Forging the Fate of Cellular Therapies for Cardiovascular Disease.

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The field of cell therapy for cardiovascular disease has progressed at an uneven rate and will likely follow the translational paths of other biological therapies. The field will require novel public–private partnerships to guide it to its ultimate applications. The long process of developing new therapeutics for cardiovascular disease may often seem like a guideless journey into the unknown. The process begins with definition of an unmet clinical need and is combined with insights into the pathophysiologic nature of disease. Whether the proposed therapy is a device, small molecule, or biological each requires extensive preclinical and clinical investigation prior to approval. While each new treatment must create its own path to approval, outcomes may be predicted by prior experiences. Cell-based therapies for cardiac and vascular disease have progressed at an uneven pace. High-profile publications suggested plasticity and potency of bone marrow–derived cells when delivered into infarcted myocardium and spurred clinical investigation seemingly before the ink dried on the original papers.1 These human studies identified a promise of therapy without harmful effects when data were aggregated.2 However, these uniformly positive small studies have not been confirmed by larger studies. This has prompted a reexamination of the underlying premise on which the original studies were based.3,4 The path by which cell-based therapeutics is being developed is illuminated by the development of other biological approaches for cardiovascular disease. The similarities allow for educated guesses as to the outcome of this therapeutic journey. Will cells ever be widely used as first-line therapy for cardiac and vascular diseases? Which diseases will be the first approved targets for cell-based therapies? How might the development process be improved? I think the answers to these questions may be informed by the fates of other biologics, including antibodies, gene therapy, and peptides. Few academic …