Cerebral Dopamine Neurotrophic Factor (CDNF) (Herantis Pharma, Finland), a potential treatment for Parkinson disease (PD), showed motor function improvement in a 12-month phase 1/2 study in individuals with moderate disease. CDNF is a multimodal, natural protein that has shown potential to slow or even reverse loss of dopaminergic neurons. If found effective, CDNF could be among the first disease-modifying treatments for PD.
Although participants reactions varied, at the 12-month mark the data suggests an overall improvement in their motor symptoms as measured by Unified Parkinson Disease Rating Scale (UPDRS). This may suggest a potential slowing of disease progression in some participants.The study gathered preliminary nonstatistical data to support an investigative assessment of CDNF’s potential efficacy; such as measuring the severity of motor symptoms with the (UPDRS).
The study also evaluated biological signals in the brain, as measured by dopamine transporter positron emission tomography (DAT PET) imaging which provides an indirect measure of dopaminergic function in the brain. Reactions varied, but a promising biological signal was seen in some patients where significant increases in DAT PET signaling was observed in the target infusion area of CDNF.
Treatment emergent adverse events (TEAE) were commonly mild and transient, and reduced compared with the first period. Similarly, serious adverse events (SAEs) were also less frequent during the second treatment period, and all participants who previously experienced SAEs have since recovered.
The 2-part study with 17 participants encompassed an initial 6-month period in which all participants were given either placebo or CDNF at 1 of 2 dose levels. Then is followed by an extended 6-month where all participants received 1 of the 2 dose levels of CDNF on a monthly basis, including the previous placebo group participants. Treatment was administered via a dose delivery system provided by Renishaw Neuro Solutions Ltd, that was implanted into the brain at the beginning of the study.
F. Stephen Benesh, MD, and Shruti P. Agnihotri, MD
Jason A. Ellis, MD; Benjamin W. Y. Lo, MD; Chirag G. Bhatia, BS; Yona Feit; Steven Mandel, MD; and Dana Shani, MD
Monideep Dutt, MD; Jamika Hallman-Cooper, MD; Ekta Bery, MD; Mohammed Shahnawaz, MD; and Grace Gombolay, MD