CDNF in Parkinson’s Disease
About 7 million people globally are diagnosed with Parkinson’s Disease, a progressive neurodegenerative disorder characterized by slowness of movement, rigidity, and resting tremor. There is no cure.
Parkinson’s disease is the second most common neurodegenerative disease only after Alzheimer’s. It is caused by the death of dopamine-producing nerve cells in a region of midbrain called substantia nigra for an unknown reason. While there are a number of treatments for the motor symptoms of patients, two major unmet clinical needs remain: disease modification, i.e. slowing down or stopping disease progression, and treatment for non-motor symptoms of the disease, such as sleep disturbance, depression, anxiety, and cognitive function.
Herantis’ CDNF has been studied and shown efficacious in several preclinical models of Parkinson’s diseasea2. CDNF has both protected and regenerated dopamine-generating cells suggesting a potential for disease modification. Signals of efficacy are also seen in non-motor symptoms based on preclinical data3.
Herantis has submitted regulatory applications to start a phase I-II clinical study in the first half of 2017 in 18 patients with Parkinson’s disease. In the clinical study CDNF will be administered directly into the brains of the patients using a device specially designed for such procedure. Implantation of the device requires a similar surgical procedure as Deep Brain Stimulators, which are commonly used for the treatment of Parkinson’s disease. The primary outcome of the clinical study is related to assessing the safety and tolerability of the treatment, with several exploratory endpoints to assess its efficacy.
2 Lindholm et al, Nature 448: 73-77, 2007; Airavaara et al. Cell Transplant. 21: 1213-1223, 2012.
3 Unpublished data