A breakthrough in Parkinson’s disease research came to light this week when researchers reported successfully growing stem cells from the skin of a patient with a rapidly progressing form of the disease. The cells, which mimic the features of Parkinson’s, should help scientists study the disease more accurately, investigate why certain nerve cells die, and find out which compounds reduce expression of the proteins behind the disease. Their report was published online on 23 August in the journal Nature Communications.
The study was led by the University of Edinburgh in collaboration with University College London (UCL), and made possible by a £300,000 grant from the charity Parkinson’s UK.
The researchers took skin samples from a patient diagnosed with one of the most progressive forms of Parkinson’s disease. Compared with the general population, people with this form of the disease have twice as many of the genes that code for the protein alpha-synuclein, and can be diagnosed in their early 30s.
From the skin cells the team produced induced pluripotent stem cells that could then be differentiated into neurons or brain cells. The resulting neurons produced twice the amount of alpha-synuclein compared to those from a family member without the disease, showing that the generated neurons retained features of the disease.
The researchers anticipate that the ability to make these neurons will make it much easier to test the effectiveness of new drugs designed to slow or halt Parkinson’s disease.
Senior author Dr Tilo Kunath, of the Medical Research Council Centre for Regenerative Medicine at the University of Edinburgh, told the press that the study provides an “ideal platform” to help better understand Parkinson’s disease and discover disease-modifying drugs.
“Current drugs for Parkinson’s alleviate symptoms of the condition. Modelling the disease in a dish with real Parkinson’s neurons enables us to test drugs that may halt or reverse the condition,” said Kunath.
Lead author Dr Michael Devine, from UCL’s Institute of Neurology, said understanding the progressive form of Parkinson’s will give insight to all different forms. The protein alpha-synuclein is implicated in nearly all types of the disease.
“As this type of Parkinson’s progresses rapidly it will also make it easier to pick up the effects of drugs tested to prevent nerve cells targeted by the disease from dying,” said Devine.
Dr Kieran Breen, Director of Research and Development at Parkinson’s UK, said even though the genetic mutation in this study results in the progressive form of the disease, the research is potentially a “huge breakthrough” for Parkinson’s disease.
“This is just the kind of innovative research that Parkinson’s UK is committed to funding as we move closer to a cure,” said Breen.
Written by Catharine Paddock PhD