Identifying New Therapeutic Options for Treatment of Scleroderma

A recent study lead by Northwestern Medicine scientists has shown potential leads on new treatments for scleroderma, a chronic, connective tissue disease with symptoms that can range from mild to life threatening.

The health issue is estimated to affect about 300,000 Americans, in which approximately one-third of patients have the systemic form of the disease. This can make the health issue particularly difficult to diagnose and treat. Fibrosis, or scarring, known as a hallmark of the disease, can create progressive tightening of the skin and lungs that can cause serious organ damage, and in some cases, even death.

Fortunately, researchers believe they've discovered some new therapeutic options that involve the identification of a specific protein and its role in fibrosis.

"Our results show how a damage-associated protein called fibronectin (FnEDA) might trigger immune responses that convert normal tissue repair into chronic fibrosis in people with scleroderma," said lead study author Swati Bhattacharyya, Ph.D., and research assistant professor in Medicine-Rheumatology, via a press release. "We also found that FnEDA, which is undetectable in healthy adults, was markedly increased in the skin biopsies of patients with scleroderma."

Though the factors responsible for fibrosis in scleroderma remain relatively unknown, the disease remains one with a high mortality rate and few effective treatments.

To examine the connection between immunity and fibrosis in scleroderma, scientists studied skin biopsies of scleroderma patients to better identify responsible factors for persistent scarring. Findings showed that FnEDA was highly elevated.

Researchers next used genetically engineered mice lacking the protein. They discovered that these mice did not develop the skin fibrosis. Yet FnEDA triggered an immune response in skin cells that caused prevention of the skin fibrosis in the mice.

While the study focused primarily on scleroderma, certain uncovered mechanisms may reveal future information regarding pulmonary fibrosis and liver cirrhosis.

"This pioneering study using state of the art experimental approaches is the first to identify an innate immune pathway for scleroderma fibrosis," Dr. Varga added, via the release. "We expect that the results will shift our thinking about the disease, and hopefully open new avenues for its treatment."

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More information regarding the findings can be seen via the journal Science Translational Medicine.