Chemogenetics, a technique to remotely control neurons, enters human clinical trials in China

Chemogenetics Enters Human Trials

Chemogenetics, a technique enabling remote control of specific neurons using inert drugs, has advanced to human clinical trials. Bryan Roth, one of the technique's inventors, disclosed on August 13th that seven such trials are underway in China. This marks a notable progression for the technology from basic neuroscience research to potential human application.

How Chemogenetics Works

Chemogenetics, including DREADDs (designer receptors exclusively activated by designer drugs), involves inserting a synthetic receptor gene into neurons. Once expressed, these neurons can be excited or inhibited by a specific designer chemical. This allows for genetically targeted therapy where the effect can be controlled and titrated by adjusting the dosage of the chemical, similar in concept to optogenetics which uses light.

Implications for Neurological Disorders

The entry of chemogenetics into human trials suggests potential new treatment avenues for various neurological and neurodegenerative conditions. Conditions such as epilepsy, Parkinson's disease, and chronic pain are cited as areas where this technology could offer therapeutic benefits. This adds to a growing array of advanced tools for affecting the human brain, beyond traditional small molecules or electromagnetism.

Advancements in Neurological Therapies

The field of neurological treatment has expanded significantly, moving beyond older methods like small molecules and electromagnetism. Newer approaches include peptides, antibodies, focused ultrasound, and neuron-engineering therapies such as optogenetics (currently limited to the retina), gene-editing, and cell therapies. Chemogenetics represents a distinct category among these, as it involves directly changing or adding neurons, often irreversibly, for therapeutic effect.