New on the BBC web site today (10th October 2013). The discovery of the first chemical to prevent the death of brain tissue in a neurodegenerative disease has been hailed as an exciting and historic moment in medical research. In tests on mice, the University of Leicester showed all brain cell death from prion disease could be prevented. Professor Giovanna Mallucci of the MRC Toxicology Unit at Leicester says the hope is to arrest the process of brain cell death.
This process is thought to take place in many forms of neurodegeneration, so safely disrupting it could treat a wide range of diseases. Hopefully, a resulting medicine could treat Alzheimer’s, Parkinson’s, Huntington’s and other diseases. More work is needed to develop a drug that could be taken by patients.
The team at the university’s Medical Research Council Toxicology Unit focused on the natural defence mechanisms built into brain cells. However, many neurodegenerative diseases involve the production of faulty or “misfolded” proteins. These activate the same defences, but with more severe consequences. The misfolded proteins linger and the brain cells shut down protein production for so long that they eventually starve themselves to death. This process, repeated in neurons throughout the brain, can destroy movement or memory or even kill, depending on the disease.
It is the first time that any form of neurodegeneration has been completely halted, so it is a significant landmark. It shows that the process being targeted has serious potential. If it can be successfully developed, which is not guaranteed, the prize would be huge.
In Parkinson’s the alpha-synuclein protein goes wrong, in Alzheimer’s it’s amyloid and tau, in Huntington’s it’s the Huntington protein. But the errant protein is irrelevant here as the researchers are targeting the way a cell deals with any misfolded protein.
It means one drug could cure many diseases and that really would be something to get excited about.
Oral Treatment Targeting the Unfolded Protein Response Prevents Neurodegeneration and Clinical Disease in Prion-Infected Mice
Science Translational Medicine 9 October 2013: Vol. 5, Issue 206, p. 206ra138 DOI: 10.1126/scitranslmed.3006767