Introduction: There remains a lack of effective regenerative therapies to treat traumatic spinal cord injury (SCI). On a global level, patients living with SCI, their families, and the healthcare system are impacted by the extensive financial burden associated with the management of SCI. Clinical trials are crucial to assess the real-world efficacy of the different emerging neuroregenerative approaches that have shown promise in the pre-clinical stages of research.
Areas covered: This perspective summarizes and discusses potential solutions to several key challenges that clinical investigators evaluating novel therapeutic strategies to treat SCI might face: 1) the challenges in patient recruitment and meeting enrollment targets with adequate statistical power; 2) the loss of patients to follow-up; 3) the heterogeneity in patient presentation and recovery trajectories; 4) the complex multifaceted pathophysiology of SCI that renders investigations of single therapeutic approaches difficult; 5) the challenge of capturing positive treatment effects of investigative therapies; 6) the high costs associated with conducting clinical trials; 7) the implementation of current guidelines for treating SCI to optimize care delivery and clinical trial conduct; 8) the shift in SCI patient demographics reflective of an aging population; and 9) navigating regulatory bodies to translate therapies into the clinic.
Expert opinion: There are challenges when conducting SCI clinical trials that span broadly across medical, social, political, and economic considerations. Thus, we should employ an interdisciplinary approach when addressing these challenges in order to facilitate the evaluation of novel treatments for SCI.
Keywords: capturing positive treatment effects; clinical trial challenges; high costs; loss to follow-up; multifaceted pathophysiology; patient heterogeneity; patient recruitment; traumatic spinal cord injury.