Planning for clinical trial of fully-biologic, cell generated graft (CGEM) for ACLrepair

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PROJECT SUMMARY/ABSTRACT Introduction: Skeletal Tissue Engineering Laboratories (STEL) Technologies, LLC is a regenerative medicine company spun out of the University of Michigan. STEL’s first product, the CGEM graft, is a unique fully biologic “off-the-shelf” tissue-engineered graft for repair of the anterior cruciate ligament (ACL) that allows the patient’s body to regrow native ACL tissue, resulting in improved knee stability and function for the ACL injuries. Significance: More than 350,000 ACL reconstruction surgeries are performed each year in the U.S. [1]. In addition to the high cost of the procedure, these patients are at high risk of developing osteoarthritis (OA) within a decade after surgery. With the increase in pediatric ACL injuries, the long-term costs and quality of life implications will be an increasing healthcare concern until innovative new treatments are available [2]. The current golf standard for ACL repair is either non-surgical, conservative treatment or surgical repair. The current grafts used for repair don’t restore normal function. Early onset OA remains a risk and is seen in about 37% of treated cases [2]. Goals of Proposed Research: This R34 clinical trial planning grant is focused on preparing our team and technology for a successful first-in-human clinical trial. Regenerative medicine projects are tremendously complex and expensive and have not been embraced by the Venture Capital (VC) community. To date, our research has been supported by a combination of NIH and state economic development grants, but to ensure success with the next stage of development, we must make significant investments in the team and planning of our clinical trial program. There is significant enthusiasm from the orthopedic community and the proposed project will advance the translatability of this technology for use in human patients and help our team attain a higher probability of commercial success by clearing three key milestones: · AIM 1: Develop a detailed clinical study protocol and budget for a Phase I clinical trial at University of Michigan. · AIM 2: Establish Investigator Manual of Operating Procedures, data safety and monitoring plan (DASP) and statistical analysis plan to support IRB approval of Phase I clinical trial. · AIM 3: Assemble the Documentation Needed for the Investigational New Drug (IND) Submission to the Food and Drug Administration (FDA). Successful completion of the above milestones will help our team design and prepare for a high-impact clinical trial program. We have assembled the management talent and technical expertise necessary to help design the clinical study and prepare the documentation for a successful first-in-human trial at the University of Michigan. STEL’s “off-the-shelf” CGEM graft for ACL repair represents a significant advancement for regenerative medicine and a transformative innovation for the treatment of soft tissue orthopedic injuries.