Academy of Finland
Funding decision
Applicant / Contact person Saarma, Mart
Organisation University of Helsinki
Project title CDNF and its Domains: Mode of Action and Novel Treatment for Parkinsonís Disease
Decision No. 310891
Decision date 28.04.2017
Funding period 01.09.2017 - 31.08.2021
Funding 448 849
WebFOCUS Report
Project description
Parkinsonís Disease (PD) is a neurodegenerative disorder, in which dopamine (DA) neurons in the brain die. Treatments are available that can improve the symptoms of PD in most patients, but no disease-modifying therapy exists. Neurotrophic factor GDNF has been efficient in animal models of PD, but has failed in clinical trials. We have discovered a new neurotrophic factor CDNF and found that it protects and restores the function of DA neurons more effectively than GDNF. Despite promising results with CDNF in animal models of PD, the CDNF-based treatment has some drawbacks. CDNF requires direct delivery to the patient's brain through invasive surgery, as like most proteins it cannot pass through the blood-brain barrier (BBB). We found that C-terminal domain of CDNF (C-CDNF) protects DA neurons and passes BBB when administered systemically. We hypothesize that systemic delivery of C-CDNF will offer a completely new, safe and effective disease modifying treatment for PD patients.