Cedars-Sinai researchers use experimental drug to reverse systemic sclerosis effects

Researchers at Cedars-Sinai Health Sciences University have used an experimental drug to reverse the effects of systemic sclerosis, a life-threatening autoimmune disease also known as scleroderma. The study, published in JCI Insight, focused on a synthetic noncoding ribonucleic acid (RNA) molecule called TY1.

In laboratory tests using immune cells from patients, TY1 reduced inflammation and DNA damage. When administered to mice with conditions mimicking systemic sclerosis, the drug improved heart function and physical activity while reducing organ scarring. Systemic sclerosis affects approximately 100,000 people in the United States, causing scarring in the skin, lungs, heart, and other organs. Currently, there is no known cure, and treatments are limited to symptom relief. Investigators are now preparing an investigational new drug application.