THE JOURNEY OF A POTENTIAL ALZHEIMER THERAPY FROM THE LABORATORY BENCH THROUGH THE PATENT OFFICE AND INTO THE CLINIC
People with rheumatoid arthritis (RA) do not tend to develop Alzheimer's disease (AD). While it has been commonly assumed that RA patients' usage of nonsteroidal anti-inflammatory drugs (NSAIDs) helps prevent AD onset, NSAID clinical trials have proven unsuccessful in AD patients. To
determine whether intrinsic factors within RA pathogenesis itself underlie RA's protective effect, we investigated hematopoietic colony-stimulating factors, upregulated in RA, on the pathology and behavior of transgenic AD mice. We found that granulocyte-macrophage colony-stimulating factor
(GM-CSF) rapidly reversed cognitive impairment and reduced cerebral amyloidosis in AD mice. Because these findings were novel, we filed a patent application for a method to treat cognitive impairment. We also developed and submitted for patent on a novel device, which we used to reduce reagent
amounts during our histopathology of the AD mice. A retrospective analysis of bone marrow transplant patients at Moffitt, who also garner cognitive deficits from the high-dose chemotherapy, showed GM-CSF to be associated with improvements in cognition, and resulted in filing a Continuation-in-Part
patent application. Because recombinant human GM-CSF has been FDA approved and used safely for over 20 years to treat leukopenia, we initiated a clinical trial in AD patients. Future research is aiming to investigate GM-CSF in other neurodegenerative diseases.