Clinical innovation for neurodegenerative diseases

Abstract

Neurodegenerative diseases, including Alzheimer’s disease (AD), Parkinson’s disease, amyotrophic lateral sclerosis and Huntington’s disease, are devastating diseases, with huge unmet medical needs. Although it has been estimated that the pharmaceutical industry has already spent billions of US dollars on developing a treatment for AD, the output so far has been dismal. We, like many others, hope that this long history of failures will soon be replaced by success. Whilst symptomatic treatments are valuable, especially those with clearly observable functional outcomes (e.g., l-DOPA in Parkinson’s disease), disease modification in terms of halting, slowing or preventing the evolution of neurodegenerative disorders remains the Holy Grail and no disease modifier product has yet been approved in any neurodegenerative disorder – with the possible exception of riluzole for amyotrophic lateral sclerosis. High cost, long timelines and low probability of success have led to reluctance by the pharmaceutical industry to invest in this area, despite the huge rewards that a successful treatments would reap [1]. Some companies have already opted to leave this area; others have decided to ‘reef the sail’. In contrast, publically funded efforts are growing. During the first decade of the 21st century, AD national plans were launched in a variety of different European countries. The US President signed the National AD Project Act in January 2011. The first European-wide Neurodegenerative Disease Research was launched in 2012. In 2013 the NIH total expenditure on AD research will reach an all-time high of US $529 million. A common belief is that public–private partnerships (PPPs) will generate breakthroughs in preclinical and clinical areas [2]. The Innovative Medicine Initiative (IMI) represents one such collaboration, which joins European governments and pharmaceutical companies together to speed up the development of better and safer medicines for patients. The IMI PharmaCog project was launched in 2010 and is already reporting data related to improved early preclinical and clinical experimental designs for predicting the cognitive properties of new drug candidates [101].