New UK trial aims to find answers more quickly for Alzheimer's disease

Multi-site trial will test several drugs simultaneously and drop those not showing clear benefit.

An innovative national trial has been launched that could cut years from the search for treatments that work for people with Alzheimer’s disease, the most common type of dementia. Minister of State for Health and Social Care Alison McGovern MP marked the launch with a visit to the trial site at Charing Cross Hospital in London. 

The AD-SMART trial is led by the UK Dementia Research Institute (UK DRI) at Imperial College London and by Alzheimer’s Research UK, in partnership with the National Institute for Health and Care Research (NIHR), the Medical Research Council (MRC) and Health Data Research UK (HDR UK). Ongoing funding has been committed by the UK government. 

Alzheimer’s affects around 600,000 people in the UK and there are currently no NHS treatments that can slow or stop the underlying disease.  

Applying lessons from cancer to dementia 

AD-SMART (Alzheimer's disease Systematic Multi-arm Adaptive Randomised Trial) will test multiple drugs simultaneously, with separate groups of participants each beginning treatment with a different drug. Unusually, all groups will be compared with one single group of participants taking a placebo.  

Researchers will examine whether the medicines can lessen symptoms such as memory and thinking problems and help people maintain their quality of life as the disease progresses. As the trial continues, regular checking of results will allow any drug that doesn't show clear benefit to be dropped quickly and replaced with another candidate.  

For too long, Alzheimer’s research has moved at the pace of one drug, one trial, one answer, sometimes taking a decade or more before we know if we’ve found something that works. We are applying advances from cancer and other conditions to accelerate testing of treatments for people with Alzheimer’s disease. Professor Paresh Malhotra Study lead, Group Leader at the UK DRI Centre for Care Research & Technology at Imperial College London

The trial will start with two repurposed medicines already approved for use to treat type 2 diabetes and attention deficit hyperactivity disorder (ADHD), Because the drugs are already approved for other conditions and have a well-established safety profile, if they prove successful, they could reach people living with Alzheimer’s much faster than a medicine developed from scratch. 

As well as repurposed drugs, the trial is also designed to test new potential treatments coming through the research pipeline.  

Study lead Professor Paresh Malhotra, Group Leader at the UK DRI Centre for Care Research & Technology at Imperial College London, said: “For too long, Alzheimer’s research has moved at the pace of one drug, one trial, one answer, sometimes taking a decade or more before we know if we’ve found something that works. We are applying advances from cancer and other conditions to accelerate testing of treatments for people with Alzheimer’s disease." 

Professor Siddharthan Chandran, Director of the UK Dementia Research Institute, said: “We already know this model works. The SMART approach, used by us in Motor Neurone Disease in MND-SMART, has shown that testing multiple drugs at once, in one smart trial design, can speed up the testing of new treatments for neurodegenerative conditions. Now, we’re doing the same for Alzheimer’s." 

“And because of the way SMART trials are designed, biotech and pharmaceutical companies can also bring their drugs straight into the trial. That means more treatments tested more quickly, and for people living with Alzheimer’s, that could make all the difference.” 

Breakthroughs that change people’s lives 

Dementia is one of the most pressing medical and societal challenges, affecting almost one million people in the UK. According to Alzheimer’s Society, this number is projected to rise to 1.4 million by 2040. 

The UK Prime Minister, Andy Burnham MP, has spoken publicly of his own father's battle with Alzheimer's disease. He said of the trial: “I know how it feels to lose a loved one to dementia and have seen first-hand the devastating impact this illness has on families. That’s why I’m proud to see this trial being launched in the UK, so that cutting-edge treatments can reach patients here as soon as possible." 

He added: “I have made dementia research a national priority. I want Britain to be the leading nation, making the breakthroughs that change people’s lives.” 

UK Science Minister Chris McDonald MP said: “AD-SMART will help to uncover new treatments faster by exploring how existing drugs might be used to treat dementia, giving hope that we might be able to find ways to improve patients’ quality of life in the future.” 

Dr Sheona Scales, Director of Research at Alzheimer’s Research UK, said: “For people living with Alzheimer’s disease, time is precious. We urgently need more treatment options that can help people live better for longer and stay connected to the people they love." 

I have made dementia research a national priority. I want Britain to be the leading nation, making the breakthroughs that change people’s lives. Andy Burnham MP The UK Prime Minister

AD-SMART is designed to find effective treatments sooner. As a multi-arm, multi-stage platform trial, by testing several drugs at once against a single shared placebo group, more participants receive an active treatment than would do in a traditional trial.  

Results are reviewed at regular stages, and drugs showing no benefit are dropped and replaced with new ones, meaning that fewer people receive ineffective treatments. Sharing one placebo group can also more than halve the time needed to evaluate treatments, which reduces costs. 

Minister’s visit 

On Wednesday 21 October, Alison McGovern MP, Minister of State at the Department of Health and Social Care, visited the AD-SMART trial site at the Imperial Memory Unit, Charing Cross Hospital, part of Imperial College Healthcare NHS Trust. During the visit, the Minister met Professor Paresh Malhotra, a trial participant and members of the AD-SMART trial team. 

The trial is being delivered by a team at Imperial College London, the UCL Innovative Clinical Trials Unit and the MRC Centre of Research Excellence in Clinical Trial Innovation (UCL), the University of Oxford and Dementias Platform UK (DPUK), with support from leading Alzheimer’s experts across the UK. 

More: UK DRI at Imperial