Once a Program has been established, working groups meet in workshops and through internet-based conferences to identify key challenges in finding treatments for diseases. These open exchange meetings help investigators share the results of their experiments and design innovative approaches that best leverage their experiments to address these challenges. Current investigators and invited participants identify key rate-limiting steps in which solutions may lead to pre-clinical and then clinical proof-of-principle studies of the feasibility for a therapeutic strategy. Their discussions, experiments and data sharing iteratively inform and enrich the work of the individuals in each collaboration and Program.
We enrich collaborations by funding institutions and core platforms that are valuable across our Programs. Some of these centralized activities include microarray gene studies, proteomics, micro and macro anatomical and functional imaging, high throughput systems for drugs, behavioral measures, the organization of clinical trials and databases. The investigators in our various Programs may collaborate through these platforms, as well as around common denominators of disease. For example, the manipulation of cancer stem cells to halt their malignancy may involve the knowledge gained in promoting the growth of stem cells for repair after stroke, spinal cord injury or damage to peripheral nerves. Also, the up and down regulation of specific genes and proteins may cause one cell to become malignant, but serve to enable regeneration of another cell after injury.
We aim to develop therapeutic interventions from preclinical studies through the stages of safety and potential efficacy trials in patients when a Program's investigators find a robust effect, especially if found in more than one disease model. Evidence for potential efficacy is derived from understanding basic mechanisms of action and both biological and behavioral effects associated with that action.