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International Clinical Trials Day: The power of clinical research to find a cure

Dr Laura PhippsKirsty MaraisClaire LucasRobin BrisbourneKaty StubbsEd PinchesGlyn Morris

As International Clinical Trials Day conveniently falls in the middle of Dementia Awareness Week, it is the perfect opportunity to talk about how clinical trials are changing the future for dementia.

Dementia doesn’t discriminate between age, ethnicity or wealth, and there are currently 850,000 people living with dementia in the UK. With no new treatments for Alzheimer’s disease in 12 years, and no treatments currently available for many other forms of dementia, it is becoming increasingly important to tackle this condition head-on with high-quality dementia research.

But what exactly is dementia research?

Well, I’m glad you asked! Dementia research aims to defeat dementia by targeting 3 different steps: 1) understanding what the condition is 2) discovering what biological processes are affected and how 3) preventing these processes from going wrong.

We can put a big old tick next to that first one, because we know what dementia is. Dementia is a term used to describe a collection of symptoms that can include memory loss, confusion and an inability to process information. Dementia can be caused by a number of different diseases, some of which will be familiar to you, like Alzheimer’s disease, and others may not, like frontotemporal dementia. The point at which we become a little unstuck is on the next two steps; trying to figure out what is going wrong, and how we can make it right.

The beauty of research is that these next steps can be done in lots of different ways, and if the study is well designed, we will always learn something about what’s going on. This is crucial, because even though the end-game is certainly to have a disease-modifying treatment, the road to this goal cannot only be drug development. It is also vitally important to keep learning about how damage is caused in these diseases to give new clues to how to stop them in their tracks.

So why aren’t all your researchers trying to cure dementia?

As a biomedical research charity, we fund research into four main areas: prevention, treatment, diagnosis and causes of dementia. We need to know the causes so that we can prevent it, and we need to discover ways of diagnosing it earlier, so that we have a better chance of treating it sooner. Understanding how vital brain processes are becoming damaged underpins progress in all of these important areas.

For example, in 2011 Prof Stuart Pickering-Brown was one of a team of world-class scientists who discovered the gene C9orf72 – now known to be the most common genetic cause of frontotemporal dementia. The discovery of this faulty gene has allowed research to progress, so that scientists can investigate more about how it is causing damage. The hope is this research will lead to the development of a treatment that could protect brain cells from C9orf72.

Ok, got it! But with all this research, why aren’t more treatments available?

Even when a promising treatment is developed, it needs to jump through lots of important hoops before it gets to patients. These measures are put in place so that researchers and clinicians can be confident that the treatment helps people, and doesn’t cause any unwanted side-effects.

The media may be tempted to report the next “dementia breakthrough” when a treatment is showing beneficial results in mice, but has yet to get anywhere near a patient, let alone improve their symptoms in a way that can be replicated in everyone with dementia. The confusion that this brings for people who are reliant on the news to inform them about progress within dementia research means that the very people who are most hopeful that a treatment is right around the corner, are the ones that might be left not knowing what to believe.

This becomes more difficult when claims are made about vitamin juices or a specific blend of spices that can ‘cure dementia’, raising hopes often despite a distinct lack of robust research evidence. Anecdotal evidence can often be an important clue to areas of more research, but it doesn’t replace the controlled and measured environment of a research study or clinical trial.

How possible treatments are tested - Click to enlarge

How possible treatments are tested – Click to enlarge

For example, at the University of Cambridge, Dr Hornberger and his team are investigating whether the ‘bonding’ hormone oxytocin can help with some of the challenging behavioural symptoms of frontotemporal dementia. Dr Hornberger’s trial is a double-blind, placebo-controlled trial. This jargon explains how Dr Hornberger is trying to eliminate one of the main factors that could influence the outcome of his results: the placebo effect.

As the trial is ‘double-blind’, neither the patient nor the clinician know who is receiving the hormone, and who is receiving the dummy treatment or ‘placebo’. This is so that if both groups of patients experience the same effect, Dr Hornberger will know that oxytocin doesn’t provide any more benefit that a placebo. It is measures like these that ensure results are accurate and reliable, providing a solid foundation for future research.

This dementia research sounds really interesting, can I get involved?

I thought you’d never ask! Join Dementia Research is a national system that allows people with and without dementia to register their interest in volunteering for research. It gives you the opportunity to take part in research that is happening all across the country, ranging from answering questionnaires and memory tests online to having brain scans, or even taking part in drug trials. To find out more or to register for Join Dementia Research, go to www.joindementiaresearch.nihr.ac.uk, or call our Dementia Research Infoline on 0300 111 5 111 and we will be happy to help you.

If you have any other questions about dementia or dementia research, please do not hesitate to contact us by telephone on 0300 111 5 111 or email at infoline@alzheimersresearchuk.org.

One Response to International Clinical Trials Day: The power of clinical research to find a cure

  1. Dr Robert Peers 25 May 2015 at 4:58 am #

    Unfortunately, if we assume that AD affects all ethnic groups, without finding references on the subject, we are in serious danger of missing or discounting important variations in incidence, that are actually seen in those groups.

    That is, AD may strike a particular ethnic group, like Latinos in Northern Manhattan, but the important part of the story is that they appear to be at much higher risk than Caucasians in the same part of the city. How so?

    The Amish, in America, appear to have no AD at all–apparently only vascular dementia. How so?

    The Nigerian Yoruba people have half the AD risk of genetically similar African Americans in Indiana. How so?

    The Arab community of Wadi Ara, in Israel, are at high risk. How so?

    The annual incidence of AD in Pittsburgh is 4 times higher than in the carefully matched Ballabgarh district in Northern India [near New Delhi]. How so?!!!!

    The true and inquisitive epidemiologist looks at both place and time, and Dr Gunter Robert Jones claims to have found AD to be almost unknown in the 1800s, from his intense study of old medical journals and texts, and autopsy results. So is AD a new disease?

    Finally, what are we to make of twin studies, when one reads about one identical twin NOT getting the disease? [See PubMed: ROBERTSON E AND DAVIDSON E].

    Surely the answer to what causes AD lies in finding the answers to these questions!!!

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