Designing drugs to target brain disorders has many challenges. Drugs have to be designed so that they can cross the blood-brain barrier before entering the brain. There is a lack of pre-clinical models that accurately recapitulate the main features of human brain diseases leading to difficulty in translating findings to humans. In addition, drug discovery for neurodegenerative diseases presents an extra challenge as significant neuron loss occurs before symptoms appear, meaning clinical trials have often been performed on populations in which the disease has already had a large effect.

Given these challenges, from 2009 - 2014 there was a 52% reduction in the number of brain-focussed drug development programs run by large pharmaceutical companies. In 2018, Pfizer completely pulled out of drug development research for dementia.

In response, research groups at universities and smaller start-up biotech companies have developed new approaches to developing drugs for brain disorders. The international chemistry news site ‘Chemistry World’ recently interviewed Prof Michael Kassiou on new research strategies he is utilising to advance drug discovery for neurodegenerative diseases, including using a phenotypic approach to screen drug candidates.

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