TABLE 6-2 Criteria for Drug Therapies Entering a Clinical Trial

  • The therapeutic window is not unrealistically restrictive.

  • The therapy improves both structural and functional outcomes.

  • The study is clinically relevant and has been replicated in an independent laboratory.

  • Improvements are seen in multiple animal models, with clinically relevant end points.

  • Major findings are published in reputable peer-reviewed journals.

  • The safety of the treatment has been confirmed.

 

SOURCE: Adapted from Dietrich, 2003.

published and concerns over constraints in meeting the needs of the sponsoring funding agencies may be among the reasons for the lack of replication studies. The spinal cord injury research community needs to embrace and encourage these studies, which can be incorporated into broader studies that not only replicate a previous study but also include novel elements in the experiment. The National Institute of Neurological Disorders and Stroke (NINDS) identified the need for these types of studies by establishing the Facilities of Research Excellence in Spinal Cord Injury (FOR-SCI) funding mechanism in 2002 (NINDS, 2002). The Miami Project to Cure Paralysis is one of the two recipients of a FOR-SCI contract and is conducting studies to review and replicate studies in the areas of neuroprotection and axonal regeneration after a spinal cord injury. The second FOR-SCI contract is with the Reeve-Irvine Research Center and stipulates a focus on interventions to promote regeneration in the chronic setting. Similar contracts should be established for replication studies in other areas of relevant research.

Furthermore, it is critical that research findings, including those from replication studies and studies with negative or inconclusive results, be published in peer-reviewed journals with details about the study design, quantitative end points, and statistical analyses (Dietrich, 2003). Not only should positive study conclusions be presented, but a forum also should be generated to enable peer review of negative conclusions, especially those pertaining to replication studies. These efforts would enable the scientific community to scrutinize the data and would provide information to the spinal cord injury patient population on the results of preclinical and clinical testing of all novel therapies.



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