The Trump administration welcomes the medicine called Leikorin to reduce some symptoms of autism, but experts who examine or treat autism almost evenly agree that medicines should be studied further before it is deployed to children or adults.
Leikovorin, also called folinic acid, is a synthetic form of vitamin B9, which requires a prescription. It is often administered to patients with cancer along with chemotherapy in the form of IV.
On Monday, the Office for Products and Drug Control announced that it is in the process of approving the version of the tablet for certain patients with autism.
Many researchers asked whether there was a premature approval, given that only a few small tests – mainly conducted outside the United States – indicated that leikovorin was effective in children with autism.
Several experts informed NBC News that FDA approval could give false hope to families, since not all children with autism can apply for the recipe, and even if they do it, the probability of seeing the results are unclear.
Scientists have been looking for decades for drugs that could it is significant to reduce autism symptomsBut few have reached a strict threshold for safety and efficiency, which is usually installed by FDA. Until Monday, the agency approved two drugs for the treatment of irritability associated with autism, but not one of which concerned the difficulties associated with autism about communication, social problems or repeating behavior.
“This is not that scientists have just looked at their navel for 20 years without looking at autism treatment. They have, but the standards were very high to approve it, ”said Alisia Halladya, chief director of autism science. Its organization, which finances the actual data from the study of autism, does not recommend Leikororin as treatment And he says that additional studies are needed.
Leikovorin “There are no criteria for satisfying the approval of FDA, but still this administration simply does it in any case. Therefore, I would definitely not call it a victory, ”Halladia said.
She added that the way the medicine was advertised at the White House briefing on Monday – as a breakthrough for families with autism – does not reflect the details of the approval of the FDA.
FDA said that in the press, the drug is approved for patients with a deficiency of folic cerebral folate, a rare neurological state characterized by a low level of vitamin B9 (folate) in the brain. Some researchers suspect that the condition is associated with autism, although not all patients with autism have.
(Halladya said that some scientists believe that about 10% to 30% of patients with autism have a condition, although this speed can be much lower.)
Leukorin can help the folate reach the brain, which theoretically can improve oral communication or reduce the symptoms of autism, such as irritability or repeating behavior. But there is no evidence that this completely eliminates the symptoms.
“I still hope that Leikovorin can be an instrument in our tool set that can help a group of patients,” said Dr. Rachel Fallmer, associate professor of the Pediatrics Department at the North -Western University of Finberg. “But I don’t know if we are in a place where we can say that this will definitely help all people with autism.”
President Donald Trump said at a briefing that approval “gives hope to many parents with autistic children, which can improve their lives.” Mehmet Oz, the administrator of the services of Medicare and Medicaid, called the action of “salvation of life.” And the FDA Commissioner Marty Makari calculated that “hundreds of thousands of children, in my opinion, will win.”
However, in a press release, after a briefing, the Ministry of Health and Social Services stated that Leikovorin “is not a medicine” from autism and “can only lead to an improvement in the deficit related to speech, for the subsidence of children.”
David Mandell, a professor of psychiatry at the University of Pennsylvania, said that the folate hypothesis was based on relatively weak science.
“We have no good, large-scale studies that show that any significant part of autistic people has a deficiency of folate,” he said.
It was shown that Leikovorin has relatively minimal side effects in patients with cancer, although high doses can cause problems with the gastrointestinal tract or increase the risk of cramps in people taking anti-seeson drugs. It is part of the general regime for colorectal and other gastrointestinal crayfish, where it is used to enhance the effect of a 5-fluururatsil chemotherapeutic drug. In more rare cases, it is used to mitigate the side effects of another chemotherapy called methotrexate with high doses.
Halladya said that Leikovorin’s tests were examined by side effects, but did not specifically evaluate the safety of the medicine. According to her, the dosage also differed from the study to the study, and some participants in the study received behavioral therapy, which complicates the determination of whether Leikorin was responsible for improving their symptoms.
Mandell said that in the largest of these tests there were 80 participants – too small to get certain results.
“If you were looking for the approval of FDA, for example, you would like hundreds of children in these trials,” he said.
Leikovorin must remove the final obstacle before it is available to some patients with autism. FDA said she was working with GSK, a brand manufacturer, Leikovorina, to update the drug label. GSK stated in his statement that drugs about drugs have yet to be submitted in order to include a new indication of autism.
On Monday, Lake said that Leikovorina would be covered with Medicaid and that private insurance companies will probably follow an example.
But Mandell said that he was worried that the expectation of a Medicaid reduction can force many families to pay from a pocket and make some buy additives from folinic acid on the Internet without a recipe. Online duties are less likely to be checked by quality, and it may be difficult to confirm what dosage they contain.
Dr. William Dahut, the main researcher at the American Oncology Society, said that it is quite possible that interest in leakovorin for autism can also affect the accessibility of the drug for cancer patients.
“In the past, we saw the lack of this drug, and if in the future there may be a shortage in the future,” he said in an email.
