I know that results in mice do not always translate to humans. I know that. I know that clinical trials can take years and that there are many hurdles between the first promising result in an animal model and the actual development of some form of treatment. Nevertheless I could not stifle a surge of hope when I heard about recent research suggesting that GM-CSF treatment reversed cognitive impairment and amyloid build-up in Alzheimer mice.
I have watched two family members succumb to Alzheimer’s disease. To me the news that something not only halted progression but actually reversed some of the cognitive impairment is huge. I know…its only in mice, but it’s the most promising news about this disease that I’ve heard in a while. And there’s more, the effective factor, GM-CSF, is already available in the drug Leukine, which is currently used to reduce risk of infection in cancer patients.
Rheumatoid arthritis is associated with a decreased risk of Alzheimer’s disease. The study by Boyd et al, published in the Journal of Alzheimer’s Disease, investigated whether the increased expression of GM-CSF (granulocyte-macrophage colony stimulating factor) in rheumatoid arthritis might play a part in this protective effect. They treated normal mice and Alzheimer mice with GM-CSF and examined the effect on the development of amyloid plaques, a prominent characteristic of Alzheimer’s that is caused by the extracellular accumulation of amyloid protein in the brain tissue. The authors also evaluated the performance of treated and untreated mice in a series of cognitive tests.
GM-CSF treatment reduced amyloid plaque development and improved cognitive performance. Cognitive tests were performed on normal mice and Alzheimer mice, both divided into treated and untreated groups. GM-CSF treated Alzheimer mice performed as well as normal mice on the cognitive tests, suggesting that the treatment was actually able to reverse previous damage.
The fact that GM-CSF is already approved for use in humans is another boon. This report states that clinical trials in Alzheimer’s patients may begin this year. I hope that this turns out to be one case of a discovery in mice that delivers results in humans as well. Up until now, there hasn’t been much good news for Alzheimer’s patients and their families.
Here’s the paper:
Boyd TD, Bennett SP, Mori T, Governatori N, Runfeldt M, Norden M, Padmanabhan J, Neame P, Wefes I, Sanchez-Ramos J, Arendash GW, & Potter H (2010). GM-CSF upregulated in rheumatoid arthritis reverses cognitive impairment and amyloidosis in Alzheimer mice. Journal of Alzheimer’s disease : JAD, 21 (2), 507-18 PMID: 20555144