1. Addressing the Unmet Needs of AMI Patients
Andrew Pecora, MD, FACP, Chief Medical Officer; NeoStem, Inc., New York City
Drug Discovery & Development - January 31, 2012
In the United States and other developed nations, heart disease kills more people
every year than cancer and costs nearly twice as much to treat. 1 For these patients,
adult stem cell biology signifies a new medical frontier.
Coronary heart disease (CHD) is the leading cause of death in the United States for
both men and women.2 CHD is caused by a buildup of plaque in the coronary arteries
leading to the heart. This buildup causes the arteries to narrow and, as a result,
myocardial ischemia—a restriction in blood supply to the heart—can occur. The
interruption of blood supply to a part of the heart can result in myocardial infarction
(MI) or acute myocardial infarction (AMI), commonly known as a heart attack. 3
Significant advances have been made globally in the prevention and early treatment
of myocardial ischemia and infarction, reducing morbidity, mortality and cost.
Despite these advances, 160,000 patients in the United States experience AMIs that
place them at risk for cardiomyocyte dysfunction leading to heart failure and other
adverse cardiac-related events, including death.4
There is an urgent need for novel therapeutic options for the prevention and early
treatment of myocardial ischemia and infarction. Adult stem cell therapy has the
potential to limit or prevent the adverse consequences of a large AMI. If confirmed to
be safe and effective, cell therapy may revolutionize the approach to cardiovascular
disease.
In some clinical studies, patients who received coronary artery injections of adult
stem cells derived from their own bone marrow after a myocardial infarction showed
improvements in left ventricular function not seen in patients given a placebo. The
larger the initial infarct size, the greater the effect of the infusion. There seems to be
a safety advantage of autologous cells over allogeneic cells; it also appears that
autologous transplanted cells remain in the heart and potentially continue to work
years later, as demonstrated in preclinical animal experiments.
Clinical trials using adult stem cell infusion as a treatment approach to AMI are
ongoing. Successes in pilot studies warrant large-scale longitudinal studies to examine
the potential effects of progenitor-cell administration on morbidity and mortality.

2. One example of an adult stem cell-based therapeutic application in the treatment of
AMI is AMR–001. AMR-001, manufactured by Amorcyte, Inc., is an autologous, bone
marrow-derived cell therapy designed to treat AMI patients with their own stem cells.
In a Phase 1 trial, AMR–001 showed a dose–related significant improvement in
perfusion. The study results demonstrated that patients receiving 10 million and 15
million cells showed significant improvement in resting perfusion rates at six months
as compared to patients receiving 5 million cells and control. The data also showed
that patients receiving 10 million or more cells showed a trend towards improvement
in ejection fraction, the percentage of blood pumped out of a ventricle with each
heartbeat, end systolic volume, infarct size and tissue death due to loss of adequate
blood supply at six month follow–up. No study–related significant adverse events were
reported. Having completed the Phase 1 trial with promising results, AMR-001 entered
its Phase 2 clinical trial in early 2012.
Intracoronary infusions of progenitor cells have shown preliminary successes in
preventing post-MI heart failure in clinical trials.5 Larger trials will be needed to
determine if past successes translate into reductions in clinical end points. If later-
phase studies are successful, it would open up a whole new way of treating heart
disease. Investigation into the use of adult stem cells in the treatment of AMI may
provide a promising future for regenerative therapeutics in the treatment of
progressive cardiomyocyte dysfunction and in addressing one of the leading causes of
death globally.
About the Author
Dr. Pecora is the chief medical officer at NeoStem, Inc., a biopharmaceutical
company engaged in regenerative science and the therapeutic development of cell-
based therapies. He is the former chairman and chief executive officer of Progenitor
Cell Therapy.
References
1. American Diabetes Association. Economic costs of diabetes in the U.S. in 2002.
Diabetes Care. 26:917–932, 2003.
2. Feero WG, et al. Genomics of Cardiovascular Disease. N Engl J Med. 2011;
365:2098-2109.
3. National Heart, Lung, and Blood Institute. NHLBI What Is Coronary Heart Disease?
National Institute of Health, 2011. Available at
http://www.nhlbi.nih.gov/health/health-topics/topics/cad/. Accessed January 27,
2012.
4. Amorcyte: About Amorcyte. http://www.amorcyte.com/about.htm. Accessed
January 27, 2012.

3. 5. Tongers J, et al. Stem and progenitor cell-based therapy in ischaemic heart
disease: promise, uncertainties, and challenges. Eur Heart J. 2011; 32(10):1197.
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