WASHINGTON (AP) – When President Donald Trump’s administration announces it Reuse old common drugs As a new treatment for autism, it was Surprising to many experts – Includes doctors who proposed ideas to the country’s best health authorities.
Dr. Richard Fry told The Associated Press that he was talking to federal regulators about developing his own customized version of the drug. Children with autismsuppose that more research is needed.
“So I was kind of surprised they approved it from the gate without doing any further research or doing anything,” said Frye, an Arizona-based child neurologist who has a book and online education business focusing on experimental treatments.
This is another example of the Trump administration’s accidental development on Monday Autism announcementcritics say it raised unproven drugs that require far more research before it was approved as a reliable treatment for complex brain diseases.
A spokesman for the Republican administration did not immediately respond to a request for comment Wednesday morning.
The country’s leading autism groups and researchers quickly distance themselves from the decision to leucovorin, a derivative of vitamin B, and call its use “very weak” and “very small.”
“We’ve seen a lot of people who have had a lot of trouble with us,” said David Mandel, a psychiatrist at the University of Pennsylvania.
Mandel and other researchers say the evidence suggests Autism is primarily rooted in geneticshas opinions from other factors, including the age of the child’s father.
Nevertheless, more and more doctors are prescribing drug therapy, reusable versions, which are used for chemotherapy, and ordering new formulations from pharmacies.
Many researchers agree that the drugs will guarantee additional research, especially for patients with folic acid deficiency or vitamin B9 deficiency in the brain, which may play a role in autism. But for now, they say it should only be taken in carefully controlled clinical trials.
“We often say that our job is to stay between the yellow lines,” said Dr. Lawrence Gray, a pediatric development expert at Northwestern University. “When people decide to go outside the current guidelines, they’re outside of it, and no one knows what’s going to happen there.”
Evidence of leucovorin has not been resolved
The use of leucovorin in autism starts with established science, but soon becomes uncertain terrain.
When metabolized, the drug is converted to folic acid. This is essential for healthy prenatal development and is recommended before and during pregnancy. However, it is not known to be much less of its role after birth.
This issue attracted the attention of Fry and others over 20 years ago. This suggests that some people with autism have low levels of folic acid in their brains due to antibodies that block the absorption of vitamins.
The theory that links autism and folic acid levels has been largely abandoned, but after studies have shown that siblings of people with autism can have low lobes without symptoms of the condition.
“I honestly thought this had disappeared as an autism theory and I was shocked to see it reappearance,” Mandel said.
In 2018, Frye and his colleagues published a study of 48 children who performed better on several language measures in children taking leucovorin than those taking placebo.
Four small studies in other countries, including China and Iran, showed similar results despite using a variety of doses, indicators and statistical analyses, but researchers say it is problematic.
Fry struggled to obtain funding to continue within the traditional academic system.
“I decided to move out of academia to become more innovative and actually do this,” he said.
Researchers saw an opening to approach Trump’s best health authorities
Earlier this year, Fry and several other researchers formed a new entity, the Autism Discovery Coalition, pitching their work to Trump administration officials. Health Secretary Robert F. Kennedy Jr.
“After Kennedy came in, we hoped they were friendly with autistic scientists,” he said.
August meeting with the Director of the National Health Bureau Jay Battacharya It quickly led to further discussion Food and Drug Administration About testing a proprietary refined version of Leucovorin.
A new formulation of the drug decades ago means a new patent, allowing Fry and his yet-to-formed pharmaceutical company to charge far more than the cheap generics currently on the market.
“We have a lot of investors who are excited about Leukovorin and want to do something high quality for children with autism,” he said.
However, the FDA announcement on Monday may have given up on that plan. Instead of previewing the new version, the agency said it would simply update the labels for common drugs to refer to its use in improving folate brain levels, including those with autism. It is expected that more doctors will prescribe it and encourage insurance companies to cover it.
Promising autism treatments often fail after more research
Experts who have spent decades treating autistic patients say it’s important to proceed carefully and systematically.
Gray recalls other experimental treatments that initially looked promising by failing in large-scale studies.
“Small studies often find highly motivated groups,” Gray said. “But when these treatments are moved to larger studies, the initial positive findings often fade away.”
Of the challenges facing leucovorin, there is no consensus on which parts of autistic patients carry folate blocking antibodies targeted by the drug.
Fry screens patients for antibodies using tests developed in the lab at State University of New York. Like many specialized tests, they have not been reviewed by the FDA.
Gray says the only way to clearly test antibodies is to extract cranial fluid from children with autism through a spinal tap.
“This is a huge limiting factor in running these large randomized controlled trials,” Gray said.
Online sources promote interest from parents
The Trump administration has debated on rapid pursuit leucovorin, but interest in the drug continues to swirl online, including forums and social media groups. Parents of children with autism.
Bryan Noonan of Phoenix discovered the drug earlier this year after asking ChatGpt for the best autism medication options for his 4-year-old son.
The FDA has never approved the drug for the underlying cause of autism, but the chatbot has directed Noonan to Frye’s study.
After evaluation and confirmation blood tests, the boy began preparing the drug from the compounding pharmacy in June.
Within a few days, Noonan says he saw an improvement in his son’s ability to make eye contact and form sentences.
“He’s not cured, but these are just areas of improvement,” Noonan said. “It was a big deal for us.”
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