29^th European Nephrology Conference

Emerging Therapies for Polycystic Kidney Disease

Emerging therapies for polycystic kidney disease are offering new hope for managing and potentially altering the course of this genetic disorder. Current research focuses on targeting the underlying molecular mechanisms driving cyst formation and kidney enlargement. One promising approach involves the use of small molecules and biologics that inhibit pathways such as the mTOR (mechanistic target of rapamycin) pathway, which is implicated in cyst growth. Drugs like tolvaptan, which targets vasopressin receptors, have shown efficacy in slowing disease progression by reducing cyst volume and preserving kidney function. Dietary and lifestyle modifications are also being considered as adjunct therapies. Gene therapy approaches will also be explored, including the potential of CRISPR technology to edit mutations in the PKD1 and PKD2 genes. These groundbreaking techniques hold the promise of correcting genetic defects at the molecular level, potentially offering a long-term solution for patients with genetic forms of the disease.