When his team of researchers injected the GM-CSF into the brains of mice with Alzheimer’s, it cut the amyloid plaque in half. When the compound was injected under their skin, the mice improved their scores navigating a water maze immensely.
GM-CSF has been FDA-approved and in use since 1991 as the prescription drug Leukine, which stimulates growth of white blood cells in transplant patients and in cancer patients undergoing chemotherapy. In 2009, Potter and co-researchers looked back at how chemotherapy patients fared cognitively after treat-ment; those who had taken GM-CSF fared far better than those who did not.
Its long-standing safety record made the next step easier.
In 2011, Potter and his colleagues at the University of South Florida Health Byrd Alzheimer’s Institute launched a Phase II clinical trial to look at the safety and efficacy of the drug in Alzheimer’s disease patients.
This fall, a similar trial will get underway at CU.