The failure rate of clinical trials for Alzheimer’s – why we need to raise our game


By Dr Simon Ridley | Thursday 03 July 2014

Dementia is the name for a collection of many different conditions, of which Alzheimer’s disease is the most common. Alzheimer’s is characterised by a gradual decline in memory and changes in behaviour and communication. In the later stages, people often forget their friends and family as well as how to walk and feed themselves and require round-the-clock care. Whilst there are some treatments available that can help with the symptoms of Alzheimer’s, there is currently no cure.

Current estimates are that 820,000 people in the UK are affected by dementia and the ageing population means that this number is expected to rise to over 2 million by 2050. The economic impact of dementia is enormous, costing the UK £23bn a year and £360bn worldwide.

These numbers alone are enough to show why we need to do more to find effective treatments for people with dementia. As the UK’s leading dementia research charity, Alzheimer’s Research UK is funding over £22m of high-quality research projects across the UK, from basic to clinical.

Research focus

Current research focuses on all aspects of dementia, from understanding the molecular changes that occur in the brain through to using animal models and stem cells to study the effects of certain drugs or genetic changes. Alzheimer’s Research UK also supports research into identifying the genetic risk factors behind the condition.

There is also investment at the clinical level, using high-tech scanning equipment to detail brain changes in people with dementia and finding ways to improve diagnosis. As a whole, these different threads of research will come together to increase our understanding of the causes of diseases like Alzheimer’s and reveal the best approaches to help those affected.

The review published in Alzheimer’s Research and Therapy today highlights a key problem – that too many drugs are failing at the clinical trial stage and that we still have a long way to go to find effective new treatments for Alzheimer’s. However we need to learn from these disappointments and use these results to inform research feeding back into the early drug development pipeline.

This study, and others like it, stress the need for governments, both here and overseas, pharmaceutical and charity sectors to increase investment and research into Alzheimer’s disease and other dementias. Dementia research is severely underfunded compared to other conditions and the impact of this lack of investment is highlighted by the statistic that while there are 108 ongoing trials for Alzheimer’s drugs there are 1438 trials for new cancer treatments.

Drug discovery pipeline

One of the major problems that we face is the threat that historic failures in Alzheimer’s trials will discourage industry from continuing to invest in this important area. We need to work hard to ensure that there is a healthy drug discovery pipeline, primed by important work being done in academic labs across the world.

We also need to explore how to incentivise research and development into dementia to encourage the pharmaceutical industry to continue to invest. This may include changing the regulatory environment to provide more generous licensing and exclusivity, and cutting down the time it takes to get approval for clinical trials.

Despite these past failures, I believe the future of Alzheimer’s research is looking stronger than ever. There is increasing awareness of dementia among the public and policy makers, and the G8 Dementia Summit in December put dementia at the top of the political agenda, targeting finding a cure or disease-modifying treatment by 2025.

Last month, Alzheimer’s Research UK launched the ‘Defeat Dementia’ campaign, pledging to raise £100m over the next five years. With the backing of the UK Prime Minister David Cameron, this campaign will see greater investment in research that feeds into all stages of the drug discovery pipeline. Our grant funding and new Stem Cell Research Centre, along with a network of Drug Discovery Institutes housed in academic centres in the UK and beyond, will bring together academic researchers and drug discovery experts to ensure that promising targets are fast-tracked towards the clinic.

We have formed cross-sector collaborations such as the Dementia Consortium to support researchers in developing early findings into validated drug targets, and have launched a £20m Global Clinical Development Fund to support more early stage clinical trials. I firmly believe that programs such as these will keep the need for effective dementia treatments as a key focus of scientific research.

This is a cross blog post with Alzheimer’s Research & Therapy.

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About the author

Dr Simon Ridley

Simon joined Alzheimer's Research UK in January 2009. As Head of Research he was responsible for the delivery of funding programmes. Simon follows new developments in dementia research and is a regular media spokesperson on research matters.

Simon has extensive experience as a researcher and has worked in industry. He studied biochemistry at Trinity College Dublin and obtained his PhD from Cambridge University. He no longer works for Alzheimer's Research UK.