Alzheimer's Treatment within Reach after Successful Drug Trial

Friday, 11th November 2016

There are 850,000 people with dementia in Britain, and this figure is anticipated to reach a million within a decade. Alzheimer’s is the most common form of the condition but hopes have recently been raised that, with the development of a new drug - verubecestat - which targets a key element of the disease, an effective treatment could at last be possible.

Trials have suggested that the drug – a product of the pharmaceutical company Merck – ‘switches off’ the toxic amyloid proteins, the production of which leads to the sticky plaques seen in the brains of Alzheimer’s patients. What is not yet known but will be the focus of future trials is whether these brain plaques are the cause of the disease, or just a symptom.

If verubecestat is indeed shown to successfully slow the pace of mental decline, it could be the first new treatment for Alzheimer’s to be licensed in more than a decade.

A spokesman for Merck said: ‘There are very limited therapeutic options available for people with Alzheimer’s disease, and those that exist provide only short-term improvement to the cognitive and functional symptoms. They do not directly target the underlying disease processes.’

Many scientists believe that Alzheimer’s is caused when accumulating proteins kill off healthy neurons, eventually degrading memory and cognition, and causing changes to personality. The new therapy is designed to prevent this process.

32 patients with early stage Alzheimer’s received the drug, called daily for a week. Healthy volunteers were also given the drug. Samples taken from the fluid surrounding the brain showed the drug had reduced the levels of two compounds that are known to contribute to the development of abnormal amyloid proteins.

Merck has, however, issued a word of caution. Whilst they believe verubecestat is successfully targeting the build-up of plaques in the brain, they are as yet uncertain as to whether this will necessarily convert into cognitive benefits for patients. ‘What we have to be worried about’, they say, ‘is that the plaques have set off other pathologies: that it is too late.’

Rosa Sancho, head of research at Alzheimer’s Research UK, welcomed ‘a wave of potential new treatments currently being tested for dementia, with the results of these studies hotly anticipated over the course of the coming months and years.’

Previous attempts to develop similar drugs have foundered largely due to side-effects, such as liver toxicity and eye problems but the new drug appears to have few side-effects and it will be the first of its kind to reach this stage of development. Trials will include well in excess of three thousand patients, with the disease at different stages of development. Initial results are expected next summer.