In September 2007, Advanced Cell Technology completed the acquisition of Mytogen, Inc. and its Myoblast program for the treatment of heart failure. The Myoblast program has successfully completed Phase I human clinical trials utilizing the therapy safely in over forty patients. Safety of the company’s myoblast therapy has been demonstrated in four independent studies. Moreover, the company has been permitted to apply its myoblast therapy without the use of accompanying arrhythmia preventatives usually required for similar programs. The FDA has reviewed the “end-of-Phase I” data and will allow Advanced Cell Technology to proceed with a Phase II human clinical trial. The company plans to begin the Phase II human clinical trial for the treatment of heart failure in approximately 160 patients. To that end, ACT and Catholic Healthcare West (the eighth largest hospital system in the nation and one of the largest not-for-profit hospital providers in the West) announced that the two parties have entered into a letter of intent (LOI) for a proposed exclusive business arrangement to establish a clinical trial research site and a North American regenerative medicine interventional cardiology training center for the aforementioned ACT Phase II myoblast cellular transplantation clinical study. The Phase II human clinical trial will utilize 3-Dimensional Guided Catheter-Based Delivery of Autologous Skeletal Myoblasts for Ishemic Cardiomyopathy (CAuSMIC). The trial, which will be open for enrollment in the next few months, will be led by principal investigator Dr. Dib and will target patients who are not eligible for angioplasty or coronary artery bypass surgery and who continue to have poor quality of life despite receiving optimal medial therapy or cardiac resynchronization therapy with a pacemaker or defibrillator device. If the Phase II human clinical trial is successful, the company expects to proceed with a pivotal Phase III trial.
The Myoblast Program may prove particularly beneficial for patients that have experienced a serious heart attack and have a high risk of heart failure. 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 technology 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 National Heart, Lung, and Blood Institute (NHLBI), a division of the National Institutes of Health (NIH), approximately 5 million people in the United States have congestive heart failure (CHF) and an estimated 400,000 new cases are diagnosed each year. Roughly 50% of CHF patients die within 5 years. The annual number of deaths directly from CHF increased from 10,000 in 1968 to 42,000 in 1993, with another 219,000 related to the condition. CHF is the first-listed diagnosis in 875,000 hospitalizations, and the most common diagnosis in hospital patients 65 years and older.