Regenerative Medicine: Could Parkinson's be the First Neurodegenerative Disease to be cured?

Abstract

Parkinson's disease is one of the most insidious neurodegenerative diseases in developed countries. Today, human pluripotent stem cells are produced from embryonic or adult cells, multiplied, differentiated into neural cell lines and ultimately transplanted into disease animal models or patients. Nowadays, DOPAminergic neurons derived from human pluripotent stem cells and human parthenogenetic cells are being clinically tested in China and Australia, respectively. More importantly, good manufacturing practices have been developed and the neurons obtained have been successfully tested in nonhuman primates by teams in Europe, USA and Japan. However, there is a need for translational clinical studies with small molecules tested in vitro, as well as testing of the the efficacy of additional therapies.

Lay abstract

The high prevalence of sporadic cases of Parkinson's disease is a serious health problem in developed societies. A safe regenerative therapy is urgently needed, and is among the most promising treatments. In several years, we can expect to see results from clinical studies using stem cells from across the globe. It is probable that the definitive cure will require more than one therapeutic approach, as discussed in this review.

Graphical abstract

The success of regenerative medicine depends on the restoration of organic homeostasis. Removing prion-like proteins from the body is especially important in sporadic cases of Parkinson's disease, which accounts for 90% of cases. Small molecules identified as enhancers of neural stem cell regeneration should be tested. Reduced doses could potentially be achieved, and unwanted side effects avoided, by a nasal application.

Keywords:

• clinical trials
• DOPAminergic precursors
• glycobiology
• human embryonic SC
• human induced pluripotent SC
• human parthenogenetic neural SC
• human pluripotent SC
• neurodegeneration
• Parkinson's
• prion-like diseases
• regenerative medicine

Acknowledgments

The author greatly appreciates English writing support provided by MT Martín Sánchez.

Financial & competing interests disclosure

The author is a state employee of the Spanish government's Ministry of Science, Innovation and Universities. The author has no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.