GDNF and cell transplantation: dual therapies for Parkinson’s
Parkinson’s is a complex condition and it is becoming increasingly clear that the future of disease-modifying therapies for Parkinson’s will rely on the use of multiple treatment approaches, as opposed to a single therapy.
Scientists in Australia have recently published pre-clinical research exploring this idea – and assessing the effects of treating models of Parkinson’s with a combination of different therapies.
Dopamine replacement cell transplantation represents one experimental method by which dead or dying neurons in people with Parkinson’s are replaced by dopamine producing stem cells. Researchers have developed robust techniques allowing for vast amounts of stem cell-derived neurons to be grown in cell culture, that could in theory be transplanted into the brains of people with Parkinson’s. These cells could replace the neurons that have been lost to the condition, taking up the lost function. But whether the Parkinson’s affected brain is a nurturing environment for these cells to flourish is an important consideration for this therapy going forward. The replacement cells will need a lot of support to maintain their health and increase their chances of survival.
The Australian researchers explored this therapy idea by introducing the protein ‘glial cell line-derived neurotrophic factor’ (or GDNF) to the brains of rats to provide a more supportive environment for transplanted dopamine replacement cells. GDNF is a ‘neurotrophic factor’ – a protein that nurtures and supports neurons and acts rather like fertiliser for the transplanted cells.
By introducing GDNF into the animal models of Parkinson’s before transplanting the dopamine replacement cells, the researchers found that the injected neurons survived, but they made fewer connections with surrounding cells than if the GDNF was given after the cells were transplanted.
The results demonstrate the potential of manipulating neurotrophic factors in combinatorial therapeutic approaches for Parkinson’s.
Cure Parkinson’s has been a long-term supporter of research for both GDNF and the TRANSEURO cell transplantation clinical trials, currently taking place in Cambridge and Sweden.