In 2010, scientists showed that higher rates of two severe kidney diseases in African Americans could be attributed to variants in a gene called APOL1. Today at Vertex, we’re focused on discovering and developing medicines aimed at treating the underlying cause of these APOL1-mediated kidney diseases. The speed of scientific progress in this area is incredibly exciting. Just 10 years after that discovery, Vertex has developed an investigational drug, which is already in a Phase 2 clinical trial.
To help increase awareness of these diseases, including APOL1-mediated focal segmental glomerulosclerosis (FSGS), we’re happy to share the next video in a series — Vertex Forward: Exploring APOL1-Mediated Kidney Diseases.
I hope you’ll take a few minutes to watch and learn more about the genetic link to FSGS and our ambitious goals of bringing a potential new medicine to patients.
The story of apolipoprotein L1 (APOL1) is a story of genetic evolution. We all have the APOL1 gene. But over the past 10,000 years, it evolved in particular ways in people who lived in sub-Saharan Africa in order to protect them from disease.
Having two APOL1 genetic variants protects people from the parasite that causes African sleeping sickness. But it also puts them at increased risk of kidney disease. In the case of FSGS, there's a 17-fold increase of occurrence. That’s because these APOL1 variants, when triggered by another event such as infection, encode a protein that damages certain parts of the kidney that filter blood. In APOL1-mediated FSGS, the consequences are severe, and 50% of patients progress to kidney failure within five years.
At Vertex, we are highly motivated to help people with APOL1-mediated kidney diseases by developing new medicines that target the underlying cause of their disease. I’m excited to see what the future holds for serious diseases like APOL1-mediated FSGS and beyond, and I’m so proud to be part of a team working tirelessly to get there.