CDNF in Parkinson’s Disease

About 7 million people suffer from Parkinson’s disease, a progressive neurodegenerative disorder characterized by slowness of movement, rigidity, and resting tremor. There is no cure.

CDNF

Parkinson’s disease (PD) is a slowly progressing, incurable neurodegenerative disease caused by the death of dopaminergic neurons in the midbrain. Common first symptoms of the disease include tremors, rigidity, and slowness of movement. While the motor symptoms can be treated with medication the progression of the disease cannot be prevented. The benefits of medication may be lost with disease progression or their side effects can become unmanageable. The progressing disease is also associated with non-motor symptoms such as sleep problems, depression, and anxiety, which are not alleviated by current Parkinson’s drugs. It is estimated that 7 million people worldwide suffer from Parkinson’s disease.

Herantis’ CDNF is a novel drug candidate for the treatment of neurodegenerative diseases. Naturally present in the human blood circulation and cerebrospinal fluid, CDNF is a protein with neuroprotective and neurorestorative properties, patented internationally by Herantis. Encouraged by strong preclinical proof-of-concept Herantis has launched a first-in-human, randomized Phase 1-2 clinical study with CDNF in the treatment of Parkinson’s disease (PD).

CDNF administration has been safe in preclinical studies and in first clinical treatments. In disease models2, CDNF has protected and regenerated dopamine-generating cells in the midbrain suggesting potential for disease modification of PD. It has also shown efficacy in non-motor symptoms of PD3. CDNF drug development is based on research at the University of Helsinki, led by professor Mart Saarma.

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Clinical development

The first-in-human, phase 1-2 clinical study in 18 patients with Parkinson’s disease is ongoing at three study sites in Sweden and Finland. Details on the clinical study have been published on the ClinicalTrials.gov web site.

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. The clinical study has received funding from the European Union’s research and innovation program Horizon 2020 under grant agreement number 732386. The name of the EU funded project is TreatER, and the project has its own web site.

Introductory video on CDNF

Herantis has prepared an introductory vide on CDNF, which is available here.

2 Lindholm et al, Nature 448: 73-77, 2007; Airavaara et al. Cell Transplant. 21: 1213-1223, 2012.
3 Unpublished data