NBC News
The Trump administration is promoting a medication called Leucovorina to reduce some symptoms of autism, but experts who investigate or treat autism almost unanimously coincide that the medicine must be studied more thoroughly before being administered to children or adults.
Leucovorina, also known as folinic acid, is a synthetic form of vitamin B9 that requires medical recipe. It is often administered to cancer patients along with chemotherapy intravenously.
The Food and Medicines Administration (FDA, in English) announced Monday that it is in the process of approved a table version for certain autism patients.
Many researchers questioned whether the approval was premature, since only a few small -scale essays – the majority made outside the United States – have demonstrated the efficacy of leukovorine in children with autism.
Several experts declared NBC News that FDA’s approval could give false hopes to families, since not all children with autism can opt for a recipe and, even if they do, the probability of obtaining results is uncertain.
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Scientists have been looking for a drug that can significantly reduce the symptoms of autism, but few have met the rigorous safety and efficiency requirements that FDA usually establishes. Before Monday, the agency had approved Two drugs to treat the irritability associated with autismbut none that will address communication difficulties, social challenges or repetitive behaviors related to the syndrome.
“It is not that scientists have spent 20 years looking at the navel without investigating the treatments for autism. They have done it, but the standards to obtain approval have been very strict,” said Alycia foundy, scientific director of the Autism Science Foundation. Its organization, which finances research on evidence -based autism, does not recommend leukovorine as a treatment and affirms that more studies are necessary.
The leucovorina “does not meet the criteria to obtain the approval of the FDA, but still this administration is doing anyway. Therefore, I would not say that this is precisely a victory,” said foundy.
He added that the way in which the medicine was promoted at a press conference held on Monday at the White House – such as a great advance for families with autistic children – does not reflect the details of the approval of the FDA.
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The FDA said in a press release that the medication has been approved for patients with folate brain deficiency, a rare neurological condition characterized by low levels of vitamin B9 (folate) in the brain. Some researchers suspect that this condition is related to autism, although not all patients with autism suffer.
(Foundy estimates that Between 10% and 30% of autism patients suffer from this condition).
Leucovorina can help the folate reach the brain, which in theory could improve verbal communication or reduce the symptoms of autism, such as irritability or repetitive behaviors. However, there is no evidence to eliminate symptoms completely.
“I still have the hope that leucovorina can be a tool in our arsenal that helps a group of patients,” said Dr. Rachel Follmer, an attached professor of pediatrics at the Faculty of Medicine of Feinberg of the Northwwestern University. “But I don’t know if we have yet reached the point where we can affirm that this will definitely help all people with autism.”
President Donald Trump said at the press conference that the approval “gives hope to many parents with autistic children that it is possible to improve their lives.” Mehmet Oz, administrator of the Medicare and Medicaid service centers, described the measure as “Salvadora”. The FDA Commissioner Marty Makary estimated that “in my opinion, hundreds of thousands of children will benefit.”
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However, in a press release after the press conference, the Department of Health and Human Services (DHS) said that leucovorina “It’s not a cure” For autism and “it can only lead to improvements in speech -related deficits in a subgroup of children.”
David Mandell, professor of psychiatry at the University of Pennsylvania, said that the folate hypothesis is based on relatively weak scientific data.
“We do not have large -scale reliable studies that demonstrate that a significant part of autistic people have folate deficiency,” he said.
It has been shown that leukovorine has relatively minimal side effects in cancer patients, although high doses can cause gastrointestinal problems or increase the risk of seizures in people who take anticonvulsive medicines. It is part of a common regime for colorectal cancer and other types of gastrointestinal cancer, where it is used to intensify the effect of the 5-fluorouracil chemotherapeutic drug. In more rare cases, it is used to mitigate the side effects of another chemotherapeutic drug called high dose metrexate.
Foundy affirmed that leukovorine trials for autism analyzed side effects, but did not specifically evaluate the security of the medication. The doses also differed from one study to another, he said, and some trial participants received behavioral therapy, which made it difficult to determine if the leukovorine was responsible for the improvement of their symptoms.
Mandell said that the greatest of these essays had 80 participants, A too small number To produce definitive results.
“If the approval of the FDA was sought, for example, hundreds of children in these trials would be needed,” he said.
Leucovorina must overcome a last obstacle before being available for some patients with autism. The FDA said that it is working with GSK, the manufacturer of the brand version of the Leucovorina, to update the medication label. GSK said in a statement that it is still necessary to submit a medicine application to include the new indication for autism.
Oz said Monday that Leucovorina recipes will be covered by Medicaid and that it is likely that private insurance companies will follow their example.
But Mandell commented that he worries that the cuts planned in Medicaid can force many families to pay from their pocket and take some to buy folinic acid supplements on the Internet without a medical recipe. It is less likely that internet supplements have been subjected to quality tests and it may be difficult to confirm the dose they contain.
Dr. William Dahut, scientific director of the American society against cancer, declared that it is possible that interest in leukovorine for autism may also affect the availability of the medication for cancer patients.
“We have seen shortage of this medicine in the past, and if the interest increases, It is possible that scarcity occurs in the future“He said in an email.
