treatment of spinal cord injuries are being explored, including efforts to prevent or reduce the adverse consequences during the acute phase of the injury and combination strategies designed to remyelinate nerve fibers, promote nerve fiber growth, and prevent cell death. During the development and verification of new therapies, researchers and regulators make decisions regarding when the data are sufficient to indicate that the intervention is efficacious and safe and can move on to the next stage of the process. The challenge is to develop therapies in a timely fashion without undermining future scientific endeavors and, most importantly, without endangering patient safety.

CRITERIA FOR VALIDATING A NOVEL THERAPY

The spinal cord injury research community is making substantial progress in developing novel therapies that may soon be ready for clinical trials. However, many of the alternative therapies that individuals with spinal cord injuries are using are not recommended options or standards of care because they have not been proven to be safe and efficacious (see Appendix F). An overriding concern that arises when a researcher contemplates translating a successful laboratory therapy to the clinical setting is the extent of preclinical data needed to justify proceeding with testing in studies with humans (Ramer et al., 2000; Kleitman, 2004). A coordinated and methodical approach is needed to verify the safety and effectiveness of therapies and treatments that are proceeding through the research pipeline. A 2003 article published in the Journal of Rehabilitation Research & Development describes a set of criteria that should be considered before a treatment can enter into a clinical trial (Table 6-2) (Dietrich, 2003). To meet these criteria, the author recommends that a coordinated effort among the spinal cord injury research community be mobilized to quickly respond to new scientific findings. As discussed in detail in Chapter 7, an enhanced research infrastructure and network is needed to facilitate collaborative research efforts.

Verification of a Therapy’s Preclinical Effectiveness in Replicated Studies with Animals

Preclinical testing provides data on whether a therapeutic intervention holds promise for the treatment of spinal cord injuries in humans. However, because the nature and severity of spinal cord injuries vary between individuals, a wide spectrum of behavioral and functional deficits exist, with no one outcome occurring among those with spinal cord injuries.

An emphasis on the replication of preclinical studies (replication studies) between laboratories is needed. Difficulties in getting replication studies



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