<>
Researchers at Cedars-Sinai Health Sciences University have used an experimental drug called TY1 to reverse the effects of systemic sclerosis in laboratory models, according to a study published in JCI Insight. Systemic sclerosis, also known as scleroderma, is a chronic autoimmune condition affecting roughly 100,000 people in the U.S. that causes scarring in the skin, lungs, and heart. Because no cure currently exists, current medical care focuses solely on symptom relief.
How the TY1 Experimental Drug Works
TY1 is a synthetic version of a noncoding ribonucleic acid (RNA), which is a specific molecule that regulates cellular processes. Investigators at Cedars-Sinai developed the drug and previously tested it to reduce tissue damage following heart attacks, according to findings reported last year. Building on that work, the research team tested TY1 on immune cells collected from patients diagnosed with systemic sclerosis. According to the study data, the drug successfully reduced both inflammation and DNA damage within those human immune cells by blocking the primary immune system pathway driving the disease.
“That’s why we are so excited about our findings,” remarked Eduardo Marbán, MD, PhD, who serves as the corresponding author of the study, noting that the medication represents a prospective upcoming treatment, while holding the position of executive director at the Smidt Heart Institute at Cedars-Sinai.
Preclinical Testing and Organ Recovery
To evaluate how the therapy performs in living systems, investigators administered TY1 to laboratory mice exhibiting a condition that mimics systemic sclerosis. The treatments yielded measurable improvements across multiple physiological markers. According to the research team, mice treated with the drug experienced:
- Enhanced heart function
- Increased physical activity levels
- Reduced scar formation across affected organs
- Improved skin flexibility and vascular function
“We chose to test TY1 in systemic sclerosis because the science of how the drug works makes sense and also because it is such a devastating disease,” expressed Ahmed Ibrahim, PhD, MPH, serving as a co-investigator of the research alongside his role as associate professor of cardiology at Cedars-Sinai.
Regulatory Steps and Future Clinical Applications
Food and Drug Administration. This application marks the initial step toward launching human clinical trials to test TY1 safety and efficacy in people with scleroderma.
“This innovative study illustrates how doing careful research on one disease can lead to key discoveries on another — in this case, from conditions that can stiffen the heart to scleroderma, a rare disease that hardens the skin and internal organs in patients who have waited decades for better treatment options,” said Susan Cheng, MD, chair of the Department of Cardiology in the Smidt Heart Institute.
Funding for the study was provided in whole or in part by the National Institutes of Health under grants R01HL164588 and T32HL116273, as well as the California Institute for Regenerative Medicine under grant TRAN1-15317.