Promising Alzheimer’s drug may also improve memory in Down syndrome and normal aging —


A brand new examine exhibits {that a} potential remedy for Alzheimer’s illness may enhance cognitive perform in individuals with Down syndrome.

The drug sargramostim (GM-CSF, which stands for granulocyte-macrophage colony-stimulating issue) is the primary to indicate reminiscence enchancment in Alzheimer’s sufferers in a section II scientific trial. GM-CSF is a standard human protein that’s secure and well-tolerated with over 30 years of FDA-approved use for different issues.

A multidisciplinary workforce on the College of Colorado Anschutz Medical Campus studied the protection and tolerability of GM-CSF remedy and its results on conduct and mind pathology in a mouse mannequin of Down syndrome and in mice present process typical getting old. The outcomes reported within the journal Neurobiology of Illness counsel that GM-CSF has potential applicability to people.

“Folks with Down syndrome are at increased danger for Alzheimer’s illness and former work confirmed that GM-CSF improves cognition and mind pathology in Alzheimer’s illness sufferers. This new examine exhibits that GM-CSF additionally, unexpectedly, improves cognition in mice that do not need Alzheimer’s illness,” mentioned senior creator Huntington Potter, PhD, professor of neurology on the College of Colorado College of Medication, director of the College of Colorado Alzheimer’s and Cognition Heart and director of Alzheimer’s illness analysis on the Linda Crnic Institute for Down Syndrome. All three teams are positioned on the CU Anschutz Medical Campus.

He provides, “Discovering a remedy which will assist kids and younger adults with Down syndrome to develop their bodily and psychological capabilities is essential to bettering their well being and actions of day by day dwelling.”

The analysis workforce, led by Md. Mahiuddin Ahmed, PhD, found that remedy with GM-CSF, which has pro-inflammatory, anti-inflammatory and immune regulatory properties, reverses studying and reminiscence deficits, the lack of sure nerve cells, and different abnormalities within the mind in a mouse mannequin of Down syndrome and in addition improves cognition in regular getting old mice. The human model of GM-CSF/sargramostim has already been proven to be efficient in bettering cognition in individuals with mild-to-moderate Alzheimer’s illness and in most cancers sufferers. The findings assist the speculation that GM-CSF/sargramostim could promote neuronal restoration from damage or from neurological illness by way of a number of mechanisms, a few of which evidently improve cognitive perform.

The subsequent step is to find out whether or not this remedy is secure, tolerable and efficacious in individuals with Down syndrome.

The CU Anschutz Medical Campus workforce was just lately awarded a grant from the Nationwide Institutes of Well being/Nationwide Institute on Ageing to check sargramostim remedy in younger adults with Down syndrome who don’t present proof of Alzheimer’s illness. They’ll examine its security and potential efficacy concerning cognitive perform, high quality of life measures and biomarkers related to neuronal injury.

“We’re breaking new floor in learning sargramostim for a number of, totally different issues — Down syndrome and Alzheimer’s illness,” Potter mentioned. “We hope that this remedy, already confirmed to be secure for different ailments, will significantly enhance cognitive perform in individuals with Down syndrome.”

The brand new NIH-funded undertaking, co-directed by Potter and Peter Pressman, MD, from the CU division of neurology, will leverage collaborations between analysis groups on the CU Anschutz Medical Campus and at Colorado State College. They’ll work carefully with the Linda Crnic Institute for Down Syndrome, which is an affiliate of the International Down Syndrome Basis (GLOBAL). GLOBAL is the main Down syndrome group efficiently advocating for Down syndrome analysis funding on the NIH.

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Supplies supplied by College of Colorado Anschutz Medical Campus. Unique written by Julia Milzer. Be aware: Content material could also be edited for type and size.