Mesoscale Nanotechnology: A Novel Therapeutic Strategy in Polycystic Kidney Disease
Abstract
Autosomal Dominant Polycystic Kidney Disease (ADPKD) is the most common inherited disease of chronic kidney disease and need for dialysis. The only approved treatment for ADPKD is only modestly effective when used in high-risk patients and has significant side effects, which reduce compliance. Despite substantial advances in our understanding the causes of PKD, some promising drugs failed clinical trials. One of the most promising classes of drugs, mTOR inhibitors, has shown highly effective therapeutic effects in animals. In humans, however, mTOR inhibitors exhibit side effects that prevent the administration of doses that are high enough to benefit patients. Recently, a new nanoparticle technology was developed to specifically target the renal tubules—the structures of the kidney affected by ADPKD. These nanoparticles can encapsulate many types of drug molecules and deliver them directly to the renal tubules. This technology allows drugs, or any other molecules, to bypass the organs of the body where they cause side effects and be deposited in the kidneys. The particles also release drugs over several months, potentially providing long-lasting therapeutic effects. Our collaborative project will be conducted by a team of investigators that includes both physician-scientists who study ADPKD, as well as a nanomaterials engineer who synthesizes nanoparticles for drug delivery applications. Our goal is to develop a transformative therapy to treat polycystic kidney disease by targeting promising pathways including mTOR and miR-17, to the kidneys using tubule-targeted nanoparticles. This approach has the potential not only to provide an important therapy for ADPKD, but it is a promising new platform to find treatments for other kidney diseases.
Document Details
- Document Type
- DoD Grant Award
- Publication Date
- Dec 05, 2021
- Source ID
- W81XWH2110188
Entities
People
- Edgar Jaimes
Organizations
- Sloan-Kettering Institute
- United States Army