A clinical trial that is presently being conducted in Russia is making use of a special stem cell population collected from menstrual blood. These menstrual blood stem cells are being used to treat heart failure patients and to date these stem cells appear to be quite safe for human patients.
To deliver the stem cells for this clinical trial the researchers used a catheter-based retrograde delivery technique in which simply means that angioplasty technology that is also used to place stent into coronary blood vessels was used to slowly deliver the cells to the heart. Essentially, a guidewire is inserted into the femoral artery in the patient’s groin. The guidewire is moved through the artery, all the way to the coronary sinus. There, and catheter is slipped over the wire and threaded to the end of the guidewire. While in the coronary sinus, dye is injected in order for the cardiologist to view the coronary tree of the heart. Throughout this procedure, the cardiologist is guided by a kind of live X-ray known as a fluoroscope. The guidewire is then inserted into the coronary artery that is blocked and a small balloon is threaded over the wire to expand the clogged vessel. Alternatively, a stent can be placed to prop open the vessel.
In the case of stem cell delivery, the guide wire is threaded with a small balloon filled with stem cells that are slowly released into the blood vessel. The stem cells will then move from the blood vessel into the heart muscle. This delivery methods is safe and because the cells are released slowly, they do not block the blood vessel.
In this clinical trial, the RECOVER-ERC trial, patients with congestive heart failure when treated with the menstrual blood-based stem cells and evaluated in this phase 2 study. These stem cells are the proprietary interest of Medistem Inc., and they are known as Endometrial Regenerative Cells or ERCs.
Medistem CEO, Alan Lewis, said of this trial, “To date, all stem cell trials in the cardiac space use bone marrow and adipose tissue sources. Unlike the painful and highly invasive process of collecting bone marrow and adipose stem cells, our collection processes involves [sic] extraction of a small amount of menstrual blood from young, healthy donors. In our FDA-cleared manufacturing protocol, one donor generates 20,000 doses. ERCs are administered without tissue matching or the requirement for immune suppressive drugs.”
The ERCs are delivered to the point-of-care as a cryogenically preserved allogeneic therapy that is ready to use, without the need for further manipulation by the physicians who will deliver them.
Lewis noted that, “This feature could make it practical for clinicians to effectively deliver stem cell therapy to large numbers of heart failure patients.”
Amit Patel, the director of cardiovascular regenerative medicine at the University of Utah, said, “This is the first time that the minimally-invasive catheter-based retrograde delivery technique has been used in the context of a ‘universal donor’ stem cell.” Patel noted that this particular delivery technique could be performed by any licensed cardiologist, which is in contrast to “other stem cell delivery techniques that require extensive user training and complex equipment that is not readily available.”
The RECOVER-ERC trial is a 60-person, double-blind, placebo controlled study that divides patients with congestive heart failure into three groups. One group receives 50 million ERCs, the second 100 million and the third 200 million. The main efficacy endpoints are assessed at six months after the stem cell treatment.
To date, 17 patients have been treated with the ERCs and there have been no treatment associated adverse reactions reported. The principal investigator, Leo Bockeria, is chairman of the Bakloulev Center in Russia. Safety oversight for this clinical trial is provided by the independent Data Safety Monitoring Board or DSMB. DSMB is chaired by Warren Sherman, M.D., Director of Cardiac Cell-Based Endovascular Therapies at Columbia University.
See Bockeria, L, et al., Journal of Translational Medicine 2013, 11:56.