Dopaminergic neurons for Parkinson's therapy

http://www.nature.com/nbt/journal/v30/n1/full/nbt.2077.html?WT.ec_id=NBT-201201


* Olle Lindvall1

DOI:
doi:10.1038/nbt.2077


A differentiation protocol guided by developmental principles produces more-authentic dopaminergic neurons for transplantation in patients.

A recent report in Nature by Studer and colleagues2 describes the conversion of human embryonic stem cells (hESCs) into substantia nigra dopaminergic neurons that ameliorate Parkinson's disease symptoms in animal models without forming tumors. From the clinical perspective, this new differentiation protocol, which generates large numbers of transplantable dopaminergic neurons of the correct phenotype, represents a major advance toward the first application of hESC-derived dopaminergic neurons for grafting in patients.

A critical issue for clinical translation is safety. The protocol for generating dopaminergic neurons should be fully chemically defined, and the components of animal origin eliminated. The potential for graft-induced dyskinesias after transplantation should be assessed in appropriate animal models.