Repurposing tocilizumab in Scleroderma patients may prevent early lung disease

April 2, 2021

Research led by Michigan Medicine’s Scleroderma Program and published in Arthritis & Rheumatology found that tocilizumab, an anti-inflammatory drug used to combat rheumatoid arthritis, can prevent lung disease in patients with systemic sclerosis if detected early enough in the disease course, according to a news release.

Systemic sclerosis is an autoimmune disease and the most serious form of scleroderma, the tightening and thickening of the skin. It can affect internal organs and lung disease is its leading cause of death, according to study author Dinesh Khanna, MBBS, MSc, director of Michigan Medicine’s Scleroderma Program.

“Some people have minimal lung disease; some people have life-threatening disease. The amount of lung scarring plays a major factor in those health outcomes,” says Khanna. “When used in patients early in their disease course, those that usually have had the disease five years or less, our study found that tocilizumab preserved lung function over the course of 48 weeks.”

When left undiagnosed and untreated, patients with systemic sclerosis can suffer a rapid decline in lung function. And unfortunately, this lung disease is irreversible.

Khanna’s research, referred to as the focuSSced trial, was a phase 3, randomized placebo-controlled trial seeking to understand the impact of tocilizumab on lung function preservation in patients with mild, moderate and severe amounts of lung scarring.

The research team found that of the 210 trial participants, tocilizumab treatment over the course of 48 weeks, compared to the placebo, stabilized forced vital capacity, which is the total amount of air exhaled during forced breathing.

The FDA just recently approving tocilizumab for slowing the rate of decline in lung function in adult patients with systemic sclerosis-associated interstitial lung disease. However, more research is needed to better understand this complex illness.

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