Doctors are now warning about the rapid development of cheap ‘autism drugs’ to relieve specific symptoms.
The American Academy of Pediatrics (AAP) said Friday that it does not recommend routine use of leucovorin in children with autism.
The drug, which costs $2.50 per pill, is derived from folic acid, a nutrient prescribed to pregnant women to ensure proper brain development in the fetus.
Traditionally given to cancer patients since the 1950s to protect their cells from the damaging effects of chemotherapy, leucovorin has also been given off-label to children with autism.
In particular, it has been shown to improve the brain’s language pathways, allowing previously non-verbal children to speak for the first time when taken daily.
But the AAP warned against routine use because of “very limited evidence” that leucovorin’s benefits outweigh its potential risks and unclear data on dosing in children.
But neurologists who have studied leucovorin’s effects on autistic children told the Daily Mail that ‘the opposite is true’ and that leucovorin is ‘one of the safest drugs’ compared to other drugs given to autistic children, such as antipsychotics, which have many potential side effects.
The controversy comes as the FDA is in the process of approving leucovorin for children and adults with cerebral folate deficiency (CFD), which occurs when the brain is unable to absorb folic acid, a naturally occurring form of folate found in foods such as leafy greens.
The American Academy of Pediatrics has warned against the routine use of leucovorin, which has been proven to improve language skills in children with autism, citing ‘very limited evidence’ (file photo).
A recent study found that three out of four people with autism suffer from CFD, which can contribute to the language and behavioral problems seen in many autism cases.
Approval could take years if the FDA requires additional safety testing, but interest and demand have grown since health officials within the Trump administration endorsed the drug at a press conference last month.
Kristin Sohl, PhD, an autism expert at the University of Missouri who worked on the AAP’s guidelines, said: told Reuters There is ‘very limited evidence’ for the use of leucovorin for autism.
She said: ‘We don’t know for whom this drug works. We don’t know if it’s safe. We don’t know what dose to start with.
‘If this is something that can help autistic children develop, that would be great, but we also don’t want to throw things at someone because we don’t know what to do.’
Dr. Sol said that although demand for leucovorin care has increased, he has only prescribed leucovorin once.
Leucovorin is derived from folic acid (also known as vitamin B9), an essential nutrient that aids in DNA synthesis and helps replicate and repair cells throughout the body.
Folic acid, in its naturally occurring form, is found in foods such as leafy green vegetables, beans, and lentils.
Folic acid is a precursor to the DNA and RNA needed to replicate a cell’s genome (the set of instructions that help the cell function properly). This is why most prenatal vitamins contain folic acid.
Research on leucovorin dates back to the early 2000s. At the time, scientists Dr. Vincent Ramaekers, of the University of Liège in Belgium, and Dr. Edward Quadros, of the State University of New York Downstate, took blood and spinal fluid samples from children with autism and found that while their blood had normal levels of folate, their cerebrospinal fluid, which protects the brain and spinal cord, had much less.
This suggested that something was blocking folic acid uptake by the brain.
Researchers also found that 75 percent of children with autism have autoantibodies in their blood, which are types that mistarget and attack the body’s tissues and organs.
Nathaniel Schumann (left), 12, spoke only two to three words at a time before starting leucovorin. He can now speak in complete sentences. And 10-year-old Meghan Dumesnil (right) has shown ‘dramatic’ speech improvement since starting the medication.
These autoantibodies are known to block a protein called folate receptor alpha, a protein that transports folate from the blood to the brain and placenta.
Studies have shown that leucovorin can clear chemical blockages in the brains of children with autism, which helps improve language skills.
However, unlike other forms of folic acid, such as vitamin supplements, leucovorin can cross the blood-brain barrier (a semipermeable layer lining the inner surface of blood vessels in the brain that protects the brain from potentially harmful substances in the blood).
This means that it can penetrate the brain and have a direct effect.
The FDA’s move to approve leucovorin is based on an analysis of data from about 40 cases showing improvements in behaviors, such as language, in children with autism who took leucovorin.
Dr. Richard Frye, a pediatric neurologist at Rossignol Medical Center in Arizona, has seen nonverbal children on leucovorin not only begin speaking in full sentences in a matter of weeks, but also display less aggression and anxiety.
Dr. Richard Frye, a pediatric neurologist at Rossignol Medical Center in Arizona, told the Daily Mail that leucovorin is ‘one of the safest medications’ for children with autism.
The Daily Mail spoke to parents of several children who had previously been treated by Dr Frye and were prescribed leucovorin. A boy from Missouri could barely speak before taking leucovorin. Two weeks later he told his father for the first time that he loved him.
Another 12-year-old boy, about six weeks after starting Dr. Frye’s trial of leucovorin, went from saying only one or two pieces at a time to being able to tell his family in full sentences what was bothering him.
Dr Frye told the Daily Mail in response to the AAP’s announcement that ‘the facts are quite the opposite’ about leucovorin.
‘Leucovorin is one of the safest drugs available,’ he said. ‘Leucovorin was approved for use in the 50s to protect the body from the side effects of chemotherapy. So we have more than 70 years of experience in safety.’
He pointed out that the folic acid in leucovorin is water-soluble, so excess levels are excreted out of the body through urine, the ‘body’s natural safety mechanism’, and that there is no upper safety limit for synthetic folic acid like this one.
Autism experts told the Daily Mail that while leucovorin is safe and effective, it is not a cure for autism and children with the condition usually require additional treatment (stock image).
He compared it to other drugs used for autism: risperidone (Risperdal) and aripiprazole (Abilify), both of which are antipsychotics.
“These substances are known to cause weight gain, diabetes, and tardive dyskinesia (a neurological disorder that causes involuntary facial and jaw movements),” Dr. Frye said.
In addition to the 40 case studies cited by the FDA, Dr. Frye pointed to five case studies involving more than 250 children, ‘similar to the number of children used in registration studies to obtain FDA approval for currently approved treatments for autism.’
“In conclusion, leucovorin is much safer and more effective than the drugs currently approved for the treatment of autism,” he added.
However, he emphasized that leucovorin is not a cure for autism and often needs to be used in combination with other treatments and medications, such as risperidone and aripiprazole.
