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. Results from a new drug study are promising, for treatment and relief.
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 says, "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.
University of Utah Rheumatologist Dr. Tracy Frech recently finished a phase two trial of a drug called Anabasum. It doesn't suppress the immune system like other drugs, but improves tissue function.
Frech says, "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.
Buma says, "This tsunami that I felt had taken over my body was calm."
Frech says, "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 design of the next stage of the trial."
Carolyn now takes Anabasum as a regular medication. She has no side effects and says she continues to improve.
Phase three of the trial will include a larger group of patients and will probably measure additional outcomes like improved lung function.