Novel drug may offer new hope for Parkinson’s sufferers

Parkinson’s disease affects between 7 and 10 million people worldwide. Symptoms like loss of motor control, mood disorders, cognitive impairment and problems with speech and swallowing are mostly caused by low levels of a chemical called dopamine in the brain. Current treatments focus on increasing dopamine levels or controlling the actual symptoms.

But these treatments don’t stop the disease from progressing. Dopamine-producing neurons in the brain continue to die and all treatments eventually become ineffective. However, initial studies have shown that a protein called cerebral dopamine neurotrophic factor (CDNF) may be able to alleviate Parkinson’s symptoms and even stop its progression. This investigational drug is the focus of the partly EU-funded project TreatER in its clinical study for the treatment of the disease.

The project is currently testing natural CDNF in a first-in-human, randomised, Phase 1-2 clinical study being conducted at three European university hospitals. Since CDNF is a protein, the body won’t transport it to the brain if it’s taken as a pill or injection. In this study, the investigational treatment is being administered directly into the brain using an implanted drug delivery system suited for Parkinson’s patients.

Now, a non-invasive CDNF development programme is also being launched by project partner Herantis to broaden the application of CDNF in Parkinson''s disease and possibly also other neurodegenerative disorders.

“Non-invasive CDNF is an important expansion to our existing patent estate and strengthens the profile and value of our CDNF program,” says Herantis CEO Pekka Simula in a news release posted on ‘GlobeNewswire’. “Based on broad and robust preclinical data, we believe CDNF can make a significant difference in the treatment of Parkinson’s disease and we look forward to exploring the new possibilities a non-invasive administration could offer to provide the best product for the benefit of patients.”

CDNF and its mechanisms

CDNF is naturally present in human blood and cerebrospinal fluid. In preclinical studies, it has been shown to alleviate both motor and non-motor symptoms of Parkinson’s and even stop the disease’s progression.

The studies have demonstrated that this potent neuroprotective factor works via a number of mechanisms that are relevant to the disease. It protects cells from endoplasmic reticulum stress, which can eventually trigger cell death. When they receive CDNF, stressed cells recover and dopamine-producing neurons begin producing the chemical again. CDNF also reduces the impact of toxic proteins such as alpha synuclein, and alleviates inflammation of brain tissue that helps prevent dopamine-producing neurons from degenerating and dying.

“We have previously shown that CDNF protects and recovers neurons from degeneration, neuroinflammation and endoplasmic reticulum stress, critical contributors to many neurodegenerative diseases,” explains project coordinator Prof. Mart Saarma from the University of Helsinki. “Our recent discoveries provide an opportunity to target numerous indications beyond Parkinson’s with simpler administration and broader distribution while maintaining the full potential of CDNF.”

Now halfway through its 3-year term, TreatER (Clinical study in Parkinson’s disease with two unique goals: 1) Proof-of-concept of CDNF protein for disease modification; 2) Validation of clinically tested device for intracerebral drug delivery) is making great strides towards its goal of developing a novel treatment for Parkinson’s disease.

For more information, please see:
TreatER project website