Currently, monitoring and data collection in Parkinson’s disease clinical trials combine subjective patient-reporting tools such as diaries and questionnaires with periodic neurological and mobility assessments in a clinical setting that only capture a specific moment in time. Unfortunately, this approach fails to give a full, real-time picture of disease progression and the impact of a patient’s daily experience with Parkinson’s symptoms such as uncontrolled tremors and muscle movement, rigidity, restless sleep and loss of balance.
"Parkinson's disease is incredibly complex, with symptoms often varying hour to hour through the course of the day," said Seth Ettenberg, President and CEO of BlueRock Therapeutics. "New tools and approaches are needed to ease the reporting burden on patients in trials and to measure and assess disease progression more effectively. We are excited to be working with the pioneering teams at Rune Labs and Emerald Innovations to harness the power of their digital health technologies to collect an unbiased, objective, and continuous measure of disease impact on function that will help us develop more effective therapies."
BlueRock is working to change the standard of care for treating Parkinson’s disease and is currently testing a first-in-class stem cell-based therapy, bemdaneprocel (BRT-DA01), in a Phase 1 clinical study. Results from the Phase 1 study are expected to be announced in the second half of 2023. The company is also enrolling patients in a two-year global non-interventional study focused on gaining a better understanding of patients’ day to day experience with Parkinson's disease.
In the non-interventional study, the Rune Labs and Emerald technologies will be used to monitor the daily activities and measure key markers of disease progression in 50 patients and monitor their daily activities.
Rune Labs' StriveStudy platform works directly with the wearable Apple Watch and Apple's Movement Disorder API and will be used to record real-time measurement of general mobility, tremor intensity, dyskinesia, and involuntary muscle movements of patients in the non-interventional study. BlueRock can leverage StriveStudy to monitor patient compliance by tracking how often they wear the Apple Watch in the study, as well as to improve the study experience for patients by streamlining data collection.
"This collaboration is shifting the paradigm for Parkinson's disease drug development," said Brian Pepin, CEO of Rune Labs. "To give hope to Parkinson's patients, we need to push neurology drug development into the future, and we believe that Rune Labs' precision neurology platform can support this goal, much as precision medicine pioneers drove the oncology field forward."
Emerald Innovations’ Emerald monitoring biosensor belongs to a new class of sensors called “invisibles” that extract health metrics from radio signals, without wearable devices. Placed in the homes of study participants, the Emerald sensor continuously analyses the surrounding radio signals using artificial intelligence. In this study it will collect data on patients’ gait speed, mobility, and sleep quality.
"Emerald is built on a decade of research and has demonstrated strong correlation with the gold standards in Parkinson's, while allowing clinical trials to reach robust evidence faster and with a smaller number of patients. Our collaboration with BlueRock enables a data-driven clinical trial in Parkinson's while patients go about their normal lives," said Dr. Dina Katabi, Co-Founder of Emerald Innovations.
BlueRock will compare data from the use of both technologies to standard measurement and feasibility tools to gauge the feasibility of their use for future clinical studies for bemdaneprocel (BRT-DA01).
About the bemdaneprocel (BRT-DA01) Phase 1 TrialThe primary objective of the Ph1 trial is to assess the safety and tolerability of bemdaneprocel (BRT-DA01) transplantation at one-year post-transplant. The secondary objectives of the trial 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.
In the United States, the trial was initiated at Weill Cornell Medicine with Dr. Harini Sarva, M.D. as the Principal Investigator (PI) and first surgeries being performed by Dr. Viviane Tabar, Chair of the Department of Neurosurgery at Memorial Sloan Kettering Cancer Center. Dr. Tabar has financial interests related to BlueRock. Memorial Sloan Kettering (MSK) has institutional financial interests related to BlueRock. Additional Neurology sites were added at the University of California, Irvine, under the guidance of Dr. Claire Henchcliffe, M.D., D.Phil., F.A.A.N., F.A.N.A. (PI), and at the University Health Network (UHN), under the guidance of Dr. Lozano, O.C., M.D., Ph.D., F.R.C.S.C., F.R.S.C., F.C.A.H.S. (PI) and Dr. Alfonso Fasano, M.D., PhD., Chair in Neuromodulation and Multi-Disciplinary Care, UHN and UoT.
More information about the Phase 1 trial is available at clinicaltrials.gov (NCT#04802733)
More information about the non-interventional study is available at clinicaltrials.gov (NCT#05363046)
About Parkinson's DiseaseParkinson'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% 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). According to the Parkinson's Foundation, more than 10 million people worldwide suffer from Parkinson’s disease, with one million living in the United States. DA01 is being clinically evaluated in a subset of patients with Parkinson's disease, where regular medications are no longer fully effective.
About BlueRock Therapeutics LPBlueRock Therapeutics LP is a clinical stage cell therapy company focused on creating cellular medicines to reverse devastating diseases. Our cell+gene platform is harnessing the power of cells to create a pipeline of new medicines for patients suffering from neurological, cardiovascular, immunological, and ophthalmic diseases. Our lead clinical program, bemdaneprocel, (BRT-DA01) is in Phase 1 clinical trials for Parkinson’s disease. We were founded in 2016 by Versant Ventures and Bayer AG and became a wholly owned, independently operated subsidiary of Bayer AG in late 2019 as a cornerstone of its newly formed Cell & Gene Therapy Platform. Our culture is defined by the courage to persist regardless of the challenge, the urgency to transform medicine and deliver hope, integrity guided by mission, and community-mindedness with the understanding that we are all part of something bigger than ourselves.
About BayerBayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2022, the Group employed around 101,000 people and had sales of 50.7 billion euros. R&D expenses before special items amounted to 6.2 billion euros.