NRT · Phase II clinical trial of PDGF-BB for the neurological regeneration and recovery in Parkinson´s disease

The goal of this project is to perform a European phase II clinical trial to demonstrate that PDGF-BB acts as a unique regenerative therapy for Parkinson’s Disease (PD), aimed at reversing the course of the disease, by promotion of endogenous brain repair mechanisms. Current pharmacological therapies for PD carry side-effects and become less effective as the disease progresses. This causes immense suffering for patients in their daily lives. There is currently no established disease-modifying, regenerative or neuroprotective treatment for PD. Such a therapy would revolutionize the capacity to effectively treat PD patients and could dramatically reduce the multi-billion € annual cost to Europe. The project will conduct a multi-centre clinical trial in PD using intracerebroventricular administration of PDGF-BB. To obtain the most relevant information from the clinical trial, state-of-the-art disease rating scales and brain imaging technology will be used to monitor changes related to restoration of brain function. Planning and conduct of the clinical study will involve investigators and advisors with extensive expertise in the disease-area and experience with participating in clinical studies. To further advance the understanding of the regenerative mechanism, preclinical studies will be performed. We will set up a framework of partners to ensure proper governance of the clinical study, compliant with fundamental ethical principles of European regulations concerning studies on “advanced therapies medicinal products”. PD patient associations and experts in ethics will assist the project with patient information and recruitment strategies. Internal training sessions, external meetings and dissemination of results will be coordinated by a communication expert firm. A robust and realistic commercialization strategy including IP, health economy and reimbursement will be developed.