Date: 04 March 2019
A pioneering, NIHR supported neurodegeneration study into Parkinson’s disease was the subject of BBC2 documentary called ‘The Parkinson’s Drug Trial: A Miracle Cure’, on Thursday 28 February.
The documentary concerned a randomised double-blind placebo controlled phase 2 trial, Extended Treatment with Glial Cell Line-Derived Neurotrophic Factor in Parkinson’s Disease, which was hosted at North Bristol Trust - focusing on the experience of some of those who took part in the study.
The trial attempted to regenerate dying brain cells in patients with Parkinson’s disease. It tested whether increasing levels of the naturally occurring protein Glial Cell Lined Derived Neurotrophic Factor (GDNF) directly into the brain via a specially-made port in the side of their head could reverse their condition.
As part of the study, brain scans were taken before and after the trial to assess brain cell functionality. After nine months, results showed that in comparison to those that had received the placebo, patients that had received GDNF showed improvement in key areas of the brain affected by the condition. By 18 months all participants had opted to receive GDNF and showed improvements compared to their results at the start of the study.
During the study, NIHR Clinical Research Network (CRN) funded staff ensured that patients and their families were supported to manage the demanding visit scheduling for the study. Despite the challenges of recruiting to an early phase experimental study, the team were able to recruit to target and maintain patients’ engagement throughout.
The study has shown that GDNF could potentially be used as treatment to restore damaged brain cells in patients with Parkinson’s. Further research now aims to find an effective treatment period and dose of GDNF to improve the lives of patients living with the condition.
Dr Camille Carroll, NIHR Clinical Research Network (CRN) National Specialty Lead for Neurodegeneration said:
"This exciting study exemplifies the best elements of delivering research in neurodegeneration in the UK. An innovative therapeutic agent and delivery system, designed by a team driven by a passion to improve the lives of those living with Parkinson’s. A dedicated clinical research team committed to the care and support of study participants - and the enthusiasm, dedication and commitment of the study participants – willing to take such a huge step into the unknown.
"This study has overcome barriers and challenges in so many domains. Its successful conclusion, with that all-important message of hope, is a tribute to all involved."
Parkinson’s disease affects around 10 million people worldwide and is the second most common neurodegenerative disorder. The condition is progressive and leads to the degeneration of dopamine generating brain cells.
You may also be interested in