Stem Cell Clinical Trials for Spinal Cord Injury: Readiness, Reluctance, Redefinition

Abstract

A wealth of scientific and clinical research has focused on the use of stem cells as a potential therapy for spinal cord injury (SCI), culminating most recently in the initiation of clinical trials. However, with the urgency that scientists and clinicians have undertaken to move forward with novel therapies for this devastating injury, the perspectives of stakeholders who live with a SCI have been left behind. Translational research in this rapidly growing field therefore overlooks a critically important viewpoint. We address this concern with a qualitative study of the perspectives on experimental stem cell treatments from individuals who have actually suffered a spinal cord injury. Using focus groups and interviews, we engaged individuals with thoracic and cervical SCIs at sub-acute and chronic stages post-injury. We found four major themes that inform the progression of stem cell research to clinical trials: 'readiness', 'the here and now', 'wait and see', and 'informed hope'. Taken together, the data suggest a profound difference related to target timing of stem cell clinical trials and the perspectives about timing from those who are the end-beneficiaries of therapy. To bridge this gap, we conclude with a number of considerations for the timing disparity of trials and recommendations for improving informed consent.

Dissociations in the meaning of risk between health-care professionals and individuals with spinal cord injury

“…12,[14][15][16] Our results underscored the need to consider a patient's perception of risk as a dynamic rather than static phenomenon when designing clinical trials. Further research has interrogated the views of health-care professionals (HCPs) and injured individuals about the evidence needed to move from bench to bedside considering different risk frameworks.…”

“…9,12,[14][15][16] Definitions and meanings of risk vary, however, and understanding these in terms of hazard, exposure, consequence and probability is complex. 6 In this study, we identified three major differences in perspectives about risk between HCPs and ISCIs.…”

“…7,8 They analyze risks within levels of uncertainty, 9,10 and consider, on a theoretical level, and without reference to a practical definition, which risks would be acceptable to the SCI population on micro, meso and macro levels. 9,11,12 The micro level is defined by relationships between researchers, physicians and their participants, the meso level by oversight from institutional review boards and ethics committees, and the macro level by regulatory structures such as those from the Food and Drug Administration and other government bodies.…”

“…For example, if there is a risk of neurologic deterioration after stem cell transplantation, do the ISCIs who incur this risk worry about it to the same degree and for the same reasons as the researchers studying the technology? There is evidence that risk perceptions vary between professionals and patients, and may be influenced by both hope 12 and hype in the stem cell context. [13][14][15][16][17] Studies of risk communication generally speak to the need for incorporating and addressing personal values in the informed consent process.…”

Ethics and Policy Issues for Stem Cell Research and Pulmonary Medicine

“…Further, potential participants may be particularly vulnerable to hype, desperation, and the therapeutic misconception (not appreciating the diff erence between usual clinical care and research). [81][82][83] Appropriate and Equitable Access…”