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The promise of cell therapies in treating chronic diseases
The practice of medicine continues to evolve with advances in science, biology and technology, and in many cases, their convergence. We see evidence of these advancements all around us, from the use of monoclonal antibodies to treat autoimmune diseases and cancer to new devices that monitor a failing heart and shock it back into normal function.
Healthcare in general is entering a crisis phase due to the rising cost of treating patients with chronic diseases. People are living longer with these diseases, and due to medical breakthroughs, dying less from acute diseases. Regenerative medicine, however, is at the forefront of a vital, emerging trend in healthcare. With the introduction of adult stem cell therapies, we are now seeing new promise in the management and even treatment of chronic disease.
Countless lives have been saved with the advent of bone marrow transplantation, which many people do not realize is actually a type of stem cell application. Rebooting the body's immune system through cell therapies, as opposed to designing and administering traditional billion-dollar pharmaceuticals, is heralding a new era in healthcare and can have a significant impact on healthcare costs.
Evidence of this transformation is all around us. We continue to see more developmental-stage, cell-based products in clinical trial pipelines, as well as recent market activity by numerous large pharmaceutical companies. In 2009, Pfizer Inc. announced an initiative with Athersys, a clinical-stage biopharmaceutical company, for the development of a stem cell therapy to treat inflammatory bowel disease. In April 2010, Dendreon received U.S. Food and Drug Administration (FDA) approval for Provenge, an immunotherapy for prostate cancer that uses a patient's own white blood cells to attack cancer cells. Later that year, Cephalon made a $130- million, cash-upfront payment to Mesoblast, a developer of innovative biological products for regenerative medicine, for the rights to specific stem cell technology products. These ventures exemplify the growing trend toward and investment in cellular therapies, which hold the promise to reverse some chronic diseases—so it's like the patient never even had the disease.
A prime example of a chronic disease that can be managed through cell therapy is cardiovascular disease. Despite many advances in the medical field, heart disease remains the leading cause of death in the United States. Adult stem cell therapies, however, are currently being developed for the treatment of damaged heart muscle following an acute myocardial infarction. The therapies work to stop the progression of heart failure by replacing damaged heart muscle with new muscle cells.
Within the cardiovascular space, we see two major areas of opportunity with adult stem cell therapies. Adult stem cells hold the ability to spare cardiac tissue that has sustained an injury from ischemia, but has not yet died. Cardiac tissue is spared through the prevention of apoptosis (programmed cell death) and revascularization at the cellular level. The prevention of apoptosis helps in preserving cardiomyocytes and their function, allowing them to compensate for nonfunctioning cardiomyocytes.
Additionally, we have seen a different cell type, cardiac progenitor cells (CPC), which hold the potential to actually generate new cardiomyocytes after cardiac tissue death. Developments in cell-based therapies, like the therapies mentioned earlier, can offer hope for patients who are in heart failure or have chronic myocardial ischemia and for patients who have had large heart attacks.
With this new class of therapy comes a host of challenges, including regulatory, business and biological challenges. Although the regulation of drugs and medical devices has long been a function of the FDA, the regulation of cellular therapies that employ cells from within our own bodies is a new hurdle. As of late, the topic of FDA regulation when it comes to stem cells has sparked opposing viewpoints. With that being said, we have seen great collaboration from regulatory agencies when it comes to cell-based therapies here in the United States as well as abroad, and we expect to continue seeing that collaboration when additional stem cell therapies are proved to be safe and effective in clinical trials.
As more trials assessing the therapeutic uses of stem cells are underway and proved successful, we will start to see these therapies being delivered to the market in a cost-efficient way through the management of distribution, sales and reimbursement.
Additionally, clinical trials will provide us with the information needed to determine whether allogenic stem cells, which are those foreign to you, or autologous stem cells, which are ones that are your own, should be used for particular therapies.
There are distinct advantages to using both cell types. While allogeneic cells will not necessarily cause damage, it is inherently safer to use autologous cells rather than ones coming from another person. Many times, people who are diagnosed with a disease and in turn require a bone marrow transplant have a hard time finding a healthy match. With autologous cells, however, there is less of a chance of rejection.
Much like there is no one drug to treat every disease, there is no one cell type to treat every disease. To that end, the challenge of whether to use allogeneic or autologous stem cells will have to be decided on a case-by-case basis.
The future of stem cell therapies relies in part on the ability to educate the public on the benefits of these therapies. When talking about stem cells, many people still revert back to the previous controversy over the use of embryonic stem cells that involve the destruction of a human embryo. The use of adult stem cells in research and therapy, however, is not considered to be controversial. Greater public understanding is necessary to drive more interest in the field, both from an investment standpoint and from the general public. Market awareness seems to follow a direct path from a state of skepticism to a gold rush, and cell therapy is already making its way along that path.
Once these therapies are proved to be safe and effective, adult stem cells will change the expected paradigm of chronic or progressive heart failure along with a host of other chronic diseases. We expect that this paradigm shift in healthcare will help to control the rising costs of treating chronic disease, spanning from cardiovascular disease to cancer to neurologic disease to autoimmune disorders. When you consider that the United States already spends more than $200 billion per year managing diabetes alone, it becomes clear how important the management of these conditions really is, not only to the health of our country but also to the future of our economy. We look forward to a whole new era of medical innovation with a world of possibilities.
Dr. Andrew Pecora is the chief medical officer and director of Neostem Inc., an international biopharmaceutical company with adult stem cell operations in the United States. Pecora served as chairman and CEO of Progenitor Cell Therapy leading up to its merger with Neostem. He was appointed to NeoStem's board of directors in December 2011. Pecora is also the founder of Amorcyte, a Neostem company pursuing cell-based therapies for the treatment of cardiovascular disease, and its first chairman and CEO. Additionally, Pecora serves as vice president of cancer services and chief innovations officer at the John Theurer Cancer Center at Hackensack University Medical Center (HUMC) and co-managing partner of the Northern New Jersey Cancer Center (NNJCC), the private physicians practice group affiliated with the HUMC. Pecora received his medical degree from the University of Medicine and Dentistry of New Jersey, graduating with honors. He went on to complete his medical education in internal medicine at New York Hospital and in hematology and oncology at Memorial Sloan-Kettering Cancer Center in New York City. He is board-certified in internal medicine, hematology and oncology. He also received a degree in medical management.