Participants in the trial, conducted at the UCLH Leonard Wolfson Experimental Neurology Centre (NIHR UCLH Clinical Research Facility), will have the rare inherited form of Alzheimer’s disease.

The drug being looked at – known as E2814 – will target tau protein found in the brain, known to be responsible for disease progression. It is the first time a treatment targeting tau is being trialled in people with the inherited form of Alzheimer’s disease.

The trial is being carried out by the Dominantly Inherited Alzheimer Network Trials Unit (DIAN-TU), an international collaboration sponsored by Washington University School of Medicine to test new experimental therapies for Alzheimer’s. E2814 is the first drug to be tested for their DIAN-TU tau study.

DIAN-TU’s Chief Investigator in the UK is Dr Cath Mummery, consultant neurologist at the NHNN and head of clinical trials at the UCL Dementia Research Centre in the UCL Queen Square Institute of Neurology.

People who inherit mutations that cause Alzheimer’s disease almost invariably develop the cognitive impairments of the disease at an earlier and more predictable age, often developing symptoms around the same age their parents did: in their 50s, 40s and even 30s.

The DIAN-TU tau next-generation program will evaluate three anti-tau drugs in clinical studies and has selected E2814 as the first investigational anti-tau drug. The clinical study aims to determine whether these drugs can slow or stop the progress of Alzheimer’s disease.

Dr Mummery said: “As we’ve learned more about Alzheimer’s, we understand that tau plays a critical role in disease progression alongside amyloid; this is the first anti-tau treatment we will study in these families with genetic forms of Alzheimer’s disease and this important work advances the field towards our goal of discovering an effective treatment for this devastating disease.

“What is gratifying is that this is an example of collaboration between UCLH and UCL, with the candidate drug developed at the Dementia Research Centre and now entering clinical trials at UCLH.”

If it is shown to be effective in the DIAN-TU trial, E2814 may be beneficial for people with the more common, sporadic forms of Alzheimer’s disease in the older population by, at least, slowing further progression of the disease.

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  • National Hospital for Neurology and Neurosurgery, UCLH