Stem Cell Therapy for Spinal Cord Injuries

Stem cell-based therapies are an emerging branch of medicine with the purpose of restoring tissue function for patients with serious injuries, such as a spinal cord injury. As a result, scientists and engineers are increasing research efforts in the field of regenerative medicine. Due to the delicate nature of stem cells, producing the large quantity required for a successful therapy has proved challenging. In recent years, research has shown the potential of stem cell-based therapies, and thus there is a need for the commercialization of these treatments. The proposed facility targets the demand for spinal cord injury treatments and can support production for both clinical trials and a commercial release. Bioreactors designed specifically for the culture and growth of stem cells have flexibility in their ability to support different stem cell lines for various therapies. Small reactors in parallel can easily adapt to changes in production size. This process also takes advantage of the best options currently available for purification and preservation to maximize the product yield. Due to the strict regulations set in place by the FDA and lack of adequate funding, there is an untapped market for stem cell therapies for spinal cord injuries. Approximately 250,000 people in the United States suffer from spinal cord injuries, varying in severity, and this patient base increases at a rate of 12,000 new injuries every year (“Spinal Cord Injury Facts and Figures”, 2009). Future markets include expansion into Europe and Asia. There are two steps to this proposal: the upstream process and the downstream process. The upstream process includes the scale-up, differentiation, and purification of human embryonic stem cells; the downstream process consists of the scale-up of neurons for injection. The upstream process will be built initially and yield enough cells for clinical trials, without incurring the capital costs of building the entire plant. Upon success of the clinical trials, the downstream process will be built for maximum production. The profitability of this proposal is based on running 26 batches a year at 1.02x1010 cells per batch or 2.66x1011 cells per year. By targeting 5,000 patients, two percent of the current market, and charging $45,000 per dose, a profitable profile can be created. Assuming 50% production capacity the first year and a ten-year plant life, the ROI, NPV, and IRR of the proposal are 226.09%, $961,892,600, and 242.81% respectively. Using a 50% production capacity allows for higher profit margins upon expansion. The proposed plan will meet the need of this growing market. Disciplines Biochemical and Biomolecular Engineering | Chemical Engineering | Engineering Comments The PDF for this record is suppressed until September 2016. This working paper is available at ScholarlyCommons: http://repository.upenn.edu/cbe_sdr/60 Department of Chemical and Biomolecular Engineering Senior Design Reports (CBE) ______________________________________________________________________________ University of Pennsylvania Spring 2014 ______________________________________________________________________________ Stem Cell Therapy for Spinal Cord Injuries Priya Kumar Diana Moock University of Pennsylvania University of Pennsylvania Adam Muncan Michelle Sorkin University of Pennsylvania University of Pennsylvania Project Advisor: Dr. Miriam Wattenbarger, University of Pennsylvania Project Recommendation: Dr. Tiffany Rau, Eli Lilly 01 April 2014 Department of Chemical and Biomolecular Engineering University of Pennsylvania 220 S. 34th Street Philadelphia, PA 19104 Dear Professor Fabiano and Dr. Wattenbarger, Following is our complete report for CBE Senior Design: "Stem Cell Therapy for Spinal Cord Injuries." For this project, we were given the assignment of designing a manufacturing facility for the production of a stem cell-based, regeneration treatment for spinal cord injuries that would allow patients to fully recover from their injuries. Currently, there is no treatment in the market for spinal cord injuries that would yield this kind of significant functional improvement. Our treatment would meet this need. Further, we were tasked with creating a custom design for the bioreactor within which the stem cells for the therapy would be cultured. The stem cells are very sensitive to shear stresses and require very precise control of pH, dissolved oxygen levels, and temperature. There are vessels on the market that can be used to grow these fragile cell lines. However, our company aims to create and optimize a reactor for use under cGMP conditions. This proposal targets two percent of the current market for spinal cord injured patients in the United States (5,000 patients). To determine the economic feasibility of this proposal, net present value, return on investment, and internal rate of return were analyzed. In order for this project to be profitable in the first year of operation, the minimum number of patients treated must be 680, a very conservative target. The pursuit of this project is economically feasible, yet further research is required to verify the success of these proposed processes on a large scale. We recommend that the necessary research be explored with the intention that this proposal be carried out in full in future years.