What are the prospects of stem cell therapy for neurology?

BMJ 2008; 337 doi: (Published 28 November 2008) Cite this as: BMJ 2008;337:a1934
  1. Siddharthan Chandran, consultant neurologist
  1. 1Anne McLaren Laboratory for Regenerative Medicine and Cambridge Centre for Brain Repair, Department of Clinical Neurosciences, University of Cambridge, Cambridge CB2 0SZ
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    Effective treatments for neurological diseases have proved elusive. Siddharthan Chandran examines the potential of stem cells to provide the answers

    The use of stem cells for regenerative medicine has captured the public’s imagination and fuelled rising expectations of clinical benefit. Clinicians increasingly need to manage patients’ expectations of what stem cells may, or indeed may not, offer. This article examines some key problems that need to be resolved before the promise of stem cells for regenerative neurology can be realised.

    Regenerative medicine is not a new discipline. It can be summarised as treatments that seek to restore structure and function after injury and, by this definition, includes solid organ transplantation. Stem cells are routinely used in haematology (blood stem cells for malignancy), plastic surgery (cultured autologous keratinocytes for skin loss or burns), and orthopaedics (autologous chondrocyte transplantation for articular cartilage defects1). But their potential in many other conditions has yet to be realised. The lack of restorative treatment for patients with progressive neurological disorders such as Parkinson’s disease, multiple sclerosis, and motor neurone disease highlights the vast unmet clinical need. Stem cells are destined to deliver new treatments for these disorders, through improving the understanding of disease pathogenesis, accelerating drug discovery and, in some instances, by direct cell based therapy. However, several challenges need to be overcome before stem cell based therapies are a clinical reality.

    What are stem cells?

    Stem cells are cells that can both self renew and generate specialised functional progeny. Harnessing these twin properties allows the generation of almost unlimited numbers of cells for experimental study and treatment (fig 1). They are often classified by developmental stage of origin and range of phenotypic potential. Human embryonic stem cells are derived from the inner cell mass of the blastocyst at around four to five days after conception and are …

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