Cerebral dopamine neurotrophic factor (CDNF) is an unconventional neurotrophic and neurorestorative factor for dopamine neurons discovered in the laboratory of Mart Saarma at the Institute of Biotechnology, HiLIFE. The first paper was published in Nature in 2007.
Currently, CDNF is being tested in phase one and two clinical trials for the treatment of Parkinson’s disease at three leading university hospitals in Finland and Sweden. Since this is the first-in-human study, its primary endpoint is the safety of CDNF, and the study has recruited patients with advanced disease who are not optimally suited for neurorestorative treatment.
Even with this challenging background, exciting initial data announced in February 2020 suggests that CDNF is safe and shows promising signals of biological activity in dopamine transporter positron emission (PET) imaging in some patients. This supports the hypothesis of CDNF’s disease-modifying potential. The treatments will continue, and further results are expected in autumn 2020.
Parkinson’s disease is the second most common neurodegenerative disorder, affecting about seven to ten million people. Currently, no treatment exists that can slow down or stop the disease progression. In Parkinson’s disease, the midbrain dopamine neurons degenerate and die, causing major motor symptoms as well as non-motor symptoms.
The sponsor of the clinical study, Herantis Pharma Plc., is a spin-off company started by Eero Castrén, Heikki Rauvala and Mart Saarma from the University of Helsinki. The researchers would like to gratefully acknowledge the contribution of the study participants.
Lindholm P, Voutilainen MH, Laurén J, Peränen J., Leppänen V-M, Andressoo J-O, Lindahl M, Janhunen S, Kalkkinen N, Timmusk T, Tuominen RK and Saarma M. (2007) Novel neurotrophic factor CDNF protects and rescues midbrain dopamine neurons in vivo. Nature, 448: 73-77.
Huttunen HJ and Saarma M (2019) CDNF Protein Therapy in Parkinson’s Disease. Cell Transplantation. 28(4):349-366. DOI: 10.1177/0963689719840290