α-Lipoic acid treatment prevents cystine urolithiasis in a mouse model of cystinuria

Excess urinary cystine can lead to painful stone formation. There is no current effective treatment, but here Pankaj Kapahi, Marshall Stoller and colleagues have found that α-lipoic acid can prevent or even reverse formation of these stones in a mouse model. Cystinuria is an incompletely dominant disorder characterized by defective urinary cystine reabsorption that results in the formation of cystine-based urinary stones. Current treatment options are limited in their effectiveness at preventing stone recurrence and are often poorly tolerated. We report that the nutritional supplement α-lipoic acid inhibits cystine stone formation in the Slc3a1 −/− mouse model of cystinuria by increasing the solubility of urinary cystine. These findings identify a novel therapeutic strategy for the clinical treatment of cystinuria.