Experimental Drug Appears to Ease Scleroderma, Doctor Says

An experimental drug for scleroderma appears to reverse the crippling, disfiguring tissue disease, which afflicts about 400,000 Americans and kills rapidly in severe cases.

Relaxin, made by Palo Alto-based Connectics Corp., has promise both for controlling little-understood scleroderma and for helping researchers battle other autoimmune diseases, said Dr. James R. Seibold of the University of Medicine and Dentistry of New Jersey-Robert Wood Johnson Medical School.

“I think the way the scientific community looks at it is, if we make a breakthrough in scleroderma, it will open the doors in many other diseases,” such as rheumatoid arthritis and lupus, Seibold said Friday.

The school is one of 13 U.S. sites where 120 patients have received relaxin, also called ConXn, according to John L. Higgins, chief financial officer at Connectics, a 5-year-old biotechnology company developing rheumatology and dermatology medications.

Scleroderma primarily strikes women. It causes certain cells to produce excess collagen, making scar tissue build up and thickening and hardening the skin. That makes fingers clumsy and joints so tight and sore that patients increasingly have trouble feeding, bathing and dressing themselves, let alone working. In about 70,000 Americans, it spreads through the body, hardening vital organs and often killing within five years.

Although existing medications ease pain and control complications of scleroderma, relaxin is the only drug being developed to reverse the disease, according to Karl Kastrof, executive director of the Scleroderma Foundation, which is “quietly optimistic” about the drug.

Relaxin, administered via a stomach pump worn on a belt, decreases collagen production and accelerates breakdown of the structural protein.

In the most recent research phase, 70% of those getting a low dose saw a significant decrease in skin scarring and an increase in ability to function--as did about half as many patients getting a placebo.

Independent researchers said that the drug, a genetically engineered version of a hormone prevalent in pregnant women, shows promise but that further research is needed.