Global partnerships to meet the dementia challenge


By | Monday 29 September 2014

Eleven more years. That is the G8 summit declaration; a cure or disease-modifying treatment for dementia by 2025. Fortunately, our scientists are not short on the drive or inspiration needed to step-up to this challenge and push forward with their pioneering research. Here, at Alzheimer’s Research UK, we fund biomedical research to find a cure. However, financial support is not the only face to our work. We are committed to backing initiatives that will bring us closer to delivering effective treatments for people with dementia as soon as possible. That is why we have joined the Coalition Against Major Diseases (CAMD), led by the Critical Path Institute in the USA.

Coalition against Major Diseases

We’re committed to backing initiatives that bring us closer to delivering effective treatments as soon as possible.

Sounds like a gang of superheroes doesn’t it? While wearing lab coats and suits, not spandex, the CAMD is an international team that brings together funding bodies, pharmaceutical companies, academics and drug regulators with one core goal: to accelerate clinical trials for neurodegenerative diseases, starting with Alzheimer’s and Parkinson’s. As we have previously blogged about, there is a high failure rate of Alzheimer’s drugs in clinical trials; of over 400 drugs that have entered patient trials, less than 100 have reached the end phase of the process. None of these that made the finish line yielded a cure for Alzheimer’s.

There are several potential reasons for these failures and we would like to address as many of them as possible through our research funding and policy work. Now, we want to join the CAMD to tackle the issue of clinical trial design and drug regulation.

Fail to plan, plan to fail

When a pharmaceutical company, or a group of academics, plans a clinical trial, it is essential that they state what they are looking for before the trial begins. For example, the organisers will predict that memory and thinking problems will improve in a trial for a new Alzheimer’s drug. If this prediction is not met, then the trial will have failed.

It is important to state what you predict at the beginning of an experiment, so that you can’t ‘cherry pick’ outcomes based on the results. So, as a far-fetched example, if memory problems do not improve but all patients suddenly become really good at singing, the researchers cannot pretend that this was the original intention. Making accurate predictions is also essential if the effects of drugs are to be properly detected and analysed.

Where does the CAMD come in? Every clinical trial has a placebo group; a set of patients given a dummy pill, so that the effect of the drug can be compared to a baseline. The CAMD has managed to get data on the placebo groups from lots of different trials and companies, which is no easy feat. By combining all of this data, the CAMD has built up an idea of what Alzheimer’s progression is actually like when no active treatment is used. This means that when designing a clinical trial, scientists can make more accurate predictions about the influence of their drug. More accurate predictions mean a greater chance of scientists being able to measure a real benefit from a new drug when they analyse their data.

Selecting the right tools for the job

Shaking up clinical trial design also involves looking at what biological changes are measured during the course of a trial. As well as looking at the memory and thinking skills of those in the trial, researchers will take blood samples, cerebrospinal fluid and do brain scans to look at any biological changes the drug causes. The CAMD will look at all these different tests, to work out which are the most suitable for detecting the effect of a drug. Do blood levels of toxic proteins indicate how well a drug is working or is it better to do a brain scan? This information will improve the design of trials, again hopefully allowing real benefits of a potential new drug to shine through. As we fund lots of research into biomarkers of disease, we are very keen to make sure that these can be of use in clinical trials to find a cure for dementia.

We are delighted to join this partnership. It is essential that the researchers we fund work together, to share their knowledge, and highlight potential biological pathways to target for treatment. Equally important is that we work with others to create a more transparent drug discovery process, whether that be in the trial design or the regulation of drugs that reach the market.

Our global Dementia Consortium, in collaboration with MRC Technology, Eisai and Lilly aims to couple academics with industrial drug discovery experts to improve the translation of promising dementia breakthroughs into potential new drugs. Through our involvement in CAMD we will accelerate and improve the next stage, that of clinical trial development to see a cure for dementia by 2025.

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Team: Science news