Trump’s administration uses a medicine called leukovorin to reduce some of the symptoms of autism, but experts who are examining or treating autism almost equally agree that the medication should be further examined before it is released in children or adults.
Leucovorin, also known as folin Aid, is a synthetic form of vitamin B9 that requires a prescription. This is often prescribed for cancer patients in combination with chemotherapy in IV.
The Food and Drug Administration announced Monday that it confirms the tablet version to certain autism patients.
Many investigators questioned whether the confirmation was too early, given that only a few small tests – mostly in the US – pointed out that leucovorin was effective for children with autism.
Several NBC News experts have said that the FDA approval can give a false hope for families, as not all children with autism can get a prescription and, even if they do, the chances of seeing the results are unclear.
Scientists have been looking for a medicine for decades that can meaningfully reduce the symptoms of autism, but only a few match the strict limit of safety and efficiency, which is usually determined by the FDA. By Monday, the agency confirmed two drugs to treat autism irritability, but none of them were dealing with autism -related communication difficulties, social challenges or recurrent behavior.
“It is not the case that scientists have only been staring at their belly buttons for only 20 years, without looking at the treatment of autism. They have, but the standards were very high to confirm it,” said Alycia Halladay, a chief scientific officer of the Autism Science Foundation. Its organization, which funding evidence -based autism, does not recommend leukovorin as treatment and says more research is needed.
Leucovorin “has no criteria to meet the FDA approval, but nevertheless the administration still does it. So I wouldn’t call it a victory,” said Halladay.
She added that the way the medication was mentioned on Monday in the White House briefing – as a breakthrough for autism families – does not reflect the FDA approval details.
In a press release, the FDA said the drug was confirmed in patients with brain folate deficiency, a rare neurological condition characterized by low levels of vitamin B9 (folate) in the brain. Some researchers suspect that the condition is associated with autism, although not all autism patients have it.
(Halladay estimated about 10-30% of autism patients.)
Leucovorin can help folram achieve the brain, which in theory can improve oral communication or reduce the symptoms of autism such as irritability or recurrent behavior. However, there is no evidence that this completely eliminates the symptoms.
“I still hope that leucovorin can be a tool in our tool set, which can help a group of patients,” said Dr. Rachel Follmer, Associate Professor of Pediatrics at the Feinberg Medical School of Northwest University. “But I don’t know if we can still say that this will certainly help anyone with autism.”
President Donald Trump said at the briefing that the confirmation “gives hope to many parents with autistic children that it could be possible to improve their lives.” Medicare and Medicaid service center administrator Mehmet Oz called the action “rescue rescue”. And FDA Commissioner Marty Makary estimated that “I think hundreds of thousands of children will benefit.”
However, in a press release, the Department of Health and Human Services said that leucovorin “is not a remedy for autism” and “can only improve language -related deficiencies associated with a subgroup.”
David Mandell, a professor at the University of Pennsylvania, said the folate hypothesis is based on relatively weak science.
“We do not have good, large-scale studies that show that any large part of autism has a lack of folate,” he said.
Leukovorin has been shown to have relatively minimal side effects in cancer patients, although high doses can cause gastrointestinal problems or increase the risk of seizures in people taking medication. It is part of the general colorectal and other gastrointestinal cancer regime when used to enhance the effect of chemotherapy 5-fluorouracil. In rare cases, it is used to relieve the side effects of another chemotherapy called high doses of methotrexate.
Halladay said side effects were given in Leukovorin autism, but did not specifically assess the safety of the drug. According to her, the doses were also different from the study to the study, and some of the study participants received behavioral therapy, making it difficult to determine whether leukovorin is responsible for improving their symptoms.
Mandell stated that the biggest of those attempts were 80 participants – too small to get the final results.
“If you are looking for a FDA approval, for example, you want hundreds of children in these tests,” he said.
Leucovorin should clean the final obstacle until it is available to some autism patients. The FDA said she was working with GSK, a Leucovoror brand engine manufacturer, to update the drug label. GSK said it still needs to submit a drug application to include a new autism reference.
Oz said on Monday that Leucovori recipes will be awarded Medicaid and that private insurance companies are likely to follow the example.
But Mandell said she was worried that Medicaid’s reduction could force many families to pay out of pocket, and encourage some of the Forinic Acid Supplements online without a prescription. Internet supplements are less likely to have been tested in quality, and it can be difficult to confirm at what dose they are present.
Chief Research Officer of the American Cancer Society dr. William Dahut said interest in leukovorin on autism can also affect the availability of drugs in cancer patients.
“In the past, we have seen a shortage of this medicine and, if interest is increased, there may be a disadvantage in the future,” he said. In the letter.
This article was originally published in nbcnews.com