CellCept (mycophenolate mofetil) is a medication marketed by Genentech and approved to prevent organ rejection in transplant patients (liver, kidneys and heart) by working to suppress the immune system.

As an immunosuppressive, CellCept is also prescribed to treat such autoimmune and inflammatory diseases as rheumatoid arthritis and lupus nephritis (lupus that affects the kidneys). It has also been investigated as a potential alternative to prednisone, a glucocorticoid, for the treatment of sarcoidosis.

Glucocorticoids like prednisone are often used as a first-line treatment for active sarcoidosis, and prednisone is FDA-approved  to treat pulmonary sarcoidosis. However, such corticosteroid treatments can have severe side effects, especially with long-term use, and many patients either do not tolerate or respond to them.

Mycophenolate mofetil, the main ingredient of CellCept, is one among the non-steroid medications being considered as an alternative to prednisone in treating sarcoidosis.

How CellCept works

CellCept is a precursor of mycophenolic acid (MPA), an inhibitor of a protein called inosine monophosphate dehydrogenase (IMPDH) 2, which is involved in the production of guanosine nucleotides. Guanosine nucleotides have various functions in the cell, but mainly act as signaling molecules. Lymphocytes, a type of immune cell, are most affected by CellCept because other cell types use a slightly different protein to produce guanosine nucleotides called IMPDH 1 and are less sensitive to CellCept.

CellCept in clinical trials

CellCept has not been tested in randomized clinical trials as a sarcoidosis treatment. But several small studies suggest it may be beneficial to patients with this disease.

One study retrospectively looked at 37 pulmonary sarcoidosis patients who either did not respond to or tolerate other immunosuppressive agents. It reported that mycophenolate mofetil treatment enabled these patients to reduce the dose of prednisone required to control their disease symptoms of the disease, but overall did not show signs of improving lung function. In a subgroup analysis, however, patients intolerant of other immunosuppressives showed “some improvement” in forced vital capacity (FVC) and diffusing capacity for carbon monoxide (DLCO), parameters of lung function, when treated with mycophenolate mofetil. Mycophenolate mofetil “appears to have potential as a second line agent for some pulmonary sarcoidosis subjects,” the researchers wrote. CellCept provided no benefit in patients who had not responded to a prior agent that suppresses the immune system.

Another retrospective study suggested that mycophenolate mofetil is beneficial in combination with prednisone (or other corticosteroids) in patients with chronic pulmonary sarcoidosis. Its data showed a mean FVC improvement of 8.5 percent in 10 treated patients, who were also able to significantly lower to less than 10 mg/day corticosteroid doses used to maintain stable disease or improve lung symptoms.

case study found mycophenolate mofetil treatment to be beneficial to an array of  patients with cutaneous, central nervous system, and renal sarcoidosis.

Another case study of a 41-year-old man with cutaneous and bone marrow sarcoidosis found he responded to a corticosteroid-sparing treatment combination of mycophenolate mofetil and methotrexate.

Additional information 

CellCept is toxic at high doses and has to be dosed carefully. Neutropenia, or abnormally low levels of white blood cells called neutrophils, is a known side effect but one less pronounced than with other agents that suppress the immune system. Other side effects include diarrhea and nausea, which can be dose limiting.

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