Experimental Therapies and Ongoing Clinical Trials to Slow Down Progression of ADPKD
Maria V. Irazabal,
Vicente E. Torres.
The improvement of imaging techniques over the years has contributed to the understanding of the natural
history of autosomal dominant polycystic kidney disease, and facilitated the observation of its structural progression.
Advances in molecular biology and genetics have made possible a greater understanding of the genetics, molecular, and
cellular pathophysiologic mechanisms responsible for its development and have laid the foundation for the development
of potential new therapies.
Therapies targeting genetic mechanisms in ADPKD have inherent limitations. As a result, most experimental therapies at
the present time are aimed at delaying the growth of the cysts and associated interstitial inflammation and fibrosis by
targeting tubular epithelial cell proliferation and fluid secretion by the cystic epithelium.
Several interventions affecting many of the signaling pathways disrupted in ADPKD have been effective in animal
models and some are currently being tested in clinical trials.
Keywords: Autosomal dominant polycystic kidney disease, AMP-activated protein kinase (AMPK) activators, cystic
fibrosis transmembrane conductance regulator (CFTR) inhibitors, mammalian target of rapamycin (mTOR) inhibitors,
somatostatin analogues, Src inhibitors, peroxisome proliferator activated receptors (PPARγ, vasopressin V2 receptor
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