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In ‘failure’ there is knowledge

The UK DRI is sorry to learn that Merck is discontinuing a phase 3 clinical trial of its BACE inhibitor drug known as Verubecestat, which was hoped to treat people with prodromal Alzheimer’s disease.

Clinical trials are vital experiments in our quest to treat neurodegenerative diseases and when they fail it is important we understand why.

Although the trial is closing, it’s essential that researchers are given full access to information and analysis generated by it so we can then look closely at the data. We should learn and understand why this trial failed, and then take this knowledge back to the lab bench as we use new approaches to understand dementia.

This latest setback is one of several failures to treat or slow Alzheimer’s disease by targeting amyloid. Because of the complexity of these trials and of the disease itself, we cannot say whether or not the overall approach of targeting amyloid, as in the Merck trial, is still a viable approach. It may be, particularly if we can get to patients long before they have symptoms. It certainly teaches us that simple assumptions of the disease mechanisms that provided the rationale for these trails needs revisiting.  

This and similar trials are a stark reminder of how much we still have to learn about Alzheimer’s disease. That is why the national UK Dementia Research Institute was launched last year – the single biggest investment in dementia research in the UK. We will concentrate on the fundamental nuts and bolts of how neurodegenerative diseases arise and progress, so we can diagnose and treat them early. We recognise there is a huge gap in our knowledge of the biological mechanisms that underpin dementia – and by focusing on this we will identify new targets for treatments.

We’re encouraged to hear that Merck remains committed to developing novel therapies for neurodegenerative diseases. We need to all be in this together to conquer dementia and improve the lives of millions of people.

John Hardy (UK DRI Professor, UCL), Bart De Strooper (UK DRI Director) and Adrian Ivinson (UK DRI COO)