New research is breaking ground in repurposing an FDA-approved drug as a potential treatment in Down syndrome and normal aging. The results suggest that GM-CSF may improve cognitive function in people with Down syndrome. The drug sargramostim (GM-CSF, which stands for granulocyte-macrophage colony-stimulating factor) is also the first to show memory improvement in Alzheimer's patients in a phase II clinical trial. GM-CSF is a normal human protein that is safe and well-tolerated with over 30 years of FDA-approved use for other disorders.
