ACT updates myoblast program for heart failure treatment

Sep 30 2010

Advanced Cell Technology ("ACT"; OTCBB: ACTC), a leader in the field of regenerative medicine, provided an update today on the status of its myoblast program. The program has successfully and safely completed Phase I human clinical trials in more than forty patients in over four independent studies. The Company is in the process of evaluating the data and is working with its scientists to evaluate the size and score of a potential Phase II trial. ACT began its myoblast program for the treatment of heart failure, advanced heart disease, myocardial infarction, and ischemia with the acquisition of Mytogen Inc in 2007.

“We believe our phase II study would represent one of the most advanced stem cell studies conducted to date”

The Phase I trials were conducted on a myoblast stem cell therapy involving transplantation of expanded autogolous myoblast (adult progenitor stem cells) derived from a small biopsy of skeletal muscle in the leg. The technology allows for expansion of the myoblast into hundreds of millions of cells over the course of two to three weeks. The surplus is then transplanted back into the patient's scarred heart tissue. The transplanted cells are incorporated into the muscle tissue, are entrained in the beat of the heart, and from preliminary data, appear to also enhance revascularization of the infarcted area.

Data collected from the trials suggests that the transplanted myoblast often improve heart function, and can lead to a significant improvement in quality of life for the patient. In the Phase 1b study, the company was able to score patients using the New York Heart Association Quality of life test, and the average observed improvement was significantly better than typical standard of care therapies in use today. According to the National Institute of Health, approximately five million people suffer from congestive heart failure, with an estimated 400,000 new cases diagnosed each year. Of the total number afflicted with the disease, 50% will die due to related complications within five years.

"We believe our phase II study would represent one of the most advanced stem cell studies conducted to date," said William M. Caldwell IV, ACT's Chairman and CEO. "In addition to evaluating our clinical options, the Company is also considering the most effective way to finance the cost of the study, which could include grants, partnerships or capital provided by foundations. We are also in negotiations with several countries in Europe to conduct a similar trial. The market potential in the EU is even greater than in the U.S. by up to 50% in patient population size by some estimates."

The FDA has given ACT clearance to proceed with a Phase II trial, which is expected to begin in the first half of 2011. The open-enrollment trial will target patients who are not eligible for angioplasty or coronary artery bypass surgery and have a poor quality of life despite the help of a pacemaker or defibrillator device.