With original findings reported in Nature Communications, a newly tested compound, RU.521, may provide the key to understanding and combating autoimmune conditions such as Aicardi-Goutieres Syndrome, and Lupus.
Cyclic GMP-AMP synthase, also known as cGAS, work like an alarm system in the body. It is one of several natural enzymes that detects and notifies our immune systems of invaders. In certain cases, however, the production of cGAS can cause the body to attack itself, leading to severe inflammation. By preventing the release of cGAS, researchers hope to be able to reduce and limit these symptoms. RU.521 appears to be a promising piece of this puzzle.
Though currently only tested in mice, the results of the study are positive. If nothing else, they will assist in the understanding of how our bodies battle invading organisms, and sometimes our own. Researchers tested over 1000 chemical compounds, and discovered four that target and inhibit the production of the cGAS enzyme. Due to its specific feature and effectiveness, researchers continued to study the compound designated RU.521.
Normally, when the body detects damage, or foreign organisms, macrophages are on the scene to clean it up. In simplest terms, macrophages eat dangerous, and damaged cells. The enzymes cGAS, and interferon, aid the macrophages in detecting and signaling their targets. When these enzymes mistakenly point out healthy cells, or respond too greatly to cells in our own bodies, the results can be anythign from uncomfortable to life-threatening.
In order to test RU.521, researchers exposed macrophages to damaged DNA molecules Left to their own devices, this encounter would trigger production of cGAS and interferon. The production of these enzymes would only serve to exacerbate the problem. When in the presence of RU.521, however, no interferon was produced, and researchers were able to determine that cGAS had been inhibited to produce this result.
In the aforementioned animal test groups, models were created to replicate Aicardi-Goutieres Syndrome. Both Lupus, and Aicardi Syndrome present autoimmune disorders and challenges, among other similarities in symptoms. The results of the animal study confirmed the earlier findings form the in vitro research. RU.521 seems to powerfully and precisely reduce cGAS and interferon production.
RU.521 is not a cure for autoimmune conditions, but researchers are hopeful that it will provide a firm foundation for our future understanding of diseased like Lupus, and Aicardi Syndrome. With continued research it may yet become a crucial ingredient to more effective therapies.