Also Known As: Mycophenolate Mofetil, Cellcept
Mycophenolate mofetil (MMF) (brand names CellCept, Myfortic) is an immunosuppressant and prodrug of mycophenolic acid, used extensively in transplant medicine. It is a reversible inhibitor of inosine monophosphate dehydrogenase (IMPDH) in purine biosynthesis, to be specific guanine synthesis, which is necessary for the growth of T cells and B cells. Other cells are able to recover purines via a separate, scavenger, pathway and are, thus, able to escape the effect.
In general, mycophenolate is used for the prevention of organ transplant rejection. Mycophenolate mofetil is indicated for the prevention of organ transplant rejection in adults and renal transplant rejection in children over 2 years; whereas mycophenolate sodium is indicated for the prevention of renal transplant rejection in adults. Mycophenolate sodium has also been used for the prevention of rejection in liver, heart, and/or lung transplants in children older than two years.
An immunosuppressant that has drastically decreased the incidence of acute rejection in solid transplant recipients, mycophenolate is increasingly utilized as a steroid sparing treatment in immune-mediated disorders including immunoglobulin A nephropathy, small vessel vasculitides, and psoriasis.
Its increasing application in treating lupus nephritis has demonstrated more frequent complete response and less frequent complications  compared to cyclophosphamide bolus therapy, a regimen with risk of bone marrow suppression, infertility, and malignancy. Further work addressing maintenance therapy demonstrated mycophenolate superior to cyclophosphamide, again in terms of response and side-effects. Walsh et al. even propose that mycophenolate should be considered as a first-line induction therapy for treatment of lupus nephritis in patients without renal dysfunction, suggesting that mycophenolate will be encountered more frequently in medical practice.
MMF is a less toxic alternative to azathioprine.
MMF is also used in the treatment of autoimmune diseases, such as pemphigus vulgaris, and systemic lupus erythematosus. Suppressing T cells and B cells stops them from attacking healthy cells, but also weakens their ability to defend against infections.
The chemical name for mycophenolate mofetil is 2-morpholinoethyl (E)-6-(1,3-dihydro-4-hydroxy-6-methoxy-7-methyl-3-oxo-5-isobenzofuranyl)-4-methyl-4-hexenoate. Its empirical formula is C23H31NO7 and molecular weight 433.50. Mycophenolate mofetil is morpholinoethyl ester of mycophenolic acid, which is used to mask the carboxyl group. Mycophenolate mofetil is reported to have pKa values 5.6 for the morpholino moiety and 8.5 for the phenolic group.