NSJ-Spine january 2004

peutic goal and the target of intensive basic and clinical research. Cellular repair of the human CNS poses significant scientific, translational, regulatory, and ethical issues. The challenges in the early trial design and implementation of stem cell–based transplantation to treat neurodegenerative disorders are measurable. Fortunately each area of cell transplantation therapy is being actively addressed by academic and industry groups, and interest continues to grow. This 2-part manuscript will discuss the preclinical and clinical issues involved in the design of a clinical trial testing cellular therapy for the lysosomal storage disorders. Part I will focus on preclinical issues related to host and donor cells, preclinical efficacy, noninvasive monitoring of transplanted stem cells, and immunotolerance. Part II will cover clinical aspects related to the host environment, clinical trial design, regulatory issues, and ethics. Successful Neural Transplantation Requires Choosing the Right Host and Donor Cells