UK scientists have, for the first time, generated live nerve cells from a patient with a rapidly progressing form of Parkinson's disease.
The breakthrough, by a team of researchers from the University of Edinburgh and University College London (UCL), should help scientists to finally understand why these nerve cells (neurons) die. It is hoped that this may then lead to the development of new drug treatments.
Dr Tilo Kunath, from the University of Edinburgh, said: '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'.
The neurons were generated from skin samples taken from a patient with a rare, early onset form of Parkinson's disease. People with this form of the disease are often diagnosed in their early thirties, and its progression is unusually rapid.
Researchers intend to identify the crucial events leading to cell death by comparing these cells with neurons similarly generated from a person without Parkinson's disease.
'Understanding such a progressive form of the disease will give us insight into different types of Parkinson's', said Dr Michael Devine from UCL. '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'.
A genetic anomaly, present in patients with this rapidly progressing form of the disease, means they have three copies of the α-synuclein gene, rather than the usual two. This leads to the production of twice as much α-synuclein protein than normal, which is the key protein implicated in all types of Parkinson's disease. Therefore findings drawn from these cells will be relevant to all forms of the disease.
Dr Kieran Breen from Parkinson's UK, who funded the research, said: 'Although the genetic mutation that leads to this progressive form of Parkinson's is rare, this exciting study has the potential to bring about a huge breakthrough in Parkinson's research'.
The research is published in Nature Communications.