The ability to effectively treat brain disease, including the neurodegenerative diseases such as Dementia, Alzheimer's, Parkinson's, and Huntington's disease, together with neurodysfunctional disorders such as bipolar disorder, epilepsy and migraine, represents a major challenge.
Any approach to therapy must entail a thorough understanding of brain function and how different kinds of nerves and support tissues that make up the brain function normally, as well as insights into why things go wrong. Only then can we begin to think about the best way to develop new therapies aimed at a cure, or at least alleviating the symptoms.
Successful development of drugs aimed at targetting brain disease also needs candidate drugs to be tested at an early stage on patient-derived nerve cells. While it is not feasable to isolate brain cells from patients, the development of induced puripotent stem cell (iPSC) technologies have enabled any cell from a patient to be reprogrammed into a nerve cells that can be used to study the disease and for drug testing.
This approach has started to uncover the defects in neurological disorders and point to ways in which drugs might be used to restore brain function and alleviate symptoms.
The identification of neuronal stem cells has also led to the prospect that adult brain tissue can be repaired, either by activating resident stem cells, or by implanting stem cells that would then restore damaged tissue.