Brain and central nervous system (CNS) disorders are seen as one of the final frontiers of modern medicine. However, progress has been slow with drug development in the area declining since the 1990s due to fewer early stage trials being started and a high attrition rate for CNS medicines.1 With this high level of risk, many pharmaceutical companies have been discouraged from pursuing drug development in this area.
The unmet need is stark. More than one billion people worldwide suffer from CNS diseases, with the incidence of neurological disorders growing faster than any other disease class in the US and the EU.1
However, while the number of people living with these conditions grows, clinical advances are not keeping pace.
“At a time when many companies are shifting their focus away from CNS, Takeda is accepting this challenge head-on," said Emiliangelo Ratti, Head of the CNS Therapy Area Unit. “As a core therapeutic area, our mission is to provide medicines for people with psychiatric, neurological and rare CNS disorders for whom there are no treatments available. To do this, we are harnessing the latest scientific breakthroughs, innovative technologies and external partnerships to discover and develop medicines for CNS disorders associated with high unmet need.”
CNS disorders are complex multifaceted conditions that affect the spectrum of mental health. It is therefore vital that Takeda respect this multifaceted nature and take a patient-centric approach to CNS drug discovery and development.
A key aspect for Takeda has been looking at past failures and successes in CNS drug development and using this information to optimize research. Ratti notes that there are a number of aspects that can negatively impact CNS research.
These include; drug targets being poorly related with the biology observed in patients, enrolled CNS patient populations being too heterogeneous, inadequate measurement tools used for assessing changes in disease severity and drug effect and clinical trial design and implementation being too complex and sub-optimal.
These insights have provided Takeda with a foundation to build on our vision of providing medicines for people with psychiatric, neurological and rare CNS disorders for whom there are no treatments available. Ratti notes that in 2017, Takeda will continue to realize this vision with the ultimate aim of improving the lives of people with CNS disorders.
What is the Central Nervous System (CNS)?
The CNS is made up of the brain, the spinal cord, and the optic nerves. The CNS controls thought processes, guides movement, and registers sensations throughout the body.
The CNS is considered separate from the peripheral nervous system (PNS), although the two systems are highly intertwined. The term PNS refers to any part of the nervous system that lies outside of the brain and spinal cord. A major difference between the CNS and PNS involves regeneration. The majority of the PNS has the ability to regenerate, whereas the CNS does not have this capability.
Disorders that affect the CNS are common, involving 25-30% of the population and include neurological and psychiatric disorders, such as Alzheimer’s and Parkinson’s diseases, major depression and schizophrenia.
1.Kesselheim AS, et al. Two decades of new drug development for central nervous system disorders. Nature Reviews Drug Discovery. 2015 14, 815–816.