Cellular therapy for childhood neurodegenerative disease. Part II: clinical trial design and implementation

Neurosurg Focus. 2008;24(3-4):E23. doi: 10.3171/FOC/2008/24/3-4/E22.

Abstract

Cellular replacement therapy attempts to improve functioning of the diseased human central nervous system (CNS). In this second installment of a 2-part review, the authors discuss the major challenges to the translation of in vitro and animal studies of neural stem cell (NSC) therapy in the clinical setting. This analysis details the problems unique to the design of clinical trials using human NSCs, outlines patient selection practices, describes surgical techniques for cellular transplantation, and reviews the regulatory issues and ethical concerns in trials involving neurologically impaired children.

Publication types

  • Review

MeSH terms

  • Child
  • Clinical Trials as Topic*
  • Humans
  • Neurodegenerative Diseases / therapy*
  • Neurons / physiology
  • Stem Cell Transplantation / ethics
  • Stem Cell Transplantation / methods*
  • Stem Cells / physiology*