A new study from the University of Colorado Anschutz Medical Campus has found that a drug originally used to treat Alzheimer’s disease may also help improve memory and learning in people with Down syndrome.
The drug is called sargramostim, or GM-CSF, which is short for granulocyte-macrophage colony-stimulating factor.
GM-CSF is a natural human protein that has been safely used for over 30 years to treat other health problems. Recently, it became the first drug to show memory improvement in people with Alzheimer’s during a phase II clinical trial.
Now, researchers have found that it could also help people with Down syndrome, who are at a higher risk of developing Alzheimer’s disease later in life.
The research team, made up of experts from different fields, tested the drug in mice that were bred to have Down syndrome as well as in normal aging mice. They found that GM-CSF treatment improved memory and learning in both types of mice. It also reduced the loss of important brain cells and other brain issues seen in Down syndrome.
Dr. Huntington Potter, one of the senior researchers on the team, said the results were surprising and exciting. Not only did the drug improve brain function in mice with Down syndrome, but it also helped normal aging mice, which means it could have benefits beyond just treating Alzheimer’s disease.
He explained that helping young people with Down syndrome develop better physical and mental abilities is a major goal. Improving their brain function can help with daily living and overall health.
The drug works by affecting the immune system. It has both pro-inflammatory and anti-inflammatory effects, and it can help the brain recover from damage caused by aging or disease.
Previous research has shown it helps cancer patients and people with mild to moderate Alzheimer’s. This study adds to the idea that GM-CSF might support brain repair in multiple ways.
Next, the team will begin testing the drug in young adults with Down syndrome who do not yet show signs of Alzheimer’s. They want to find out if it is safe and if it can improve thinking and memory, as well as overall quality of life. The project is being funded by a grant from the National Institutes of Health and the National Institute on Aging.
Dr. Potter said the team is making exciting progress by studying this drug for both Alzheimer’s and Down syndrome. Since sargramostim is already considered safe for other illnesses, there is hope it could soon be used to help people with Down syndrome live fuller, more independent lives.
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The study is published in Neurobiology of Disease.
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