Abstract
Cell therapy for stroke may benefit from translational research guidelines to aid its entry into the clinic as a safe and effective treatment. Failures of clinical trials using neuroprotective drugs in stroke patients, despite promising laboratory findings, led to conference meetings under the banner of Stroke Therapy Academic Industry Round table, or STAIR, whose main goal was to formulate recommendations designed to enhance the success of translating preclinical research on neuroprotection into clinical application. In 2007, a group of scientists from academia and industry with interest in cell therapy and stroke, together with representatives from the National Institute on Neurological Disorders and Stroke (NINDS) and the Food and Drug Administration, convened to outline STAIR-like criteria called Stem cell Therapeutics as an Emerging Paradigm in Stroke, or STEPS, that will serve as a guide in the conduct of laboratory research and clinical trials of restorative therapies, in particular cell therapy for stroke (3). The preclinical arm of STEPS specifically calls for the use of appropriate species for stroke modeling, the need for standardization of functional tests and outcomes, the requirement for cell imaging and tracking, the incorporation of safety indices, and the prerequisite for determining mechanisms underlying restorative therapy (1). A stringent parameter for testing any experimental treatment is the replication of results across independent research laboratories, a critical translational bridge alluded to in the STEPS meeting, which coincidentally was solicited by the NINDS in its recent request for grant proposals.
To this end, a consortium consisting of multiple institutions with expertise in cell therapy and stroke is being pursued by the independent laboratories of Chopp, Steinberg, and Borlongan, in coordination with clinical input from Hess and Kondziolka and a biostatistics data coordination center by Lu. These investigators highlight the rationale for establishing such consortium and its purpose for evaluating the therapeutic potential of cell therapy for stroke in the present commentary (2). In view of cell therapy being marketed as “medical tourism” around the world, and the lifting of the US federal ban on embryonic stem cell research, which is likely to gain further public attention and scrutiny with respect to the safety and efficacy of this treatment, this raises the urgency for an orderly transition of laboratory findings into the clinic. The preclinical STEPS group serves not as an impediment to clinical trials of restorative treatment, but rather as a consultative faculty of scientists and clinicians who possess the desire of optimizing the therapeutic potential and assuring the safety of cell therapy for stroke. Whereas the current affiliations of this STEPS consortium are limited to the US, the near-term goal is to receive memberships from academic, industry, and legislative organizations worldwide working on restorative therapy.