Does the Unproven Autism Drug Backed by Trump Really Work?

"According to Martin Makary, head of the US Food and Drug Administration (FDA), the drug leucovorin will help hundreds of thousands of kids with autism. But a day after Makary praised leucovorin's powers at a White House event, some specialists are warning that the science to warrant Makary's enthusiasm is far from solid. Those researchers say that the drug's efficacy has not been established, that scientists don't know how much of the drug to give or how people should take it, and that safety data in children are lacking."

"According to the FDA's current plans, leucovorin will be available to only a minority of autistic people. All of this has led to widespread confusion, say clinicians, who also worry about the expectations created by Makary and other officials in the administration of US president Donald Trump."

"I've heard from a lot of families, says psychologist Catherine Lord at the University of California, Los Angeles. The major thing they say is, What is this? What do we do?' I don't want to get everyone's hopes up that this is a magic cure, says Rebecca Schmidt, a molecular epidemiologist at the University of California, Davis. It's not for everybody."

"At an announcement on 22 September, Makary announced the upcoming approval of leucovorin, a form of the vitamin folate, by saying it would open the door to the first FDA-recognized treatment pathway for autism. People with low levels of folate in the protective fluid surrounding the brain and spine can sometimes exhibit traits associated with autism, including challenges in social communication. This condition, called cerebral folate deficiency, could be due to rogue antibodies that attack the body's own proteins in t"

Martin Makary, head of the US Food and Drug Administration, announced that leucovorin will help hundreds of thousands of children with autism. Some specialists say the supporting science is weak and efficacy has not been established. Researchers report unknown dosing, unclear administration routes, and insufficient pediatric safety data. FDA plans indicate that leucovorin will be accessible to only a minority of autistic people. Clinicians report widespread confusion and worry that official statements have created unrealistic expectations. Families are seeking guidance about whether and how to use the drug. Leucovorin is a form of folate linked to cerebral folate deficiency and possible autoimmune antibodies.

Read at www.nature.com