Regulatory aspects of small molecule drugs for heart regeneration

Even though recent discoveries prove the existence of cardiac progenitor cells, internal regenerative capacity of the heart is minimal. As cardiovascular disease is the leading cause of deaths in the United States, a number of approaches are being used to develop treatments for heart repair and regeneration. Small molecule drugs are of particular interest as they are suited for oral administration and can be chemically synthesized. However, the regulatory process for the development of new treatment modalities is protracted, complex and expensive. One of the hurdles to development of appropriate therapies is the need for predictive preclinical models. The use of patient-derived cardiomyocytes from iPSC cells represents a novel tool for this purpose. Among other concepts for induction of heart regeneration, the most advanced is the combination of DPP-IV inhibitors with stem cell mobilizers. This review will focus on regulatory aspects as well as preclinical hurdles of development of new treatments for heart regeneration.