Marbán Lab

The Marbán Laboratory focuses on two disease themes. The first builds on the long-standing interest of Eduardo Marbán, MD, PhD, in the molecular basis of excitability to create novel, biologically based treatments for cardiac arrhythmias.

The Marbán Lab was the first to create a de novo biological pacemaker, work that is now proceeding toward first-in-human clinical testing. The laboratory is also developing extracellular vesicles as therapeutic agents for refractory ventricular tachycardia.

The second broad category of research in the Marbán Laboratory is in cell therapy and regenerative medicine, which the team has pursued for 16 years. The Marbán Lab was first to isolate and characterize a type of adult progenitor cell known as cardiosphere-derived cells (CDCs), which are now in advanced clinical trials. The first manuscripts defined the engraftment, differentiation and functional benefit of CDCs in murine and porcine models of myocardial infarction. Subsequently, the work has gone from proof of concept through completion of five clinical trials in patients with heart failure and/or Duchenne muscular dystrophy. These trials include ALLSTAR (the ALLogeneic Heart STem Cells to Achieve Myocardial Regeneration trial for moderate heart failure after myocardial infarction) and HOPE-Duchenne (the Halt cardiomyOPathy progrEssion in Duchenne muscular dystrophy trial). Two additional clinical trials of allogenic CDCs—Regress-HFpEF (Regression-Heart Failure with Preserved Ejection Fraction trial for heart failure with preserved ejection fraction) and ALPHA (ALlogeneic CDCs for Pulmonary Hypertension therApy)—are ongoing.

In the process of characterizing CDCs, the laboratory has shown that the mechanism of benefit is largely indirect, mediated by the secretion of extracellular vesicles (exosomes). CDC exosomes are currently being developed as next-generation therapeutic candidates for a variety of clinical indications. A major effort currently in the lab is mining the contents of CDC exosomes to identify defined noncoding RNA species, which may themselves be viable therapeutic candidates. Work in the lab spans from mechanistic discovery science through advanced clinical trial design and analysis, a continuum to which trainees are rigorously exposed.

The Marbán Laboratory is affiliated with the Smidt Heart Institute, Cardiology Department and Barbra Streisand Women's Heart Center.