* Banner image courtesy of Dr Sally Lowell - MRC Centre for Regenerative Medicine, University of Edinburgh

A research paper recently published in the Journal of Parkinson's Disease discusses an impending clinical trial in Parkinson's disease (PD) using parthenogenetic stem cells for transplantation into PD patients.

Background:

Stem cell breakthroughs for the treatment of PD in recent years have shown that it is possible to make dopamine cells from embryonic stem cells and transplant them to positive effect into the brain of animal models, replacing cells lost to the disease. Human embryonic stem cells have been manipulated in the laboratory to produce a new generation of dopamine cells that behave like native dopamine cells when transplanted.

These cells then '...functioned equally well as the cells that are naturally found in the brain' ".

Malin Parmar - Lund Stem Cell Center, Sweden

Dopamine cells made from human embryonic stem cells are paving the way for a new treatment for Parkinson's. This potentially unlimited supply, sourced from stem cell lines, has opened the doors to the next step in clinical application - human trials. As with many such exciting research avenues however we must react with caution and evaluate the opportunities provided to PD patients. Without this, the patient community is left trying to interpret complex scientific issues on its own, and individual patients cannot make informed decisions on whether they should seek to participate in the planned trials or not. The authors of the paper (detailed below) have identified several key questions that need discussion ahead of any stem cell-based trial in Parkinson's:

  1. What is being transplanted, and what is the proposed mechanism of action?
  2. What are the pre-clinical safety and efficacy data supporting the use of the proposed stem cell product?
  3. Should arguments concerning ethics, risk-mitigation or trial logistics outweigh concerns regarding the expected efficacy of the cell and constitute a primary justification for choosing one cell type over another in a clinical trial?

Find the answers to these and further questions in the full article : Journal of Parkinson's Disease, vol. 6, no. 1, pp. 57-63, 2016.

Abstract:

'Recent news of an impending clinical cell transplantation trial in Parkinson’s disease using parthenogenetic stem cells as a source of donor tissue have raised hopes in the patient community and sparked discussion in the research community. Based on discussions held by a global collaborative initiative on translation of stem cell therapy in Parkinson’s disease, we have identified a set of key questions that we believe should be addressed ahead of every clinical stem cell-based transplantation trial in this disorder. In this article, we first provide a short history of cell therapy in Parkinson’s disease and briefly describe the current state-of-art regarding human stem cell-derived dopamine neurons for use in any patient trial. With this background information as a foundation, we then discuss each of the key questions in relation to the upcoming therapeutic trial and critically assess if the time is ripe for clinical translation of parthenogenetic stem cell technology in Parkinson’s disease.'

Authors: Roger A. Barker - John van Geest Centre for Brain Repair, University of Cambridge, UK; Malin Parmar, Agnete Kirkeby, Anders Björklund - Wallenberg Neuroscience Center and Lund Stem Cell Center, Sweden; Lachlan Thompson - Florey Institute for Neuroscience and Mental Health, University of Melbourne, Australia; Patrik Brundin - Center for Neurodegenerative Science, Van Andel Research Institute, USA.