At a recent BRAIN 전문가 회의 in Bethesda, Bryan Roth of UNC School of Medicine revealed that seven clinical trials in China are testing chemogenetic therapies in humans. Roth, who pioneered the DREADDs technology two decades ago, explained that the approach uses a designer receptor that responds to the antipsychotic drug clozapine with picomolar affinity. Introducing the receptor gene via a viral vector lets researchers silence specific neuronal populations when the drug is administered.

Dirk Trauner of the University of Pennsylvania highlighted DREADDs as a “more precise knife,” potentially reducing off‑target effects seen with conventional small‑molecule treatments. Although these tools have long been staples in basic neuroscience, they have not yet entered clinical practice.

The Chinese trials target intractable epilepsy, Parkinson’s disease, and neuropathic pain, offering a less invasive alternative to brain surgery. While gene‑therapy vectors carry immune‑reaction risks, the staggered start of studies suggests early safety signals.

If the trigeminal pain trial succeeds, it could pioneer circuit‑based treatments for a wide range of neuropsychiatric disorders.