Gladstone researchers create a drug that replicates the cellular effects of low-oxygen therapy to treat inherited mitochondrial diseases.

For most people, living at high altitudes—where oxygen levels are lower than at sea level—can offer health benefits, such as reduced rates of heart disease and improved endurance. However, for individuals with inherited mitochondrial diseases, who often do not survive beyond childhood, low-oxygen environments like those at high elevations could be life-saving, potentially prolonging their lifespan and alleviating symptoms.

Now, researchers at Gladstone Institutes have developed a drug that replicates the effects of low-oxygen exposure. In mice with Leigh Syndrome—the most common childhood mitochondrial disease—the drug, called HypoxyStat, extended lifespan more than threefold and reversed brain damage and muscle weakness, even when administered in the disease’s late stages.