Potter, Huntington PHD

INCLUDE Grants​

GM-CSF/sargramostim treatment to improve cognition in Down syndrome

Potter, HuntingtonPressman, Peter Scott

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.

Neurovascular unit dysfunction in Down syndrome revealed by TBI

Huang, MingxiaPotter, HuntingtonSullivan, Kelly D

Many of the neurological conditions that co-occur in people with Down syndrome (DS) arise from brain cell alterations that are subtle and progressive in nature, making their categorical detection a challenge for researchers and clinicians. We postulate that the DS brain is predisposed to traumatic brain injury (TBI)-induced acute and long-term disabilities based on our findings that a mouse model of DS is hypersensitive to a single mild TBI and exhibits long-term impairment. This transformative research project aims to use mild TBI as a sensitizing probe to visualize the subtle and hard-to-detect DS-associated cellular and molecular alterations with the overarching goal of discovering new therapeutic strategies for people with DS.