At The University of Texas Health Science Center at Houston (UTHealth), rheumatologist Shervin Assassi, M.D., is developing a blood test to determine which drugs work best for scleroderma patients with lung disease.
Assassi, whose work is supported with a $1.4 million Department of Defense medical research grant, is testing biomarkers that predict medication response in patients with scleroderma, an autoimmune disease affecting about 130,000 people in the United States.
Scleroderma patients often develop interstitial lung disease (also called pulmonary fibrosis) and have difficulty breathing and performing everyday tasks, said Assassi, an associate professor at McGovern Medical School at UTHealth.
"Medications that generally dampen the immune response such as cyclophosphamide or mycophenolate mofetil are used to treat scleroderma-related interstitial lung disease," he said. "However, patients respond very differently to these medications. Some show significant improvement in their lung function while others will continue to experience worsening disease, despite treatment."
Because these medications can have life-threatening side effects, doctors need to know ahead of time if they are going to work, Assassi said.
The research project capitalizes on the valuable biospecimens and clinical information collected in the recently completed Scleroderma Lung Study II, he said. This randomized controlled study showed that both cyclophosphamide and mycophenolate mofetil were modestly effective in the overall group.
However, the treatment effect was highly variable. The new project will link the molecular data generated in the biospecimens in the Scleroderma Lung Study II to lung outcomes to develop prediction models that identify patients who are likely to respond to immunosuppression.
"This can ultimately lead to more effective and safer treatment strategies for persons with scleroderma-related interstitial lung disease," he said.
Assassi's collaborators include David Elashoff, Ph.D., and Donald Tashkin, M.D., at the University of California, Los Angeles, and Jeffrey Browning, Ph.D., at Boston University.