State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies - A Workshop
Regenerative medicine holds the potential to create living, functional cells and tissues which can be used to repair or replace those that have suffered irreparable damage due to disease, age, traumatic injury, or congenital defects. The field of regenerative medicine is broad and includes research and development of gene and cell therapies, tissue engineering, and non-biologic constructs. Although regenerative medicine has the potential to result in health and economic benefits, this relatively new field has struggled to enact policies and procedures that will assure patients that novel therapies are both safe and effective.
The potential applications of cellular therapies are broad, ranging from islet cell transplantation and regeneration to cure Type I diabetes, to regenerative neurobiology approaches to treat injuries and degenerative diseases like spinal cord injury and amyotrophic lateral sclerosis. Other areas of ongoing cellular therapy research include restoring vision through cell regeneration in the retina, repairing or restoring function in the musculoskeletal system, and improving or restoring function in various organs including the heart, liver, lungs, and kidney. Although the novel and exciting research findings in cellular therapies are promising, they would benefit from an improved understanding of the underlying biology and basic science involved in the differentiation, engraftment, behavior, and survival of implanted cells in vivo. This knowledge will help overcome scientific and technical hurdles relating to assessing and ensuring successful long term outcomes of cell therapies, controlling cell differentiation, and refining processes for production on a scale that is clinically effective and commercially sustainable. Research in these areas may also benefit from lessons and best practices identified by previous successes in regenerative cell-based therapies, such as hematopoietic stem cell transplants for the treatment of blood cancers. Increased communication and collaboration across fields may facilitate sharing of these lessons to help inform and advance ongoing research.
Current cellular therapies in regenerative medicine use several delivery approaches, including introduction of cells that have been modified, expanded, or genetically manipulated into diseased tissue, with or without supporting biologic or non-biologic materials such as key signaling molecules or scaffolding to facilitate the delivery and success of these therapies. Much of the research on these approaches is carried out using in vitro or rodent models, neither of which closely mimics the complex environment of the human body. Translating promising research from these models to clinical studies is a challenging process and as the field matures, it will be helpful to develop guidelines for the safe and proper use of regenerative medicine advances, highlight translational barriers, and the explore the regulatory environment. Additionally, the study and use of regenerative medicine therapies are complicated by ethical and social debate taking place around the use of adult, embryonic, and induced pluripotent stem cells.
On October 13, 2016, the Forum on Regenerative Medicine hosted a public workshop with the goal of highlighting opportunities and challenges associated with developing regenerative medicine cellular therapies and related technologies. Stakeholder groups, including research scientists, clinicians, patients, payers, regulators, and representatives from pharmaceutical and biotech companies, presented their perspectives and participated in discussions during the workshop.