Corporate Banner
Satellite Banner
Scientific Communities
Become a Member | Sign in
Home>News>This Article

Study Leads to Alzheimer's Breakthrough

Published: Thursday, October 10, 2013
Last Updated: Thursday, October 10, 2013
Bookmark and Share
Researchers at the Medical Research Council Toxicology Unit have used an orally-administered compound to block a major pathway leading to brain cell death in mice, preventing neurodegeneration.

The team had found previously that the build up of misfolded proteins in the brains of mice with prion disease over-activates a natural defence mechanism in cells, which switches off the production of new proteins. This mechanism would normally switch back ‘on’ again, but in these mice the continued build-up of misshapen protein keeps the switch turned ‘off’. This is the trigger point leading to brain cell death, as the key proteins essential for nerve cell survival stop being made. 

Originally, the team injected a protein that blocked the ‘off’ switch of the pathway into a small region of the brain, and by doing this were able to restore protein production, and halt the neurodegeneration. The brain cells were protected, and protein levels and synaptic transmission (the way in which brain cells signal to each other) were restored allowing the mice to live longer. This led the scientists to predict that compounds able to block this pathway would also protect brain cells.

In the new study, published in Science Translational Medicine, the researchers gave by mouth a drug-like compound against the pathway to prion infected mice, hoping to block the off-switch in the same way.  The compound, which had originally been developed by GlaxoSmithKline for a different purpose, was able to enter the brain from the bloodstream and halt the disease, throughout the whole brain. However, this compound, despite protecting the brain, also produced weight loss in the mice and mild diabetes, due to damage to the pancreas.*

The researchers studied mice with prion disease because these mouse models currently provide the best animal representation of human neurodegenerative disorders in which the build up of misshapen proteins is linked with brain cell death.  These include Alzheimer’s and Parkinson’s as well as prion diseases.  Another paper in Nature Neuroscience last month highlighted this pathway as a potential therapeutic target in treating Alzheimer’s.

Professor Giovanna Mallucci, who led the team, said, “Our previous study predicted that this pathway could be a target for treatment to protect brain cells in neurodegenerative disease.  So we administered a compound that blocks it to mice with prion disease. We were extremely excited when we saw the treatment stop the disease in its tracks and protect brain cells, restoring some normal behaviours and preventing memory loss in the mice.

“We’re still a long way from a usable drug for humans – this compound had serious side effects. But the fact that we have established that this pathway can be manipulated to protect against brain cell loss first with genetic tools and now with a compound, means that developing drug treatments targeting this pathway for prion and other neurodegenerative diseases is now a real possibility.”

Professor Hugh Perry, chair of the Medical Research Council's Neuroscience and Mental Health Board, said, “Misshapen proteins in prion diseases and other human neurodegenerative disorders, such as Alzheimer’s and Parkinson’s, also over-activate this fundamental pathway controlling protein synthesis in the brains of patients. Despite the toxicity of the compound used, this study indicates that, in mice at least, we now have proof-of-principle of a therapeutic pathway that can be targeted. This might eventually aid the development of drugs to treat people suffering from dementias and other devastating neurodegenerative diseases.”

Further Information

Join For Free

Access to this exclusive content is for Technology Networks Premium members only.

Join Technology Networks Premium for free access to:

  • Exclusive articles
  • Presentations from international conferences
  • Over 3,500+ scientific posters on ePosters
  • More Than 5,200+ scientific videos on LabTube
  • 35 community eNewsletters

Sign In

Forgotten your details? Click Here
If you are not a member you can join here

*Please note: By logging into you agree to accept the use of cookies. To find out more about the cookies we use and how to delete them, see our privacy policy.

Related Content

Genetic Signature Linked to Cancer Prognosis Identified
The results of the analysis of 8,161 tissue samples could in the future help clinicians decide how best to treat a patient as well as aid the development of new targeted treatments.
Monday, October 10, 2016
Potential Urine Test for CJD
Researchers at the Medical Research Council (MRC) Prion Unit at UCL have found that it may be possible to determine whether or not a person has sporadic Creutzfeldt-Jakob Disease (sCJD) by testing their urine for the presence of abnormal prion proteins.
Tuesday, October 04, 2016
Genes Essential to Life Discovered
Genes critical for life are discovered in humans and mice as part of large-scale phenotyping study.
Thursday, September 15, 2016
Gene Linked to Hearing Loss Identified
Researchers have identifed a gene associated with age-related hearing loss.
Monday, August 22, 2016
World's Most In-Depth Study to Detect Alzheimer's Disease
A multisite team will see the most thorough and vigorous testing for Alzheimer's ever performed on volunteers.
Monday, August 22, 2016
Discovery of Key Component of HIV Yields New Drug Target
Scientists from the MRC Laboratory of Molecular Biology in Cambridge and University College London have discovered an essential feature of HIV that the virus uses to infect cells whilst avoiding detection by the immune system. This discovery presents a new drug target and the opportunity to re-evaluate existing treatments for HIV to improve their efficacy.
Thursday, August 11, 2016
MRC Technology, Alzheimer’s Association Collaborate
MRC Technology (MRCT), an independent medical research charity based in London, and the Alzheimer’s Association in Chicago have entered into an agreement to review and monitor the Association’s grant-funded research portfolio.
Thursday, January 21, 2016
Study Shows Blocking Brain Inflammation Could Help Alzheimer's
The research was jointly funded by the Medical Research Council (MRC) and Alzheimer’s Research UK.
Saturday, January 09, 2016
A Fundamental Protection Mechanism Against Formalin In Mammals is Revealed
Formaldehyde, or formalin, is well known to all of us as a common chemical used in many industrial processes and also as a preservative, remarkably we also produce formaldehyde in our bodies.
Wednesday, September 30, 2015
Liver Regrown from Stem Cells
Scientists have repaired a damaged liver in a mouse by transplanting stem cells grown in the laboratory.
Wednesday, July 22, 2015
MRC, GSK and Five Leading UK Universities Collaborate
Collaboration to crack difficult disease areas.
Thursday, July 16, 2015
‘Mini Bile Ducts’ used to Discover New Drugs that could Prevent Liver Damage
An experimental cystic fibrosis drug has been shown to prevent the disease’s damage to the liver, thanks to a world-first where scientists grew mini bile ducts in the lab.
Tuesday, July 14, 2015
First RNAi Meiosis Screen Reveals Genes Essential to Generate Eggs
Screening techniques developed leading to the discovery of genes essential for meiosis in mammals.
Wednesday, July 08, 2015
Study Identifies New Way to Kill the Malaria Parasite
Scientists have discovered new ways in which the malaria parasite survives in the blood stream of its victims, a discovery that could pave the way to new treatments for the disease.
Tuesday, July 07, 2015
Making Vaccines More Effective In The Elderly
Compound shown to restore the immune system’s inbuilt memory.
Tuesday, November 11, 2014
Scientific News
How it Works: Advanced Data Analysis Using Visualization
Visualisation of data can be used to help molecular biologists tackle the vast datasets their experiments create.
Unravelling the Role of Key Genes and DNA Methylation in Blood Cell Malignancies
Researchers from the University of Nebraska Medical Center have demonstrated the role of Dnmt3a in safeguarding normal haematopoiesis.
Salford Lung Study - The First Real World Clinical Trial
In this podcast, we learn about the Salford Lung Study and its potential to revolutionize the way we assess new drugs and treatments around the world.
NIH Researchers Unveil New Wound-Healing Role for Protein-Folding Gene in Mice
The study found that topical treatment of an Hsp60-containing gel dramatically accelerates wound closure in a diabetic mouse model.
50-Year-Old Bacteria Could Be Alternative Treatment Option for Cancer
Researchers have developed a non-toxic strain of Salmonella to penetrate and target cancer cells.
Promising Blood Test Fails to Yield Clues About Best Strategies for Bladder Cancer Treatment
Penn Medicine research challenges previous findings on utility of neutrophil-to-lymphocyte ratio as a biomarker.
Fighting Cancer with the Power of Immunity
Researchers at MIT have used a combination of four different therapies to activate both of the immune system’s two branches, producing a coordinated attack that led to the complete disappearance of large, aggressive tumors in mice.
A Simple Tool for Clinical and Postmortem Toxicological Analysis
In this study, GC-MS is used for the determination of clozapine, and five antidepressants in human plasma, serum and whole blood.
Identification of Individual Red Blood Cells by Raman Microspectroscopy
In this study, Raman Microspectroscopy was used to identify individual red blood cells.
Influential Cancer Researcher Receives Agilent Thought Leader Award
Biologist Scott Lowe receives award in recognition for his contributions to cancer biology.
Scroll Up
Scroll Down
Skyscraper Banner

Skyscraper Banner
Go to LabTube
Go to eposters
Access to the latest scientific news
Exclusive articles
Upload and share your posters on ePosters
Latest presentations and webinars
View a library of 1,800+ scientific and medical posters
3,500+ scientific and medical posters
A library of 2,500+ scientific videos on LabTube
5,200+ scientific videos