The Forum on Regenerative Medicine of the Health and Medicine Division (HMD) of the National Academies of Science held a workshop entitled, “State of the Science in the Field of Regenerative, Medicine: Challenges of and Opportunities for Cellular Therapies”. Experts and stakeholders in academic and industry research, patient advocacy, and law and ethics addressed both the promise and challenge of regenerative medicine.
Cell, tissue, and organ damage due to illness, injury, or aging is a growing health concern. Regenerative therapies for craniofacial, neurological, and muscular tissues damaged from tumors or injury could change the course of painful, debilitating, and even fatal diseases. Scientists have made key discoveries, such as cell reprogramming, that have propelled regenerative medicine forward, but formidable scientific, technological, and regulatory hurdles remain.
Lorenz Studer of the Sloan Kettering Institute discussed challenges with verifying cell types, understanding the stages of cell maturation most compatible with transplantation, accurately recapitulating human physiology and pathology in animal models, coaxing cells to adopt a three-dimensional architecture and to communicate with each other and different cell types, and overcoming the persistent threat of host rejection. Furthermore, for these therapies to become commercially viable, researchers will have to overcome manufacturing challenges in scalability, reproducibility, and quality control that will require significant technological advances, workforce training, and financial investments.
A persistent theme through the workshop was the length of time required for significant advances to occur. Studer recounted his two-decade journey in dopamine neuron regeneration for the treatment of Parkinson’s Disease. Ultimately, Studer and colleagues were able to graft the neurons into a mouse model of Parkinson’s Disease and demonstrate symptomatic relief. Anthony Oro of Stanford University described a similar 18-year effort in skin regeneration for the treatment of epidermolysis bullosa, a potentially fatal connective tissue disorder that can result in severe and painful blistering and squamous cell carcinoma. Currently, Oro’s work is in early-phase, proof-of-concept clinical trials.
Pat Furlong, President of Parent Project Muscular Dystrophy, gave a particularly compelling presentation from the perspective of a patient advocate. Furlong integrated current challenges and pitfalls of regenerative therapies in muscular dystrophy with her personal story of losing two sons to the disease. Furlong stressed the importance of exercising caution and restraint when communicating with patients and relatives about therapies still in the experimental stage.
NIDCR houses an active program in tissue engineering and regeneration and director Martha Somerman is a forum member. Furthermore, several AADR members are actively involved in and making significant contributions to regenerative research for periodontal disease, temporomandibular joint disorders, and other diseases of the dental, oral, and craniofacial complexes. AADR staff will continue to monitor and communicate future HMD activities in regenerative medicine.