After a lightning round of proposals and reviews, an international team of scientists led by Dr. Camillo Ricordi was granted immediate FDA authorization for a 24-patient clinical trial to test the safety and exploratory efficacy of umbilical cord-derived mesenchymal stem cells (UC-MSCs) to block the life-threatening lung inflammation that accompanies severe cases of COVID-19. The therapy uses cells from discarded umbilical cords, with just one cord saving as many as 10,000 lives.
The trial will be based at the University of Miami Health System and Jackson Health System in Miami, Florida. It is the result of a collaborative, international, academic initiative sponsored by The Cure Alliance, a non-profit group of scientists and innovators dedicated to sharing knowledge and accelerating cures for all diseases. In response to the COVID-19 pandemic, The Cure Alliance has pivoted all resources to fighting the virus. The clinical protocol has already been shared with academic institutions throughout the world.
Together with Dr. Ricordi, engineers here at Biorep have designed a Stem Cell Isolator that will be used in the second wave trials which will start after proof of safety and early efficacy with the current trial. Validation runs on the cell products obtained with the Stem Cell Isolator are underway prior to using them clinically. In a time of global pandemic that has not been experienced for generations, Biorep was poised and ready to use our experience and expertise to develop a device that will enable Dr. Ricordi’s team of researchers to pursue this cutting-edge trial. Our team is both humbled and honored to be a part of this effort, with potentially life-saving treatments for patients with COVID-19.
Our contribution to this trial and working jointly together with medical professionals to create solutions, fit perfectly within our mission statement:
Biorep enables advancements in medical technologies by offering the single source solution for healthcare professionals and researchers worldwide through a collaborative commitment to the design, development, and manufacturing of innovative biomedical products to improve the quality of life for patients.
Collaboration being one of our core values, we work directly with researchers on-site to discuss their requirements, and begin the engineering process by creating sketches to begin taking their idea to reality. With this information, our team of engineers take these requirements and sketches and begin our development process. The sketches are turned into engineering models and drawings, and come to life in our R&D lab with rapid prototyping techniques. These prototypes are then provided directly to the researchers for their input and feedback to ensure they meet their requirements. With their continued input and guidance, Biorep creates a product to enable the researchers to continue their cutting edge research in a more efficient and innovative way. We always strive to evaluate and innovate our current products to ensure they meet the needs of today, look forward to the future, and wherever it takes us next.
Biorep was called upon by researchers at the University of Miami to engineer a machine to isolate stem cells from fat tissue; this product originated in this collaboration. The Stem Cell Isolator as a device, helps these researchers perform these trials in a novel, and innovative way in an important stage in the process: stem cell isolation. Originally intended for isolated stem cells to be turned into insulin producing cells used for diabetes research (Biorep was founded to enable researchers to identify a cure for Type I Diabetes), we worked directly with the Cure Alliance to re-purpose this machine for their COVID-19 clinical trials. Dr. Giacomo Lanzoni, PhD, a Research Assistant Professor at the University of Miami, and the Diabetes Research Institute, explains that “The stem cell isolator performs mechanical tissue dissociation and gives rise to fragments of a homogeneous size (based on the cutting screen pore size and shape). In the absence of exogenous enzymes, this mechanical and automated process enables the generation of fragments that largely preserve stem and progenitor cell niches.” This product is now patent-pending, as we have made an effort to engineer something truly innovative with mechanical properties unique to both the device itself, and the process for which it was created.
Dr. Ricordi, the principal investigator, is the Stacy Joy Goodman Professor of Surgery and Director of the Diabetes Research institute(DRI) and Cell Transplant Center at the University of Miami Miller School of Medicine. “We are very grateful to the FDA’s Center for Biologics Evaluations and Research, Office of Tissues and Advanced Therapies for performing four rounds of reviews in a record time — one week,” said Dr. Ricordi.
The FDA had previously authorized the testing of UC-MSC cell products in patients with Type I Diabetes and Alzheimer’s Disease at the University of Miami as part of other clinical trials. For the COVID-19 trial, Dr. Ricordi enlisted additional experts from around the world with extensive experience in infectious diseases, pulmonary medicine and critical care, while others provided expertise in cell-based product development and their use in clinical trials. The cell therapy is administered intravenously.
“There is no time to waste,” said Dr. Ricordi. “Patients who die from COVID-19 have a median time of just 10 days between first symptoms and death. In severe cases oxygen levels in the bloodstream drop, and the inability to breathe pushes patients towards their end very quickly; any intervention that might prevent that trajectory would be highly desirable.”
COVID-19 and pneumonia, like other highly contagious viruses, cause in many people, severe lung inflammation, making breathing difficult. Oxygen levels in the bloodstream drop and vital organs begin to fail. The science suggests the severe lung inflammation can be blocked with a simple IV infusion of Umbilical Cord Mesenchymal Stem Cells (UC-MSCs), preventing the life-threatening progression of COVID-19. Pre-clinical and early clinical testing in pilot clinical trials indicate that this cell type can have a significant life-saving effect on patients affected by the most severe cases of COVID-19, associated with a powerful inflammatory reaction and associated cytokine storm leading to Acute Lung Injury (ALI) and Acute Respiratory Distress Syndrome (ARDS).
These trials are highly-anticipated as people across the country and around the world, watch with anticipation with the hope of a successful outcome. Dr. Ricordi and his team have already been interviewed and featured in several news stories, to help share the potentially ground-breaking efforts that are already underway. Please watch the MSNBC story here, coverage for the first transplant here, and even international coverage here. For more information about The Cure Alliance and their continuing coverage of COVID-19 and these stem cell trials, please visit here.
We are hopeful for the successful outcome of these clinical trials, and we are inspired by the highly competent medical professionals leading these trials. We wish to thank all of them for allowing us to be a part of this experience and effort to put our engineering expertise to work in a very meaningful endeavor. Biorep will share more information and the results of these trials, as more information becomes available.
Acknowledgments:
We thank the supporters of The Cure Alliance for this COVID-19 initiative, especially Barilla, the Italian Food Company, that has been The Cure Alliance major philanthropic supporter since the beginning of this initiative. We also thank the Diabetes Research Institute Foundation for supporting the DRI Team and the DRI cGMP Advanced Cell and Biologic Product Manufacturing Facility, that will provide the UC-MSC products used in this clinical trial.
Biorep would also like to mention and thank the key contributors of these trials and this important work: Giacomo Lanzoni (1), Elina Linetsky (1), Diego Correa (1), Roger Argelio Alvarez (2), Antonio Marttos (2,3), Khemraj Hirani (1), Shari Messinger Cayetano (1), Jose Guillermo Castro (2), Michael John Paidas (2), JoNell Efantis Potter (2), Xiumin Xu (1), Marilyn Glassberg (4), Jianming Tan (5), Amit Nilkanth Patel (6), Bradley Goldstein (7), Norma Sue Kenyon (1), David Baidal (1), Rodolfo Alejandro (1), Rodrigo Vianna (2,3), Arnold Caplan (8) and Camillo Ricordi (1,2)
- Diabetes Research Institute, Cell Transplant Center, University of Miami Miller School of Medicine, FL, U.S.A.
- University of Miami Health System and Jackson Health System, FL, U.S.A.
- Miami Transplant Institute, FL, U.S.A.
- University of Arizona College of Medicine, AZ, U.S.A.
- Fuzhou General Hospital, China
- University of Utah, UT, U.S.A.
- Duke University, Durham, NC, U.S.A.
- Case Western Reserve University, Cleveland, OH, U.S.A.
