Navigating the Manufacturing Process and Ensuring the Quality of Regenerative Medicine Therapies Proceedings of a Workshop (2017) / Chapter Skim
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5 Considerations for Improving and Regulating Regenerative Medicine Products
5 Considerations for Improving and Regulating Regenerative Medicine Products
Pages 55-66
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From page 55... ...
• MSC surface markers that the researchers in that field have agreed upon and proposed to use as potential CQAs, do not reveal significant differences between cell lines and their func tion. (Bauer)
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From page 56... ...
The regulatory landscape for regenerative therapies is evolving, and this chapter explores relevant issues including approaches to developing standards and establishing and enforcing regulations that meet the needs of patients. The speakers in this session were Robert McBurney, the president and chief executive officer of Accelerated Cure Project for Multiple Sclerosis and co–principal investigator of the iConquerMS Patient-Powered Research Network; Steven Bauer, the chief of the cellular and tissue therapy branch in the Center for Biologics Evaluation and Research at FDA; and Katherine Tsokas, the senior director for global regulatory affairs at ohnson & J Johnson.
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From page 57... ...
iConquerMS currently has 36 individuals on its governing board, research committee, and engagement committee, 22 of whom are lawyers, scientists, neurologists, and biorepository experts who have MS. Speaking about the regenerative medicine opportunities in MS, McBurney highlighted three areas in particular: rebooting the "confused" immune system with hematopoietic stem cells or MSCs; repairing the damaged myelin sheath with oligodendrocytes regenerated from MSCs or iPSCs; and regenerating lost nerve cells and reforming appropriate connections using MSCs or iPSCs.
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From page 58... ...
Early results from using autologous hematopoietic stem cells to treat MS have spurred the development of another upcoming trial, called the BEAT-MS trial. McBurney said he hopes that the BEAT-MS trial will carefully develop analytical aspects so that the researchers can ensure that the cells are in the best possible state prior to transfusion.
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From page 59... ...
The raw materials for the consortium came from Bauer's laboratory, which manufactured MSCs from eight different human bone marrow donors obtained from commercial sources. The consortium has subjected this material to a variety of analytical methodologies, including gene expression and epigenetic analyses and in vivo and in vitro models of immunosuppression and wound repair.
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From page 60... ...
This type of assay might also be useful for evaluating the impact of a manufacturing process, particularly tissue culture conditions, on desired biological activity. In fact, consortium researchers examined the effects of different manufacturing conditions on osteogenic activity, as predicted by morphological changes, finding differences according to the manufacturing conditions (Marklein et al., 2016)
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From page 61... ...
FDA's CMC guidance recommends that sponsors refer to USP sterility tests, which provide the details needed to conduct those assays. The challenge, she said, is to make sure the sponsor can apply that test to its particular product and follow the procedure exactly.
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From page 62... ...
One way that regulators could help sponsors during the development of a product is by providing additional feedback or assessment tools, Tsokas said, noting that such information would help sponsors understand whether they are implementing regulations and guidance appropriately. Another helpful approach, she said, would be to increase the number of USP monographs and specific checklists for standardized platform activities in order to help sponsors have confidence they are meeting regulatory requirements.
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From page 63... ...
It also requires regulators, industry, and academia communicating with one another and working together to determine where to make improvements that will help industry develop advanced therapies for the benefit of patients worldwide. DISCUSSION Increasing Collaboration Among Stakeholders FDA, NIST, and various standards determining organizations such as ASTM International, the American Type Culture Collection, the Standards Coordinating Body, and the National Institute for Innovation in Manufacturing Biopharmaceuticals are working together to determine where standards for regenerative medicine product manufacturing can be developed.
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From page 64... ...
When communicating with patient advocates regarding regulatory policies, FDA tries to engage actively by including the advocates in some of the agency's advisory panels. FDA is at the intersection of many different interests, Bauer said, but its interaction with patient advocacy groups is very highly valued.
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From page 65... ...
The issue of unproven stem cell therapies came up at a recent public workshop held by FDA in September 2016, Bauer said. The workshop focused on the scientific considerations and challenges to informing the development of human cells, tissues, and cellular- and tissue-based products subject to premarket approval and included speakers who discussed approaches to educating consumers about clinics offering unproven therapies.
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