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

Date:
March 29, 2022
Source:
University of Colorado Anschutz Medical Campus
Summary:
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.



FULL STORY ==========================================================================
A new study shows that a potential treatment for Alzheimer's disease
may also 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 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.

A multidisciplinary team at the University of Colorado Anschutz Medical
Campus studied the safety and tolerability of GM-CSF treatment and its
effects on behavior and brain pathology in a mouse model of Down syndrome
and in mice undergoing typical aging. The results reported in the journal Neurobiology of Diseasesuggest that GM-CSF has potential applicability
to humans.

"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. All three groups are located on the CU Anschutz Medical Campus.

He adds, "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, led by Md. Mahiuddin Ahmed, PhD, 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. The findings support the
hypothesis that GM-CSF/sargramostim may promote neuronal recovery from
injury or from neurological disease through multiple mechanisms, some
of which evidently enhance cognitive function.

The next step is to determine whether this treatment is safe, tolerable
and efficacious in people with Down syndrome.

The CU Anschutz Medical Campus team was recently awarded a grant from
the National Institutes of Health/National Institute on Aging to study sargramostim treatment in young adults with Down syndrome who do not
show evidence of Alzheimer's disease. They will investigate its safety
and potential efficacy regarding cognitive function, quality of life
measures and biomarkers associated with neuronal damage.

"We are breaking new ground in studying sargramostim for multiple,
different disorders -- Down syndrome and Alzheimer's disease," Potter
said. "We hope that this therapy, already proven to be safe for other
diseases, will greatly improve cognitive function in people with
Down syndrome." The new NIH-funded project, co-directed by Potter and
Peter Pressman, MD, from the CU department of neurology, will leverage collaborations between research teams on the CU Anschutz Medical Campus
and at Colorado State University. They will work closely with the Linda
Crnic Institute for Down Syndrome, which is an affiliate of the Global
Down Syndrome Foundation (GLOBAL). GLOBAL is the leading Down syndrome organization successfully advocating for Down syndrome research funding
at the NIH.


========================================================================== Story Source: Materials provided by University_of_Colorado_Anschutz_Medical_Campus. Original written by
Julia Milzer. Note: Content may be edited for style and length.


========================================================================== Journal Reference:
1. Md. Mahiuddin Ahmed, Athena Ching-Jung Wang, Mihret Elos, Heidi
J. Chial,
Stefan Sillau, D. Adriana Solano, Christina Coughlan, Leila Aghili,
Paige Anton, Neil Markham, Vanesa Adame, Katheleen J. Gardiner,
Timothy D.

Boyd, Huntington Potter. The innate immune system stimulating
cytokine GM-CSF improves learning/memory and interneuron and
astrocyte brain pathology in Dp16 Down syndrome mice and improves
learning/memory in wild-type mice. Neurobiology of Disease, 2022;
168: 105694 DOI: 10.1016/ j.nbd.2022.105694 ==========================================================================

Link to news story: https://www.sciencedaily.com/releases/2022/03/220329114719.htm

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