CellCept (mycophenolate mofetil) is an oral medication developed by Genentech (a member of the Roche group) that can improve lung function in people with scleroderma.
How CellCept works
Scleroderma is a condition where scar tissue develops in the skin and possibly in such organs as the heart, intestinal tract, and lungs. Scar tissue in the lungs can cause lung fibrosis, also known as interstitial lung disease (ILD), which can lead to breathing difficulties. Lung problems remain one of the biggest contributors to reduced quality of life and mortality in patients with scleroderma.
The development of scar tissue in scleroderma is thought to be partly due to inflammation because of an abnormally active immune system. CellCept is a medication that suppresses the immune system by stopping immune cells from multiplying. It is used off-label as a treatment for scleroderma.
CellCept in clinical trials for scleroderma
Several observational studies suggest that CellCept is effective in treating lung disease in people with scleroderma.
Researchers from the University of California recently conducted two studies that looked at the effect of different immune-suppressing medications for the treatment of ILD in scleroderma. The first of these studies, the Scleroderma Lung Study compared the effect of cyclophosphamide to placebo. Cyclophosphamide is another immune-suppressing therapy commonly used to treat ILD in scleroderma.
The Phase 2 (NCT00883129) Scleroderma Lung Study 2 compared CellCept to cyclophosphamide and placebo in treating ILD in scleroderma.
Researchers analyzed patients who received CellCept daily (at a maximum daily dose of 1.5 g) for two years in the second study and compared them to patients who received placebo in the first study.
They concluded that CellCept was able to significantly improve lung function in patients with scleroderma. Both groups of patients were of similar scleroderma subtype and had similar skin involvement. While the ages and baseline lung functions of the patients differed slightly between groups, researchers made adjustments for this during the comparisons.
Another Phase 3 clinical trial (NCT02896205) is investigating the effect of CellCept at a dose of up to 2 grams per day compared to placebo on lung function in people with scleroderma. The study is being carried out at the Postgraduate Institute of Medical Education and Research in Chandigarh, India. It was scheduled to be completed in July 2017, but results have not been made public.
Additional details
Besides lung function, CellCept may also improve skin symptoms in scleroderma.
Common side effects of CellCept include nausea, vomiting, diarrhea, swelling of the ankles and feet, and high blood pressure. CellCept also weakens the immune system in general, leaving patients at a higher risk of getting infections. While on CellCept, regular blood tests are necessary to watch for more serious side effects.
Women should avoid getting pregnant while on CellCept, as this medication increases the risk of miscarriage and birth deformities. Both men and women are advised to use reliable contraception while taking CellCept.
CellCept is also used to treat other conditions such as myasthenia gravis and to prevent organ transplant rejection.
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