China Launches 7 Human Chemogenetic Trials for Brain Disorders, Using DREADDs Activated by Clozapine
Updated
Updated · Chemical & Engineering News · Aug 14
China Launches 7 Human Chemogenetic Trials for Brain Disorders, Using DREADDs Activated by Clozapine
1 articles · Updated · Chemical & Engineering News · Aug 14
Summary
At least seven Chinese clinical trials are testing chemogenetic therapies in humans for intractable epilepsy, Parkinson’s disease and neuropathic pain—the first known clinical use of DREADD technology.
The approach inserts a designer receptor gene into targeted neurons via viral vectors, then uses clozapine to selectively suppress those cells and their circuits, potentially with fewer off-target effects than conventional drugs.
Three trials use adeno-associated virus delivery, bringing gene-therapy risks including severe immune reactions; several recent deaths in early-stage gene-therapy studies in China underscore the safety stakes.
Bryan Roth, who developed DREADDs 20 years ago, said six studies appear to have started months after the first epilepsy trial, suggesting investigators may already have seen early signs the approach was tolerable.
If successful, the trials could offer an alternative to brain-removal surgery in some severe cases and open a path to circuit-based treatments for a far wider range of neuropsychiatric diseases.