Goal 3: Advance Translational Research

Better disease models

Many diseases of the heart, lung and blood systems are studied using animal models, often with genetically engineered mice. However, while mice get models, humans get diseases. Too many grants are devoted to curing models, a practice encouraged by many high profile journals who want to see “proof” in a standard model of disease. Much less time, effort and money will be wasted on developing ineffective therapies if focus could be placed on developing new disease models in cells, tissues, animals or humanized animals that better capture what is happening in patients. Developing and validating better models is a critical challenge for successful translation.

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Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Critical Challenge (CC)

Details on the impact of addressing this CQ or CC :

Morphologic appearance is necessary but insufficient as a criterion for validating a model. Comparative “omics” should be strongly encouraged. It is a waste of resources to develop therapies based on the effects of yet another gene knock out in something as unphysiological as atherosclerotic lesions in an ApoE knock out mouse or asthma in a mouse with eosinophilic airspace disease that largely ignores the bronchial tree or the infusion “stem cells” on cardiac function in a mouse with an experimental myocardial infarct. These are simply prominent examples of widely used standard models with little real utility. Therapies based on such approaches are likely to fail. Better models that more realistically capture phenotypic features of real diseases could lead to more promising therapies.

Feasibility and challenges of addressing this CQ or CC :

Model development is not very sexy and stands a better chance of acquiring support outside the standard study section review mechanism where proposals are judged by investigators with a vested interest in (and academic career based on) using existing models, despite their poor track records.

Name of idea submitter and other team members who worked on this idea : Jordan S. Pober

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Idea No. 393