Alemtuzumab is a therapy being tested as a possible treatment for the symptoms of scleroderma that works by targeting the immune response.

Genzyme, a Sanofi company, produces alemtuzumab. The therapy is approved to treat multiple sclerosis (under the brand name Lemtrada) and chronic lymphocytic leukemia (marketed as Campath in the U.S., and MabCampath in Europe).

How alemtuzumab works

Scleroderma is an autoimmune condition. Its symptoms are thought to be caused by the immune system mistakenly attacking healthy cells and tissues. This triggers inflammation and a chronic wound response, leading to the overproduction of the protein collagen, a key component of fibrosis or scarring.

Alemtuzumab is a type of therapy that alters the immune response, destroying immune cells thought to be involved in the abnormal response seen in scleroderma. It is a type of antibody, a protein designed to interact with a specific target, that binds to a protein called CD52.

CD52 is found on the surface of mature lymphocytes, or immune cells like T-cells and B-cells. When alemtuzumab binds to CD52, it marks that cell for destruction. As it should only affect mature lymphocytes and not the stem cells they develop from, once the therapy is stopped the patient should be able to produce new lymphocytes.

By destroying the immune cells potentially responsible for triggering an overly aggressive immune response, it is theorized that alemtuzumab could reduce or prevent the damage caused by scleroderma.

Alemtuzumab in clinical trials

A study published in 1996 in the Journal of Rheumatology reported data from a single patient, suggesting that alemtuzumab  treatment may improve skin symptoms in scleroderma.

A pilot Phase 1/2 proof-of-concept clinical trial (NCT01639573) is currently recruiting up to three children, ages 8 to 18, with drug-resistant systemic scleroderma at Children’s Hospital, Los Angeles. The trial will monitor patients for up to two years to determine whether alemtuzumab is safe, and whether prolonged administration can achieve immunoablation or the complete removal of the patient’s immune response. This study is set to conclude in January 2020.

Other information

Alemtuzumab is administered as an injection into a vein.

As it suppresses the immune system, patients taking alemtuzumab have an increased susceptibility to infections.

Common side effects of alemtuzumab are headaches, rashes, fever, nausea, thyroid problems, infections, low white blood cell counts, musculoskeletal pain, and changes in blood pressure or heart rate.

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