In January 2012, NeoStem enrolled the first patient in our PreSERVE Phase 2 trial, a multicenter, randomized, double-blind, placebo-controlled U.S. clinical trial to evaluate the efficacy and safety of a single intra-coronary infusion of at least 10 million cells of AMR-001, post STEMI, in subjects with ejection fractions of 48% or less as measured by cardiac magnetic resonance imaging (“CMR”). Patient enrollment was completed for this study in December 2013 with the first data readout expected in the third quarter of 2014.
The objective of the Phase 2 study is to determine the safety and the efficacy of AMR-001 in improving cardiac function and outcomes of patients after STEMI, a type of AMI. Read the PreSERVE Trial Overview to learn more about the trial.
The completed Phase 1 study of AMR-001 in 31 patients with damaged heart muscle following AMI showed a statistically significant dose-related improvement in myocardial perfusion. Patients who received 10 million cells (n=5) or 15 million cells (n=4) showed statistically significant improvement in resting perfusion rates at six months as compared to patients who received 5 million cells (n=6) or the control groups (n=15), as measured by Single-photon emission computerized tomography (SPECT). The study data also showed a dose-related trend towards improvement in ejection fraction (the percentage of blood pumped out of the ventricles with each heart beat), end systolic volume (the blood volume remaining in a ventricle at the end of contraction and the beginning of filling, which can be used clinically as a measurement of the adequacy of cardiac emptying), and reduction in infarct (death of tissue caused by shutting off the blood supply) size.
AMR-001 works by increasing microvascular blood flow in the heart muscle via the development and formation of new blood vessels, thereby reversing the restriction of blood supply caused by a heart attack and rescuing tissue from eventual cell death. The treatment process works as follows:
- A patient’s own bone marrow is harvested and a sterile pharmaceutical composition enriched for CD34+/CXCR4+ cells is prepared using our patented technology.
- The isolated cells are then infused via catheter into the infarct-related artery 6 to 11 days following an AMI, which we believe is the optimal time frame for cellular intervention, after the pro-inflammatory “hot phase” and prior to permanent scar formation, while the heart tissue is actively attracting CD34+/CXCR4+ cells.
- The infused cells migrate to the at-risk tissue along a hypoxia-induced Stromal-Derived Factor-1 gradient to a signal emitted from the infarct as described above, inducing angiogenesis and a resultant functional benefit.