Alzheimer’s treatment breakthrough: British scientists pave way for simple pill to cure disease

Historic ‘turning point’ hailed as UK researchers discover how to halt death of brain cells, opening new pathway for future drug treatments

Scientists have hailed an historic “turning point” in the search for a medicine that could beat Alzheimer's disease, after a drug-like compound was used to halt brain cell death in mice for the first time.

Click image above to view graphic

Although the prospect of a pill for Alzheimer's remains a long way off, the landmark British study provides a major new pathway for future drug treatments.

The compound works by blocking a faulty signal in brains affected by neurodegenerative diseases, which shuts down the production of essential proteins, leading to brain cells being unprotected and dying off.

It was tested in mice with prion disease - the best animal model of human neurodegenerative disorders - but scientists said they were confident the same principles would apply in a human brain with debilitating brain diseases such as Alzheimer's or Parkinson's.

The study, published today in the journal Science Translational Medicine, was carried out at the Medical Research Council's (MRC) Toxicology Unit at the University of Leicester.

“It's a real step forward,” team leader Professor Giovanna Mallucci told The Independent. “It's the first time a substance has been given to mice that prevents brain disease. The fact that this is a compound that can be given orally, that gets into the brain and prevents brain disease, is a first in itself… We can go forward and develop better molecules and I can't see why preventing this process should only be restricted to mice. I think this probably will translate into other mammalian brains.”

In debilitating brain diseases like Alzheimer's, the production of new proteins in the brain is shut down by a build-up of “misfolded proteins” or amyloids. This build-up leads to an “over-activation” of a natural defence mechanism that stops essential proteins being produced. Without these proteins to protect them, brain cells die off - leading to the symptoms of diseases like Alzheimer's.

The compound used in the study works by inhibiting an enzyme, known as PERK, which plays a key role in activating this defence mechanism. In mice with prion's disease, it restored proteins to protect brain cells “stopping the disease in its tracks”, restoring some normal behaviours and preventing memory loss.

Although the compound also produced significant side effects in mice, including weight loss and mild diabetes, which was caused by damage to the pancreas, Professor Mallucci said it would “not be impossible” to develop a drug that protected the brain without the side effects and that work towards doing so had been “very promising”.

The breakthrough was greeted with excitement by scientists, who nonetheless cautioned that it remained a significant proof of principle and a possible basis for new treatments, rather than a guarantee of an Alzheimer's cure in the near future.

Computer graphic of a vertical (coronal) slice through the brain of an Alzheimer patient (credit: Science Photo Library) Computer graphic of a vertical (coronal) slice through the brain of an Alzheimer patient (credit: Science Photo Library)  

Professor Roger Morris, acting head King's College London's department of chemistry, said: “This is the first convincing report that a small drug, of the type most conveniently turned into medicines, stops the progressive death of neurons in the brain as found, for instance, in Alzheimer's disease. True, this study has been done in mice, not man; and it is prion disease, not Alzheimer's, that has been cured.  However, there is considerable evidence that the way neurons die in both diseases is similar; and lessons learned in mice from prion disease have proved accurate guides to attenuate the progress of Alzheimer's disease in patients.”

“From finding the first effective drug in a mouse, to having an effective medicine in man, usually takes decades to bring to fruition, in the very few cases in which it is successful. So, a cure for Alzheimer's is not just around the corner. However, the critical point of principle made by Professor Mallucci's study is that a drug, given orally, can arrest neurodegeneration caused by amyloid in the brain.   

”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.“ 

David Allsopp, professor of neuroscience at Lancaster University said that the study had thrown up ”very dramatic and highly encouraging results“, but said that more research was needed to overcome the "problematic side-effects" and to prove the technique would be effective against other disease like Alzheimer's and Parkinson's.

There are currently 800,000 people in the UK with dementia and Alzheimer's disease is the most common cause. The number of people living with the condition is set to break one million by 2021, and represents an enormous health burden for the NHS and the social care system. Parkinson's affect 1 in 500 people and around 127,000 people suffer from the condition.

Dr Eric Karran, director of research at Alzheimer's Research UK, said: "Targeting a mechanism relevant to a number of neurodegenerative diseases could yield a single drug with wide-reaching benefits, but this compound is still at an early stage. It will be important for these findings to be repeated and tested in models of other neurodegenerative diseases, including Alzheimer's disease. While Alzheimer's is the most common form of dementia, other diseases that cause dementia are also characterised by the abnormal build-up of proteins in the brain.

“If this process is also working overtime in these conditions too, targeting it could be a promising avenue for investigation. However, what is true in animals does not always hold true in people and the ultimate test for this compound will be to see whether it is safe and effective in people with these diseases.”

PROMOTED VIDEO
Have you tried new the Independent Digital Edition apps?
News
ebooksAn unforgettable anthology of contemporary reportage
Latest stories from i100
Have you tried new the Independent Digital Edition apps?
Independent Dating
and  

By clicking 'Search' you
are agreeing to our
Terms of Use.

iJobs Job Widget
iJobs General

Psychology Teacher

Main Teacher Pay Scale : Randstad Education Leeds: Teacher of Psychology An en...

International Promotions Manager - Consumer Products

competitive + bonus + benefits: Sauce Recruitment: A global entertainment busi...

Data Warehouse & Business Intelligence Co-ordinator

£35000 per annum: Ashdown Group: This new opportunity has responsibilities des...

Geography Teacher

£19200 - £33600 per annum + pre 12 week AWR : Randstad Education Manchester Se...

Day In a Page

How could three tourists have been battered within an inch of their lives by a burglar in a plush London hotel?

A crime that reveals London's dark heart

How could three tourists have been battered within an inch of their lives by a burglar in a plush London hotel?
Meet 'Porridge' and 'Vampire': Chinese state TV is offering advice for citizens picking a Western moniker

Lost in translation: Western monikers

Chinese state TV is offering advice for citizens picking a Western moniker. Simon Usborne, who met a 'Porridge' and a 'Vampire' while in China, can see the problem
Handy hacks that make life easier: New book reveals how to rid your inbox of spam, protect your passwords and amplify your iPhone

Handy hacks that make life easier

New book reveals how to rid your email inbox of spam, protect your passwords and amplify your iPhone with a loo-roll
KidZania lets children try their hands at being a firefighter, doctor or factory worker for the day

KidZania: It's a small world

The new 'educational entertainment experience' in London's Shepherd's Bush will allow children to try out the jobs that are usually undertaken by adults, including firefighter, doctor or factory worker
Renée Zellweger's real crime has been to age in an industry that prizes women's youth over humanity

'Renée Zellweger's real crime was to age'

The actress's altered appearance raised eyebrows at Elle's Women in Hollywood awards on Monday
From Cinderella to The Jungle Book, Disney plans live-action remakes of animated classics

Disney plans live-action remakes of animated classics

From Cinderella to The Jungle Book, Patrick Grafton-Green wonders if they can ever recapture the old magic
Thousands of teenagers to visit battlefields of the First World War in new Government scheme

Pupils to visit First World War battlefields

A new Government scheme aims to bring the the horrors of the conflict to life over the next five years
The 10 best smartphone accessories

Make the most of your mobile: 10 best smartphone accessories

Try these add-ons for everything from secret charging to making sure you never lose your keys again
Mario Balotelli substituted at half-time against Real Madrid: Was this shirt swapping the real reason?

Liverpool v Real Madrid

Mario Balotelli substituted at half-time. Was shirt swapping the real reason?
West Indies tour of India: Hurricane set to sweep Windies into the shadows

Hurricane set to sweep Windies into the shadows

Decision to pull out of India tour leaves the WICB fighting for its existence with an off-field storm building
Indiana serial killer? Man arrested for murdering teenage prostitute confesses to six other murders - and police fear there could be many more

A new American serial killer?

Police fear man arrested for murder of teen prostitute could be responsible for killing spree dating back 20 years
Sweetie, the fake 10-year-old girl designed to catch online predators, claims her first scalp

Sting to trap paedophiles may not carry weight in UK courts

Computer image of ‘Sweetie’ represented entrapment, experts say
Fukushima nuclear crisis: Evacuees still stuck in cramped emergency housing three years on - and may never return home

Return to Fukushima – a land they will never call home again

Evacuees still stuck in cramped emergency housing three years on from nuclear disaster
Wildlife Photographer of the Year: Intimate image of resting lions claims top prize

Wildlife Photographer of the Year

Intimate image of resting lions claims top prize
Online petitions: Sign here to change the world

Want to change the world? Just sign here

The proliferation of online petitions allows us to register our protests at the touch of a button. But do they change anything?