An Integrated Omics Approach for Treatment of Steroid-Resistant Nephrotic Syndrome in Primary Focal Segmental Glomerulosclerosis
Abstract
The proposed research project addresses Fiscal Year 2018 Peer Reviewed Medical Research Program Topic Area of Focal Segmental Glomerulosclerosis (FSGS). FSGS is a form of kidney that results in the patient spilling a lot of protein into the urine and developing signs and symptoms of nephrotic syndrome – edema, peripheral swelling, etc. With time, the kidney function declines and the patient is destined to go on dialysis and/or on an organ transplant waitlist. Currently used therapies are only partially effective or completely ineffective in at least a subset of this population. The majority of clinical trials have also failed as they typically use a one-size-fits-all approach with many of them repurposing drugs used in other indications. New research into the pathways underlying FSGS suggest that not all FSGS patients have the same disease profile. Some patients may have elevations in certain disease-causing molecules whereas other patients may have elevations in other molecules, which also cause the same disease. Therefore, an unresearched therapy is unlikely to work, will work only partially, or a combination of this in some patients only. We have developed a molecule that lowers spilling of protein into the urine in models of FSGS. The present program is designed to understand the molecular basis underlying the activity of this molecule and determine if we can identify FSGS patients that can use such a molecule for benefit. This program is also called Precision Medicine, which is to select the right therapy for the right patient every time.
Document Details
- Document Type
- DoD Grant Award
- Publication Date
- Nov 19, 2019
- Source ID
- W81XWH1910448
Entities
People
- Prakash Narayan
Organizations
- Angion Biomedica Corporation
- United States Army