Pluripotent Stem Cells & Neurodegenerative and Ischaemic Brain Diseases: Basic Science to Clinical Applications

Degenerative disorders of the central nervous system (CNS), such as Alzheimer’s disease (AD), Parkinson’s disease (PD) and Huntington’s disease (HD), represent a growing public health challenge worldwide. The ability to accurately diagnose and track neurodegenerative disorders is becoming increasingly important as we enter a new clinical era of disease-modifying therapies. Such treatments may be most effective in the earlier stages of the disease before neurodegeneration becomes too widespread, therefore, it may be important to identify subjects at early stages of the disease. Valid cellular models of these diseases are also needed to examine pathogenesis and test novel treatments at the preclinical stage. The recent advancement in stem cell technology, particularly in pluripotent stem cell biology, has opened up new vistas for neurodegenerative disease research. Both human embryonic stem cells and induced pluripotent stem cells possess the inherent ability to produce all cell types including neuronal tissues in large numbers. The greatest potential for induced pluripotent cells derived from affected individuals is likely to be their utility for modelling and understanding the mechanisms underlying neurodegenerative processes, and for searching for new treatments, including cell replacement therapies. However, much work remains to be done before pluripotent cells can be used for preclinical and clinical applications. In this chapter we discuss briefly, the clinical challenges in early diagnosis of these diseases, the progress so far in the use of stem cells for various neurodegenerative diseases at the levels of discovery, transition and translation, and the challenges of generating specific neural cell subtypes including the recent interest in the use of pluripotent stem cells to model these diseases in the dish. Progress in these areas will substantially accelerate effective application of pluripotent stem cells.

Keywords: Pluripotent stem cells, Cnetral nervous sytem, Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, ischaemic brain, reprogramming, clinical trials.