SIU 2018: Cell Therapy and Constructs – Why is it Not Yet in Clinical Use?
The available regenerative platforms in his center include cell therapy, biomaterials, tissue engineering, devices, and small molecules. The cell therapy platform includes both autologous (from the same person) and allogeneic (from a donor) stem cell transplants, usage of placental cells, muscle progenitor cells, and testicular tissue. In the biomaterials platform, there are projects involving natural and synthetic materials including collagen, hyaluronic acid, and many others.
Tissue engineering involves ambitious projects trying to create urethras, bladders, vaginas, anal sphincters, and many more organs. Through tissue engineering, using cells, biomaterials and signaling factors, whole organs are attempted to be created, and eventually implanted within the patient. Adding cells to various scaffolds gives the necessary platform for tissue regeneration.
There have already been some attempts in creating whole organs through tissue engineering. For instance, bladder creation has been attempted in seven spina bifida patients with failing bladders, who have been involved in successful phase two trials, with more than 15 years of follow-up. Other organs include an engineered urethra in five pediatric patients, who have had traumatic urethral strictures and have been followed for up to 72 months up until now, with promising results. Lastly, four patients with vaginal aplasia have been implanted with an engineered vagina, being functional over eight years after surgery, with all patients reporting being sexually active.
It is important to remember that the entire process from idea, until FDA approval and being given clearance to be used clinically in a standardized manner, takes approximately 20 years! This is a long process, beginning from the scientific discovery, going through process development, validation and manufacturing, and then through the regulatory and clinical phase, up to the final commercialization and logistic issues.
However, it is important to remember that successful discovery does not equal successful technical transfer. Many times, the clinical needs of patients do not match the market needs and this can halt the entire process of development. Furthermore, scientific or positive clinical outcomes do not mean that the product will be successful in the market, causing the entire project to be canceled. Additionally, successful phase one trials do not necessarily mean successful phase 2 and phase 3 trials. The developers need to consider the clinical outcomes, the cost, and the market competitiveness. Unfortunately, another major hurdle is the lack of standardized regulatory requirements around the world, making global harmonization an ideal that is far from being realized. Lastly, there is uncertainty in the reimbursement of these novel products, making investors hesitant to keep investing in the product.
Dr. Yoo concluded his talk stating that to accelerate the development timeline, we must engage experts from the various fields of regulation, clinical, and industry in the early stages of the scientific discovery, and try to get maximal co-operation and consensus in every step of this long journey.
Presented by: James J. Yoo, MD Wake Forest Institute for Regenerative Medicine, Wake Forest School of Medicine, Winston Salem, North-Carolina, USA
Written By: Hanan Goldberg, MD, Urologic Oncology Fellow (SUO), University of Toronto, Princess Margaret Cancer Centre Twitter: @GoldbergHanan at the 38th Congress of the Society of International Urology - October 4- 7, 2018 - Seoul, South Korea