Traversing the Translational Trail for Trials

Clinical trials for neurological disorders do not readily lend themselves to using a simple and direct outcome measure (clinical endpoint) to detect whether a drug, cell transplant, or rehabilitation strategy provides a meaningful (functional) benefit to patients. The central nervous system (CNS) is the most heterogeneous tissue of the body with hundreds of different cell phenotypes, each of which is capable of responding to a particular treatment in some specific and perhaps unpredictable or incomplete manner. In this review, the focus is on rehabilitation strategies, and spinal cord injury (SCI) will be used as a model CNS disorder.

• 1
  RCTs usually provide the strongest evidence for a treatment effect that most everyone will accept with a high degree of confidence.
• 2
  The largest degree of recovery occurs within the first few weeks and months after the initial damage.
• 3
  Increasing the likelihood that patients with sensorimotor complete SCI can regain function over as little as 2 spinal cord segments can make a dramatic improvement in their ability to independently complete many self-care activities