Is this a cure for Alzheimer's? Gene therapy could help to prevent brain-clogging plaques from forming

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Alzheimer's disease could be stopped by gene therapy, scientists discovered.

Inserting a gene may prevent the formation of clumps of protein which are found in the brains of dementia sufferers, experts believe.

The plaques - built up of amyloid-beta peptide - are thought to trigger the death of brain cells.

But the PGC-1-alpha gene helped to prevent the lumps of protein and kept mice with a healthy brain, a new study found.

Inserting the PGC-1-alpha gene into the brains of mice helped to stop protein build-ups - found in the brains of dementia patients - from forming, experts discovered

It is involved in metabolic processes in the body, including the regulation of sugar and fat. 

Experts hope the findings may lead to new treatment for dementia for millions of people around the world.

Imperial College London researchers used a modified virus to deliver the PGC-1-alpha gene to brain cells.

They injected the virus into two areas of the brain in mice susceptible to Alzheimer's disease. 

The animals were treated at the early stages of Alzheimer's disease, when they still had not developed amyloid plaques.

The areas targeted were the hippocampus and the cortex, as these are the first regions to develop amyloid plaques in Alzheimer's disease.

The plaques - built up of amyloid-beta peptide (pictured yellow) - are thought to trigger the death of brain cells and cause dementia

Damage to the hippocampus affects short-term memory, and leads to a person forgetting recent events, such as a conversation or what they ate for breakfast.

The hippocampus is also responsible for orientation, and damage results in a person becoming lost on familiar journeys, such as driving home from the shops.

The cortex is responsible for long-term memory, reasoning, thinking and mood.

Damage can trigger symptoms such as depression and struggling to work out simple everyday tasks. 

After four months, the mice who received the gene had very few protein build-ups, compared with the untreated mice, who had multiple plaques in their brain.

The treated mice also performed as well in memory tasks as healthy mice including challenges such as replacing a familiar object in the mouse's cage with a new one.

It was also discovered there was no loss of brain cells in the hippocampus of the mice who received the gene treatment.

They also had a reduction in the number of glial cells, which in Alzheimer's disease can release toxic inflammatory substances that cause further cell damage.

Lead researcher Dr Magdalena Sastre hopes the new findings may one day provide a method of preventing the disease, or halting it in the early stages.

She explained: 'Although these findings are very early they suggest this gene therapy may have potential therapeutic use for patients. 

Scientists hope the findings may lead to new treatment for dementia for millions of people around the world (stock)

'There are many hurdles to overcome, and at the moment the only way to deliver the gene is via an injection directly into the brain. 

'However this proof of concept study shows this approach warrants further investigation.

'We are still years from using this in the clinic. However, in a disease that urgently needs new options for patients, this work provides hope for future therapies.'

Dr Doug Brown, from the Alzheimer’s Society, said: 'It’s still early days for this gene therapy approach.

'While it has shown promise in mice, we’re still not sure whether this will be suitable for people. 

'Using gene therapy could prove to be useful further down the line in dementia research and we look forward to seeing if this will be an effective way of developing future treatments.'

Dr David Reynolds, from Alzheimer’s Research UK, said: 'There are currently no treatments able to halt the progression of damage in Alzheimer’s, so studies like this are important for highlighting new and innovative approaches to take us towards that goal. 

'This research sets a foundation for exploring gene therapy as a treatment strategy for Alzheimer’s disease, but further studies are needed to establish whether gene therapy would be safe, effective and practical to use in people with the disease. 

'The findings support PGC-1-alpha as a potential target for the development of new medicines, which is a promising step on the road towards developing treatments for this devastating condition.'

Dementia affects 47.5 million people across the world - of which Alzheimer's is the most common form.

The Alzheimer's Society estimate one in three people over 65 will develop dementia and numbers will grow as the population ages

There is no known cure, but current drugs can help to treat symptoms of the disease.

The study was published in the journal Proceedings of the National Academy of Sciences.