Stem cell research and therapy understandably accounts for much excitement and caution within the life science community and general population. The potential applications of stem cell therapy, from regenerating organs and limbs to fighting cancer, seem endless. The Food and Drug Administration (FDA) recognizes the exciting potential benefit that stem cell therapy poses, but also weighs the potential risks and defends its stance on regulations in an article recently published in The New England Journal of Medicine.
Safe use and efficacy of stem cells certainly exists. For example, the successful use of stem cells derived from peripheral blood or bone marrow for hematopoietic reconstruction is well established. However, expanded use into new areas does not have strong empirical support. The FDA argues that “despite the absence of compelling evidence from adequate, well-controlled clinical trials, some practitioners assert that stem cells have a unique capacity to restore health because they can sense their environment and differentiate in a manner that repairs any defect…[this] assertion… is not based on scientific evidence.”
The FDA admittedly worries about the lack of evidence, stating that “the literature is replete with instances of therapeutic interventions pursued on the basis of expert opinion and patient acceptance that ultimately proved ineffective or harmful when studied in well-controlled trials comparing them with the standard of care.” The FDA expresses the dangers of therapies derived from anecdotal success stories and cited a time in the United States’ history in which such therapies were rampant, and patients unfortunately suffered the consequences. Researchers and companies developing therapeutic products argue that current and/or additional regulations will stifle creativity and delay the cycle of getting potential life-saving therapies to patients. According to the FDA, this fear stems from the lack of familiarity with the available pathways for developing cellular therapy products and the lack of a “systematic, facilitated approach to assembling the clinical data necessary to support the licensure of stem cell therapies produced by individual practitioners at different sites.” The FDA closes its argument by restating its mission of facilitating the development of safe and efficacious therapies that are readily available for patients while maintaining a process that supports future scientific advances.
Navigating the current regulations surrounding stem cell therapies is a necessary step in the product development cycle. However, expedited pathways do exist for the treatment of life-threatening diseases that currently have an unmet medical need. Pearl Pathways is equipped to support you during this process and alleviate the burden that regulations seemingly impose. Contact us today for support.