Designer receptor therapy long used in the lab crosses over into humans

Key Insights

- In a first-in-human trial, researchers found that chemogenetic therapy appears safe in several people with epilepsy uncontrolled by medication.

- The study was not built to test efficacy, but the therapy may have reduced people’s seizures as long as they stayed on a companion drug.

- Researchers are excited about the possibility of changing the activity of specific circuits in an adult’s brain.

A chemogenetic therapy has shown promise for treating epilepsy that does not respond to drug treatment (New Engl. J. Med. 2026 DOI: 10.1056/NEJMc2606815). The three-patient study is the first time that researchers have used designer receptors, commonly referred to as DREADDs, to change the activity of an adult’s brain.

The trial is one of at least seven testing DREADDs in humans in China, news of which stunned researchers at a US National Institutes of Health Brain Research through Advancing Innovative Neurotechnologies (BRAIN) Initiative meeting in August. Although the new results come from a pilot study rather than a formal clinical trial, neuroscientists call its hints at safety and efficacy exciting.