2011 Annual Report: Novel Stem Cell Technique Shows Promise in Treating Disease


Cell replacement therapy seeks to restore function in the body by replacing cells that are lost due to disease — or treatment for disease, as can occur with certain treatments for cancer — with new, healthy cells. In Parkinson’s disease, this means replacing dopamine cells in the brain, the main type of cell that degenerates in this disease.

In November, Center investigators published a study on a new strategy for using embryonic stem (ES) cells to graft human dopamine neurons into preclinical models of Parkinson’s disease. Historically, ES cells have shown the ability to become dopamine-producing neurons in the test tube, but have not been effective at treating Parkinson’s disease when transplanted into a living organism. However, the new technique has revealed promise in three animal models of Parkinson’s, reflecting the potential for dopamine cells’ survival and function in the brain.