Cell transplantation for Huntington's disease: Should we continue?

Cell transplantation for Huntington's disease has developed over the last decade to clinical application in pilot trials in the USA, France and the UK. Although the procedures are feasible, and under appropriate conditions safe, evidence for efficacy is still limited, which has led to some calls that further development should be discontinued. We review the background of striatal cell transplantation in experimental animal models of Huntington's disease and the rationale for applying similar strategies in the human disease, and we survey the present status of the preliminary studies that have so far been undertaken in patients. When we consider the variety of parameters and principles that remain poorly defined – such as the optimal source, age, dissection, preparation, implantation, immunoprotection and assessment protocols – it is not surprising that clinical efficacy is still unreliable. However, since these protocols are all tractable to experimental refinement, we consider that the potential for cell transplantation in Huntington's disease is greater than has yet been realised, and remains a therapeutic strategy worthy of investigation and pursuit.