This damage can lead to kidney failure requiring people with FSGS to undergo dialysis and/or a kidney transplant. Each kidney is made up of ~1 million tiny filters called glomeruli. When glomeruli become damaged or scarred (sclerosis), proteins begin leaking into the urine (proteinuria). The word “focal” means that only some of the glomerular filters become scarred and “segmental” means that only some sections of the glomerulus become scarred. Our Focal Segmental Glomerulosclerosis (FSGS) research is intended to improve the lives of those living with this rare disease.
The onset of FSGS can occur at any age, depending on the underlying cause. The hallmark sign of FSGS is proteinuria. As proteinuria progresses, people with FSGS may experience severe swelling around the eyes, hands and feet, and abdomen causing sudden weight gain. Those with FSGS often feel exhausted, even after relatively simple tasks. Other symptoms of FSGS include low blood albumin, high cholesterol, and high blood pressure.
Recent evidence demonstrates that the TRPC5-Rac1 pathway, a critical regulator of cellular motility, forms a vicious cycle that drives pathogenic remodeling of the actin cytoskeleton in podocytes. The cause of FSGS is the result of podocyte dysfunction and injury, a result of dysregulation of the podocyte cytoskeleton. Many familial and sporadic forms of FSGS have been linked to genetic or environmental dysregulation of Rac1 signaling within the podocyte, highlighting the importance of Rac1 as a driver of kidney disease.
Focal Segmental Glomerulosclerosis research shows activation of the TRPC5-Rac1 pathway causes podocyte loss and breach of the filtration barrier, leading to proteinuria, the hallmark of FSGS. Inhibition of TRPC5 and subsequent suppression of Rac1 signaling offers a potential point of therapeutic intervention to restore podocyte integrity and halt disease progression.
FSGS is diagnosed by a renal biopsy, but Focal Segmental Glomerulosclerosis research is being conducted to discover novel biomarkers that may allow for non-invasive ways to diagnose and treat FSGS.
Of adult nephrotic syndrome cases are caused by FSGS
Of pediatric nephrotic syndrome cases are caused by FSGS
Average timeframe after within which people typically progress to ESKD
Number of people who progress to ESKD annually in U.S.
Of patients experience recurrence of FSGS after first kidney transplant
Of people with FSGS experience recurrence in subsequent transplants if they lost their first
There are no approved treatments for FSGS. The current treatment includes steroids and renin angiotensin system inhibitor drugs (ACE inhibitors or ARBs) to control blood pressure and reduce proteinuria. Other medications may be used to suppress the immune system. Once a person progresses to ESKD, the only treatment options are dialysis and transplantation. Our Focal Segmental Glomerulosclerosis research is working to improve the lives and create treatments for those living with FSGS.
Living with FSGS
I've learned that I have to be my own best advocate-- with doctors and others in the medical community, pharmacists, family, and others. I am the one who knows what it's like to live in my body and have this disease.
Living with FSGS
I have learned that your body is constantly adjusting it never stays the same and you must focus on staying healthy.
Caregiver of Daughter with FSGS
What I have learned about living with kidney disease is that the disease is only part of the problem. That there is so much more work that needs to be done to just live with kidney disease. A lot more education, research, funding, and advocating. That there must be more options and better treatments.
Living with FSGS
Often, we don't look sick and, on some days, may not act sick but kidney disease leads to widespread ailments. Some days my kidney disease has no impact on my daily living while on other days it feels as if the disease is in control of my abilities.
In this video, Jill H. talks about living with and managing FSGS, including her experience with dialysis and kidney transplants.
Our lead product candidate, GFB-887, is designed specifically to treat people with kidney diseases associated with an over-activation of the TRPC5-1 pathway.
We are working vigorously to pioneer disease modifying-treatments that bring hope and renewed quality of life to people living with kidney diseases.