In my clinical neuropsychology course, our professor alerted us to a newly-published article with potentially huge implications. Anyone who has known someone with Alzheimer's Disease (AD)understands just how devastating it is. Currently, there are no known cures for neurodegenerative diseases such as AD and Parkinson's Disease (PD) - only drugs that can slow down the process of degeneration. An article published in the journal Science Translational Medicine by Moreno et al. (2013) may represent a huge step toward treatment of these diseases.
At a basic level, several neurodegenerative diseases are thought to be due to accumulations of misfolded proteins in brain cells, which result in the discontinuation of protein synethesis. If cells aren't producing protein, they die, resulting in neurodegeneration. Using this knowledge, researchers
infected mice with prion disease (another neurodegenerative disease), and then orally administered a drug which inhibits the function of a protein kinase that mediates the process of shutting-down protein synthesis. Thus, the goal was not to get rid of the misfolded proteins, but to allow the synthesis of new proteins to continue despite their presence.
Amazingly, prion-infected mice treated at both early and later stages of the disease were completely free of confirmatory clinical signs of prion disease after treatment. Some of the mice did exhibit some early-indicator signs, however. Control mice, who were infected with prion but not treated, were all terminally sick at this point.
The drug did have some side effects. Most significantly, the treated mice experienced weight loss of 20% of their body mass. In addition, their blood sugar was elevated (but not to the point of diabetes). The authors note, however, that problems such as these are managed relatively routinely, and that any negative effects would need to be weighed against the potential benefits of protecting our brains. I certainly think most people would tolerate some weight loss and elevated blood sugar in order to keep their brains intact (although 20% weight loss is alarming).
These findings are extremely significant in that we now have a drug that is known to stop the process of neurodegeneration in mice and potentially humans as well. However, there are still many hurdles to jump before translating the treatment to humans. Researchers note that because long-term treatment would be needed in humans, fine-tuning of these drugs is crucial. Specifically, research is needed to determine when the drug needs to be administered to be most effective and how to minimize serious side effects. In addition, clinical trials on humans, once begun, will take years. Although there are still many challenges to overcome, these results suggest it is likely that 20-somethings like myself will see treatments for diseases such as AD and PD in our lifetimes.
Moreno, J. A., Halliday, M., Molloy, C., Radford, H. Verity, N., Axton, J. M., . . . Mallucci, G. R. (2013). Oral treatment targeting the unfolded protein response prevents neurodegeneration and clinical disease in prion-infected mice. Science Translational Medicine, 5 (206), 1-10.