Drug combination that reduces oxygen levels in tissues improves Parkinson's symptoms in mice
In a new study, Broad Institute researchers report that two small-molecule chemicals that reduce oxygen delivery to cells improved symptoms of neurodegeneration in a mouse model of Parkinson's disease. The scientists had previously found that excess oxygen in the brain is associated with neuronal death in Parkinson's—and that reducing the oxygen that mice breathe could help reverse symptoms.
A new study from the Broad Institute reveals that a drug combination reducing oxygen levels in tissues can improve symptoms of Parkinson's disease in mice. Researchers found that two small-molecule chemicals, GBT440 and PT2399, when combined, led to extended lifespan, alleviated symptoms in a mouse model of Leigh syndrome, and delayed the progression of neurological symptoms in a mouse model of Friedreich's ataxia.
These conditions are rare genetic disorders caused by mitochondrial malfunction. The combination drug regimen, dubbed "hypoxia in a pill," shows promise as a potential treatment for Parkinson's and certain mitochondrial disorders, although more preclinical and clinical testing is required.
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