Abstract 3502: First in Man Transendocardial Injection of Autologous Aldehyde Dehydrogenase-Bright Cells in Heart Failure Patients (FOCUS-Bright)
Background: Stem cell therapy has been investigated in ischemic heart failure (IHF) patients. Although several clinical studies have utilized autologous bone marrow (BM) mononuclear cells, it is uncertain if a specific BM cell subtype may have greater therapeutic effect. This is the first in man study to assess the safety and efficacy of transendocardial injection of autologous bone marrow derived aldehyde dehydrogenase-bright cells (ALDHbr cells) in patients with severe IHF.
Methods: 20 patients with IHF (NYHA/CCS II/IV) with no option for revascularization were randomized (1:1) into treatment (transendocardial injection of 2.38±1.3 million ALDHbr cells) or placebo controls (transendocardial injection of saline). Primary endpoint was safety. Efficacy endpoints (reversibility in SPECT and MVO2) were also evaluated at baseline, 3 and 6 months follow-up. Comparisons were made by student t-test or Wilcoxon Signed-Rank test.
Results: There were no major peri-procedural complications. Adverse events were evenly distributed within treated and controls. At 6 months, treated patients had improved myocardial perfusion when compared to controls (reversibility 2.6±4.5% vs 8.0±5.2% respectively; p=0.012) (Fig A⇓). Delta MVO2 was higher in the treated patients vs controls; though not statistically significant (2.2±2.9 mL/kg/min vs 0.6±3.6 mL/kg/min respectively, p=0.304) (Fig B⇓).
Conclusions: Transendocardial injections of ALDHbr cells in patients with severe IHF are safe and can improve myocardial perfusion.