Injection to halt the progress of Alzheimer’s 'available within decade'

Henry Bodkin
An injection to the spine which prevents certain forms of the disease taking hold could be available in under 10 years - Getty Images Contributor

An injection capable of halting the progress of Alzheimer’s could be available to patients within a decade, Britain’s leading dementia organisation predicts.

The Alzheimer’s Society says a series of recent breakthroughs in treatments that disrupt harmful genes has brought scientists to a “tipping point” in their fight against the disease.

For decades, researchers have sought without success a treatment for Alzheimer’s based on targeting damaging proteins that build up in the brain.

However, the “remarkable” results of a recent trial which set out to silence the troublesome genes which regulate proteins in children with a rare spinal condition, has convinced scientists they could adopt the same approach in people at high risk of dementia

In an interview with The Daily Telegraph, Dr James Pickett, head of research at the Alzheimer’s Society, said an injection to the spine which prevents certain forms of the disease taking hold could be available in under 10 years.

The treatment, using so-called “molecular scissors”, would not alter a person’s fundamental genetic code, but rather the way specific genes known to play a role in dementia communicate.

Such a drug would principally benefit around 18,000 people in the UK with a high risk of hereditary Alzheimer’s, approximately 2 per cent of the overall population of those with the degenerative disease.

These include early onset Alzheimer’s which can affect people as early as in their thirties.

There is currently no cure for any form of the disease.

“2019 is a tipping point for dementia gene therapy,” said Dr Pickett.

“There are lots of different pieces of the puzzle coming together.

“We’ve got all of this genetic knowledge, like cancer researchers did 30 years ago, and we’re now investing in understanding it and exploiting it.”

Currently scientists know of 25 genes which significantly increase a person’s risk of Alzheimer’s, up from just one in 2012.

Researchers increasingly believe that the expression of these genes could be manipulated by means of CRISPR gene editing technology, which exploits a process used by the immune system to cut up the DNA of invading bacteria.

The technology gained widespread publicity last month after He Jiankui, a Chinese scientist, claimed to have used it to help create the world’s first genetically-edited babies.

The development would have been illegal in the UK and was roundly condemned by the scientific community.

By contrast, the proposed Alzheimer’s treatments would be in the form of “messenger therapies”, targeting the way genes regulate the activity of damaging proteins, such as tau and Apo, in the brain.

Children suffering from spinal muscular atrophy who were given that style of treatment are currently still able to walk and are not relying on a ventilator, years after they were expected to have progressed to a debilitated state.

Similarly, a gene therapy trial at University College London aimed at reducing the levels of toxic protein in Huntington disease patients has also returned promising results.

“What we learn from one disease we can take on to another,” said Dr Pickett.

“There is going to be a leap of faith moment.

“There is a global network of people with dominantly inherited Alzheimer’s disease genes and these unfortunate people are very willing to take part in trials.”

He said the priority should be to attract CRISPR scientists currently working on the technology in other fields, such as fertility, to turn their attentions towards dementia in order to improve its precision.

Dr Pickett added that for non-familial or “sporadic” Alzheimer’s disease, the form suffered by the majority of patients which usually develops after 65, preventative treatments remain “further away”.

However, he pointed to a current trial targeting tau which concludes in January 2020 as a potential building block for more broadly applicable therapies.

The new-found optimism follows a series of blows to Alzheimer’s research over recent years where high-profile trials returned disappointing results.

In January the pharmaceuticals giant Pfizer announced it was pulling out of research and development into an Alzheimer’s drug.

There are currently around 850,000 people in the UK living with some form of dementia.

Around one in 14 over the age of 65 will develop the disease, with one in six over the age of 80.

Case study: Husband diagnosed with Alzheimer's at 36

Daniel was diagnosed with dementia in September 2017 after experiencing problems with his short term memory

Last year, Daniel Bradbury, 31, was diagnosed with a rare form of Alzheimer’s caused by a mutation in the gene called PSEN1.

He inherited the gene from his father, Adrian, who died from the condition himself aged just 36.

Daniel struggles with his mobility and short-term memory as a result of his condition, and is now unable to work his previous job as a landscape gardener. 

He has also experienced changes to his behaviour and mood.

Daniel’s wife, Jordan - a medical photographer - said: “It breaks my heart to see my husband so unwell. For Daniel, it's mentally exhausting. His brain is dying and he's mentally exhausted by that. It's kind of mental torture for you too because you're seeing your loved one decline.”

“I wouldn't wish this condition on anybody. It's this massive wedge and elephant in the room and you try and have these non-dementia days but it's here, you see it, you hear it, you feel it in our house. It's like this horrible dark cloud over our family all the time.”

There is a 50 per cent chance that Daniel and Jordan's two-year-old twins, Lola and Jasper, have inherited the PSEN1 gene.

Jordan is worried not only for her husband, but also for her children.

Jordan believes that if something doesn’t change in the development of Alzheimer treatment, history will simply repeat itself. "I'm going to be caring for my children who will have Alzheimer's in their early thirties. I could lose my entire family unit before I retire. That for me is a very scary feeling, a very scary reality,” she adds.

"I want there to be something available for my children should they need it.”

The hope for many Alzheimer sufferers and their relatives is that a cure will eventually be found, not just a treatment to slow down the disease.

“Treatments are great, but for me, as somebody who is watching their partner live with the condition and manage it every day, nothing short of a cure will do.”