CellCept (mycophenolate mofetil) is an oral immune-suppressing treatment developed by Genentech (a member of the Roche group), commonly used to treat patients with myasthenia gravis.

How CellCept works

Myasthenia gravis is an autoimmune condition in which the immune system mistakenly produces antibodies (proteins that are part of the immune system and are usually made to attack viruses and bacteria) that destroy acetylcholine receptors found on muscle cells, leading to muscle weakness.

CellCept works by stopping these antibodies from forming. It directly interferes with DNA replication in immune cells, which slows down the formation of new cells. CellCept reduces the levels of antibodies that attack acetylcholine receptors, thereby improving symptoms of muscle weakness.

CellCept in clinical trials for myasthenia gravis

A small study has shown that more than half of myasthenia gravis patients responded well to CellCept.

But not all trials have been successful in demonstrating the short-term benefits of this medication. A Phase 3 clinical trial (NCT00285350) analyzed 80 patients with myasthenia gravis who received 20 mg a day of corticosteroids in addition to either 2.5 g a day of CellCept or placebo over three months. The results did not show any difference between patients receiving CellCept with corticosteroids and patients receiving corticosteroids only.

A separate Phase 3 trial (NCT00683969) in 176 patients who took corticosteroids for at least a month prior to receiving either 2 g of CellCept a day or a placebo for nine months also did not find any difference between the two groups of patients.

However, another study found that 80 percent of myasthenia gravis patients on CellCept achieved good outcomes after taking the medication long-term (at least two years). A majority of these patients also managed to reduce the amount of corticosteroids that they needed to control their symptoms.

Further details

Improvements in myasthenia symptoms usually are only experienced a few months to a year after starting CellCept.

Common side effects of  CellCept are stomach pain, diarrhea, vomiting, lower limb swelling, and high blood pressure. Patients also have a higher risk of serious infections, as CellCept weakens the overall immune system.  Regular blood tests are required so that a doctor can monitor the patient for serious side effects.

Women who are pregnant or are planning to conceive are at higher risks of miscarriage or of having a child with birth defects if they are taking CellCept.

While the evidence for miscarriage and birth defects resulting from future fathers taking this medication is not as strong, men using CellCept are still advised to use reliable contraceptive methods.

In addition to myasthenia gravis, CellCept is used to prevent organ rejection after organ transplantation.

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