CDNF for the treatment of Parkinson’s Disease
CDNF in patients
CDNF is a novel drug candidate that has the potential to stop the progression of Parkinson’s disease by protecting neurons from degeneration and restoring the functions of already damaged neurons. CDNF’s safety and tolerability was studied in a Phase 1-2 randomized, placebo-controlled, double-blind, multicenter clinical study conducted in patients with Parkinson’s disease in three university hospitals in Sweden and Finland. CDNF was delivered directly to the brain using a surgically brain-implanted drug delivery device. The 12-months treatment period was recently successfully completed and the results demonstrated that CDNF is safe and well tolerated. The patients are currently in a 1-year follow-up study. More information about the clinical study is available at www.treater.eu.
As a brain-implanted drug delivery device would not be suitable for early-stage patients, Herantis is currently exploring other less invasive, more patient-friendly ways to deliver CDNF to patients. Any concrete plans for further clinical studies will be announced at the appropriate time by the company.
Parkinson’s disease is an incurable, progressive brain disorder estimated to affect over seven million patients worldwide. It is caused by the degeneration of dopamine-producing neurons in the brain. The underlying reasons behind degeneration of dopaminergic neurons is not yet fully understood. It is however known that the accumulation and aggregation of alpha-synuclein in the affected brain areas is at the core of the disease, and that the neuronal degeneration is associated with a cell level phenomenon called ER stress. The typical symptoms of the disease include tremors, slowness of movement, muscle stiffness, and impaired balance. As the disease progresses, other possible symptoms include problems with cognition, sleep and speech, depression, and severe constipation.
The drug candidate CDNF is a natural human protein, which has been shown to protect neurons, and help degenerating neurons restore their functionality. This is based on several mechanisms, including alleviation of ER stress and reduction of alpha-synuclein aggregation and toxicity. Both are highly relevant to Parkinson’s disease.
In Parkinson’s disease models, CDNF restores the function of already damaged neurons and protects healthy neurons from degeneration. Based on this, CDNF has the potential to alleviate both motor and non-motor symptoms of Parkinson’s disease as well as stop the further progression of the condition. For more detailed information on the mechanism of action of CDNF, please see here.
Introductory video on CDNF
CDNF in media
EU success story article about CDNF: Novel drug delivered direct to brain may halt Parkinson’s