An existing cancer drug eases kidney damage in a rare inherited disease — in mice

University of Regensburg researchers have cracked why nail-patella syndrome — a rare genetic disorder marked by unusual nails and kneecaps, but whose real threat is kidney failure — destroys the kidneys: faulty LMX1B protein gets flagged and prematurely shredded by the cell's recycling system, so kidney filtering cells run out of it. Treating mice with bortezomib, an already-approved cancer drug that blocks that shredding, stabilized the protein and significantly reduced kidney damage. Not yet a human therapy, but the first credible therapeutic lead for a disease with none.