Actemra (tocilizumab), a medication produced by Genentech, is approved by the U.S. Food and Drug Administration (FDA) to treat rheumatoid arthritis. It is being investigated as a potential treatment for systemic scleroderma.

Systemic scleroderma is a rare autoimmune condition characterized by the thickening, hardening, and scarring of the skin. It is caused by the accumulation of excess collagen in the skin and internal organs, such as the heart, lungs, kidneys, and intestinal tract, leading to organ damage.

How Actemra works

Actemra works by directly blocking the action of a protein called interleukin-6 (IL-6). Interleukins are proteins produced by white blood cells to regulate the immune response.

IL-6 plays a major role in the disease process of systemic scleroderma. Found at high levels in blood and skin lesions of patients with this condition, it appears to be associated with greater disease activity and higher mortality.

By preventing the activation of the immune system via the inhibition of IL-6, Actemra may stop or reduce the excessive production of scar tissue in systemic scleroderma.

Clinical trials for systemic scleroderma

A Phase 2/3 clinical trial (NCT01532869) investigated the efficacy and safety of tocilizumab in 87 patients with systemic scleroderma. During its first part, patients were randomized to weekly injections of tocilizumab at 162 mg or a placebo for 48 weeks. During the trial’s second, open-label period, all patients received 162 mg of tocilizumab as injections under the skin for an additional 48 weeks.

Results from the first 48 weeks showed mild improvements in skin symptoms (the primary trial outcome) in treated patients  compared to those who received placebo, although these changes were not significant. There was also some evidence of better lung function in those taking tocilizumab. But no differences between the two groups were seen in terms of disability, itching, fatigue, or overall disease severity.

More than 90 percent of patients in both groups also had side effects, with those given tocilizumab experiencing more incidences of serious infections compared to placebo (16 percent versus 5 percent) and one death in the tocilizumab group reported to be in relation to the treatment. Results from the first part of the trial, which ended in 2015, were published in The Lancet.

Data from weeks 48 to 96, a longer-term observation, also found that those taking tocilizumab were more likely to develop serious infections but no new side effects were observed. Results from this open-label part of the trial — also showing improved skin scores and stabilized lung function in people moving from placebo to tocilizumab treatment — were published in the Annals of Rheumatic Diseases. Among patients continuing on treatment, a maintenance of benefits was observed.

A randomized, double-blind, and global Phase 3 trial (NCT02453256) of tocilizumab in more than 200 patients with systemic scleroderma began in 2015 and is ongoing. Like the earlier trial, this study consists of an initial 48-week period, where patients receive either weekly injections of tocilizumab (162 mg) under the skin or a placebo, followed by an open-label period —  weeks 48 to 96 — where all are given the tocilizumab regimen. Researchers aim to assess any changes in skin symptoms, lung function, and other disease-related laboratory parameters.

The trial is being carried out at 106 locations worldwide and is expected to be completed in February 2019.

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