It is a derivative of the natural product anabaseine that acts as a partial agonist at neural nicotinic acetylcholine receptors (nAChRs).

[1][2] Activation of the α7 nAChR has been shown to have neuroprotective effects and to improve cognitive function, making it an attractive target for drug development.

Both GTS-21 itself and its demethylated active metabolite 4-OH-GTS-21[3] display nootropic[4] and neuroprotective effects,[5][6][7][8] and GTS-21 is being investigated for the treatment of Alzheimer's disease,[9][10] nicotine dependence,[11] and, most significantly, for schizophrenia.

A recent study investigated the cholinergic anti-inflammatory pathway (CAP) in rheumatoid arthritis (RA).

They used the α7 nicotinic acetylcholine receptor (α7nAChR) agonist GTS-21 to study its role in reducing synovial inflammation in a mice model of collagen-induced arthritis (CIA).

[18] Several other trials focusing on a range of health issues including Alzheimer's, schizophrenia, autism, ADHD, and nicotine use were either discontinued or withdrawn.

In contrast, db/db mice treated with GTS-21 demonstrate significant reductions in plasma BUN and creatinine compared with db/db controls (P < 0.05 vs. db/db vehicle).

However, more research is needed to fully understand its safety and efficacy, and to determine the optimal dosing and administration regimens.