Unlocking the Potential of Regenerative Medicine
Posted on January 17th, 2012 in Critical Limb Ischemia (CLI)
Dear Friends of Aastrom,
Regenerative medicine is the term most often used to describe research and drug development involving the use of stem cells to stimulate the body to rebuild and repair damaged tissue. Based on the results of multiple research programs in the U.S. and other countries, regenerative medicine has the potential to cure or reverse the symptoms of many diseases that affect different tissues in the body, including vascular, muscle, organ, bone and nerve tissues.
Today, the field of regenerative medicine encompasses many approaches to treatment, including:
- therapies administered by injection to promote tissue healing and organ regrowth;
- the growth of new tissue and organs in a laboratory for implantation in patients; and
- the use of biocompatible materials or small molecules to support the body’s natural ability to regenerate tissue.
At Aastrom, our approach to regenerative medicine is to use a patient’s own cells to develop a therapy that includes the optimal range and ratio of cells to treat severe, chronic ischemic cardiovascular diseases such as critical limb ischemia (CLI) and dilated cardiomyopathy. The product we are developing, ixmyelocel-T, is personalized and custom-made for each patient from a small amount of his or her own bone marrow cells. Our production process expands the numbers of certain key cell types – CD90+ mesenchymal cells, CD14+auto+ monocytes and alternatively activated macrophages – which, according to our research, seem to play an important role in tissue remodeling, immunomodulation and the growth of new blood vessels.
At the November 2011 American Heart Association Scientific Sessions, Aastrom reported positive results from the RESTORE-CLI Phase 2 clinical trial for ixmyelocel-T. In this study, patients in the treatment arm showed a 62% reduction in risk relative to placebo in the primary efficacy endpoint of time to first occurrence of treatment failure (p = .0032) (Click here to read more about these results). We plan to initiate the REVIVE Phase 3 clinical trial for ixmyelocel-T this quarter, making Aastrom the leading regenerative medicine company in the field of CLI and one of the few companies to have successfully advanced a cell therapy product to late-stage clinical development.
While there have been many important advances in regenerative medicine in recent years, developing new therapies in this field has been challenging. Aastrom’s success in advancing ixmyelocel-T is the result of a number of key decisions and efforts by the Aastrom team:
- We are developing ixmyelocel-T as a treatment for severe, chronic ischemic cardiovascular diseases for which there are no other treatment options.
- We use only adult human stem cells from each patient’s own bone marrow. As a result, our production process is not associated with the ethical issues surrounding the use of embryonic stem cells. In addition, because we use the patient’s own cells to develop ixmyelocel-T, there is minimal risk of rejection.
- We collect a small amount of bone marrow and administer ixmyelocel-T rapidly in an out-patient procedure.
- We use a proprietary production process and a centralized cGMP manufacturing facility to ensure that every patient receives a safe, consistent, high-quality product derived from his or her own cells.
- Our development team is following a clear regulatory path established by other autologous cellular therapies that have been approved by the FDA in recent years.
These attributes distinguish ixmyelocel-T from other experimental cell therapies and have contributed to our success to date, but much work remains to be done. To unlock the potential of regenerative medicine to improve human health in the years ahead, we believe swift action in several key areas is needed:
- We must encourage closer collaboration among leading academic research centers to identify and advance promising early-stage drug candidates to clinical-stage research conducted by industry partners.
- Additional sources of non-dilutive federal funding are needed to support research, early-stage drug development and job creation.
- We must continue to clarify and expedite the regulatory process for regenerative medicines by taking into account the unique production requirements and mechanisms of action of these therapies.
- Finally, we must continue to expand science education in the U.S. to train the next generation of innovators in cellular and regenerative medicine.
- Action will require careful planning, coordinated efforts and government action to be effective. But if we take action now, I feel confident that we can realize the full potential of regenerative medicine in the coming decade and position the U.S. to maintain and expand our global leadership position in this rapidly evolving and highly promising field.