Efficacy and Safety: Pre-clinical Models and Non-invasive Monitoring
Numerous in vitro and in vivo models and assays lack predictive utility, causing a resource drain as therapies are disregarded part way through development. Increasing their success rate (currently below 20%) requires the TERM community to identify and abandon failed models, learn from them and devise new model systems. Many regenerative medicine therapies require more complex measures of toxicity and especially efficacy than conventional pharmaceuticals.
For the industry to thrive, screening systems and appropriate prediction models for cells and biomaterials must be devised with reliable, predictive utility.
One major barrier for assessing the success of regenerative therapies is the lack of non-invasive imaging strategies to monitor in vivo tissue repair and correlate functional outcomes with implant behaviour. Post-transplant information regarding cell localisation, survival, migration and differentiation could provide data to elucidate mechanisms of repair (which may be different to preclinical models) or reasons for graft failure (i.e. incorrect cell localisation). In contrast to small animal models, our ability to track transplanted cells in the clinic is currently limited.
This can be due to:
Inadequate tissue penetration
Toxicity and safety concerns (i.e. viral introduction of transplant-cell tracers).
Strategies to overcome these challenges will be discussed. This will result in the identification of key targets for the TERM community to address whilst also emphasizing the huge potential for routine clinical use of non-invasive monitoring for regeneration and monitoring of therapeutic implantation.