Discovery ‘could hold key to Alzheimer’s
treatment’
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
