Chemogenetic brain therapies enter the clinic as China leads DREADD trials

US researchers at a NIH BRAIN Initiative meeting were stunned to learn that seven Chinese trials are testing DREADDs—designer receptors activated by a designer drug—in humans to suppress targeted brain activity for conditions like epilepsy, Parkinson’s disease, and neuropathic pain. The approach uses viral delivery to express receptors in specific neurons; when the drug is administered, activity in those circuits diminishes. While potentially offering circuit-specific therapies with fewer off-target effects, gene-therapy vectors carry immune risks. A trigeminal neuropathic pain trial has the most potential, and success could open circuit-based neurotherapies for many neuropsychiatric diseases. The pace suggests China is leading in this frontier.