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Stem cells and regenerative medicine 

Stem cells and regenerative medicine

Stem cells and regenerative medicine

Alexis J. Joannides

, Roger Pedersen

, and Siddharthan Chandran

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date: 24 April 2017

There is a great and unmet need for treatments that will deliver restorative solutions to patients with diseases hitherto considered irreparable. The twin properties of self-renewal and specialization common to stem cells offer unprecedented opportunities for regenerative medicine through the generation of unlimited numbers of defined human cells on the scale necessary for experimental study, drug discovery, and disease modelling. The field is fast developing, with the first wave of putative treatments necessarily drawn from autologous and adult stem cells, but the outstanding question remains whether these will lead to meaningful regenerative therapies.

Requirements for regenerative therapy

(1) A prerequisite for any regenerative therapy is the generation of scalable and enriched numbers of defined cell types appropriate to the target condition. The application of developmentally based insights offers the most rational route to generate functional cell types, and this approach is starting to yield success. (2) Preclinical work-up requires demonstration of sustained stem-cell mediated functional recovery in appropriate models of injury and this remains in its infancy, although for some diseases considerable progress and proof of concept has been achieved. (3) General principles include the need for ensuring appropriate distribution, connectivity, survival, and function of stem cells in the context of injury, without the hazards of tumour generation or immune rejection.

How might the promise of stem cells be realized?

(1) Consideration of three major target conditions for regenerative medicine—Parkinson’s disease, heart failure, and diabetes—emphasize distinct and common challenges that must be overcome in order to realize the stem cell promise. (2) The emergence of novel approaches to induce pluripotency from differentiated somatic cells, along with insights based on human embryonic stem cells and increased recognition of endogenous stem cells, offers a range of mechanisms through which stem cells may be therapeutic. (3) In addition to classic cell/tissue replacement approaches, the ability of stem cells that include patient-specific material to model disease and enable drug discovery is likely to lead to significant therapeutic advances through the promotion of endogenous repair.

How long will it take before stem cell treatments are available?

The time it will take to deliver clinically useful stem cell treatments will vary from disease to disease and reflects the need for cell-based therapeutics to be competitive against established treatments. The history of haematological stem cell medicine, from which much of the template of regenerative medicine is borrowed, suggests an incremental and combinatorial approach to treatment, emphasizing the need for sophisticated clinical trial design to ensure correct clinical evaluation of putative regenerative stem cell based therapies.

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