Dopamine-producing cells created from induced pluripotent stem cells and transplanted into rats with Parkinson's disease, successfully alleviated symptoms in a recent study.
Parkinson's disease is a neurodegenerative disorder characterised by the loss of neurons which produce the neurotransmitter dopamine. When these neurons die, patients experience motor deficits due to loss of dopamine in the brain. While drug treatments are available to replace the dopamine lost, tolerance often builds up reducing the efficacy of these drugs over time. US researchers have now developed a cell therapy in which induced pluripotent stem cell derived progenitor cells can differentiate into the neurons that create dopamine.
'Patients with Huntington's disease or multiple system atrophy or even Alzheimer's disease could be treated in this way for specific aspects of the disease process,' said senior author Dr Jeffrey Kordower.
The study, published in Nature Regenerative Medicine, determined that cells created from induced pluripotent stem cells transplanted into rats with Parkinson's after 17 days of differentiation, worked better at alleviating symptoms of the disease compared to those left to differentiate for 24 or 37 days. Immunocompromised rats were used to prevent the rats' immune system rejecting the donor cells.
Researchers from the Arizona State University determined that the more cells applied, the faster the rats recovered from their motor deficits. This was due to the cells being able to produce tyrosine hydroxylase – the enzyme responsible for making dopamine.
The findings from this proof-of-concept study are soon to be put to the test in a clinical trial of patients with a specific type of Parkinson's disease caused by a mutation in the Parkin gene.
'We cannot be more excited by the opportunity to help individuals who suffer from this genetic form of Parkinson's disease, but the lessons learned from this trial will also directly impact patients who suffer from sporadic, or non-genetic forms of this disease,' Dr Kordower said.
Sources and References
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Optimising maturity and dose of iPSC-derived dopamine progenitor cell therapy for Parkinson's disease
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Designer neurons offer new hope for treatment of Parkinson's disease
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Stem cell therapy set for human trials after reversing Parkinson's in rats
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Designer neurons bring hope for treatment of Parkinson's disease
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Aiming to treat Parkinson's by replacing neurons, Aspen Neuroscience nabs $147M
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