Abstract
Lost in Translation: Progress and Challenges in Advanced Therapies to Treat CVDs
Highlights
While gene-based therapies are rapidly advancing in many disease areas, targeting cardiovascular diseases (CVDs) is complex and challenging
The consortium delivering the enduring “autologous bone marrow cell therapy in acute myocardial infarction” (BAMI) trial, where patients with an acute ST-elevation myocardial infarction received an intracoronary bone marrow mononuclear cell (BM-MNC) infusion within 2–8 days following acute revascularisation by primary percutaneous coronary intervention (PCI), was unable to report definitive conclusions regarding the efficacy of BM-MNC therapy, principally due to low recruitment and event rates.[3]
Buoyed on by anecdotal proclamations of success, we await the publication of defining data from recent pioneering surgeries where Human induced pluripotent stem cells (hiPSCs)-derived cardiomyocytes have been administered to patients with heart disease via direct intracoronary injection and a cellularized tissue-engineered graft.[5]
Summary
While gene-based therapies are rapidly advancing in many disease areas, targeting CVDs is complex and challenging. Despite expansive and largely positive outcomes in pre-clinical studies using cell-based therapies to treat myocardial injury by targeting cardiomyocyte regeneration and/or endogenous neovascularisation, autologous cell therapy trials in patients with acute myocardial infarction or heart failure have largely failed to live up to predicted expectations over the past decade.
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