Potential breakthrough in the treatment of Parkinson’s disease – drug candidate CDNF could stop the progression of the disease and improve symptoms

Potential breakthrough in the treatment of Parkinson’s disease – drug candidate CDNF could stop the progression of the disease and improve symptoms

The novel drug candidate CDNF, discovered at the Institute of Biotechnology, University of Helsinki, could be a disease-modifying treatment of Parkinson’s disease in addition to alleviating its symptoms. Such a breakthrough would also have significant social implications. In Finland, approximately 14 000 patients suffer from Parkinson’s disease, and thus far, there is neither a cure nor a treatment that can slow the progression of this disease.

CDNF could be a breakthrough in the treatment of Parkinson’s disease. Currently in its first clinical study in Finland and Sweden, the first results indicate that CDNF can protect and even restore dopaminergic neurons, slow the progression of Parkinson’s disease, and alleviate its symptoms.

CDNF is a protein naturally found in the human brain and circulation. It plays an important role in neuronal survival. Parkinson’s disease is caused by the loss of dopamine-producing neurons; thus, CDNF targets the root cause of the disease. This new drug candidate differs from known anti-Parkinson’s drugs in that it targets true disease modification in addition to symptomatic relief. Going forward, it could even stop disease progression.

“We are on the verge of a major breakthrough.”

In preclinical studies, CDNF has been shown to protect neurons from degeneration and to restore impaired neurons. This suggests potential to relieve the symptoms of Parkinson’s disease and stop its progression. Currently available drugs and therapies are only able to alleviate the symptoms of the disease.

“Based on the mechanism of action of CDNF, treatment should be started as early as possible,” points out Pekka Simula, CEO of Herantis Pharma, the company developing CDNF.

In this manner, the course of the disease could be stopped at an early stage. The earlier a patient receives treatment for symptomatic relief and arresting disease progression, the more normal life the patients could live despite a serious brain disease.

The current study only includes patients with advanced disease. This was a requirement from the regulatory authorities since the primary endpoint of a first-in-human clinical study, such as the ongoing CDNF study, is always safety. So far, the results suggest that CDNF is safe and well-tolerated and also suitable for use with other anti-Parkinson’s drugs. In the next clinical trial, Simula hopes that earlier-stage Parkinson’s disease patients will be involved.

“If the initial efficacy signals are confirmed with future results, we would consider this a big step forward in the treatment of Parkinson’s disease,” says Simula. “However, it can already be said that we are on the verge of something significant. In this first clinical study, brain imaging results have shown restored dopamine activity by even dozens of percent in some patients.”

Effective treatment at home

A new drug candidate would also have a significant social impact if the symptoms and progression of Parkinson’s disease can be relieved, halted, or cured. If symptomatic relief and improvement are feasible, patients’ needs for various services, such as serviced housing or hospital care, would decrease. Disabling brain diseases come at a huge economic cost: Parkinson’s disease alone costs the EU approximately € 15 billion a year.

Further studies are now needed to confirm the efficacy of CDNF. At the same time, the development of a second-generation CDNF drug candidate is underway to allow for more straightforward administration of CDNF, allowing for easy treatment of the disease at home.

“The most promising second-generation xCDNF molecules are currently being developed and tested to remain in the patient’s bloodstream for as long as possible, ensuring that as much of the drug enters the brain as possible. If this is successful, the drug will be able to be administered in the future, for example, like an insulin injection.”

Along with this drug development, the research team is also investigating whether the CDNF drug candidate could be beneficial for other neurodegenerative diseases.

CDNF drug development is based on scientific research by Professor Mart Saarma’s group at the University of Helsinki. The Finnish company Herantis Pharma has patented CDNF internationally.

Parkinson’s is an incurable, serious brain disease

In Finland, Parkinson’s disease affects about 14 000 patients. The symptoms of Parkinson’s disease are caused by the loss of dopamine-producing neurons in the brain. This degeneration of neurons leads to a continued decrease in dopamine levels in the brain, resulting in various movement-related (motor) symptoms for the patient. So far, there is no cure for the disease, which means that the neurodegeneration will continue for the rest of the patient’s life. Indeed, at present, Parkinson’s disease inevitably leads to severe disability when the symptoms of the disease deprive the patient of the ability to live a normal life. Symptomatic treatment of Parkinson’s disease has been available since the 1960s, but to this day no drug offers disease modification nor complete symptomatic relief. CDNF is a very promising drug candidate, representing a big leap in the treatment of this severe brain disease.