SALT LAKE CITY – Imagine your skin and internal organs slowly thickening and hardening and there is no cure.
100,000 Americans, mostly women, have Systemic Sclerosis.
"Every morning, you wake up seeing what things you could do, what things you couldn't do, how far your hands were turning inward," says patient Carolyn Buma.
"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.
It doesn't suppress the immune system like other drugs, but improves tissue function.
"Not only does it have anti-fibrotic effects, or scarring effects but also probably improves profusion, or blood flow to the different organs," said Dr. Frech.
Patients reported renewed skin tone and feeling and sleeping better.
"This tsunami that I felt had taken over my body was calm," said Buma.
"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," says Dr. Frech.
Carolyn now takes Anabasum as a regular medication. She has no side effects and says she continues to improve.
Phase 3 tests will include a larger group of patients and will probably measure additional outcomes like improved lung function. That phase of tests begins within six months.
OPTIONS: If left untreated, systemic sclerosis can be fatal within a few years, but there are treatment options that slow progression. They include blood pressure medication, medication to aid breathing, physical therapy, light therapy, such as ultraviolet A1 phototherapy, and nitroglycerin ointment to treat localized areas of tightening of the skin.
If this story has impacted your life or prompted you or someone you know to seek or change treatments, please let us know by contacting Jim Mertens at jim.mertens @wqad.com or Marjorie Bekaert Thomas at firstname.lastname@example.org.