Intermittent Bilateral GDNF for Parkinson's Disease

• Research type

  Research Study

• Full title

  A Placebo-Controlled Randomised, Double-Blind Trial to Assess the Safety and Efficacy of Intermittent Bilateral Intraputamenal Glial Cell Line-Derived Neutotrophic Factor (GDNF) Infusions Administered via Convection Enhanced Delivery (CED) in Subjects with Parkinson's Disease

• IRAS ID

  93130

• Contact name

  Alan Whone

• Sponsor organisation

  North Bristol NHS Trust

• Eudract number

  2011-003866-34

• Research summary

  Assessing the benefit and safety of administering intermittent GDNF infusions in PD. At present only drugs which improve the symptoms of Parkinson's disease (PD) are available. What is needed is a treatment which slows or reverses disease progression. The aim of this research is to test such a treatment. Previous animal model and pilot open label PD patient investigations showed that continuous glial cell line-derived neurotrophic factor (GDNF) infusions delivered into the brain improve motor symptoms and, as assessed by brain scans and post-mortem assessments, restore dopamine nerves. However, at the beginning of the last decade, a placebo-controlled multi-centre trial failed to demonstrate clinical benefit, despite witnessed improvements in brain scans. In addition, a study in monkeys occurring at the same time raised questions over safety. Since that point further human investigations have not been performed. In significant part, we believe, the above issues were due to a failure in the way GDNF was surgically delivered. We have now developed an in-house device which animal model studies suggest will allow GDNF to be given much more reliably to the putamen area of the brain. We feel that this now allows for definitive testing of GDNFs effects in humans. We propose conducting a placebo controlled trial of intermittent GDNF infusions in 42 patients at our centre. We anticipate that the information gained from this study, if successful, will rapidly lead to a large multi-national trial with the prospect of a new disease-slowing therapy being available to PD patients within 5 years.

• REC name

  South West - Central Bristol Research Ethics Committee

• REC reference

  12/SW/0047

• Date of REC Opinion

  24 Sep 2012

• REC opinion

  Further Information Favourable Opinion