Targeting the Toughest Diseases (Sponsored Content)
Aug 9, 2023
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NBA great Alonzo Mourning recounts his fight against kidney disease and discusses breakthroughs in genetic research, the role of the APOL1 protein in preventing African sleeping sickness and its link to kidney disease, and Vertex Pharmaceuticals' groundbreaking research and efforts to develop transformative therapies.
APOL1-mediated kidney disease affects a significant percentage of African Americans, Latinx, and Afro-Caribbean communities, and Vertex Pharmaceuticals is researching small molecule therapies to address this unmet medical need.
Understanding the genetic basis of APOL1-mediated kidney disease sheds light on why certain populations are disproportionately affected, opening the door for targeted research and potential treatments.
Deep dives
APOL1-mediated kidney disease: The Genetic Abnormality and Its Impact
APOL1-mediated kidney disease is a type of kidney disease caused by a genetic abnormality known as the APOL1 gene. This gene mutation developed thousands of years ago in sub-Saharan Africa as a protective mechanism against African sleeping sickness. However, when individuals inherit two risk variants of APOL1, their chances of developing kidney disease increase tenfold. The disease, characterized by scar tissue development on the kidney filters, affects a significant percentage of African Americans, Latinx, and Afro-Caribbean communities. While chronic kidney disease has no cure, options like dialysis and kidney transplants offer temporary relief. Vertex Pharmaceuticals is researching APOL1-mediated kidney disease, hoping to develop small molecule therapies to address this unmet medical need.
Uncovering the Genetic Basis of APOL1-mediated Kidney Disease
Dr. David Friedman and his team at Harvard Medical School and Vertex Pharmaceuticals identified the APOL1 gene as the primary cause of kidney disease in African American families. Through genetic mapping and advances in technology, they unearthed the specific mutations in APOL1 that lead to kidney disease. A key breakthrough came with the 1000 Genomes Project, which provided a comprehensive database of genetic variations across diverse ancestries. Understanding the genetic basis of APOL1-mediated kidney disease sheds light on why certain populations are disproportionately affected. This knowledge opens the door for targeted research and potential treatments.
Vertex's Efforts and Future Promises for APOL1-mediated Kidney Disease
Vertex Pharmaceuticals focuses on diseases with substantial unmet needs and well-understood underlying causes. This aligns with their efforts in researching APOL1-mediated kidney disease, aiming to develop investigational small molecule therapies. The company has made significant progress in evaluating potential therapies in experimental settings and preliminary studies with patients. While the road to finding a treatment is challenging, Vertex remains committed to raising awareness about APOL1-mediated kidney disease and advocating for preventative measures like genotyping. By expanding diagnosis rates and providing precise therapies, Vertex aims to make a lasting impact on the lives of kidney disease patients.
The battle against humanity’s most challenging diseases is happening at the intersection of business and medicine. A new six-episode podcast called Targeting the Toughest Diseases explores how Vertex Pharmaceuticals, a Boston-based biotech company, is using innovative tools, methods, and a unique philosophy to search for treatments and cures. Produced by Bloomberg Media Studios and Vertex, the podcast’s latest episode features NBA great Alonzo Mourning recounting his fight against kidney disease, and how future generations of patients may have an easier time of it. You can subscribe today on Apple Podcasts, Spotify or wherever you listen to your favorite podcasts.