According to a story from BioSpace, a recent study conducted by the biotechnology company Goldfinch Bio in collaboration with academic partners is highlighting the importance of proteinuria in determining outcomes in focal segmental glomerulosclerosis (FSGS), a rare disease that impacts the kidneys. Proteinuria is a condition in which protein is lost in the urine. Findings from the research indicate that reducing or halting proteinuria can improve the chances of kidney survival in these patients.
About Focal Segmental Glomerulosclerosis
Focal segmental glomerulosclerosis (FSGS) is responsible for roughly a sixth of nephrotic syndrome cases in children and adults. This condition can be acquired (due to obesity, drug abuse, vasculitis, or diabetes) or can be the result of heritable genetic mutations affecting several genes; in some cases, no known cause can be identified. Nephrotic syndrome describes a group of signs and symptoms that appear due to damage to the kidney. Symptoms include excess fluid within the body, puffy eyes, swelling throughout the body (edema), anemia, and shortness of breath. focal segmental glomerulosclerosis is the most common cause of nephrotic syndrome in adults, and is characterized by scarring in the glomeruli. In some adults, the condition results in kidney failure and excess proteins detected in the urine (proteinuria). As a result, dialysis may be necessary in many people with the condition. A kidney transplant is also an option in some cases. To learn more about focal segmental glomerulosclerosis, click here.
About The Study
This study was a retrospective examination of data from a trial of 138 patients with steroid-resistant focal segmental glomerulosclerosis. The analysis determined that patients who saw a reduction in proteinuria as a result of treatment saw significantly slowed disease progression, and their risk of death or end-stage kidney disease also decreased. The patients in this study were being treated with a combination of mycophenolate mofetil and dexamethasone or cyclosporine, as well as lisinopril or losartan.
These findings are relevant to Goldfinch because they are developing GFB-887, which is being developed with a mechanism to reduce proteinuria. It is classified as an inhibitor of transient receptor potential canonical channel 5 (TRPC5), a pathway that is overactivated in this disease. This overactivity damages cells called podocytes, which normally help prevent excess protein loss in the urine. GFB-887 is currently being tested in a phase 2 trial.
Learn more about this study here.