BlueRock Therapeutics is all set to initiate Phase I Study of MSK-DA01 Cell Therapy for advanced Parkinson’s disease (PD) after it received permission from Health Canada.
A progressive neurodegenerative disorder, PD is caused by damage to nerve cell in the brain, causing lower dopamine levels. The deterioration of motor and non-motor symptoms is because of dopamine-producing neuron loss.
The ‘Phase 1 Safety and Tolerability Study of MSK-DA01 Cell Therapy for Advanced Parkinson’s Disease (PD)’ trial plans to enrol ten patients in the US and Canada, with the University Health Network being the Canadian trial site.
Evaluating the safety and tolerability of DA01 cell transplantation at one year post-transplant will form the primary objective of the trial.
As secondary objectives, the evidence of transplanted cell survival and motor effects at one and two years after transplant, continued safety and tolerability at two years and transplantation feasibility, will be analysed.
BlueRock president and CEO Emile Nuwaysir said: “We’re excited to initiate this study with our expert collaborators at University Health Network in Toronto as we believe this trial could shift the treatment paradigm for PD patients in Canada and around the world.
“Our therapy is intended to replace the midbrain dopaminergic neurons lost in the degenerative condition to rebuild the neural circuit, and thereby restore motor control to Parkinson’s patients.
“If successful, this could demonstrate for the first time that the effects of degenerative disease are, in principle, reversible.”
This is the first of its kind trial in Canada to assess pluripotent stem cell-derived dopaminergic neurons in patients with PD, BlueRock noted.
University Health Network Stereotactic and Functional Neurosurgery RR Tasker Chair Andres Lozano said: “Exploring the potential for a cell therapy that could improve Parkinson’s rather than simply slowing its progress is a monumental step. Few therapeutic approaches to date have been designed to truly restore function.
“This is an important step to advance treatment using a novel strategy for a disease that does not yet have a cure and could change lives for many patients.”