The (Translational) Road Less Traveled.

Abstract

An anonymous editorial in Nature Biotechnology asserted that cell therapy for heart disease has been a failure and should not continue. In actuality, the development of CD34+ cells for treatment of refractory angina was a well-informed and well-designed pathway toward Food and Drug Administration (FDA) approval to address an important unmet clinical need. Contrary to what was asserted in the Nature Biotechnology editorial, trials of CD34+ cell therapy for refractory angina not only met their primary end points but were based on a clear mechanism of action. > I shall be telling this with a sigh > > Somewhere ages and ages hence: > > Two roads diverged in a wood, and I— > > I took the one less traveled by, > > And that has made all the difference . > > Robert Frost (1874–1963), “The Road Not Taken” In April 2017, an anonymous editorial was published in Nature Biotechnology entitled “A Futile Cycle in Cell Therapy: Should a Cell Therapy for Heart Disease With Scant Evidence of Efficacy Continue to be Tested in Humans?”1 The authors cite a single, phase 2 study in patients with recent acute myocardial infarction—the PreSERVE-AMI trial—as evidence that “cell therapy for heart disease” has been a failure and that the field should focus more on “bona fide cardiomyocytes or progenitors that may differentiate into heart muscle and defined paracrine factors that may tip the balance from fibrosis toward repair.” We take issue with several points in the editorial, the primary being the anonymous author or authors’ apparent disregard of basic principles of therapeutic development, the failure to distinguish between types of heart disease, and a poor understanding of the available evidence in the field, particularly as it relates to the cell type under study. The test agent in PreSERVE-AMI was the autologous CD34+ cell.2 To date, there have …