New Kidney Research Focuses on Rare Disease, FSGS

FSGS is a rare condition that often leads to kidney failure

Jul 15, 2015, 10:00 ET from DUET study

ROCKVILLE, Md., July 15, 2015 /PRNewswire-USNewswire/ -- In normal, healthy kidneys millions of tiny filters called "glomeruli" perform the essential function of helping the kidneys function by keeping protein and cells in the bloodstream. For people with a rare condition called focal segmental glomerulosclerosis (FSGS), these filters are changed by FSGS and do not work properly. The result is a number of serious side effects. Although there are a few treatments that address these side effects, there is no FDA-approved medication for FSGS. For this reason, doctors are now participating in the DUET study for FSGS to learn more about a potential new treatment.

FSGS causes changes to the filters in the kidneys that keep nutrients and other good things in the body and keep wastes out. This can cause symptoms like large amounts of protein leaking into the urine (proteinuria), swelling caused by extra fluid in the body, high blood pressure (hypertension), high cholesterol (hyperlipidemia), low blood protein, and a risk for infection and blood clots.

There are two types of FSGS. Primary FSGS is when the cause is unknown, and secondary is when FSGS is caused by an infection or disease.  According to the National Kidney Foundation, FSGS can affect children and adults. 

Proteinuria is a significant concern for people with FSGS. The most common treatments to help reduce proteinuria and improve kidney function are drugs that suppress the immune system and steroids, which carry their own major side effects. People with FSGS may also be encouraged to make diet changes to limit their salt and protein intake. These treatments only address the side effects of FSGS and do not address the root of the problem. Most people with FSGS will eventually require dialysis or a kidney transplant as the result of kidney failure. Approximately 1,000 FSGS patients per year receive kidney transplants.

A clinical trial called the DUET study for people with FSGS is evaluating the safety and effectiveness of an investigational medication. The goal is to learn whether the investigational medication may decrease proteinuria. People with primary FSGS may be eligible to participate in the study if they are between the ages of 8 and 75 and if they do not have a history of heart disease, diabetes, hepatitis, HIV, or organ transplant.

The information learned from the DUET study may help doctors develop treatment options for people with FSGS in the future. To learn more about the DUET study and to see if there is a study doctor located near you visit the study website