Scientists turn off Alzheimer’s protein in human brain cells

Scientists have succeeded in neutralising a gene associated with Alzheimer’s disease in human brain cells for the first time. The researchers, from the biomedical research organisation Gladstone Institutes in San Francisco, turned off a protein linked to the apoE4 gene.

In doing this, they have prevented it from being able to damage nerve cells, which in turn leads to dementia. It is hoped that the move could pave the way for treatment options in patients at high risk of Alzheimer’s, before the condition becomes too advanced.

Approximately one in four people carry the apoE4 gene and having just one copy of it doubles the risk of developing Alzheimer’s. Those who have two copies see their chances of being diagnosed with the condition increase by 12 times.

Study participants with two copies of apoE4 helped lead to the breakthrough, as nerve cells donated by them were analysed by scientists. They were then compared to cells from those who did not have the gene.

So far, the compound that turns off the protein responsible for apoE4 has only been tested on cells in laboratory conditions. Plans are underway to improve the substance and make it safe for testing on patients.

Lead author Yadong Huang said: “Drug development for Alzheimer's disease has been largely a disappointment over the last ten years.”

With 46.8 million people in the UK living with dementia, and Alzheimer’s being the most common form, the battle is on to find a cure. Despite a large number of institutions being involved in research and big pharmaceutical companies funding studies, there has not yet been an effective treatment brought to market.

Many of the drugs that seem to have potential in the lab are unable to realise it when it comes to clinical trials. One of the reasons for this, it is thought, is down to treatment beginning too late in the development of the disease, when brain cells have already died.

Research that leads to a better understanding of Alzheimer’s and earlier diagnosis is key to tackling the condition, but is not a solution on its own. A huge body of work is being undertaken, helping to build up a clearer picture of the challenges involved in tackling a situation that is slowly becoming an epidemic.

One of the most promising drugs going through trials at present is taken as a tablet and known as LMTX. It has been developed by scientists at the universities of Oxford and Aberdeen and contains a protein that dissolves the protein tangles in the brain that clump together and form plaques.

So far, it has been tested on 800 patients from 12 different countries, with good results. Subjects have seen their brains recover so much that in nine months their MRI scans bear a resemblance to those of healthy people.

Patients who have been taking LMTX have been able to carry out everyday tasks that are usually difficult for people with Alzheimer’s. They include bathing and dressing themselves, as well as remembering the date and correctly naming objects.

While more research into the drug needs to be done, initial signs are good and being able to live an independent life for longer could make a huge difference to Alzheimer’s sufferers of the future.