Trump team backs an unproven drug for autism - but does it work?

"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."

"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 this case, proteins that ensure import of folate into the brain."

Leucovorin, a form of folate, is being proposed as a potential treatment for autism and described as possibly helping many children. Clinical evidence remains limited because trials have been small and efficacy has not been firmly established. Optimal dosing, administration methods and long-term safety data for children are unclear. Cerebral folate deficiency, in which antibodies impair folate import into the brain, can produce autism-like social-communication challenges and may respond to folinic acid in some cases. Planned FDA availability would cover only a minority of autistic people, and clinicians warn against portraying leucovorin as a universal cure.

Read at Nature