Clinical translation section: accelerating the pace from bench to bedside

Excerpt

Cell and gene therapy clinical trials have been conducted for various indications such as cancer, cardiovascular diseases and autoimmune disorders for more than 20 years. The increased understanding of immune function, cancer biology, and stem cell biology have dramatically accelerated the development of technology for cell and gene therapy in these areas. Supported by some successful clinical results, the development of many potential new technologies has produced an explosion of therapeutic pursuits in the clinic. The new technologies have produced significant challenges in the clinical translation space, including the need to develop innovative clinical trial designs, to accelerate development of therapies by minimizing the number of patients required to evaluate safety and efficacy; to develop and incorporate methods to capture important biologic effects of cell and/or gene based therapies in patients; and to dissect the impact of therapeutic combinations. Furthermore, the increasingly personalized flavor of cell and gene therapies has produced an ever-greater need for developing reliable biomarkers for selecting patients and measuring biologic effects of therapy. Finally, regulatory agencies have recognized the need for modifying acceptable evaluation metrics to respond to the increasing complexity in clinical design and interventions. Yet, despite significant advancement in the field, progress remains slow relative to discovery and the need of speeding up clinical application of basic science discoveries is still unmet. Indeed, the rapid advancement of therapeutic technologies in the laboratory; the plethora of biomarker candidates; and the recent innovations in clinical science concerning trial design, contrast with the slow development process from conception of an idea till proof of concept in the clinic. Typically, clinical trials of this nature take several years to develop, implement, complete and report. This creates a significant gap of knowledge in the field, as clinical innovation takes several years to be communicated. …