Health Canada gave a biotech venture the permission to initiate a Phase 1 clinical trial investigating the use of stem cell therapy targeting Parkinson’s Disease (PD). Titled “Phase 1 Safety and Tolerability Study of MSK-DA01 Cell Therapy for Advanced Parkinson’s Disease,” this actually represents the first time in Canada that researchers will test on humans pluripotent stem cell-derived dopaminergic neurons in patients with Parkinson’s disease. With the study hosted at University Health Network in Toronto, the venture is a clinical stage biotech company, wholly-owned subsidiary of Bayer AG called BlueRock Therapeutics LP. The stakes are considerable here as the sponsor’s President and Chief Executive Officer, Emile Nuwaysir, PhD, suggests “…this trial could shift the treatment paradigm for PD patients in Canada and around the world.”
Parkinson’s disease is a progressive neurodegenerative disorder caused by nerve cell damage in the brain, leading to decreased dopamine levels. The worsening of motor and non-motor symptoms is caused by the loss of dopamine-producing neurons. At diagnosis, it is estimated that patients have already lost 60-80 percent of their dopaminergic neurons. Parkinson’s disease often starts with a tremor in one hand. Other symptoms are rigidity, cramping and slowness of movement (bradykinesia). Parkinson’s disease is the second most common neurodegenerative disorder, impacting more than 7.5 million people, including 1.3 million in North America.
Introduction to Engineered Therapy Platform
The Bayer subsidiary is identified as a top stem cell therapeutic venture focusing on a range of therapeutic areas, including cardiovascular and neurological diseases such as PD. Its lead stem cell program focused on the regeneration of heart muscles in patients who experienced heart attacks or suffer chronic heart failure.
In development with Toronto Centre for Regenerative Medicine and University Health Network (UHN), the company was launched by Bayer and Versant Ventures. Their cell+gene platform has the potential to reshape cellular medicine, according to their website. They position that while their authentic cells enable them to actually replace cells, restore function and reverse disease, these engineered cells also help the firm deliver what they call “payloads” in novel ways, opening up the potential for enabling therapies to treat what were previously considered intractable common and rare diseases.
What is unique about these engineered cell therapies?
The company employs proprietary bioprocessing and manufacturing methods to create stable master cell banks supporting near unlimited expansion and differentiation into any cell type in the human body. Called a “Universal Pluripotent Master Cell Bank with Limitless Expansion and Broad Cell Differentiation Potential,” the company explains that first they take healthy donated human stem cells and employing Reprogramming factors create induced pluripotent stem cells; then via gene editing technology now available, they create what they call “Allo-Engineering for Immune Evasion,” leaving the ability for what is known as “Culturing and Expansion.”
The company decided to apply this advanced regenerative therapy model to Parkinson’s disease as, first and foremost, the founders here have pioneered the derivation of dopaminergic neurons that evidence robust preclinical data pointing to a restoration of motor function and boosts in dopamine release with maturity.
Their lead program, an authentic cellular therapy candidate targeting PD, now reaches clinical trials in Canada. For more on the company’s position on how this will work, follow the link to the pipeline.
This clinical trial will enroll ten patients in the United States and Canada with the Canadian trial site at University Health Network, Canada’s largest research and teaching hospital. In the USA, the study (NCT04802733) disclosure identifies that patients will undergo surgical transplantation of the dopamine-producing cells under general anesthesia into a part of the brain called the putamen.
The patients will be given medicine to partially suppress their immune system (aimed to prevent the body from rejecting the cells) for one year. The primary objective of the Phase 1 study is to assess the safety and tolerability of DA01 cell transplantation at one-year post-transplant. The secondary objectives of the study are to assess the evidence of transplanted cell survival and motor effects at one- and two-years post-transplant, to evaluate continued safety and tolerability at two years, and to assess feasibility of transplantation.
Investigator Point of View
Brad Wouters, PhD, Executive Vice-President, Science and Research with the University Health Network (UHN), reports, “We are thrilled to be the first national trial site bringing leading-edge clinical research with life-changing potential to the nearly 8 million patients world-wide who are affected by Parkinson’s.” He continued, “As the largest hospital-based research institution in Canada, UHN is uniquely positioned with both talent and clinical trial infrastructure to support this important research.”
While Andres Lozano, MD, PhD, Senior Scientist with Krembil Research Institute (part of UHN), declared, “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.” He continued, “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 our lives for many patients.”
BlueRock Therapeutics is a leading engineered cell therapy company with a mission to develop regenerative medicines for intractable diseases. BlueRock Therapeutics’ cell+gene platform harnesses the power of cells to create new medicines for neurology, cardiology, and immunology indications. BlueRock Therapeutics’ cell differentiation technology recapitulates the cell’s developmental biology to produce authentic cell therapies, which are further engineered for additional function. Utilizing these cell therapies to replace damaged or degenerated tissue brings the potential to restore or regenerate lost function. BlueRock Therapeutics was founded in 2016 by Versant Ventures and Bayer AG and capitalized with one of the largest-ever Series A financings in biotech history by Bayer AG (through its Leaps by Bayer unit) and Versant Ventures. The company was fully acquired by Bayer in 2019. BlueRock Therapeutics’ culture is defined by scientific innovation, the highest ethical standards, and an urgency to bring transformative treatments to all who would benefit.
Brad Wouters, PhD, Executive Vice-President, Science and Research with the University Health Network (UHN)
Andres Lozano, MD, PhD, Senior Scientist with Krembil Research Institute