Imagine your skin and your internal organs slowly thickening and hardening, and there’s no cure. About 100,000 Americans, most of them women, have been diagnosed with systemic sclerosis, according to the Scleroderma Foundation. Results from a new drug study are promising for treatment and relief from this rare autoimmune disease of the connective tissue.
Carolyn Buma is happy she’s able to take 16-year-old Sophie for a walk. She’d been losing mobility to systemic sclerosis.
Buma detailed, “Every morning, you wake up seeing what things you could do, what things you couldn’t do, how far your hands were turning inward. It’s a very scary thing.”
Systemic sclerosis is when the body overproduces collagen, causing scar tissue to form in the skin, muscles, joints, and internal organs. It also constricts blood flow, causing Raynaud’s syndrome in most patients. Tracy Frech, M.D., a rheumatologist at the University of Utah recently finished a Phase II trial of a drug called Anabasum. It doesn’t suppress the immune system like other drugs, but improves tissue function.
Dr. Frech explained, “Not only does it have anti-fibrotic effects, or scarring effects but also probably improves profusion, or blood flow to the different organs.”
Patients reported renewed skin tone and feeling and sleeping better.
“This tsunami that I felt had taken over my body was calm,” Buma said.
Dr. Frech said, “While the skin was the primary thing that we’re saying was the success with the drug there, lots of other information is captured, and that can help inform the next stage of the trial.”
Buma now takes Anabasum as a regular medication. She has no side effects and said she continues to improve.
The University of Utah is one of nine research hospitals in the U.S participating in this trial. Phase III will include a larger group of patients and will probably measure additional outcomes like improved lung function. Phase three begins within six months.