Parkinson’s disease breakthrough with stem cell research

By Dr. Ananya Mandal, MDAug 24 2011

A new stem cell study has raised hopes for therapy in Parkinson's disease. Researchers said they have, for the first time, generated stem cells from one of the most rapidly-progressing forms of the disease. They said the development will boost research into the condition because it will allow scientists to model the disease in laboratories and help them shed light on why certain nerve cells die. The research was led by the University of Edinburgh in collaboration with University College London (UCL).

Parkinson’s is a neurological condition that comes about when certain brain cells die and the brain fails to produce enough of the chemical dopamine, which affects movement.

The team took skin samples from a patient diagnosed with one of the most progressive types of Parkinson's. The skin cells were then used to generate brain nerve cells affected by the disease. Scientists behind the study said the ability to generate these nerve cells will make it easier to monitor the effectiveness of any new drugs that could slow or halt progress of the condition. The aim is to find drugs that can prevent the death of these key cells, known as neurons, which break down as a result of Parkinson's.

Dr Tilo Kunath, of the University of Edinburgh's Medical Research Council centre for regenerative medicine, 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. This study provides an ideal platform to gain fresh insight into the condition and opens a new area of research to discover disease-modifying drugs.”

This study showed that the patient with a form of the disease that progresses quickly and can be diagnosed in people in their early 30s. People with this form of Parkinson's have twice as many of the genes that produce a protein, alpha synuclein, compared with the general population explain researchers. Although this form of the condition is rare, the protein involved is linked to virtually all types of the disease. The study was funded with a £300,000 grant from Parkinson's UK.

Dr Michael Devine, of UCL's institute of neurology, said, “Understanding such a progressive form of the disease will give us insight into different types of Parkinson's. 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.”

The research has been published in the journal Nature Communications.

Dr Kieran Breen, from Parkinson's UK, added, “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. This is just the kind of innovative research that Parkinson's UK is committed to funding as we move closer to a cure.”