Existing drug reduced Alzheimer’s symptoms in clinical trial

Linda Brent, PhD

Executive Director, Parsemus Foundation

A hand holding a glowing brain to symbolize Alzheimer care

Scientists at the University of Colorado Alzheimer’s and Cognition Center wondered why individuals with rheumatoid arthritis are less likely to have Alzheimer’s disease. Could a natural human protein called GM-CSF, which is increased in those with rheumatoid arthritis, help Alzheimer’s patients?

A study in a mouse model of Alzheimer’s showed promising results: after a few weeks of treatment with GM-CSF, the mice had reduced amyloid deposition and their poor memory returned to normal.

Now the researchers have reported results of a randomized, double-blind, placebo-controlled phase II trial in humans. They found that the 20 participants who were given the existing GM-CSF drug Sargramostim (Leukine®), which is often used to increase white blood cells after cancer treatments, had significant improvements in memory and Alzheimer’s biomarkers as compared to the control group. Cognition memory improved by almost two points in the 30 point Mini-Mental State Exam, and brain amyloid, tangles, and neurodegeneration improved toward normal. The research was published in Alzheimer’s & Dementia: Translational Research and Clinical Interventions.

Sargramostim is the first drug to show improvement in Alzheimer’s patients in a phase II clinical trial.

A larger trial is now underway, funded in part by the Alzheimer’s Association/Part the Cloud initiative.

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