Mycophenolate mofetil (MMF) is an immunosuppressive drug designed to inhibi
t inosine monophosphate dehydrogenase (IMPDH). IMPDH is a key enzyme in the
de novo purine synthesis of lymphocytes, It is crucially important for pro
liferative responses of human T and B lymphocytes, The inhibition of IMPDH
thus leads to selective lymphocyte suppression. After successful use in var
ious in vitro and animal models, MMF was brought to clinical trial in patie
nts undergoing transplantation. The drug is rapidly and completely absorbed
following oral administration. Pilot studies of administration with cyclos
porin and corticosteroids suggested a significant reduction in the incidenc
e of organ rejection at dosages of 1 to 3 g/day, As a result of these studi
es, 3 pivotal randomised double-blind multicentre trials, involving nearly
1500 patients, were designed to investigate the effects of addition of MMF
to different standard immunosuppressive protocols on the prevention of acut
e renal allograft rejection. After 6 months, the rates of biopsy-proven rej
ection were significantly reduced in patients receiving MMF. In combination
with cyclosporin and corticosteroids, the adverse effect profile resembled
that of azathioprine. Most adverse effects were associated with the gastro
intestinal tract, the blood system and opportunistic infections. MMF offers
improved immunosuppressive therapy following renal and probably other soli
d organ transplantation. MMF has born licensed since 1995 for the preventio
n of acute renal allograft rejection in most countries. It has been used in
different combinations of immunosuppressive drugs and in various dosages a
nd regimens.