Hallmarks of Parkinson’s include damage to dopamine-producing nerve cells in a brain area called the substantia nigra, toxic accumulation of alpha-synuclein protein clumps in nerve cells, and chronic brain inflammation.
Dopamine is a neurotransmitter, or chemical that facilitates communication between nerve cells. This means it’s involved in controlling movement, cognition, learning, memory, and mood.
There are no approved therapies to prevent the progression of Parkinson’s. Treatments have focused on alleviating the disease’s movement symptoms. The more damage to dopamine-producing nerve cells, the less effective these therapies are, however.
The treatment of non-movement symptoms — including depression, anxiety, sleep disturbance, and cognitive dysfunction — remains an unmet need for Parkinson’s patients.
CDNF is a synthetic neurotrophic factor, or protein that helps nerve cells survive. The factor occurs naturally in blood and cerebrospinal fluid.
CDNF protected and regenerated nerve cells in studies involving animal models of Parkinson’s disease. It stopped, and to some extent reversed, the progression of the disease.
The video that Herantis produced explains CDNF’s modes of action and advantages. “This video briefly explains the disease and shows how our CDNF aims to protect the dopamine-producing nerve cells from degeneration. This could slow down or even stop disease progression,” Pekka Simula, Herantis’ CEO, said in a press release.
Researchers have found that CDNF can repair nerve cells to the point that they can produce dopamine again. It also reduces alpha-synuclein clumping and nerve cell inflammation.
In addition, CDNF can protect and repair nerve cells from endoplasmic reticulum stress. The reticulum is a large organelle that takes part in a number of cell functions, including protein production. Abnormal protein production can cause the endoplasmic reticulum stress associated with neurodegenerative diseases such as Parkinson’s.
Preclinical-trial research has also shown signs that CDNF can improve Parkinson’s patients’ non-movement symptoms, Herantis reported.
The clinical trial that Herantis has started will evaluate CDNF’s safety and preliminary ability to improve Parkinson’s symptoms. It hopes to enroll 18 patients in the study.
The participants will receive CDNF directly into theirs brain through an implanted drug delivery system for six months. Simula said the company is working on a non-invasive delivery method for CNDF — a breakthrough in Parkinson’s treatment.
Results of the trial are expected by the end of 2019.