Promising Alzheimer's drug may also improve memory in Down syndrome and normal aging (Sargramostim, a human protein)

Medical Xpress / CU Anschutz Medical Campus / Neurobiology of Disease ^ | Mar. 29, 2022 | Md. Mahiuddin Ahmed et al

[ConservativeMind]

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 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 suggest 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 author Huntington Potter, Ph.D.

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. 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.

"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."

[ConservativeMind]

The drug is expensive, but commonly available.

[ConservativeMind]

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[ryderann]

But can you worm your horse with it?

[AlaskaErik]

I was going to look into this, but then I forgot.

[dblshot]

Paging Dr. Kevin O’Connor, Dr. O’Conner!

[MomwithHope]

Does Pfizer make it?