ACT or ACTC.OB 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 40 patients in over 4 independent studies.
- ACT is in the process of evaluating data to evaluate the size and score of a potential Phase II trial.
The Phase I trials were conducted on a myoblast stem cell therapy involving transplantation of expanded autologous myoblast (adult progenitor stem cells) derived from a small biopsy of skeletal muscle in the leg.
- 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; ACTC.OB 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.
The Myoblast Program may provide for patients that have experienced a serious heart attack and have a high risk of heart failure.
- ACT began its myoblast program for the treatment of heart failure, advanced heart disease, myocardial infarction, and ischemia with the acquisition of Mytogen in 2007;
- The myoblast stem cell therapy involves transplantation of expanded autologous myoblasts (adult progenitor stem cells) derived from a small biopsy of skeletal muscle from a patient’s leg;
- The science allows for the expansion of myoblasts into hundreds of millions of cells over a period of two to three weeks;
- The resulting myoblasts are then transplanted back into the patient’s scarred heart tissue through the use of a catheter-based procedure;
- While the Phase I human clinical trials were focused on the safety of the therapy, the clinical data from those trials suggests that the myoblasts often improve function in the heart and can lead to a significant increase in quality of life for the patient.
According to the NIH, approximately 5 M 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.
- The market potential in the EU is even greater than in the US by up to 50% in patient population size by some estimates.
The FDA has given ACTC.OB clearance to proceed with a Phase II trial, which is expected to begin in FY/11. 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.