Translational research in spinal cord injury: a survey of opinion from the SCI community.
Much like our colleagues studying neuroprotection for acute stroke, we in the spinal cord injury (SCI) community have witnessed the preclinical emergence of numerous promising neuroprotective and neuro-regenerative treatments that have then disappointingly failed to demonstrate convincing efficacy in clinical trials. In contrast to the stroke field, the SCI community lacks guidelines to steer the preclinical development of therapies and maximize their chance of success prior to translation into expensive and laborious clinical trials. We conducted a survey of the SCI research community to garner perspectives on the question of what preclinical evidence was required before translating an experimental treatment into clinical trials. The opinions of the 324 respondents about what constitutes necessary preclinical evidence before moving to human SCI trials revealed strong support for the demonstration of efficacy in large-animal models, cervical injury models, and for independent replication of promising results. Marked differences exist between the sentiments of the respondents and the translational experience of our field. A framework for guiding the preclinical development of novel therapies prior to human translation would be helpful for ensuring clinical success. Greater dialogue on this issue is necessary to improve our chances of successfully bringing effective treatments to patients with this devastating injury.
Kwon
Brian K
BK
Combined Neurosurgical and Orthopaedic Spine Program (CNOSP), Department of Orthopaedics, University of British Columbia, Vancouver, British Columbia, Canada. brian.kwon@vch.ca
Hillyer
Jessica
J
Tetzlaff
Wolfram
W
D000818:Animals; D002985:Clinical Protocols / Q000592:standards; D002986:Clinical Trials as Topic / Q000592:standards; D004353:Drug Evaluation, Preclinical / Q000379:methods / Q000639:trends*; D019317:Evidence-Based Medicine / Q000639:trends; D006801:Humans; D023421:Models, Animal; D009488:Neurosciences / Q000379:methods / Q000639:trends*; D013119:Spinal Cord Injuries / Q000503:physiopathology / Q000628:therapy*; D011795:Surveys and Questionnaires; D057170:Translational Research, Biomedical / Q000379:methods / Q000639:trends*