Alzheimer’s disease: reviewing the immediate treatment horizon

Stuart Goodman | 2nd September 2016

With a current market valuation at $6.5 billion, and a projected direct medical cost of $1.1 trillion by 2050, Alzheimer’s disease (AD) is an area of huge unmet need.¹ There is enormous commercial potential for pharmaceutical companies to address the challenge of AD sooner rather than later, and we see many contenders racing to be the first to do so.

Unfortunately, the complexity of the disease has meant that many recent AD trials have been unsuccessful. However, the learnings from these ‘failures’ have paved the way for the robust pipeline of treatments that could potentially be coming to market in the next five years.

A recent article by BLH Consultant David Cooney explored the bleak history of Alzheimer’s disease trial failures since the turn of the century. In it, he highlighted how, despite continued investment by pharmaceutical companies into AD treatment research, AD sufferers still only have 5 FDA-approved treatments to choose from, and even these are only capable of slowing the progression of disease symptoms.

However, every trial that has been unsuccessful has been a guiding hand to the scientific community, allowing those working extensively in the disease area to progress our understanding and develop new molecules for application in new trials and investigations. In this article, Account Manager Stuart Goodman takes a look at some of the promising categories of Phase 3 treatments that could soon be available to patients with AD.

The continued focus on a beta-amyloid solution 

Whilst there is increasing evidence that AD is a complex and multi-factorial disease, many scientists continue to believe that beta-amyloid (Aβ) is the main pathogenic factor responsible for the degenerative changes that occur in the brain during AD. Consequently, manipulating the amount of Aβ in the brain still represents one of the most attractive approaches for disease intervention in AD. We can see this reflected in the five-year Phase 3 pipeline, where Aβ immunotherapy drugs, small-molecule BACE inhibitors and anti-Aβ aggregation agents are all undergoing late stage testing.

Crenezumab, gantenerumab, and solanezumab are all involved in additional prospective longitudinal investigations – the Alzheimer’s Prevention Initiative (Phase 2), the Dominantly Inherited Alzheimer Network study (Phase 2/3), and the Anti-Amyloid Treatment in AD Prevention Trial (Phase 3) respectively. These investigations are ongoing, and will examine whether anti-amyloid drugs can prevent or delay disease onset in individuals with a high chance of developing AD (as determined by genetic predisposition or PET brain scans). They represent an important shift of research focus to earlier in the disease pathway, and will go a long way toward supporting or refuting the concept of the amyloid hypothesis. 



  1.  Qian X, et al. Nature Reviews Drug Discovery. 2015;14:675-76.


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