WASHINGTON (AP) \u2014 When President Donald Trump\u2019s administration announced it would repurpose an old, generic drug as a new treatment for autism, it came as a surprise to many experts \u2014 including the physician who suggested the idea to the nation\u2019s top health officials.<\/p>\n
Dr. Richard Frye told The Associated Press that he\u2019d been talking with federal regulators about developing his own customized version of the drug for children with autism, assuming more research would be required.<\/p>\n
WATCH: Without new evidence, Trump and his health officials push unproven link between acetaminophen and autism<\/a><\/p>\n \u201cSo we were kinda surprised that they were just approving it right out of the gate without more studies or anything,\u201d said Frye, an Arizona-based child neurologist who has a book and online education business focused on the experimental treatment.<\/p>\n It\u2019s another example of the haphazard rollout of the Trump administration\u2019s Monday announcement on autism, which critics say has elevated an unproven drug that needs far more study before being approved as a credible treatment for the complex brain disorder.<\/p>\n READ MORE: WHO urges caution on autism and acetaminophen claims, saying several studies \u2018found no such relationship\u2019<\/a><\/p>\n A spokesperson for the Republican administration did not immediately respond to a request for comment Wednesday morning.<\/p>\n \u201cWe have nothing resembling even moderate evidence that leucovorin is an effective treatment for autism symptoms.\u201d<\/p>\n The nation\u2019s leading autism groups and researchers quickly distanced themselves from the decision on leucovorin, a derivative of vitamin B, calling the studies supporting its use \u201cvery weak\u201d and \u201dvery small.\u201d<\/p>\n \u201cWe have nothing resembling even moderate evidence that leucovorin is an effective treatment for autism symptoms,\u201d said David Mandell, a psychiatrist at the University of Pennsylvania.<\/p>\n WATCH: Health experts respond to Trump\u2019s claims linking autism to acetaminophen<\/a><\/p>\n Mandell and other researchers say the evidence suggests autism is mostly rooted in genetics, with input from other factors, including the age of the child\u2019s father.<\/p>\n Nevertheless, a growing number of doctors are prescribing the medication, repurposing versions used for chemotherapy or ordering new formulations from compounding pharmacies.<\/p>\n Many researchers agree the drug warrants additional study, particularly for patients with a deficiency of folate, or vitamin B9, in the brain that may play a role in autism. But for now, they say, it should only be taken in carefully controlled clinical trials.<\/p>\n READ MORE: Research doesn\u2019t show using Tylenol during pregnancy causes autism. Here are 5 things to know<\/a><\/p>\n \u201cWe often say our job is to stay between the yellow lines,\u201d said Dr. Lawrence Gray, a pediatric developmental specialist at Northwestern University. \u201cWhen people just decide to go outside of current guidelines, then they\u2019re outside of that. And nobody knows what\u2019s going to happen out there.\u201d<\/p>\n The evidence for leucovorin isn\u2019t settled<\/p>\n The case for leucovorin\u2019s use in autism begins with established science but quickly veers into uncertain terrain.<\/p>\n When metabolized, the drug turns into folate, which is essential for healthy prenatal development and is recommended before and during pregnancy. But far less is known about its role after birth.<\/p>\n READ MORE: Research doesn\u2019t show using Tylenol during pregnancy causes autism. Here are 5 things to know<\/a><\/p>\n The issue caught the attention of Frye and others more than 20 years ago, when research suggested some people with autism had low levels of folate in the brain due to antibodies blocking the vitamin\u2019s absorption.<\/p>\n The theory linking autism to folate levels was mostly abandoned, however, after research showed that the siblings of people with autism can also have low folates without any symptoms of the condition.<\/p>\n \u201cI honestly thought this had died out as a theory for autism and was shocked to see its reemergence,\u201d Mandell said.<\/p>\n READ MORE: Here\u2019s what we know about the causes of autism<\/a><\/p>\n In 2018, Frye and his colleagues published a study of 48 children in which those taking leucovorin performed better on several language measures than those taking a placebo.<\/p>\n Four small studies in other countries, including China and Iran, showed similar results, albeit using different doses, metrics and statistical analyses, which researchers say is problematic.<\/p>\n Frye struggled to get funding to continue within the traditional academic system.<\/p>\n WATCH: How RFK Jr. is shaping the conversation about autism and why advocates are pushing back<\/a><\/p>\n \u201cI decided to move out of academia to be more innovative and actually do some of this stuff,\u201d he said.<\/p>\n Researchers saw an opening to approach Trump\u2019s top health officials<\/p>\n Earlier this year, Frye and several other researchers formed a new entity, the Autism Discovery Coalition, to pitch their work to Trump administration officials including Health Secretary Robert F. Kennedy Jr.<\/p>\n \u201cAfter Kennedy got in, we thought they\u2019d hopefully be friendly to autism scientists,\u201d he said.<\/p>\n When metabolized, the drug turns into folate, which is essential for healthy prenatal development.<\/p>\n An August meeting with National Institutes of Health Director Jay Bhattacharya quickly led to further discussions with the Food and Drug Administration about testing a proprietary, purified version of leucovorin.<\/p>\n