As in any industry, an unmet need and the potential for economic gain often combine to produce two opposite results: on the one hand, such opportunity will attract legitimate experts who are authentically qualified to provide a beneficial service or product that meets a need, and, on the other hand, charlatans whose illigitimate services or products have no validity whatsoever will also be attracted to the field. Such has always been the case in most, if not all, economic sectors throughout history, especially in medical specializations, and such will probably always be the case throughout the future, given human nature and the tendency of history to repeat itself. Whether dealing with cars, jewelry or designer clothing, low quality reproductions of the most coveted styles abound and are often deliberately promoted as being something which they are not. It should come as no surprise, therefore, that the stem cell industry is no different. Fortunately, however, there is a simple “antidote” to such a danger: knowledge.

Since ancient times, free market economics have warned the buyer to beware, and fraudulent market activity is hardly a modern phenomenon. The very same market forces which allow for the possibility of deception on the part of the seller, however, also demand, and motivate, some level of intelligence and education on the part of the buyer. Predictably, therefore, deceptive products and services will often arise wherever legitimate opportunity and progress also exist, and history has repeatedly proven that this is usually not a question of “if” but rather a question of “when”. In a population of educated and well-informed consumers, however, such deception will be shortlived, as knowledgeable people will be able to tell the difference between something of quality and value, as opposed to something that is worthless and perhaps even dangerous.

Unlike with cars, jewelry or designer clothing, the consumers of stem cell therapies are often patients with life-threatening disease or illness who are desperate for any treatment whatsoever. Consequently, scientists and governments alike are working to formulate official guidelines and regulatory laws that will protect the patient by ensuring, as much as is humanly possible, the legimacy of stem cell providers, and also by penalizing those who violate such regulation. Meanwhile, however, the stem cell field is still in its infancy, and the basic premises behind such regulation are not yet globally respected. Consequently, at the moment, anyone who wishes to peddle modern versions of snake oil while masquerading as a stem cell expert is free to do so, and those who actually do engage in such unethical and medically dangerous activity are tireless in their efforts to profit from the exploitation of consumers, especially with the ease of marketing their products and services over the internet.

According to Dr. Insoo Hyun, associate professor of bioethics at Case Western Reserve University School of Medicine in Cleveland, Ohio, and the lead author of a paper outlining the commercial guidelines of stem cell therapies, “Stem cell research is progressing so rapidly and has sparked a lot of interest in translational research including among patients in hope for therapies. At the same time, legitimate science is speeding ahead and getting to the point where there needs to be more of a roadmap to take the basic knowledge to clinical applications.” Although such incidents have not yet made major news headlines, Dr. Hyun adds that it is “only a matter of time” before someone somewhere is physically harmed by bogus stem cell therapies.

According to Dr. Paul Sanberg, professor of neurosurgery and director of the University of South Florida Center for Aging and Brain Repair in Tampa, “We clearly need guidelines for around the world to make sure that appropriate research is done before clinical work is undertaken in patients. We see desperate patients all the time and want to make sure that any therapies they take come from responsible research groups.” Similarly, Dr. Darwin Prockop, chair of Genomic Medicine and director of the Texas A&M Health Science Center College of Medicine Institute for Regenerative Medicine, adds, “There is tremendous confusion about the two types of stem cells, embryonic stem cells and adult progenitor stem cells. The difference is monumental, and needs to be clarified.”

Although there do exist a number of clinics around the world which actually offer legitimate, scientifically based adult stem cell therapies, there also exist a number of clinics which do not offer anything of legitimacy, even though they purport to be legitimate. Accompanying the recent publication of stem cell guidelines is a commentary by Canadian researchers which contains an analysis of 19 websites that were found from a simple Google search, all of which advertize expensive stem cell therapies of dubious validity and safety. For example, a number of clinics in a number of countries such as China and the Ukraine claim to have treated thousands of patients for everything from Parkinson’s disease and stroke to heart conditions, but without any scientific verification or corroboration of their claims. Indeed, a team of scientists from the University of Alberta were unable to find any substantiating evidence for any of the claims made by such clinics, nor were they even able to find any verification that real stem cells were actually used in the treatments, nor what types of stem cells might have been used, nor what the source of the cells might have been (human or nonhuman, for example, adult or fetal or embryonic, etc.). Additionally, nowhere were risks or contraindications mentioned on any of the websites for any of the clinics. Given the typical cost of such therapies, which averaged around $21,500, one might assume that prospective “customers” would be interested in obtaining prior verification of the safety and efficacy of such procedures, but apparently many patients who would otherwise have no treatment whatsoever are willing to take extraordinary risks, financially as well as medically, in exchange for even a false glimmer of hope, since even false hope is better than nothing, from the perspective of someone whose very life is already at risk.

According to Dr. Hyun, the newly formulated guidelines are meant to illuminate stem cell research and to guide researchers toward responsible and accountable practices, rather than to hinder or discourage their progress. Indeed, agencies such as the U.S. FDA (Food and Drug Administration) typically perform such roles, though not all countries have such regulatory governmental agencies. Furthermore, even in countries such as the U.S., where a powerful FDA has been governing medical research and clinical practices for years, specifically for the protection of human patients who are being treated with clinical therapies, nevertheless there is still the problem that such guidelines are outdated and do not apply to the stem cell field. The stem cell field is still young enough, and so radically different from all previous specializations of medical science, that the issues which are unique to stem cells have never before been fully addressed. As Dr. Hyun explains, “Most of the time, stem cell products are presenting entirely novel products that are unpredictable in humans. Unlike drugs, you can’t just create a batch and put them on the shelf and expect they will be the same. We need uniform quality control and manufacturing. And if they’re embryonic or pluripotent stem cells, they could form unwanted tissues or tumors. So, we have to be very careful about following up and monitoring patients.”

Authored by a task force composed of stem cell specialists from 13 countries, the new guidelines address, among other topics, questions of ethical review, quality and safety, voluntary informed consent of participants in research projects, careful monitoring of volunteers, and caution in using stem cell therapies outside of a research context. Hopefully, the ethical principles which are at the very essence of such guidelines will be given serious attention and consideration by stem cell researchers throughout all countries of the world.

For those patients who are still awaiting stem cell cures for their diseases and illnesses, the field seems to be advancing much too slowly. As Dr. Hyun points out, however, not all progress is visible to the public. “For patients, it’s not surprising that there are not direct applications,” Dr. Hyun adds, “but what is often lost to the public is that so much knowledge has been gained from stem cell research. The advancements for patients are going to come sooner through these indirect routes, not direct cell-based therapy, but from the expansion of knowledge.”

In addition to the need for ethical guidelines, there is another lesson to be learned from the increasing number of bogus stem cell therapies that are springing up around the world. Namely, the necessity and urgency of such guidelines also highlights the necessity and urgency for formal, official government approval of those adult stem cell therapies which have already been proven to be safe and efficacious, so that more clinics that offer such therapies will be allowed to open in their native countries, such as the United States, instead of having to locate themselves overseas in foreign countries where they are competing against the clinics that offer bogus therapies. In other words, an updated revision of the FDA approval process, so that it is directly relevant to stem cell therapies, would allow more adult stem cell therapies to be available throughout the U.S. to more patients with various diseases and injuries, who could benefit from such therapies but for whom such therapies have not yet received FDA approval. The current FDA approval process, which was designed years ago with the specific goal of testing safety and efficacy in pharmaceutical drugs, is in many ways neither relevant nor logical when applied to the testing of stem cell therapies. Such a topic is highly complex and could constitute an entirely separate publication unto itself. Suffice it to say that a swifter, more precise and more modernized FDA approval process which is specifically tailored to stem cell therapies is desperately and urgently needed in the United States, as is its equivalent in other countries.

Meanwhile, however, as in any market, consumers must arm themselves with the power of knowledge, which is their greatest defense. Especially where the quality of a product or service can make the difference between life and death, such as with stem cell therapies, it is all the more critically important that the buyer beware.

A novel population of adult stem cells has been found to repair damaged heart muscle in an animal model, and the results suggest a wide range of therapeutic applications for human diseases and injuries. Led by Dr. John Huard, scientists at the Children’s Hospital in Pittsburgh, Pennsylvania have used myoendothelial cells, derived from skeletal tissue, to treat heart damage in mice which was similar to the damage found in humans following a heart attack.

The myoendothelial cells had been purified from human skeletal tissue, and were found not only to repair the injured heart muscle but also to stimulate angiogenesis (the growth of new blood vessels) within the heart, and the cells were also shown to reduce the formation of scar tissue following the injury, all of which dramatically improved left ventricle function. The formation of cardiac scar tissue following a heart attack is a common and serious problem and is often the cause of a second heart attack in many patients. Dr. Huard and his colleagues have now demonstrated that this particular population of adult stem cells, myoendothelial cells, adequately correct all types of damage to the cardiac tissue caused by heart attacks. In fact, at 6 weeks after injection, the myoendothelial cells were found to be 40 to 50% more effective in repairing heart muscle than were previous approaches which employed only myoblasts (muscle cells).

According to Dr. Huard, “This study confirms our belief that this novel population of stem cells discovered in our laboratory holds tremendous promise for the future of regenerative medicine. Specifically, myoendothelial cells show potential as a therapy for people who have suffered a myocardial infarction. The important benefit of our approach is that as a therapy, it would be an autologous transplant. This means that for a patient who suffers a heart attack, we would take a muscle biopsy from his or her muscle, isolate and purify the myoendothelial cells, and re-inject them into the injured heart muscle, thereby avoiding any risk of rejection by introducing foreign cells.”

Myoendothelial cells have previously been used as a therapy for numerous conditions, most recently in the repair of bladder muscle in women. Many diseases and injuries involve damaged muscle, of many varieties, and can therefore be alleviated with a treatment that not only regenerates muscle cells but which also stimulates angiogenesis and blocks the formation of scar tissue. Consequently, numerous therapeutic uses are expected for this population of myoendothelial cells, one of which includes the treatment of Duchenne muscular dystrophy (DMD), a genetic disease that strikes approximately one in every 3,500 boys and which is caused by a lack of the protein dystrophin, which gives muscle cells their structure.

As the Director of the Stem Cell Research Center at Children’s Hospital and professor and vice chair for research in the Department of Orthopedic Surgery at the University of Pittsburgh School of Medicine, Dr. Huard will begin clinical trials on humans next month.

Physicians announce their successful results for the first patient to be treated with autologous adult stem cells in a clinical study of pulmonary hypertension.

Led by Dr. Leonel Fernandez Liriano, professor of medicine at the Pontifical Catholic University School of Medicine in the Dominican Republic, the international medical team announced their 9-month follow-up results for the clinical trial, in which autologous (in which the donor and recipient are the same person) adult stem cells were extracted from each patients’ own blood and differentiated into new blood vessels.

According to Dr. Zannos Grekos, assistant clinical professor of cardiology at Nova Southeastern University and a member of the international team that developed the stem cell treatment protocol, “It goes against traditional theory that we should try to fix the existing pulmonary vasculature, but we are generating new blood vessels with impressive results.”

The clinical study represents a collaborative effort involving researchers from the Tel Aviv based company TheraVitae, and physicians from the Florida based adult stem cell company Regenocyte Therapeutic, which also includes physicians from Regenocyte’s Dominican Republic division. Patient baseline and follow-up testing are being conducted in part by the Mayo Clinic.

Karl Wagner, the 46-year-old patient who was the first to be treated, was previously described as having been in a rapid decline prior to receiving the adult stem cell therapy in February of 2008. According to Mr. Wagner, after having first been diagnosed with pulmonary hypertension, “I was being managed by medication but still had violent chest pains, heart palpitations, extreme fatigue, and severe shortness of breath. I could barely do anything with my daughters and was on oxygen almost all the time. Doctors at the Mayo Clinic gave me a three year prognosis.”

After being treated with the adult stem cell therapy, Mr. Wagner’s pulmonary artery pressure improved from 41 mm Hg, which is classified as severe pulmonary hypertension, to 24 mm Hg, which is classified as normal. The other patients who participated in the clinical trial are showing the same pattern of improvement.

According to Dr. Hector Jose Rosario, professor of cardiology and director of cardiovascular therapy for Regenocyte’s Dominican division, “This is the first time medical science has successfully reversed the disease process in pulmonary hypertension, a previously untreatable condition with a very grim prognosis.” As Dr. Grekos adds, “Using advanced engineered stem cell technology and innovative delivery methods, we’ve been able to harness the regenerative power of stem cells and literally replace the damaged blood vessels in the lungs of the pulmonary hypertension patients.”

According to Mr. Wagner, whose oxygen saturation levels are now consistently high, so that he no longer needs to be supplemented with oxygen nor is he a candidate for a lung transplant any longer, “I feel great and have a normal life again. I take my girls to school every morning and work all day. My quality of life is ten-fold what it used to be. I also am off almost all of my medications and the doctors at Mayo Clinic have given me a new prognosis.”

The autologous adult stem cell therapy used in the study is based upon several years of Regenocyte’s clinical experience in the treatment of cardiac and vascular disease. As Dr. Athina Kyritsis, chair of Regenocyte’s Scientific Advisory Board, explains, “In treating diseases like cardiomyopathy and peripheral vascular disease, we’ve had consistent success in generating viable heart tissue and growing new vessels. With the increased circulation, healing of wounds, and improvement in ejection fractions, it seemed a natural progression to approach pulmonary hypertension in the same manner. I believe we have only begun to discover what adult stem cells can accomplish in altering the course of diseases now thought to be untreatable.”

The clinical trial was conducted with support from the nonprofit Alliance for the Advancement of Adult Stem Cell Therapy and Research.

The renowned embryologist, Dr. James Thomson, of the University of Wisconsin at Madison, announced yesterday that he is merging 3 university spinoff companies into a new, single business entity. With $18 million in venture capital cash, Dr. Thomson stated at a news conference on Monday that he believes his new company will become a world leader in stem cell technology.

The three University of Wisconsin spinoffs – Cellular Dynamics, Stem Cell Products and iPS Cells – are being merged into a single new company which will still be based in Madison, Wisconsin and which will retain the name Cellular Dynamics International (CDI). According to CEO Bob Palay, CDI “intends to be the world leader in the industrialization of basic stem cell technology.”

Focused specifically on the commercialization of stem cell technology as it applies to drug testing and research, rather than to the discovery of cell-based therapies, CDI’s initial work will be centered around the development of new technology which can supply human heart cells to researchers for use in drug testing, especially for the testing of adverse reactions to pharmaceuticals.

One of the popular misconceptions about embryonic stem cells is that they might offer a cure for disease or injury within the near future, whereas nothing could be further from the truth, and this is a point which all of the stem cell authorities, including Dr. Thomson, repeatedly emphasize. As the first person in the world ever to isolate embryonic stem cells, first from a monkey in 1995 and then from a human in 1998, Dr. Thomson is idolized in embryonic stem cell laboratories throughout the world since he is widely recognized as the father of embryonic stem cell science. Additionally, when his lab announced the breakthrough in 2007 with the development of iPS (induced pluripotent stem) cells, once again his name was in newspaper headlines around the globe. Although iPS cells, which were originally transformed from ordinary skin cells, are still extremely problematic for a number of scientific reasons, they are not generally regarded as ethically controversial, as embryonic stem cells are, since the destruction of an embryo is required for the extraction of embryonic stem cells but not for the development of iPS cells. Nevertheless, as Dr. Thomson repeatedly explains, any potential cures either from embryonic stem cells or from iPS cells, for any disease or injury, are still at least another decade away, if not further, due to the numerous scientific problems that are inherent in these cells and which have yet to be resolved. Consequently, Dr. Thomson therefore believes that the greatest benefit to be derived from embryonic stem cells is not from any cure that might be developed from the embryonic stem cells themselves, but rather in the use of the embryonic stem cells for drug testing and development – i.e., in the basic use of these cells to test for adverse reactions from pharmaceuticals in the laboratory, toward the ultimate goal of developing cures from the pharmaceuticals, not from the embryonic stem cells. Currently, side effects from drugs are tested on animal cells, but rarely with great accuracy, with the result that physicians prescribe medication to patients without knowing in advance whether or not an individual patient will have side effects to the medication, and then the patient is monitored to see whether or not side effects will occur. Dr. Thomson’s business model now offers a new paradigm, in which adverse reactions to specific medications would be tested on human, not animal, cells, derived from the human embryonic stem cells, prior to prescribing a drug to a patient. As Dr. Thomson explains, “We’re very much going to be focused on products rather than long-term promises. There are things that drug companies want today.”

By sharp contrast, adult stem cells are neither problematic in the laboratory nor ethically controversial, and have already been used for years in clinical therapies for numerous diseases and injuries. Unlike adult stem cells, however, both embryonic stem cells and iPS cells have numerous biological hurdles to overcome, which include, among other problems, their inherent risk of teratoma formation. Teratomas are a very specific type of tumor and their formation, by definition, is the universal laboratory test for determining whether or not a cell, such as an embryonic stem cell or an iPS cell, is “pluripotent”. If a cell forms a teratoma, then it is recognized as being an embryonic stem cell or some other type of pluripotent cell, such as an iPS cell, whereas if a cell does not form a teratoma then it is recognized as not being an embryonic stem cell nor an iPS cell nor any other type of pluripotent cell. Since adult stem cells are multipotent, not pluripotent, they do not form teratomas nor do they exhibit the numerous other problems inherent in embryonic and iPS cells, which is why adult stem cells have already been used as clinical therapies for years in the treatment of real human patients with real diseases, whereas embryonic stem cells have never progressed beyond the laboratory stage and any hope of a clinical cell-based therapy being developed from embryonic stem cells is at least another decade away, if not further, as the pioneers of embryonic stem cell science, such as Dr. Thomson, repeatedly state.

At the news conference yesterday Dr. Thomson also predicted that within the next 20 years all drug testing will include the use of human heart cells, and according to this view he is designing CDI to be a world leader in the supply of human heart cells, developed from embryonic stem cells, which CDI will then sell to pharmaceutical companies. Additionally, CDI will also develop red blood cells and platelets from stem cells to be used in blood transfusions, which would alleviate supply shortages and also hopefully reduce some of the risks associated with human blood donation. As CEO Bob Palay cautioned, however, even for this it will still take at least another decade for these products to be developed and to pass regulatory approval. According to Palay, “For these lifesaving treatments to happen, we have to drive the cost down, quantities and qualities way up and go through the approval process to ensure the safety and effectiveness. Historically, that takes a decade or more.”

Currently CDI employs a staff of 50 individuals but is growing rapidly. The $18 million in venture capital funding, which is derived primarily from local Wisconsin investors and from the Wisconsin Alumni Research Foundation, will be used not only for industrializing a production infrastructure within the company by which human cell types are mass produced, but also for the creation of a repository of stem cells which would be a type of bio-bank in which stem cells that are engineered from DNA could be stored and used for testing individual reactions to drugs. Even without the full development of such an infrastructure and without the completion of the repository, however, CDI is already using its proprietary stem cell technology to supply heart cells to Roche and to other pharmaceutical companies. Although the global economic crisis has resulted in declines in most markets, including in most of the other major sectors of the biotech industry, stem cell companies are well positioned for growth as analysts predict that the regenerative medicine industry will constitute a $10 billion market by 2016.

At the moment, the time would appear to be ripe for startups, especially since the road ahead is a long one, at least for any enterprise based upon embryonic stem cells. As CEO Bob Palay acknowledges, the plan to build CDI into a world leader in the industrialization of basic stem cell technology is “an ambitious goal”. CDI’s stock closed today at $9.87.

Each year thousands of babies in the U.S. alone are born with defective heart valves. Now, doctors at the University Hospital of Munich are growing new heart valves from adult stem cells derived from umbilical cord blood which are designated ultimately for the replacement of such defective heart valves in the earliest stages of a newborn’s life.

From umbilical cord blood that was collected at the time of birth, cardiac surgeon Ralf Sodian and his colleagues in Munich were able to isolate those stem cells that are known to differentiate into cardiac tissue. The stem cells were then frozen and stored for 12 weeks, after which they were seeded and expanded upon a biodegradable polymer scaffold in the laboratory, from which eight new heart valves were grown. Preliminary examination with electron microscopy revealed that the stem cells had integrated into the pores of the scaffolding and not only had differentiated into cardiac tissue but also exhibited characteristics of the extracellular matrix as well. The newly engineered valves were shown to contain a wide array of proteins which included 78% as much collagen as heart valves which are formed from pulmonary tissue, 67% as much elastin, and 85% as much glycosaminoglycan, which is a carbohydrate found in connective tissue. The polymer scaffolds, which provide the architectural blueprints for the structural template of the heart valves on which the stem cells are guided in their differentiation, are designed to dissolve over time, thereby leaving behind nothing but the fully formed valve, each of which was tested for functional efficacy according to variations in blood flow volume and pressure, and all of which were found to mimic naturally occurring healthy valves. The next step, which will begin in 2009, will involve implantation of the bioengineered valves into young lambs to test how the valves change in growth and function over several years. If the valves are proven to be capable of growing as the young lambs mature and age, Dr. Sodian then expects to begin offering transplantation of these heart valves into human babies who are born with heart valve defects, using autologous (in which the donor and recipient are the same person) stem cells derived from the umbilical cord blood of each newborn.

Of all congenital heart defects, valve abnormalities are among the most common. With valves that are too narrow or do not close completely with each beat of the heart, “regurgitation” of the blood can cause a number of systemic physiological problems, depending upon the severity of the defect. In extreme cases, when a valve cannot be surgically repaired, complete valve replacement is the only solution, although valves that are transplanted into babies and children typically do not grow over time as the child grows, thereby necessitating repeated operations throughout the individual’s life. Additionally, replacement valves in the past have been fashioned either from human, animal or artificial material, all of which also pose a number of risks, not the least of which is immune rejection.

As Dr. Sodian explains, “The problem is, if you have to do surgery on a child, you have a relatively small heart valve and the child grows out of it, which means you have to do the surgery many times. The basic idea is to implant something living, functional, from your own cells which will integrate into the surrounding tissue with the potential to grow. Imagine you had a child with congenital heart disease and this child has to be operated on every 2 to 3 years. It’s very hard for children and parents. The goal is to do surgery once that would last a lifetime. If we replace a valve in a child, they will need surgery several times in their lifetime, because they will grow out of the device, so the ultimate goal is to have a construct which is able to grow with the child and only have to do the surgery once. Earlier is better, if possible.”

The field of tissue engineering in general and of heart valves in particular is still in its infancy, with various research teams around the world exploring options for growing new heart valves not only from stem cells but also from bone marrow and amniotic fluid. Within this context, the innovation and novelty of Dr. Sodian’s procedure is in and of itself worthy of attention. According to AHA (American Heart Association) spokesman Dr. Russel V. Luepker, the Mayo Professor of Epidemiology and Community Health at the University of Minnesota in Minneapolis, “The whole idea of building a scaffold is a unique idea. We generally put progenitor cells in the heart and try to get them to grow muscle cells, and they’re sitting in the middle of other cells. But to build a scaffold that looks like a heart valve, then hope and anticipate that the cord blood cells will take that hint and differentiate, I think is very innovative.” He cautiously adds, however, “I don’t think anyone has any idea if the valves would grow. One may not know until it is put into a child, and the child grows. There are obviously a lot of hurdles to overcome.”

In regard to the physiological importance of even the tiniest of heart valves, Dr. Leupker explains, “The stresses on a heart valve are enormous. They have to hold the blood back with each beat. The wear and tear on them which we see with metal and plastic valves is an issue, and those are fairly hard substances.” As Dr. Sodian adds, “Tissue engineering provides the prospect of an ideal heart valve substitute that lasts throughout the patient’s lifetime and has the potential to grow with the recipient and to change shape as needed. We showed that it is possible to do this with human cells.”

Stem cells derived from umbilical cord blood are known to be among the most versatile of all adult stem cells, having already been demonstrated to differentiate into a wide variety of tissue types, including cardiac tissue which is one of the most highly specialized and complex of all human bodily tissues since it is both muscular and electrical in nature. Additionally, stem cells derived from umbilical cord blood are ethically noncontroversial, since the destruction of an embryo is not required for the derivation of such stem cells.

In what is known as a concept study, Dr. Sodian and his colleagues reported the results of their newly pioneered procedure today at the annual meeting of the American Heart Association in New Orleans.

In a further sign of the increasing investment opportunities that are springing up throughout the stem cell industry, big pharma has now decided to capitalize upon stem cell research. According to Pfizer spokeswoman Ruth McKernan, the world’s largest drugmaker has allocated a budget of $100 million to be directed over the next five years toward developing stem cell products that will specifically target the treatment of diseases that are typically associated with aging, such as heart disease, diabetes, vision loss and hearing loss, among other ailments. While such stem cell research and development is already underway in smaller companies such as Geron and Novocell, this marks the first time that a major pharmaceutical organization has entered the field. Although its headquarters are in New York, Pfizer is dedicating its laboratories in Cambridge, Massachusetts and Cambridge, England to the development of drugs that will stimulate adult stem cells that already exist in the human body to heal injury and disease. According to Corey Goodman, president of Pfizer’s Biotherapeutics and Bioinnovation Center, the U.S. lab will focus on heart disease, cancer and diabetes, while the UK lab will research therapies for vision and hearing. Pfizer is planning to hire 70 new scientists by the end of 2009 to staff laboratories at the two locations. Although embryonic stem cells will constitute part of the research, the primary emphasis will be on stimulating the body’s own adult stem cells for self-repair, thereby slowing or possibly even reversing the aging process.

According to Alan Trounson, president of the California Institute of Regenerative Medicine, the state agency that funds stem cell research in California, “The major pharma companies are moving into the field and taking a very strong position. We feel they’re like big ships coming together with us. It’s starting to be an armada.”

Biotech companies have not been entirely immune to the recent global financial crisis, with Pfizer’s stock losing 28% this past year, most recently falling 45 cents, or 2.7%, in one day, to $16.28 where it settled at 4:15 p.m. yesterday on the New York Stock Exchange composite trading. Nevertheless, the decision to join the stem cell bandwagon offers Pfizer greater protection from economic vicissitudes than the company might otherwise enjoy.

Embryonic stem cells are believed to be capable, at least theoretically, of differentiating into all 210 cell types of the human body, although this has never actually been demonstrated. By contrast, each type of adult stem cell is more limited in its “potency”, but taken altogether, all the various types of adult stem cells can also differentiate into all 210 cell types of the human body. Additionally, adult stem cells have thus far proven to be safer and more effective than embryonic stem cells, since embryonic stem cells still remain extremely problematic in the laboratory and consequently have never advanced to the clinical stage. Purely as an area of scientific interest, however, if not yet as a candidate for clinical therapy, embryonic stem cells are attracting the curiosity of more and more pharmaceutical companies, who also recognize the more immediate benefits of adult stem cell therapies, and who are therefore allocating research funding to both fields. Indeed, other major drugmakers besides Pfizer are also taking an increasingly active interest in both types of stem cell research and technology. The Swiss pharmaceutical companies Novartis AG and Roch Holding AG, for example, as well as Johnson & Johnson of the United States, and the London-based company GlaxoSmithKline, which is the world’s second largest pharmaceutical company, second only to Pfizer, have all initiated new investments in or partnerships with other biotech companies that are developing stem cell therapies. In 2007 Novartis and Roche helped fund the Spanish company Cellerix in the use of adult stem cells from subcutaneous fat for the treatment of patients with rare skin disorders, while in July of this year Glaxo announced a four-year, $25 million stem cell project with Harvard University. Similarly, J&J’s venture capital arm took an equity interest in the U.S. company Tengion, which is growing various organs such as bladders from adult stem cells in the laboratory, and J&J also led a $25 million round of funding for Novocell, which is involved in researching possible diabetes therapies from embryonic stem cells. According to Reinhard Ambros, executive director of the Novartis Venture Fund which invests in corporations involved in the life sciences, “There will be more companies coming with good technologies that will raise more interest from venture capital people.” However, Pfizer is the first big pharma company to dedicate an entire program exclusively to stem cell research, as pointed out by John McNeish, the director of Pfizer’s Massachusetts division. According to Corey Goodman, in order to enter the stem cell field, Pfizer first underwent a major change in corporate policy which was subjected to detailed reassessment and authorization by CEO Jeffrey Kindler.

Pfizer’s approach to their new stem cell research project is based in large part upon the work of Dr. Sheng Ding of the Scripps Research Institute in California, who explains that, “People might not know that stem cells are everywhere in the body and play a role in disease. Awakening stem cells already present in the body might be very attractive for therapeutic intervention to achieve healing and repair.” In 2007, Dr. Ding cofounded the San Diego-based company Fate Therapeutics, which is collaborating with Pfizer in developing novel pharmaceutical agents that can activate and mobilize endogenous dormant adult stem cells already residing in the hearts and other organs of people, so that such stem cells will be triggered to repair tissue that is damaged by injury or disease. According to Dr. McNeish, “Most people and scientists do believe that cells will be medicines in the near future.” Dr. Brock Reeve, executive director of the Harvard Stem Cell Institute, adds, “Originally, pharma stayed away because of the timeline, knowing that [embryonic] stem cell-based therapies will be years down the road. Where things have changed in the last year is that we can now create cells of interest in particular diseases,” referring specifically to the newly developed laboratory technologies that have made the news headlines over the past year, such as the technique pioneered by Dr. Shinya Yamanaka of Kyoto University in which iPS (induced pluripotent stem) cells are created by reprogramming ordinary adult, non-stem cell, somatic cells to behave with a pluripotency seemingly equivalent to that of embryonic stem cells.

As more and more venture capital funding and for-profit companies enter the stem cell field, thereby adding to the diversification and competition of marketable products, stem cells are no longer limited to the realm of university laboratories and academia, but instead represent a growing industrial sector of the world’s stock markets.

Representatives of the adult stem cell company Mesoblast announced today that they have been awarded the 2008 Frost & Sullivan United States Stem Cell Market Technology Innovation of the Year Award. Although the company is based in Australia, the award also recognizes Mesoblast’s U.S.-based counterpart, Angioblast Systems, Inc., for its success and contribution to the stem cell industry. The selection of an award winner is based upon a number of factors which include market analysis and interviews, all of which combine to determine one company with the top industry rank.

According to Katheryn Symank, Frost & Sullivan’s industry analyst, “Angioblast’s proprietary technology has several attractive attributes that set it apart from other stem cell products, including very accurate identification and isolation. This technology allows for a cell population with up to 1000-fold greater concentration of stem cells compared to other conventional sorting methods. Moreover, due to the non-immunogenic nature of the cells, Angioblast’s highly concentrated and pure population of stem cells can provide a well-regulated, consistent batched product with stringent release criteria akin to small molecule pharmaceuticals.”

Symank adds, “Since Angioblast’s proprietary technology allows for a very pure, potent and homogenous cell population, we view the recent pharmaceutical partnering activity in the stem cell space as a major validation of Angioblast’s approach. This underscores the company’s prospects for significant commercial transactions.”

Dr. Silviu Itescu, the founder of the company, responded by stating, “We are honoured to be recognized with this prestigious award from Frost & Sullivan. We will continue to optimize and progress our innovative technology in order to produce novel therapies for major cardiac, vascular, eye and orthopedic indications with unmet clinical needs.”

Mesoblast Ltd., is an Australian biotechnology company the focus of which is the development of novel treatments for orthopedic conditions via the rapid commercialization of unique adult stem cell technologies, especially those involving mesenchymal precursor cells (MPC), which are specifically aimed at the regeneration and repair of bone and cartilage. Mesoblast has acquired a substantial interest in the U.S.-based company Angioblast Systems which is developing the platform MPC technology for the treatment of cardiovascular diseases, including but not limited to the repair and regeneration of blood vessels and heart muscle. Mesoblast and Angioblast Systems already hold a number of patents in the field.

Adult stem cells that have been derived from human adipose (fat) tissue mark the first of their kind in a new breakthrough that could offer an ideal stem cell therapy for heart patients.

According to Dr. Rodney Dilley, principal scientist at Melbourne’s Bernard O’Brien Institute, “The fact that you can do this potentially opens a whole area of heart regeneration methods. Our approach is to create a piece of heart muscle that we can use to put onto the heart to stop it from remodelling and to return its contractile function to normal.” Since heart muscle does not usually regenerate itself after injury, but instead forms scar tissue as part of the “remodelling” process, this announcement by the Australian scientists has far-reaching implications for the field of cardiology. Additionally, since most people have accumulated the storage of some body fat, adipose tissue constitutes one of the most easily accessible sources of autologous adult stem cells.

The discovery could potentially offer a treatment for a wide variety of cardiac problems, ranging from congenital heart defects to age-related heart disease.

As a medical therapy, stem cells offer, for the first time in history, the possibility of treatment and perhaps even the cure of human diseases which previously have been untreatable. Precisely for that reason, the business of stem cells is projected to be a lucrative one.

The stem cell field is estimated to become a $500 billion industry over the next 20 years, and there is hardly a nation on earth that is not targeting stem cell research and development as part of its economy. To be able to “get in at the ground floor”, in any business with this potential for growth, is a rare opportunity. Recognizing such an obvious fact, the largest pharmaceutical company in the world has now decided to seize this opportunity.

According to Dr. John McNeish, executive director of R&D at Pfizer, the pharmaceutical industry leader is scheduled to open its second regenerative medicine center in Cambridge, England, next month. The focus of its U.K. location will be iPS (induced pluripotent stem) cells and their applications in ophthalmologic and central nervous system diseases. Pfizer’s first regenerative medicine center, located in Cambridge, Massachusetts, already focuses on stem cell therapies for the treatment of heart disease and diabetes.

As Dr. McNeish announced to reporters last month at the World Stem Cell Summit that was held in Madison, Wisconsin, “Stem cells can help us make good decisions about which compounds will be more likely to be safe. These cells will be tremendous in drug discovery. They will help us understand personalized medicine, genetic variation, ethnic populations, and which biomarkers to follow.”

As the largest pharmaceutical company in the world, Pfizer employs approximately 100,000 people worldwide in the manufacture and commercialization of prescription medication, with sales of Lipitor, its cholesterol-lowering drug, exceeding $10 billion last year alone. As an indication of its commitment to the stem cell field, Pfizer’s new regenerative medicine center in Cambridge, England, is estimated to occupy a space of approximately 52,000 square feet in area.

Pfizer is not the first pharmaceutical company to enter the stem cell industry. Last year, GlaxoSmithKline (GSK), AstraZeneca and Roche Holding together launched a new drug screening initiative entitled “Stem Cells for Safer Medicines”. GSK has also announced a $25 million collaboration with the Harvard Stem Cell Institute, geared toward developing the drug screening potential of stem cell technology.

The merging of “big pharma” with stem cell R&D is perhaps the latest and most significant indication of the rapid growth of the stem cell field, though this will certainly not be the last indication of its type.

In January of 2008, Osiris Therapeutics and Genzyme began collaborating together on a multi-million-dollar Department of Defense (DoD) contract that was awarded to Osiris, the objective of which is the development of a civilian and military medical response to nuclear or radiological events.

The DoD contract specifies the development and stockpiling of Prochymal, which is a proprietary adult stem cell therapy developed by Osiris, specifically for the repair of cellular injury that might result from the “acute radiation syndrome” (ARS) that accompanies severe and sudden radiation exposure. Terms of the contract provide for the purchase of up to 20,000 doses of Prochymal by the DoD at $10,000 per dose.

According to C. Randal Mills, Ph.D., President and CEO of Osiris, “We are honored that the Department of Defense has selected Prochymal in this critical effort to better safeguard our armed forces against the potentially horrendous effects of battlefield exposure to a radiological weapon. The contract also brings into focus a substantial new market opportunity for Prochymal. We are working diligently towards licensure of Prochymal for ARS and stand ready to assist other sectors of the United States government and allied nations in their emergency preparedness efforts.”

Major General John Parker, M.D., a former Commanding General who is currently responsible for countermeasure development and acquisition and who is also a member of the Medical Countermeasure Advisory Board of Osiris, adds, “Prochymal’s unique mechanism of action and strong clinical profile make it very well suited to address the complicated injuries associated with ARS. Currently, every scenario contemplating a radiological emergency, both civilian and military, involves people suffering from the life-threatening effects of ARS without effective treatments. Today’s decision by DoD sets in motion a sound plan to change that, by expeditiously completing development of the first effective therapy for ARS.”

As Henri Termeer, Chairman and CEO of Genzyme, explains, “We are pleased to partner with Osiris in developing this innovative cell therapy to treat the potentially lethal complications of ARS for the U.S. military. With our combined first-in-class technology and development expertise, Osiris and Genzyme have the necessary resources to complete this assignment for the Department of Defense and to work with other government organizations committed to safeguarding our nation and its allies.” According to Thomas MacVittie, Ph.D., Professor of Radiation Oncology and Pathology at the University of Maryland and a member of the NIAID (National Institute of Allergy and Infectious Diseases) Medical Countermeasures and CDC Strategic National Stockpile Radiation Working Groups, who is also a member of the Medical Countermeasure Advisory Board of Osiris, “Prochymal represents a breakthrough in countermeasure development for ARS. Prochymal has demonstrated therapeutic utility in humans repairing many of the major organ systems affected by radiation injury. Where most approaches only target a single component of ARS, Prochymal has the potential to address the entire syndrome including both acute and delayed effects in multiple organ systems.”

ARS is known to damage most severely the DNA of the rapidly dividing cells in the gastrointestinal tract, the skin and bone marrow. If severe and untreated, death can result within a matter of days or months following the initial exposure. Prochymal is a highly purified formulation of mesenchymal stem cells that are cultured and expanded. Prochymal is currently in Phase II clinical trials for the treatment of Type I diabetes, and Phase III clinical trials for both the treatment of Graft vs. Host Disease and Crohn’s disease. Additionally, Prochymal has demonstrated preliminary efficacy in the treatment of heart attacks and it has demonstrated a strong safety profile in seven previous Phase I and Phase II clinical trials. Prochymal has also shown a potential ability to reverse cellular damage and improve survival in diseases that are similar to ARS.

While warfare has always been understood to have biological and health consequences for those who are involved, the anti-inflammatory and regenerative properties of adult stem cells now offer a new type of countermeasure against nuclear and radiological threats. The strong interest of the Deparment of Defense in adult stem cell therapies marks yet another historic milestone in the versatility and applicability of these potent therapies.