A study revealed that GM-CSF, which is an Alzheimer's drug, may improve cognitive function in people with Down syndrome. The drug sargramostim. GM-CSF, which stands for granulocyte-macrophage colony-stimulating factor, is the first to show memory improvement in Alzheimer's patients in 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. The study, published in the journal, 'Neurobiology,' claims that a multidisciplinary team has studied the safety and tolerability of GM-CSF treatment and its effects on behaviour and brain pathology in a mouse model of Down syndrome and in mice undergoing typical ageing.
"People with Down syndrome are at higher risk for Alzheimer's disease and previous work showed that GM-CSF improves cognition and brain pathology in Alzheimer's disease patients. This new study shows that GM-CSF also, unexpectedly, improves cognition in mice that do not have Alzheimer's disease," said senior author Huntington Potter, PhD, professor of neurology at the University of Colorado School of Medicine, director of the University of Colorado Alzheimer's and Cognition Center and director of Alzheimer's disease research at the Linda Crnic Institute for Down Syndrome.
He added, "Discovering a treatment that may help children and young adults with Down syndrome to develop their physical and mental capabilities is critical to improving their health and activities of daily living."
The research team discovered that treatment with GM-CSF, which has pro-inflammatory, anti-inflammatory and immune regulatory properties, reverses learning and memory deficits, the loss of certain nerve cells, and other abnormalities in the brain in a mouse model of Down syndrome and also improves cognition in normal aging mice. The human version of GM-CSF/sargramostim has already been shown to be effective in improving cognition in people with mild-to-moderate Alzheimer's disease and in cancer patients.