September 27, 2011 -- Regenerative medicine companies such as Athersys Inc. (NASDAQ: ATHX), Osiris Therapeutics Inc. (NASDAQ: OSIR), Mesoblast (ASX:MSB) and others are busy developing novel stem cell treatments to target a range of disease indications that impact the healthcare system. Cardiovascular disease represents a vast area of unmet need, which these industry leaders are working hard to address, along with several others in the field. By developing new therapies that could represent significant advances over current treatment options, these emerging approaches could complement currently available technologies, promoting healing in unique and powerful ways. As a result, these companies could be poised to capitalize on a huge opportunity.
Heart disease is the leading cause of death in the United States. With a population of roughly 300 million, an estimated 785,000 Americans suffered a heart attack for the first time in 2010, and about 470,000 more people experienced their second or subsequent heart attack, according to the Centers for Disease Control and Prevention (CDC). That equates to about one every 25 seconds and a rate of death equivalent to roughly one person every minute in the United States, a vivid illustration of the unmet need related to heart disease. There are a number of underlying factors contributing to this epidemic, including rising rates of obesity, diabetes, and other factors that have a heavy impact on the risk and rates of heart disease, which is projected to cause a further rise in the years ahead.
Acute Injury – Treat the Damage Now
A heart attack – clinically referred to as a myocardial infarction (“MI”) – creates rapid or acute damage to heart muscle tissue when blood flow in an artery is blocked by a clot or other obstruction, such as from plaque in the arteries that has built up over time. The obstruction cuts off the supply of oxygen and nutrients to heart muscle, causing tissue to die, and triggering significant inflammation in the region of injury. That’s about as straight-forward as any medical description can get given that “Myo” means muscle, “cardial” is a reference to the heart, and “infarction” means death of tissue due to lack of blood supply. Standard care for a patient suffering a heart attack involves restoring blood flow in the artery as quickly as possible, which typically involves balloon angioplasty (literally, inflating a special balloon in the artery to move the obstruction out of the way) and insertion of a mesh-like tube, or stent, to keep the artery open and maintain efficient blood flow.
If administered early, this treatment can help bring the heart back to acceptable levels of function, but frequently the initial ischemic injury and subsequent scarring leaves the heart forever damaged, decreasing pumping ability. Over time, this can result in further weakening of the heart, substantially reducing patient quality of life, and ultimately leading to congestive heart failure. However, regenerative medicine therapies represent a new way to potentially treat the damage that occurs following a heart attack, which could improve patient recovery. Such approaches could possibly reduce the economic impact of heart disease which is expected to soar in coming years with an aging population.
Several companies are conducting clinical trials focused on evaluating various approaches to stem cell therapy, some of which appears to be promising. Recent data from one clinical trial by Athersys, Inc. (NASDAQ:ATHX) involved administration of its proprietary biologic MultiStem® to patients that had recently suffered a heart attack. In addition to showing a consistent safety profile, investigators from the Cleveland Clinic and other participating cardiovascular treatment centers saw some promising improvement in heart function from the one year follow-up data obtained from patients that participated in the study. Many of these patients had experienced severely compromised heart function following the heart attack. Athersys plans to launch the next phase of clinical development in the next few months, while it continues to advance other Phase 2 clinical programs to treat stroke and inflammatory bowel disease (where it is partnered with Pfizer). In addition, Osiris is in the midst of completing a Phase 2 clinical trial in heart attack patients with their product Prochymal, while Mesoblast just recently announced the start of a Phase 2 study in Europe with its partner Cephalon. Each of these approaches relies on the use of manufactured stem cell therapies that are “off the shelf” products expanded and produced from young healthy donors, meaning that clinicians could administer them to the patient using relatively simple and straightforward delivery techniques. However, Athersys may have the most scalable and effective platform, and currently has the lowest valuation, and therefore could represent a compelling buying opportunity for investors. While many believe that a drug-like stem cell therapy could have significant advantages over alternatives, these are not the only approaches in development, as companies like Cytori (NASDAQ:CYTX) are using patient derived stem cells obtained from processed fat tissue, while others like Aastrom (NASDAQ: ASTM) are using bone marrow derived cells obtained from the patient.
Chronic Heart Disease – Growing by the Generation
Chronic cardiovascular disease comes in a variety of forms, but is generally distinguished into two main categories: congestive heart failure and peripheral vascular disease. Congestive heart failure (“CHF”) can be broken down into further categories, but on a broad scale is simply described as progressive weakening of the heart’s pumping ability, resulting in deterioration over time. Blood can become backed-up in other areas of the body, including, but not limited to, the lungs, liver, gastrointestinal tract, and the arms and legs. Slow and deadly, approximately 5.8 million Americans are living with CHF with more than a half million new cases diagnosed each year. CHF is far more common as we age, and is the most common diagnosis in hospital patients age 65 and over, although the risk of the disease is clearly associated with other factors, such as obesity, diabetes, and smoking. Interestingly, however, the condition can also affect children and young adults. In fact, more than 1.5 million sufferers of CHF in the U.S. are under the age of 60. Disturbingly, roughly one third of people who develop CHF die within five years.
The economic burden of heart disease is significant, and is expected to mushroom in the years ahead. A recent study by the American Heart Association projects that costs could soar from $273 billion per year in 2010, to $818 billion per year by the year 2030. However, regenerative medicine may be able to provide a big part of the solution to this growing problem, as innovative new therapies get further into clinical development and get closer to commercialization. Mesoblast was the first to demonstrate some promising clinical results in this area, when it announced data from a 60 patient clinical trial that showed an apparent reduction in mortality, hospitalization, and adverse cardiovascular events among patients that were treated with stem cell therapy versus the standard of care. It’s noteworthy that this was right around the time of its blockbuster $1.7 billion potential deal with Cephalon, which acquired development rights in the cardiovascular and neurological areas.
The next few months should be interesting, as companies continue to advance trials and generate additional clinical data. Given the recent partnering activity in the area, and with at least a couple of examples of approved products in the cell therapy space from Organogenesis and Advanced BioHealing (recently acquired by Shire for $750 million in cash), continued progress in the field could cause things to really heat up. Ultimately this should have a positive impact on company valuations, which seem out of sync with the enormous potential for these programs.
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