Polycystic Kidney Disease

Polycystic kidney diseases are a group of hereditary disorders in which benign cysts grow uncontrollably in the kidney, often ultimately leading to kidney failure and the need for renal transplant. ADPKD is the most common form of PKD, affecting more than 600,000 Americans and 12.4 million people worldwide. Tolvaptan, a vasopressin type 2 receptor blocker from Otsuka Pharmaceutical, is the only drug approved for the treatment of high-risk patients with ADPKD.

Treatment with tolvaptan ameliorates but does not prevent the progression of ADPKD – and, in addition to its high cost, tolvaptan can induce liver toxicity and causes polyuria (excessive urine production) in a high percent of patients, which has a substantial negative effect on both compliance and quality of life.  A variety of potential drug payloads, including mTOR inhibitors, cyclin dependent kinase inhibitors, epidermal growth factor receptor (EGFR) inhibitors, and peroxisome proliferator-activated receptor (PPAR) agonists, could benefit from MNP formulation for the treatment of ADPKD.