The Need for Novel Therapeutic Directions in Autosomal Dominant Polycystic Kidney Disease Patient Care
A late-2025 review maps the expanding ADPKD treatment pipeline beyond tolvaptan, including repurposed SGLT2 inhibitors, metformin and GLP-1 receptor agonists, novel molecular agents such as a polycystin-1 corrector and a microRNA-17 inhibitor, dietary strategies, and early gene therapy, signalling a shift toward disease-modifying and potentially curative approaches.
Background
ADPKD is the most prevalent genetic kidney disorder, characterized by progressive growth of kidney cysts leading to kidney failure in most patients. Management was limited to supportive measures until disease-modifying agents received regulatory approval.
Emerging therapies
At present only the vasopressin V2-receptor antagonist tolvaptan (and, in Italy, the somatostatin analog octreotide long-acting release for stage 4 CKD) is approved to slow progression. Strategies under clinical investigation include repurposed SGLT2 inhibitors, metformin, and GLP-1 receptor agonists; innovative agents such as a monoclonal antibody against pregnancy-associated plasma protein-A, a microRNA-17 inhibitor, and the polycystin-1 correcting agent VX-407; and dietary interventions such as caloric restriction and ketogenic diets.
Clinical implications
Rapid advances in gene therapy, though so far only explored experimentally, could eventually correct the underlying genetic defect and potentially reverse disease pathogenesis. Combined with pharmacologic and dietary approaches, these developments point toward more individualized, disease-modifying ADPKD care.
Category
Research
Source
Clinical Journal of the American Society of Nephrology (CJASN)
More from ASNRT News
Browse the latest news, society announcements, KDIGO guideline updates, and AJNT issue releases on the ASNRT newsroom.