A new study from the University of Colorado found that a potential treatment for Alzheimer’s disease may also improve cognitive function in Down syndrome and normal aging.
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 is published in the Neurobiology of Disease and was conducted by Huntington Potter et al.
In the study, the 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
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 next step is to determine whether this treatment is safe, tolerable, and efficacious in people with Down syndrome.
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Previous research has shown that some drugs could help fight Alzheimer’s disease.
In a study from Rush University published in Science Signaling, researchers found two drugs approved decades ago not only counteract brain damage caused by Alzheimer’s disease but also may improve cognition.
If the surprising drug combination continues to destroy a key feature of the disease, then an effective treatment for Alzheimer’s may have been hiding for decades in plain sight.
A promising series of early studies are highlighting two well-known medicine cabinet standbys—gemfibrosil, an old-school cholesterol-lowering drug, and retinoic acid, a vitamin A derivative.
Gemfibrosil, is sold as Lopid and while it’s still used, it is not widely prescribed. Doctors now prefer to prescribe statins to lower cholesterol.
Retinoic acid has been used in various formulations to treat everything from acne to psoriasis to cancer.
In the study, the two drugs are being studied for their robust impact on the brain and a potential new role that could one day thrust them into fighting what is now an incurable brain disease.
Both medications have an uncanny capability to zero in on the brain’s astrocytes, cells that originally got their name because they look like stars.
Previous studies have found that astrocytes may be responsible for the accumulation of amyloid beta (Aβ), the gooey plaque that damages neurons.
As a result, these star-like cells aid in the cascade of deleterious events that rob people of their sense of self, their memories, and ultimately steal their lives.
In the study, the team discovered that gemfibrozil and retinoic acid, when used in combination, force astrocytes to reverse their destructiveness, and instead reduce amyloid-beta in the brain—improving cognitive function.
The findings suggest that perhaps in the not-too-distant future, these drugs can be repurposed to coax astrocytes into a beneficial role, serving as Aβ “clearing machines,” eliminating the accumulation of plaques and preventing Alzheimer’s from unraveling the brain.
For more information about brain health, please see recent studies about 13 things your doctor can check to help protect brain health, and results showing new drug for Alzheimer’s disease prevention is safe and effective.
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