Metformin and adult polycystic kidney disease: research and clinical application

Autosomal Dominant Polycystic Kidney Disease (ADPKD) is the most common monogenic nephropathy. It is characterized by the onset of progressively enlarging cysts in kidneys (and in other organs, mainly in the liver) that cause the progressive deterioration of renal function. Since 2018, Tolvaptan is available for the treatment of the disease (and in Italy also Octreotide), and allows to slow the progression of the disease towards renal failure. This new drug, even representing an epochal revolution in the management of this pathology, is not without side effects as well as potential adverse events that, although rare, should not be neglected (so much so as to make it necessary to monitor, first monthly and then quarterly, some important bio-humoral parameters). Therefore, the war for the defeat of ADPKD is far from being won and new efforts are needed to increase knowledge about the complex pathophysiology of this disease in order to identify other candidate molecules for the treatment of the disease. Among these Metformin, a drug that has long been used in the treatment of type 2 diabetes mellitus, according to numerous preclinical and clinical studies, would be able to reduce the development and formation of cysts, with a slowdown in the tissue damage related to them and of the progression to end-stage renal failure.
Autosomal Dominant Polycystic Kidney Disease (ADPKD) is the most common monogenic nephropathy. It is characterized by the onset of progressively enlarging cysts in kidneys (and in other organs, mainly in the liver) that cause the progressive deterioration of renal function. Since 2018, Tolvaptan is available for the treatment of the disease (and in Italy also Octreotide), and allows to slow the progression of the disease towards renal failure. This new drug, even representing an epochal revolution in the management of this pathology, is not without side effects as well as potential adverse events that, although rare, should not be neglected (so much so as to make it necessary to monitor, first monthly and then quarterly, some important bio-humoral parameters). Therefore, the war for the defeat of ADPKD is far from being won and new efforts are needed to increase knowledge about the complex pathophysiology of this disease in order to identify other candidate molecules for the treatment of the disease. Among these Metformin, a drug that has long been used in the treatment of type 2 diabetes mellitus, according to numerous preclinical and clinical studies, would be able to reduce the development and formation of cysts, with a slowdown in the tissue damage related to them and of the progression to end-stage renal failure.