Initially, pre-clinical studies provided evidence that a patient’s own CD34+ stem cells (CD34), could stimulate new vessel formation in those patients suffering from ischemic myocardium. Consequently, a phase 1, first-in-human trial was completed providing even further evidence of feasibility, safety and increased bioactivity in those patients with CCS class 3 and 4 angina. The phase 2 study, designed to test the ability of a patient’s own stem cells (CD34 cells) to improve blood flow to the heart, could show the ability to improve heart function and reduce the possibility of events such as hospitalizations and heart failure in patients with refractory angina. A total of 167 patients were randomized and completed the injection procedure, and 162 patients completed the six-month evaluation.

“The results from this study provide the first significant evidence that a patient’s own stem cells can actually be used as a treatment for their heart disease:’ said Dr. Douglas Losordo, lead author from Northwestern University, Chicago, Illinois. “This study provides some hope for those patients with currently untreatable angina to be more active with less pain.

As a phase 2 study this program indicates how this strategy can be executed on a large scale at sites across the country. A program of this size has never been done in any stem cell cardiac study and is critically important if such a therapy is going to be effectively delivered.

It also provides information about appropriate endpoints regarding suitability and sample size of this therapy for a phase 3 efficacy study, the final study that will ultimately determine if the treatment works.