Potter, HuntingtonPressman, Peter Scott
Summary
People with Down syndrome (DS) exhibit mild to severe intellectual disability, which challenges their ability to function independently. Any improvement in their cognitive ability would greatly improve their activities of daily living, but currently there are no therapeutics available to improve cognitive function in people with DS. This proposal aims to design and complete a clinical trial in adults with DS using sargramostim, an FDA-approved drug for increasing the production and differentiation of various white blood cells, which has been shown in animal models of DS, Alzheimer’s disease (AD), and normal aging and in human studies of AD and chemo brain to be safe and associated with improved cognition.