The promise of cell therapies in treating chronic diseases

The practice of medicine continues to evolve with advancesin science, biology and technology, and in many cases, their convergence. Wesee evidence of these advancements all around us, from the use of monoclonalantibodies to treat autoimmune diseases and cancer to new devices that monitora failing heart and shock it back into normal function.

Healthcare in general is entering a crisis phase due to therising cost of treating patients with chronic diseases. People are livinglonger with these diseases, and due to medical breakthroughs, dying less fromacute diseases. Regenerative medicine, however, is at the forefront of a vital,emerging trend in healthcare. With the introduction of adult stem celltherapies, we are now seeing new promise in the management and even treatmentof chronic disease.

Countless lives have been saved with the advent of bonemarrow transplantation, which many people do not realize is actually a type ofstem cell application. Rebooting the body's immune system through celltherapies, as opposed to designing and administering traditional billion-dollarpharmaceuticals, is heralding a new era in healthcare and can have asignificant impact on healthcare costs.

Evidence of this transformation is all around us. Wecontinue to see more developmental-stage, cell-based products in clinical trialpipelines, as well as recent market activity by numerous large pharmaceuticalcompanies. In 2009, Pfizer Inc. announced an initiative with Athersys, aclinical-stage biopharmaceutical company, for the development of a stem cell therapyto treat inflammatory bowel disease. In April 2010, Dendreon received U.S. Foodand Drug Administration (FDA) approval for Provenge, an immunotherapy forprostate cancer that uses a patient's own white blood cells to attack cancercells. Later that year, Cephalon made a $130-million, cash-upfront payment toMesoblast, a developer of innovative biological products for regenerativemedicine, for the rights to specific stem cell technology products. Theseventures exemplify the growing trend toward and investment in cellulartherapies, which hold the promise to reverse some chronic diseases—so it's likethe patient never even had the disease.

A prime example of a chronic disease that can be managedthrough cell therapy is cardiovascular disease. Despite many advances in themedical field, heart disease remains the leading cause of death in the UnitedStates. Adult stem cell therapies, however, are currently being developed forthe treatment of damaged heart muscle following an acute myocardial infarction.The therapies work to stop the progression of heart failure by replacingdamaged heart muscle with new muscle cells.

Within the cardiovascular space, we see two major areas ofopportunity with adult stem cell therapies. Adult stem cells hold the ability tospare cardiac tissue that has sustained an injury from ischemia, but has notyet died. Cardiac tissue is spared through the prevention of apoptosis(programmed cell death) and revascularization at the cellular level. Theprevention of apoptosis helps in preserving cardiomyocytes and their function,allowing them to compensate for nonfunctioning cardiomyocytes.

Additionally, we have seen a different cell type, cardiacprogenitor cells (CPC), which hold the potential to actually generate newcardiomyocytes after cardiac tissue death. Developments in cell-basedtherapies, like the therapies mentioned earlier, can offer hope for patientswho are in heart failure or have chronic myocardial ischemia and for patientswho have had large heart attacks.

With this new class of therapy comes a host of challenges,including regulatory, business and biological challenges. Although theregulation of drugs and medical devices has long been a function of the FDA,the regulation of cellular therapies that employ cells from within our ownbodies is a new hurdle. As of late, the topic of FDA regulation when it comesto stem cells has sparked opposing viewpoints. With that being said, we haveseen great collaboration from regulatory agencies when it comes to cell-basedtherapies here in the United States as well as abroad, and we expect tocontinue seeing that collaboration when additional stem cell therapies areproved to be safe and effective in clinical trials.

As more trials assessing the therapeutic uses of stem cellsare underway and proved successful, we will start to see these therapies beingdelivered to the market in a cost-efficient way through the management ofdistribution, sales and reimbursement.

Additionally, clinical trials willprovide us with the information needed to determine whether allogenic stemcells, which are those foreign to you, or autologous stem cells, which are onesthat 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 inherentlysafer to use autologous cells rather than ones coming from another person. Manytimes, people who are diagnosed with a disease and in turn require a bonemarrow transplant have a hard time finding a healthy match. With autologouscells, however, there is less of a chance of rejection.

Much like there is noone drug to treat every disease, there is no one cell type to treat everydisease. To that end, the challenge of whether to use allogeneic or autologousstem cells will have to be decided on a case-by-case basis.

The future of stem cell therapies relies in part on theability to educate the public on the benefits of these therapies. When talkingabout stem cells, many people still revert back to the previous controversyover the use of embryonic stem cells that involve the destruction of a humanembryo. The use of adult stem cells in research and therapy, however, is notconsidered to be controversial. Greater public understanding is necessary to drivemore interest in the field, both from an investment standpoint and from thegeneral public. Market awareness seems to follow a direct path from a state ofskepticism to a gold rush, and cell therapy is already making its way alongthat path.

Once these therapies are proved to be safe and effective,adult stem cells will change the expected paradigm of chronic or progressiveheart failure along with a host of other chronic diseases. We expect that thisparadigm shift in healthcare will help to control the rising costs of treatingchronic disease, spanning from cardiovascular disease to cancer to neurologicdisease to autoimmune disorders. When you consider that the United Statesalready spends more than $200 billion per year managing diabetes alone, itbecomes clear how important the management of these conditions really is, notonly to the health of our country but also to the future of our economy. Welook forward to a whole new era of medical innovation with a world ofpossibilities.

Dr. Andrew Pecora is the chief medical officer and directorof Neostem Inc., an international biopharmaceutical company with adult stemcell operations in the United States. Pecora served as chairman and CEO ofProgenitor Cell Therapy leading up to its merger with Neostem. He was appointedto NeoStem's board of directors in December 2011. Pecora is also the founder ofAmorcyte, a Neostem company pursuing cell-based therapies for the treatment ofcardiovascular disease, and its first chairman and CEO. Additionally, Pecoraserves as vice president of cancer services and chief innovations officer atthe John Theurer Cancer Center at Hackensack University Medical Center (HUMC)and co-managing partner of the Northern New Jersey Cancer Center (NNJCC), theprivate physicians practice group affiliated with the HUMC. Pecora received hismedical degree from the University of Medicine and Dentistry of New Jersey,graduating with honors. He went on to complete his medical education ininternal medicine at New York Hospital and in hematology and oncology atMemorial Sloan-Kettering Cancer Center in New York City. He is board-certifiedin internal medicine, hematology and oncology. He also received a degree inmedical management.