Clinical trials for neuroregenerative therapies for spinal cord injury: what have we learnt so far?

ABSTRACT

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:

• traumatic spinal cord injury
• clinical trial challenges
• patient recruitment
• loss to follow-up
• patient heterogeneity
• multifaceted pathophysiology
• capturing positive treatment effects
• high costs

Acknowledgement

NH is supported by the Research Fund of the University of Basel for Excellent Junior Researchers. MGF is supported by the Robert Campeau Family Foundation/Dr. C.H. Tator Chair in Brain and Spinal Cord Research at UHN.

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This manuscript was not funded.