Laura Barisoni, MD, co-authored a paper titled “Inaxaplin for Proteinuric Kidney Disease in Persons with Two APOL1 Variants,” which was published in the New England Journal of Medicine (NEJM) on March 16th, with an accompanying two-minute NEJM Quick Take video that highlights aspects of the manuscript. Read the paper here.
Individuals with two toxic gain-of-function variants in the gene encoding apolipoprotein L1 (APOL1) are at greater risk for the development of progressive kidney disease, including Focal Segmental Glomerulosclerosis (FSGS), a severe form of kidney disease.
Despite the known genetic cause, therapies targeting proteinuric kidney disease in persons with two APOL1 variants (G1 or G2) are lacking. The study found that APOL1 inhibitor inaxaplin, a small-molecule compound, was associated with reductions in proteinuria over 13 weeks of treatment of in participants with two APOL1 variants and focal segmental glomerulosclerosis.
The manuscript was selected as a Science Behind the Study feature titled “Inhibiting APOL1 to Treat Kidney Disease,” written by NEJM deputy editors Dr. Winfred Williams and Dr. Julie Ingelfinger. It describes key concepts of FSGS, APOL1, and inaxaplin.
Neil R. Powe, M.D., published an accompanying editorial, “A Step Forward for Precision Equity in Kidney Disease,” which outlines why inaxaplin may be a “key, new tool in the management of the disproportionate burden of chronic kidney disease in persons of African ancestry” and describes the research as a “major scientific breakthrough with enormous implications” that along with other “reinforcing studies, may transform the lives of persons with FSGS and two APOL risk variant alleles.”