The Global Clinical Trials Fund: drugs and dummies


By Dr Emma O'Brien | Thursday 11 December 2014

Today marks one year since the G8 Dementia Summit. The last year has been a whirlwind of new investment and exciting initiatives, both from Alzheimer’s Research UK and government, all with one aim: to bring us closer to a treatment for dementia. But we mustn’t lose momentum. Today, we are pleased to announce our newest initiative, the Global Clinical Trials Fund. In our hunt to find a cure, it’s essential that we fund the crucial steps that a treatment must take on its way to patients.

Science and scurvy

The first clinical trial was conducted in 1747, by Dr John Lind. He wanted to find a way to treat scurvy, a disease historically associated with pirates and sailors, which we now know to be caused by a deficiency of vitamin C. Although Dr Lind wasn’t the first person to suggest that citrus fruits, rich in vitamin C, could treat scurvy, he was the first to compare potential remedies. He took several groups of sick sailors and gave each group a different ‘treatment’. Those that ate oranges and lemons did better than those who drank half a pint of sea-water, barley water or vinegar.

Getting to grips with the basics

These days, clinical trials are a lot more complicated to run, rarely involve vinegar and can cost millions of pounds. But they are essential for new treatments to reach patients. A lot of the work that we fund focuses on understanding the biological chain of events that lead to Alzheimer’s disease and other causes of dementia. By getting to grips with the basics, our researchers have uncovered key steps in the progression of dementia that could potentially be targeted with drugs. But there are a huge number of steps from promising experiments in cells and animals to seeing a treatment help a loved one. Very few drugs have made it that far, and those that have only treat symptoms, rather than halting the devastating damage to the brain.

Turning eureka moments into treatments

That’s why Alzheimer’s Research UK has launched a Global Clinical Trials Fund (GCTF). We are committed to defeating dementia and that means that we must fund trials that enable groundbreaking discoveries in the lab to one day help the 830,000 people living in the UK with dementia. We have the ambition to raise £20million over five years specifically for clinical trials. The funding will support trials testing new experimental drugs and those exploring the potential for drugs for other conditions to be ‘re-purposed’ to help people with dementia. But this Global Clinical Trials Fund is not just limited to drug trials. We will also fund non-drug ‘intervention’ trials, such as whether exercise in the elderly can improve symptoms.

Unlike Dr Lind’s initial study, today’s clinical trials cover three stages.


Phase I tests the treatment in a small number of healthy people. The aim is to check for dangerous side effects (more of a problem in drug trials compared to intervention trials).

Phase II trials involve a larger number of people, who are affected by the disease. They too look at safety, but also start to check whether the drug or intervention is effective in patients. Researchers may look at whether memory and thinking skills improve and could also carry out brain scans and blood tests to look for changes in the disease course. Importantly, in phase II trials the drug is compared to a dummy drug, known as a placebo. It is well known that people often experience improved symptoms when they think that they are taking medication, something known as the placebo effect. So each trial must compare the drug to a dummy pill. Neither the patients, researchers nor people administering the drug must know who is taking which.

If a drug is safe and shows some signs of being effective in phase II trials, it will enter large-scale testing often with thousands of people and multiple clinical trial sites across the world. This phase III trial is to ensure that the treatment continues to be effective in large groups of the population. These studies often take two to three years or more to complete. This stage is the most expensive to fund.

The Global Clinical Trials Fund will support phase I and phase II trials across the world, as this is where we as a charity think we can make a large impact in our goal to defeat dementia. While phase III trials are frequently funded by commercial organisations, once drugs have shown promise in the earlier trial stages, academics and those working for small or medium enterprises often struggle to get phase I and phase II trials off the ground. By supporting more ideas at this critical stage, we’re pushing breakthroughs closer to the people who need them the most.

From bench to bedside

We are proud to be funding the full spectrum of research; novel science in the lab, early-phase drug discovery via the Dementia Consortium and our network of Drug Discovery Institutes and now taking the most promising ideas further into clinical trials. With your help we can raise the funds we need to make sure that the treatments we are all so desperate for, reach the people who that need them.


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About the author

Dr Emma O'Brien