Hot on the heels of Friday’s blog – Sniffing Out Alzheimer’s, British scientists just announced a major breakthrough that could, one day, result in a treatment for Alzheimer’s, Parkinson’s, Huntington’s and other neurodegenerative diseases.
In tests on mice, researchers from the toxicology unit of the Medical Research Council showed brain cell death from prion disease could be prevented.
Professor Roger Morris, from King’s College London, said: “This finding, I suspect, will be judged by history as a turning point in the search for medicines to control and prevent Alzheimer’s disease.”
It is rare to get cautious scientists keen to describe any study as a turning point in history, let alone a study in mice.
Not only is it is early science, a lot can go wrong between a drug for mice and a drug for humans and the only published data is for prion disease, not even Alzheimer’s.
So why the excitement?
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.
The research team focused on the natural defense mechanisms built into brain cells. When a virus hijacks a brain cell it leads to a build-up of viral proteins. Cells respond by shutting down nearly all protein production in order to halt the virus’s spread.
However, many neurodegenerative diseases involve the production of faulty or “misfolded” proteins. These activate the same defenses, 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 thought to take place in many forms of neurodegeneration, so safely disrupting it could treat a wide range of diseases.
The researchers used a compound which prevented those defense mechanisms kicking in and in turn halted neurodegeneration.
The study showed mice with prion disease developed severe memory and movement problems. They died within 12 weeks. However, those given the compound showed no sign of brain tissue wasting away.
Lead researcher Professor Giovanna Mallucci says: “They were absolutely fine, it was extraordinary. What’s really exciting is a compound has completely prevented neurodegeneration and that’s a first. This isn’t the compound you would use in people, but it means we can do it and it’s a start.“
She said the compound offered a “new pathway that may well give protective drugs” and the next step was for drug companies to develop a medicine for use in humans.
Side effects are an issue. The compound also acted on the pancreas, meaning the mice developed a mild form of diabetes and lost weight. Any human drug would need to act only on the brain.
David Allsop, professor of neuroscience at Lancaster University described the results as “very dramatic and highly encouraging.”
SRxA’s Word on Health agrees. We look forward to seeing further research and how these findings could apply to diseases such as Alzheimer’s and Parkinson’s.
SRxA's Word on Health 