Diabetes and Parkinson’s Disease – Stem Cells May Make Treatment Possi…

Diabetes and Parkinson’s Disease – Stem Cells May Make Treatment Possi…

Parkinson’s disease (PD) is a degenerative disorder affecting the central nerve system. A person with Parkinson’s disease may develop muscular rigidity, tremors, and changed speech patterns. The person may also have problems using language and lose higher cognitive roles. PD may cause a person to move very slowly (bradykinesia) and may cause loss of balance. The disease is progressive and chronic. PD is not fatal, but ultimately harsh muscular problems may cause pneumonia, choking, and falls which may consequence in death. PD affects approximately 150 out of every 100,000 Caucasian individuals, and is slightly less common among African-Americans.

Parkinson’s disease is caused by decreased activity of dopamine (DA)-secreting nerve cells located in the substantia nigra (“black substance”) of the brain. DA is a neurotransmitter involved in the regulation of muscular activity in addition as various neuropsychiatric roles including cognition and behavior. A shared medical treatment for PD is L-Dopa which is converted into DA by dopaminergic neurons in the substantia nigra. Administration of L-Dopa attempts to replace the body’s supply of DA. The drug is not an optimal therapy – only a small percentage is converted to DA and the drug causes many side-effects.

PD is an ideal candidate for stem cell treatment. bright research by Wernig et al.1 (conducted in the Jaenisch laboratory at the Whitehead Institute for Biomedical Research) demonstrated successful treatment of PD in adult rats using neurons derived from stem cells. These authors derived iPS cells by reprogramming rat connective tissue cells. The stem cells were then transformed into neuronal cell types and transplanted into the brains of adult rat models of PD. The nerve cells were successfully transplanted and led to functional recovery in eight of nine rats. Importantly, no cancers that might have been caused by the transplants were detected up to eight weeks following transplantation.

An additional exceptional study was reported recently by Zhou et al.2 at the Harvard Stem Cell Institute. Using an in vivo approach in adult mice, this team directly converted mouse pancreatic exocrine cells into pancreatic endocrine β-cells. The team used an adenovirus to transfect the exocrine cells with a specific combination of transcription factors, effectively reprogramming the exocrine cells into endocrine β-cells. This dramatically breakthrough demonstrates that it is possible in certain circumstances to avoid the necessity of reprogramming a cell to an embryonic pluripotent state. Zhou et al showed that transdifferentiation is possible by using a cocktail of lineage-specific transcription factors.

Much work is needed to be done, of course. however the work of both these research teams points to exciting new possibilities for treatment of these devastating diseases.

The field of regenerative medicine is progressing at an astonishing rate. Hallowed concepts of embryology and development are being revised, nearly on-the-fly, yearly if not monthly. We are living in very exciting times.

1 Wernig M, et al: Neurons derived from reprogrammed fibroblasts functionally integrate into the fetal brain and enhance symptoms of rats with Parkinson’s disease. Proc Natl Acad Sci USA 105(15):5856-5861, 2008

2 Zhou Q, et al: In vivo reprogramming of adult pancreatic exocrine cells to beta-cells. character 455(7213):627-632, 2008

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