The Trump administration is hailing a medication called leucovorin for reducing some autism symptoms, but experts who research or treat autism almost uniformly agree the medication should be studied further before it\u2019s rolled out to children or adults.<\/p>\n\n\n\n
Leucovorin, also referred to as folinic aid, is a synthetic form of vitamin B9 that requires a prescription. It\u2019s often administered to cancer patients alongside chemotherapy in the form of an IV.<\/p>\n\n\n\n
The Food and Drug Administration announced Monday that it’s in the process of approving a tablet version for certain autism patients.<\/p>\n\n\n\n
Many researchers questioned whether the approval was premature, given that only a handful of small trials \u2014 mostly conducted outside the U.S. \u2014 have pointed to leucovorin being effective in children with autism.<\/p>\n\n\n\n
Several experts told NBC News that the FDA\u2019s approval could give false hope to families, since not all children with autism may qualify for a prescription and, even if they do, the likelihood of seeing results is uncertain.<\/p>\n\n\n\n
Scientists have searched for decades for a drug that can meaningfully reduce autism symptoms, but few have met the rigorous threshold for safety and effectiveness that\u2019s typically set by the FDA. Before Monday, the agency had approved two drugs to treat irritability associated with autism, but none that addressed autism-related communication difficulties, social challenges or repetitive behaviors.<\/p>\n\n\n\n
\u201cIt\u2019s not like scientists have just been staring at their belly buttons for 20 years, not looking at autism treatments. They have, but the standards have been very high to get it approved,\u201d said Alycia Halladay, chief science officer at the Autism Science Foundation. Her organization, which funds evidence-based autism research, does not recommend leucovorin as a treatment and says more studies are needed.<\/p>\n\n\n\n
Leucovorin \u201cdoesn\u2019t have the criteria to meet FDA approval, but yet this administration is just doing it anyway. So I wouldn\u2019t exactly call this a win,\u201d Halladay said.<\/p>\n\n\n\n
She added that the way the medication was touted at a White House briefing on Monday \u2014 as a breakthrough for autism families \u2014 doesn\u2019t reflect the details of the FDA\u2019s approval.<\/p>\n\n\n\n
The FDA said in a news release that the drug is being approved for patients with cerebral folate deficiency, a rare neurological condition characterized by low levels of vitamin B9 (folate) in the brain. Some researchers suspect the condition is associated with autism, though not all autism patients have it.<\/p>\n\n\n\n
(Halladay estimated 10%-30% of autism patients have the condition.)<\/p>\n\n\n\n
Leucovorin can help folate reach the brain, which in theory might improve verbal communication or reduce autism symptoms like irritability or repetitive behaviors. But there\u2019s no evidence that it eliminates symptoms altogether.<\/p>\n\n\n\n
\u201cI still remain hopeful that leucovorin may be a tool in our toolbox that can help a group of patients,\u201d said Dr. Rachel Follmer, an assistant professor of pediatrics at Northwestern University Feinberg School of Medicine. \u201cBut I don\u2019t know if we\u2019re at the point yet where we can say that this is definitely going to help all individuals with autism.\u201d<\/p>\n\n\n\n
President Donald Trump said at the briefing that the approval \u201cgives hope to the many parents with autistic children that it may be possible to improve their lives.\u201d Mehmet Oz, administrator of the Centers for Medicare and Medicaid Services, called the action \u201clife-saving.\u201d And FDA Commissioner Marty Makary estimated that \u201chundreds of thousands of kids, in my opinion, will benefit.\u201d<\/p>\n\n\n\n
However, in a news release following the briefing, the Department of Health and Human Services said leucovorin \u201cis not a cure\u201d for autism and \u201cmay only lead to improvements in speech-related deficits for a subset of children.\u201d<\/p>\n\n\n\n
David Mandell, a psychiatry professor at the University of Pennsylvania, said the folate hypothesis is based on relatively weak science.<\/p>\n\n\n\n
\u201cWe do not have good, large-scale studies that show that any significant portion of autistic people have a folate deficiency,\u201d he said.<\/p>\n\n\n\n
Leucovorin has been shown to have relatively minimal side effects in cancer patients, though high doses can cause gastrointestinal problems or increase the risk of seizures in people taking anti-seizure medications. It\u2019s part of a common regimen for colorectal and other gastrointestinal cancers, where it\u2019s used to intensify the effect of the chemotherapy drug 5-fluorouracil. In rarer cases, it\u2019s used to mitigate the side effects of another chemo drug called high-dose methotrexate.<\/p>\n\n\n\n
Halladay said leucovorin trials for autism looked at side effects, but didn\u2019t specifically evaluate the medication\u2019s safety. Dosages also differed from study to study, she said, and some trial participants received behavioral therapy, making it hard to determine if leucovorin was responsible for improving their symptoms.<\/p>\n\n\n\n
Mandell said the largest of those trials had 80 participants \u2014 too small to produce definitive results.<\/p>\n\n\n\n
\u201cIf you were looking for an FDA approval, for example, you\u2019d want hundreds of kids in these trials,\u201d he said.<\/p>\n\n\n\n
Leucovorin must clear a final hurdle before it\u2019s available to some autism patients. The FDA said it is working with GSK, the maker of the brand name version of leucovorin, to update the drug\u2019s label. GSK said in a statement that a drug application still needs to be submitted to include the new indication for autism.<\/p>\n\n\n\n
Oz said Monday that leucovorin prescriptions will be covered by Medicaid and that private insurance companies are likely to follow suit.<\/p>\n\n\n\n
But Mandell said he worries that anticipated cuts to Medicaid could force many families to pay out of pocket, and drive some to purchase folinic acid supplements online without a prescription. Online supplements are less likely to have been quality tested and it can be hard to confirm what dosage they contain.<\/p>\n\n\n\n
Dr. William Dahut, chief scientific officer of the American Cancer Society, said it\u2019s possible that interest in leucovorin for autism could also impact the drug\u2019s availability for cancer patients.<\/p>\n\n\n\n
\u201cWe have seen shortages of this drug in the past, and if there is increased interest, shortages may occur in the future,\u201d he said in an email.<\/p>\n\n\n\n