Dear Friends of Aastrom,
The issue of safety associated with stem cell therapies has been in the news recently. Last month, the FDA issued a consumer update describing potential risks associated with the use of stem cell therapies that have not yet been approved. Also recently, 60 Minutes ran a story about patients with severe illnesses who sought treatment with unapproved stem-cell therapies when no approved treatment options were available. In many cases, these patients paid vast sums of money and traveled to other countries to be treated with potentially dangerous and unregulated cell therapies.
These reports highlight the value of appropriate safety standards in the development of cellular therapies, standards which Aastrom maintains in all of its development, manufacturing and shipping procedures. These stories also raise an important issue about the different types of stem cells used in medical research and drug development, and the need to define clearly which cell types are being used. In many cases, the ethical or safety concerns associated with cell therapies relate to the use of embryonic stem cells, not the use of adult or autologous (“patient-specific”) stem cells which we use at Aastrom. Therapies derived from non-embryonic stem cell therapies are now approved for use in treating more than 70 medical conditions. 1, 2
In the development of our cellular-medicine product, Aastrom uses the patient’s own bone marrow, which is collected in an outpatient setting. After the sample arrives at our laboratory in Ann Arbor, each step of the production process is carefully monitored to meet or exceed the safety and quality standards established by the FDA.After production, the final product, known as ixmyelocel-T, is then shipped back to the doctor for administration to the patient. While ixmyelocel-T is not yet approved for use in the United States, more than 400 patients have been treated with our product candidates in clinical trials.
This quarter we are advancing ixmyelocel-T to a Phase 3 clinical trial for the treatment of critical limb ischemia (CLI) and next quarter a Phase 2b trial for the treatment of dilated cardiomyopathy. Our Phase 3 REVIVE clinical trial in CLI will include 594 patients who have no option for revascularization and who have existing tissue loss. We have worked closely with the FDA, the Center for Biologics Evaluation and Research (CBER) and physician experts to design this trial and ensure that our production process and safety standards comply with all state and federal regulations.
As a developer of new cellular medicines, we believe stem-cell therapies have enormous therapeutic potential and can have a major positive impact on patients with serious, untreatable health problems. We also believe rigorous laboratory and clinical standards are essential to ensuring patient safety and public trust in our products. Aastrom is committed to employing the highest standards of pharmaceutical and medical practice as we advance our therapy toward final regulatory review.
As the field of cellular medicine continues to flourish, we have an obligation to distinguish the different types of stem cells used in the production process and support regulatory requirements for research, manufacturing and patient care that reflect these differences. Only those companies that maintain their commitment to the highest standards of safety and product quality warrant our support.
*For information about the FDA consumer update on stem-cell therapies, visit: http://www.fda.gov/ForConsumers/ConsumerUpdates/ucm286155.htm
**To see the 60 Minutes story about use of unapproved therapies, visit: http://www.cbsnews.com/video/watch/?id=7394380n