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.”

Start your day with The Independent, sign up for daily news emails
News
US comedian Bill Mahr
people
Voices
Winston Churchill, then prime minister, outside No 10 in June 1943
voicesA C Benson called him 'a horrid little fellow', George Orwell would have shot him, but what a giant he seems now, says DJ Taylor
Sport
football
News
Rob Lowe
peopleRob Lowe hits out at Obama's snub of Benjamin Netanyahu
PROMOTED VIDEO
Have you tried new the Independent Digital Edition apps?
ebooks
ebooksA year of political gossip, levity and intrigue from the sharpest pen in Westminster
News
Davies (let) says: 'Everybody thought we were having an affair. It was never true!'
people'Our friendship flourished online. You can share some very revelatory moments at four in the morning…'
Arts and Entertainment
Over their 20 years, the band has built a community of dedicated followers the world over
music
News
Staff assemble outside the old City Road offices in London
mediaThe stories, the writers and the changes over the last quarter of a century at Britain's youngest paper
Life and Style
The Oliver twins, Philip and Andrew, at work creating the 'Dizzy' arcade-adventure games in 1988
techDocumentary looks back at origins of the computer-games industry
Arts and Entertainment
Krall says: 'My hero player-singer is Elton John I used to listen to him as a child, every single record
music
News
Friends for life … some professionals think loneliness is more worrying than obesity
scienceSocial contact is good for our sense of wellbeing - but it's a myth that loneliness kills, say researchers
Arts and Entertainment
The Wu-Tang Clan will sell only one copy of their album Once Upon A Time In Shaolin
musicWu-Tang Clan and The Sexual Objects offer fans a chance to own only copies of their latest albums
News
i100
Environment
Number so freshwater mussels in Cumbria have plummeted from up to three million in the 20th century to 500,000
environment
Life and Style
Models – and musicians – on the catwalk in Dior Homme for the men’s 2015/16 fashion show in Paris
fashionAt this season's Paris shows, various labels played with the city boys' favourite
News
i100
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

Recruitment Genius: Sales Consultant - Solar Energy - OTE £50,000

£15000 - £50000 per annum: Recruitment Genius: Fantastic opportunities are ava...

Recruitment Genius: Compute Engineer

Negotiable: Recruitment Genius: A Compute Engineer is required to join a globa...

Ashdown Group: PHP Web Developer / Website Coordinator (PHP, JavaScript)

£25000 - £28000 per annum + 25 days holidays & pension: Ashdown Group: PHP Web...

Recruitment Genius: Estates Projects & Resources Manager

£35000 - £40000 per annum: Recruitment Genius: Based in London, Manchester, Br...

Day In a Page

Iraq invasion 2003: The bloody warnings six wise men gave to Tony Blair as he prepared to launch poorly planned campaign

What the six wise men told Tony Blair

Months before the invasion of Iraq in 2003, experts sought to warn the PM about his plans. Here, four of them recall that day
25 years of The Independent on Sunday: The stories, the writers and the changes over the last quarter of a century

25 years of The Independent on Sunday

The stories, the writers and the changes over the last quarter of a century
Homeless Veterans appeal: 'Really caring is a dangerous emotion in this kind of work'

Homeless Veterans appeal

As head of The Soldiers' Charity, Martin Rutledge has to temper compassion with realism. He tells Chris Green how his Army career prepared him
Wu-Tang Clan and The Sexual Objects offer fans a chance to own the only copies of their latest albums

Smash hit go under the hammer

It's nice to pick up a new record once in a while, but the purchasers of two latest releases can go a step further - by buying the only copy
Geeks who rocked the world: Documentary looks back at origins of the computer-games industry

The geeks who rocked the world

A new documentary looks back at origins of the computer-games industry
Belle & Sebastian interview: Stuart Murdoch reveals how the band is taking a new direction

Belle & Sebastian is taking a new direction

Twenty years ago, Belle & Sebastian was a fey indie band from Glasgow. It still is – except today, as prime mover Stuart Murdoch admits, it has a global cult following, from Hollywood to South Korea
America: Land of the free, home of the political dynasty

America: Land of the free, home of the political dynasty

These days in the US things are pretty much stuck where they are, both in politics and society at large, says Rupert Cornwell
A graphic history of US civil rights – in comic book form

A graphic history of US civil rights – in comic book form

A veteran of the Fifties campaigns is inspiring a new generation of activists
Winston Churchill: the enigma of a British hero

Winston Churchill: the enigma of a British hero

A C Benson called him 'a horrid little fellow', George Orwell would have shot him, but what a giant he seems now, says DJ Taylor
Growing mussels: Precious freshwater shellfish are thriving in a unique green project

Growing mussels

Precious freshwater shellfish are thriving in a unique green project
Diana Krall: The jazz singer on being friends with Elton John, outer space and skiing in Dubai

Diana Krall interview

The jazz singer on being friends with Elton John, outer space and skiing in Dubai
Pinstriped for action: A glimpse of what the very rich man will be wearing this winter

Pinstriped for action

A glimpse of what the very rich man will be wearing this winter
Russell T Davies & Ben Cook: 'Our friendship flourished online. You can share some very revelatory moments at four in the morning…'

Russell T Davies & Ben Cook: How we met

'Our friendship flourished online. You can share some very revelatory moments at four in the morning…'
Bill Granger recipes: Our chef serves up his favourite Japanese dishes

Bill Granger's Japanese recipes

Stock up on mirin, soy and miso and you have the makings of everyday Japanese cuisine
Michael Calvin: How we need more Eric Cantonas to knock some sense into us

Michael Calvin's Last Word

How we need more Eric Cantonas to knock some sense into us