Exploring the State of the Science in the Field of Regenerative Medicine Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop (2017) / Chapter Skim
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7 Looking Toward the Future: Concluding Thoughts
7 Looking Toward the Future: Concluding Thoughts
Pages 65-76
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From page 65... ...
Daley and a panel of stakeholders then summarized their individual views of the key themes that emerged throughout the workshop and added their perspectives about the prospects for regenerative medicine and the roadblocks that must be addressed in order to move forward. HYPE AND THE PROMISE FOR CHANGE Daley began by proposing a paradox, prompted by a Boston Globe opinion piece by Eric Lander titled "Hype vs.
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From page 66... ...
Stem cells and cellular therapies have followed this pattern, too, said Daley. Society is already reaping the benefits of years of research and investment in some cell-based therapies, such as hematopoietic stem cell therapy, he stated, highlighting the recent progress in applying gene editing and recombinant DNA approaches to T cell modification and other hematopoietic stem cell therapies.
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From page 67... ...
. The hope that accompanied the emergence of both embryonic stem cells and iPS cells as potential new regenerative approaches to treat disease has been tempered by the decades-long research and development process that has yet to yield an FDA-approved product, Daley said, but the field is on the cusp of success, with promising therapies to treat neurologic diseases currently in clinical trials and with remarkable progress having been seen in the field of in vitro gametogenesis to treat infertility.
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From page 68... ...
acts as a steward of the field of regenerative medicine by bringing together scientists and clinicians involved in stem cell research and promoting high scientific standards through communication and the development of guidelines for the responsible conduct of research, Daley said. In May 2016 the ISSCR released an updated set of guidelines for stem cell research and clinical translation.2 The initial set of guidelines was meant to assist researchers in the controversial field of embryonic stem cells, but it has evolved into a broader effort to guide the clinical translation of stem cell therapies.
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From page 69... ...
The Forum on Regenerative Medicine can support this effort by continuing to convene stakeholders in the field and by illuminating opportunities and challenges of regenerative medicine in a responsible way, he suggested. By investing in the deepest scientific understanding of regenerative therapies, the field will continue to sustain support from the National Institutes of Health and from the investors who will carry promising research forward into the clinic.
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From page 70... ...
These themes are described below. Understanding and Characterizing Cells One major challenge facing research in regenerative medicine, Daley said, is deriving the medically relevant cell types and defining them in such a way that that they can be assigned an identity and produced with reliable potency than can enable the development of a dose–response relationship in clinical trials.
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From page 71... ...
Workshop participants discussed the challenge of determining what endpoints to measure in order to assess quality, potency, and function for regenerative cellular therapies, asking the panel how the need for a deep scientific understanding of the therapy can be balanced with innovation and the role of commercial investment in clinical translation. "We need to sustain a culture of innovation," Daley replied.
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From page 72... ...
For example, Srivastava commented, while autologous cell transplants do not risk immune rejection, they may be "too expensive and face too many regulatory hurdles to be a realistic approach." There are efforts under way to develop iPS cell banks with multiple HLA qualities that could be used for off-the-shelf products, he noted. Participants pointed out other methods on the horizon for avoiding immune rejection, including advances in conditioning therapies and the reprogramming of endogenous cells.
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From page 73... ...
In order to get these therapies to the patients who need them most, a workshop participant said, it may be necessary to develop alternative funding mechanisms. For example, reducing the cost of upfront investment through government incentives or other means may encourage companies to invest in cell-based therapies, commented a workshop participant, mentioning a UK program called the Regenerative Medicine Platform in which the government funds research in several key areas and makes the resulting data available for the entire research community.
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From page 74... ...
Regenerative approaches for these conditions will focus on harnessing the regenerative capacity of endogenous cells through reprogramming, stimulating cell division, or introducing external materials such as exosomes, he said. Refocusing on Patients While the workshop focused primarily on the state of the science of cell-based therapies, many individual speakers urged participants to keep the needs of patients in focus.
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From page 75... ...
The Forum on Regenerative Medicine was designed for this very type of cross-fertilization, and its continued work will encourage stakeholders in the field to look for commonalities and overlapping interests "that will fire the imagination" and inspire collaboration, she concluded.
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