overall, 85% of patients experienced benefits including improved speech/communication skills.
The HHS said leucovorin may be useful in children with autoantibodies to digest it, but noted that “data are limited and need to be replicated.”
A spokesperson said the country’s health agencies will support follow-up research on the effect of leucovorin on CFD, and any possible link to people with autism. Post-Market Surveillance and Safety Studies are part of the plan.
Parents see the green light
Parents interpreted the FDA announcement as a green light for the use of Leucovorin in Autism, sharing medical advice that led Facebook to create Joyce’s site. It was reinstating the ban on illegal chats, only to have it lifted again last week for further violations.
Joyce began researching the drug after watching a news program about an autistic boy in therapy showing marked improvement in speech.
He found just three studies, all by the same author, that he saw as reliable. No large trials in autistic children comparing leucovorin to placebo have been conducted.
Joyce reviewed the DRUCE record of Ract in cancer patients, where Leucovorin has been linked to insomnia, aging and depression. In children, doctors and parents say it can lead to hyperactivity, aggression and frustration.
Jose’s neurologist was aghast when he prescribed leucovorin, but the well-mannered pediatrician, who heard about the problem at a conference, was determined.
The child’s care team measured language skills before treatment and after four months, Joyce said.
He is more aware of the world around him, and more responsive, Joyce said. “It didn’t cure his autism, but it improved his health. I’m sure it’s real.”
Radwan, who gives medicine in its operation, says Botholdomdomni is not completely understood who can benefit, how much and how much it helps. So far, profits are “pretty modest,” he said.
Parents in some online communities want recognition.
Sophia urwin, 33, a single parent from Wellington, New Zealand, whose four-year-old, non-verbal son will be diagnosed with autism in 2022, is worried about desperate parents turning to folinic acid versions.
“It’s really easy to get carried away thinking something is a miracle ‘cure’ when you’ve been on ice for so long,” she said.
(Reporting by Julie Steenhuysen; Editing by Caroline Humer and Bill Berkrot)
