SALT LAKE CITY, Utah. (Ivanhoe Newswire) — 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 told Ivanhoe.
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.
Contributors to this news report include: Cyndy McGrath Supervising Producer; Wendy Chioji, Field Producer; Milvionne Chery, Assistant Producer; Roque Correa, Editor; Jason Ball, Videographer.
BACKGROUND: Systemic sclerosis (SS) is an autoimmune disorder where healthy tissue is destroyed because the immune system mistakenly thinks it’s a foreign substance or infection. SS is characterized by changes in the texture and appearance of the skin. This is due to increased collagen production. The skin thickens and hardens; other symptoms include hair loss, calcium deposits, or white lumps under the skin, small, dilated blood vessels under the skin’s surface, joint pain, shortness of breath, a dry cough, diarrhea, constipation, difficulty swallowing, esophageal reflux, and abdominal bloating after meals. The exact cause of SS is unknown but it is known that women are more likely to develop it. There is no cure, but results from a new drug study are promising. (Source: http://www.healthline.com/health/scleroderma#Overview1)
TREATMENT: Anabasum is a synthetic oral endocannabinoid-mimetic drug that specifically binds to the CB2 receptor expressed on activated immune cells and fibroblasts. In November 2016, Corbus Pharmaceuticals reported positive data results from a Phase 2 study in systemic sclerosis, showing clear signal of clinical benefit with anabasum. Pre-clinical and Phase 1 clinical studies (123 subjects) have shown anabasum to have a favorable safety and tolerability, profile. It has also demonstrated promising potency in pre-clinical models of inflammation and fibrosis. Anabasum triggers a process responsible for the resolution of inflammation and fibrosis, while reducing production of pro-inflammatory molecules. Anabasum is currently being evaluated in four separate Phase 2 clinical trials for the treatment of cystic fibrosis, diffuse cutaneous systemic sclerosis, and dermatomyositis and systemic lupus erythematosus.
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.
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