Small molecule drug candidate offers hope for rare kidney stone disease with no current treatment

Scientists at the Buck Institute for Research on Aging have shown that an orally administered small molecule, N-propargylglycine (N-PPG), can completely prevent the formation of calcium oxalate kidney stones, protect against kidney failure, and fully restore normal survival in a mouse model of primary hyperoxaluria type 2 (PH2), a rare and currently untreatable genetic disorder that causes progressive kidney failure in infants and young adults. An estimated 1,700 people suffer from the disease in the US, although experts believe many more cases go undiagnosed.