State Panel Bans Human-Animal Hybrids

In Louisiana, the State Legislature has made it a crime to create human-animal hybrids. Under the newly approved legislation, the crime would be punishable by up to ten years in prison.

Promoted by State Senator Danny Martiny, the bill passed without objection by members of the State Senate Judiciary Committee, before going to the full State Senate.

Specifically, the bill prohibits the combination of human sperm with a nonhuman egg, and nonhuman sperm with a human egg, and the development of human neural tissue within a nonhuman animal. The bill does not prohibit some common medical practices such as the use of pig valves in human heart surgery.

The creation of human-animal hybrid embryos is a common procedure in many types of experimental embryonic stem cell research, although outside of the embryonic stem cell world such practices remain highly controversial, primarily for the ethical can of worms that is unleashed by such research. Hybrid experimentation was the topic of widespread debate last year in Britain when funding for such procedures was abruptly halted, despite the fact that Parliament had passed the Human Fertilisation and Embryology Bill, which legalized the creation of human-animal hybrid embryos, in May of 2008. (Please see the related news article on this website entitled, “Human-Animal Hybrid Experimentation in the U.K. is Halted”, dated January 13, 2009). Although such hybrids are usually destroyed during the process of embryonic stem cell experimentation, the possibility of such an embryo surviving past the embryonic stage and growing to adulthood would be a bioethical nightmare, if not also a scientific one as well. Despite the immense biological interest that such hybrids present, the ability to create entire new species in the laboratory is usually a cause for grave concern within the legal and religious communities if not the scientific community.

Earlier this year, research conducted by the company Advanced Cell Technology in Worcester, Massachusetts yielded disappointing results regarding the effectiveness of hybrid techniques. When human DNA was combined with nonhuman ova (eggs) to create hybrid embryos, researchers found that the human DNA did not reprogram effectively and therefore failed to generate stem cells.

Two U.S. Adult Stem Cell Companies Form Collaboration in Asia

The adult stem cell storage company Neostem announced today that it has signed an exclusive licensing agreement with Regenerative Sciences for the commercialization of proprietary adult stem cell technology. The territory to which the licensing agreement applies is all of Asia.

Regenerative Sciences developed the technology, known as Regenexx, specifically for the treatment of orthopedic conditions. Regenexx is a minimally invasive outpatient procedure in which adult stem cells are extracted from a patient’s own bone marrow, cultured in a medium containing important growth factors from the patient’s own blood, expanded to millions of stem cells over a period of 2 to 3 weeks, and then readministered to the patient. Specifically, mesenchymal stem cells are derived from each patient’s own bone marrow, and after processing the cells are injected directly into the patient’s afflicted joints. Since the stem cells are autologous (in which the donor and recipient are the same person), there is no risk of immune rejection. The procedure has already been used to treat hundreds of patients, for whom the procedure has eliminated the need for surgery.

Regenerative Sciences will serve as a consultant to NeoStem throughout their commercialization of the technology in Asia. According to Dr. Robin Smith, CEO of NeoStem, "We are very pleased to obtain the exclusive license in Asia to Regenerative Sciences’ unique technology, and we’re delighted to establish this relationship with Dr. Centeno. The minimally invasive Regenexx procedure complements NeoStem’s established in-office stem cell collection procedures and expands our expertise into a new area. Restoring joints, bone, muscle and cartilage and combating orthopedic diseases complements our growth in the anti-aging and regenerative medicine arena. Recently, we announced acquisition of an exclusive worldwide license for innovative stem cell technology and applications for cosmetic facial expansion procedures and skin rejuvenation. With Dr. Centeno’s assistance we will be adding his stem cell expansion procedure for orthopedic regeneration to the services we are offering in Asia through prominent medical institutions with which we are partnering that we expect to come to market by the end of this year."

According to Dr. Christopher Centeno, founding CEO of Regenerative Sciences, "I look forward to working with Dr. Smith and her team at NeoStem to bring Regenerative Sciences’ important stem cell innovations to a worldwide audience of doctors and patients. I’m especially excited about NeoStem’s relationships and its expanding presence in China."

With their corporate headquarters in New York City and their laboratory banking facility in Los Angeles, NeoStem specializes in the collection, processing and long-term storage of adult stem cells for therapeutic use at a later time in the future, if and when such a therapy might be needed. NeoStem was the first company to provide adult stem cell collection and banking services to the general public.

Headquartered in Colorado, Regenerative Sciences first began performing the Regenexx joint and bone stem cell procedure in 2005. Since then, hundreds of patients have been able to avoid surgeries of the shoulder, knee and hip through the use of their own autologous adult stem cells. As stated on their website, Regenexx is "a non-surgical treatment option for people suffering from moderate to severe joint or bone pain due to injury or other conditions."

Regenerative Sciences found itself at the center of an important controversy when, in July of 2008, Dr. Centeno received a letter from the U.S. FDA (Food and Drug Administration) stating that the autologous adult mesenchymal stem cells processed with Regenexx are considered to be "drugs" since they are intended for therapeutic use. According to the FDA letter, "These cells are considered drugs because the therapeutic claims shown on your website demonstrate that they are intended for use in the diagnosis, cure mitigation, treatment or prevention of disease in man." The letter concluded by stating, "Please be advised that in order to introduce or deliver for introduction a drug that is also a biological product into interstate commerce, a valid biologics license must be in effect. Such licenses are issued only after a showing of safety and efficacy for the product’s intended use. While in the development stage, such products may be distributed for clinical use in humans only if the sponsor has an investigational new drug (IND) application in effect as specified by FDA regulations… The mesenchymal stem cells utilized in your Regenexx procedure are not the subject of an approved biologics license application (BLA) nor is there an investigational new drug application (IND) in effect. Therefore, your implantation of the mesenchymal stem cells for which a valid license or IND is not in effect appears to violate the Act and the PHS Act and may result in the FDA seeking relief as provided by law."

Regenerative Sciences responded by posting the following notice on their website, which was most recently updated on March 31, 2009: "Last summer we got a letter from the FDA stating that they felt that our Regenexx medical procedure was actually the manufacturing of a new drug. The letter made no sense, as what we’re doing is practicing medicine. We kindly wrote back stating our position and then we didn’t hear back for more than half a year. Recently, the FDA has again asserted that they believe we are manufacturing a drug. They haven’t given any credible rationale for why they believe this, as what we’re doing with adult stem cells is no different than the average fertility clinic that grows embryos in culture for re-implantation. The fertility clinic is not regulated as a drug manufacture facility. The fertility specialists fought that fight and won."

This is a critically important issue, since it illustrates the frustration that many, if not all, adult stem cell scientists in the U.S. feel, as it this stance by the FDA which poses an insurmountable, not to mention entirely illogical, hindrance to adult stem cell therapies in the United States. For anyone who has ever wondered why it is that adult stem cell therapies are available in ordinary clinics in most countries outside of the U.S., but only in a limited number of FDA-approved clinical trials within the U.S., here’s your answer: the FDA considers each person’s own autolgous adult stem cells to be a "drug", and therefore those stem cells are subject to the same multi-year, multi-million-dollar clinical trial process through which all pharmaceutically produced drugs must pass before being considered legally marketable within the United States.

The statement on the website of Regenerative Sciences continues: "ASCTA (American Stem Cell Therapy Association) is a physician organization that was formed in opposition to the FDA’s position that adult stem cells are drugs. This group heralds a much bigger movement than what we’re doing here with the Regenexx procedure. We’ve found literally an outcry by patients with chronic diseases that the FDA would stand in their way of getting safe stem cell work performed by their doctors. We agree that there are hundreds of likely unsafe stem cell outfits around the globe injecting God-knows-what into whoever has the will to pay. All the more reason for an organization to step to the forefront to establish physician-run guidelines for safe lab practices and clinical oversight… The pre-clinical research on adult stem cells is much stronger than embryonic stem cells… When I’ve posed the question to numerous doctors and experts in the field, are your own stem cells drugs? They look at me like I’m crazy, and often reply ‘Of course my stem cells aren’t drugs!’ Why would the FDA take the position that your cells are drugs… The ASCTA physician group will be getting out its lab practices guidelines meant to hold new adult stem cell practices to the highest standards to protect patients. Our goal is clear and it’s worth fighting for: Safe Stem Cells Now!"

Likewise, the ASCTA has posted the following statement on their website: "The American Stem Cell Therapy Association (ASCTA) announced today the online publication of its mission statements and charter. The organization was formed in response to the Food and Drug Administration’s (FDA) recent position that the adult stem cells found in everyone’s body are drugs, a position the ASCTA opposes. This physician organization is establishing laboratory guidelines that will allow doctors to bring adult stem cell therapy to their patients more quickly. These guidelines will be similar to those used by fertility specialists in in-vitro fertilization (IVF) labs, where many of the same cell culture techniques are used."

Dr. Centeno, one of the founding members of the ASCTA, is quoted on the ASCTA’s website where he states, "Many patients are dying or suffering daily with incurable diseases or problems that require major surgery. These patients should have access to basic adult stem cell therapy now. ASCTA is establishing guidelines which will allow the safe use of the patient’s own adult stem cells under the supervision of doctors." According to Dr. Frank Falco, another ASCTA founding member who is also quoted on the ASCTA’s website, "The FDA’s position against someone using their own stem cells is taking it too far. We are talking about a person using their own tissue to treat a degenerative disorder or process safely without the use of medications or surgery. Although we agree that oversight and standards are necessary, this should be provided through a physician organization such as ASCTA rather than by a government agency."

Not all stem cells are created equal, of course, and it is vitally important that the FDA make distinctions between embryonic, fetal and adult stem cells. In this regard, the ASCTA website continues, "Adult stem cells are different than embryonic stem cells. Adult stem cells are found in the patient’s body in various tissues. In order to obtain enough stem cells for treatment, they often need to be cultured, similar to today’s fertility treatments. Adult stem cells have undergone much more research than embryonic stem cells and therefore are closer to real world treatments. These adult stem cells are taken from the patient’s own body (autologous) and ASCTA believes that they are therefore the safest for use in treating patients." To this Dr. Centeno adds, "While the Obama administration seems to have opened the embryonic stem cell door, their FDA seems to want to slam the adult stem cell door shut."

As also stated on their website, "The ASCTA is a physician group comprised of various medical and surgical specialists whose goal is to bring safe stem cell therapy to patients by establishing laboratory and clinical guidelines." More information is available at www.stemcelldocs.org.

Additionally, in April of 2009 a patient movement called "Safe Stem Cells Now!" was formed in response to the FDA’s unfounded position that a person’s own adult stem cells are "drugs" and therefore should be regulated in the same manner. More information is available at www.safestemcells.org.

As Barbara Hanson, cofounder of www.stemcellpioneers.com, states, "Adult stem cells are cells from our own body. They are very safe. There are no moral or ethical issues. They are safer than taking aspirin and yet the FDA has classified our own stem cells as drugs that require regulation. This means that prolonged investigations, including lengthy clinical trials, will be required for each and every disease and application that adult stem cells could be used for. This could take years and years. It smells of big pharma to me and many others."

Indeed, many people feel that this stance by the FDA is purely politically and economically motivated. At the very least, it is an entirely unscientific stance, and it is merely one example of the numerous ways in which the FDA needs to update its regulations so that these regulations are relevant and applicable to stem cells, which do not fall into any of the previously existing categories for which the FDA has ever had to formulate national law in the past. Such outdated laws are precisely what is driving many of the best and most accomplished adult stem cell physicians and scientists "off-shore", to set up their laboratories and clinics anywhere at all in the world, just as long as it is outside the borders of the United States. In the end, it is the U.S. patient who suffers, since the researchers and clinicians themselves are not stopped but are merely forced to relocate to other countries. Until the FDA is able to recognize the numerous and vast differences between an autologous adult stem cell and a pharmaceutically manufactured drug, it is no wonder that adult stem cell companies such as NeoStem and Regenerative Sciences are able to form licensing agreements throughout all of Asia, but not in their own home country, the United States.

Genzyme Reports Clinical Data and Business Updates

Today the biotech company Genzyme announced updates on anticipated near-term regulatory milestones, new clinical data and plans for continued sustainable growth across each of its businesses. The company reaffirmed its estimated goal of $7 billion in annual revenue by 2011, as growth continues to be driven by an increase in sales of existing products as well as by the launch of new treatments and "continued operating leverage from the company’s global infrastructure." According to Henri A. Termeer, Genzyme’s chairman and CEO, "We are modifying our earnings presentation to provide a simpler picture for investors. We continue to meet our financial commitments, increasing the profitability of the company while investing in new therapies and bringing them to the market."

Genzyme measures its financial activities not just by earnings but also by a number of other parameters and metrics which include cash flow, return on equity, return on assets and return on invested capital, all of which are expected to increase through 2011. Return on invested capital is estimated to be approximately 13% in 2011, with approximately $5.6 billion in cash from operations through the end of 2011. Additionally, the company plans to invest approximately $1.9 billion of this projected total in the expansion and maintenance of its global infrastructure, with approximately $1 billion allocated for repurchasing shares and approximately $700 million designated for potential milestone payments that are scheduled to be made to partners.

The company reported that pipeline as well as business developments have exceeded expectations. According to the report, "The launch of Myozyme (alglucosidase alfa) has been more rapid than the launch of any of Genzyme’s other treatments for lysosomal storage disorders, driven by faster-than-expected adoption by physicians and patients and consistent support from health authorities in more than 40 countries." Genzyme also presented new data from a Phase II clinical trial of GENZ-112638, which is an oral therapy currently in development for patients with Gaucher disease type 1. Data at the end of the first year indicate that the compound either improved or stabilized the bone disease that is caused by Gaucher disease.

In the product portfolio for the hematologic oncology field, the company estimates revenue of $1 billion by 2011. In oncology and bone marrow transplant, a recently announced transaction with Bayer HealthCare extends Genzyme’s commercial presence to more than 90 countries. Cardiometabolic and renal proprietary products are currently in Phase II/III clinical trials in which 315 hyperphosphatemic dialysis patients are enrolled. Results for the primary endpoint of serum phosphorus changes are expected during the first half of next year, and the core patent estate for sevelamer will not expire until 2014.

Additionally, in December of last year Genzyme received approval from the U.S. FDA for the marketing of its proprietary adult stem cell stimulating product, Mozobil, which is a novel, proprietary small-molecule CXCR4 chemokine receptor agonist. When used in combination with G-CSF (granulocyte-colony stimulating factor), Mozobil has been shown to increase the number of stem cells circulating in the peripheral blood by mobilizing hematopoietic stem cells in the bone marrow and stimulating the cells to migrate into the bloodstream for easier collection and subsequent autologous transplantation. According to John DiPersio, M.D., Ph.D., a professor at Washington University in St. Louis, "Mozobil is an important advancement in the treatment of patients with certain types of cancer who require a stem cell transplant. This product should become an integral part of the treatment regimen for transplantation because of the benefits it offers to patients, physicians and transplant centers." Approximately 55,000 hematopoietic stem cell transplants are performed each year globally for multiple myeloma, Hodgkin’s and non-Hodgkin’s lymphoma, for which Mozobil is expected to be used in the majority of such cases. Peak annual sales of Mozobil are projected to reach $400 million. Genzyme has also submitted applications to the respective regulatory agencies in Europe, Australia and Brazil for marketing approval of Mozobil, and additional applications in up to 60 countries are planned.

In November of 2008, Genzyme announced a strategic alliance with Osiris Therapeutics for the commercialization of two adult stem cell products. According to the agreement, Osiris will commercialize the proprietary products Prochymal and Chondrogen in the U.S. and Canada, while Genzyme will commercialize the products throughout the rest of the world. Genzyme made an upfront payment of $130 million to Osiris in additon to milestone and royalty payments that will be made in the future and which are estimated to be worth approximately $1.3 billion. Prochymal and Chondrogen are therapies that use allogeneic (in which the donor and recipient are different people) adult mesenchymal stem cells derived from bone marrow. Both of the products are late-stage treatments for a wide variety of diseases.

Genzyme, the world’s largest maker of drugs for rare genetic disorders, is well known for commercializing first-in-class biotechnologies. In addition to its large orthopedic franchise, Genzyme’s products are focused on rare genetic disorders, kidney disease, cancer, transplant and immune disease, diagnostic testing, cardiovascular disease, neurodegenrative diseases, endocrinology and other medical specializations in which patient needs are not adequately met. Genzyme’s strategic alliance with Osiris marks a major expansion by Genzyme into the field of cell transplant therapies. Founded in 1981, Genzyme now has more than 10,000 employees around the world with revenues in 2007 of $3.8 billion. Its products and services are available in nearly 90 countries worldwide. In 2007, Genzyme was awarded the National Medal of Technology, the highest honor awarded by the President of the United States for technological innovation.

Genzyme’s stock closed at $60.85 today.

Cytori and General Electric Agree Upon Collaboration

Cytori Therapeutics has announced that GE Healthcare, a division of the General Electric Company, will market Cytori’s stem cell extraction products in the U.S., Canada and Mexico for 18 months, beginning in the second quarter of 2009. This agreement follows a similar arrangement that the two companies made in January and which applied to ten European countries.

Known as StemSource, the proprietary technology is used to extract adult stem cells from adipoes (fat) tissue, after which the cells are then either cryogenically preserved or made available for research purposes. The processing system would make the stem cells available for cell banking and research markets.

The agreement does not include Cytori’s Celution System, which is still being reviewed by the U.S. FDA (Food and Drug Administration) for marketing approval.

According to Christopher Calhoun, CEO for Cytori Therapeutics, "Expanding our commercialization partnership into North America will allow us to take advantage of GE Healthcare’s established stem cell banking and research customer base. Both organizations recognize there is an existing demand in the United States for these products and we look forward to making the StemSource technology available in the U.S. in the first half of 2009."

According to Konstantin Fiedler, general manager of Cell Technologies at GE Healthcare, "This agreement between GE Healthcare and Cytori is important for our growing life sciences business. Our U.S. sales team, like that in Europe, is seeing growing customer demand for adipose-related cell technology, both from our existing stem cell banking customers as well as our research customers involved in the development of cell therapies. An expanded partnership with Cytori gives our U.S. sales team access to these first-in-class products for these markets in the United States."

Founded in 1878 by Thomas Edison, the multinational conglomerate General Electric ranks as the tenth largest company in the world in terms of market capitalization, with reported revenue in 2008 of $182.5 billion. The GE Healthcare division alone employs more than 47,000 people in more than 100 countries.

Founded in 1996 and headquartered in San Diego, Cytori Therapeutics develops technology for reconstructive and aesthetic surgical procedures.

Stem Cells Return Lost Hearing

Researchers at the University of Sheffield have announced that they have differentiated fetal stem cells into the sensory hair cells of the ear, which could then be surgically inserted into the ear to restore lost hearing, at least theoretically.

According to Dr. Marcelo Rivolta, who led the study, "It will take several years before we are in a position to start doing human trials." He and his colleagues caution that the research is still in the preclinical, animal model phase, and has not yet advanced to a level where it could be performed safely and effectively in humans.

In the past, whenever auditory hair cells were damaged, permanent hearing loss would result since it was not possible to repair the damaged cells. Now, however, stem cells could be differentiated into auditory cells, thereby restoring lost hearing.

According to Dr. Ralph Holme, director of biomedical research at the Royal National Institute for Deaf People, which helped fund the research, "This research is incredibly promising and opens up exciting possibilities by bringing us closer to restoring hearing in the future."

Although the scientists used fetal stem cells, they are also investigating the ability of embryonic and adult stem cells to differentiate into auditory hair cells. Fetal stem cells have a number of inherent problems and risks, not the least of which is the formation of tumors. (Please see the related news article on this website, entitled "Fetal Stem Cell Therapy Could Prove Fatal", dated February 17, 2009). Presumably this is also the reason why iPS (induced pluripotent stem) cells were not differentiated into auditory neurons, which iPS cells are certainly capable of doing, but scientists have not yet been able to create a completely safe iPS cell which is free of cancer-causing genes and which also lacks the strong tendency to form teratomas (a type of tumor with cells and tissue from all 3 germ layers).

The auditory hair cells of the cochlea, or cochlear hair cells, function as the sensory receptors of both the auditory system and the vestibular system, not only in humans but in all vertebrates. In the past, damage to any part of the cochlear apparatus would often result in decreased hearing sensitivity, which was always considered to be irreversible. These fine hair cells are “bundled” in two sections, known as outer and inner hair cells, each of which performs a specialized function. The outer hair cells provide a purely mechanical amplification of sound, in a similar way as an acoustic preamplifier. The inner hair cells act as a more complex transducer, converting the mechanical vibrations into electrical signals that travel via the auditory nerve to both the auditory brainstem and the auditory cortex of the brain. Although the normal human hearing range is considered to be from 20 Hz to 20,000 Hz, some degree of sensorineural hearing loss is normal with advanced age.

Since all aspects of the auditory system were originally formed from stem cells, then at least in principle it should be possible to regenerate all aspects of the auditory system, given the right type of stem cell. At least conceptually, it should be possible to regenerate not only the auditory sensory receptors of the cochlear hair cells but also auditory neurons as well, regardless of where, exactly, the anatomical damage is located.

Pfizer and the University of Wisconsin Form Licensing Agreement

Once again, the pharmaceutical giant Pfizer has entered into a collaborative agreement with academia, this time licensing human embryonic stem cell patents from the University of Wisconsin at Madison. The patent technology will be developed not for clinical cell-based therapies, per se, but primarily for the testing of new drugs. Terms of the arrangement were made with the Wisconsin Alumni Research Foundation (WARF), the university’s licensing arm.

According to Ruth McKernan, chief scientific officer of Pfizer Regenerative Medicine, "Our license with WARF provides us with information and materials that will allow us to use their cell lines to explore a whole new range of therapies. Stem cells can be used to create specialized human tissue. Our scientists will determine how new medicines may be able to improve the way stem cells regenerate damaged tissues. We will be optimizing the production of cells that could, one day, be used for therapeutic purposes."

According to Wisconsin Governor Jim Doyle, "To have these two giants in the field of biopharmaceutical research and stem cell research come together brings us one step closer towards finding relief from diseases like diabetes, Alzheimer’s, Parkinson’s, multiple sclerosis and cancer."

Of course, the entire field of embryonic stem cell research was born at the University of Wisconsin at Madison, where Dr. James Thomson became the first person ever to isolate a stem cell in the laboratory, first from a nonhuman primate in 1995 and later from a human in 1998. By "these two giants", therefore, Governor Doyle is referring of course to Pfizer and also to the entire "industry" of stem cell scientists and laboratories that has sprung up around Dr. Thomson over the past decade.

Actually, as Dr. Thomson himself has stated on a number of occasions, he does not expect embryonic stem cells to yield cell-based therapies for diseases in the immediate future, but instead he expects embryonic stem cells to be most useful as a way of testing new pharmaceuticals, which in the past could only be tested in animal models since human tissue was not available. In fact, in November of last year, Dr. Thomson formed a new company, Cellular Dynamics International (CDI), precisely for this reason. 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 per se, CDI is 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. (Please see the related news article on this website entitled, "Embryologist and Stem Cell Pioneer Forms New Company", dated November 25, 2008, as originally reported in Forbes). In the past, side effects from drugs have been tested on animal cells, but rarely with great accuracy, with the result that physicians often 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 instead presents a different 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 has explained in regard to CDI, "We’re very much going to be focused on products rather than long-term promises. There are things that drug companies want today."

Still, embryonic stem cells are, by their very nature, heterologous vis a vis any living individual, and therefore neither genetic nor immune matching is possible between the embryonic stem cells and the individual who is still alive and in need of the therapy, which thereby also eliminates the possibility of matching drug reactions. By sharp contrast, iPS (induced pluripotent stem) cells, which are derivable from any living person, offer matching not only of genetic and immune profiles but also of pharmaceutical reactions as well. In this regard, even Dr. Thomson has stated, "The world has changed. Over time, these [iPS] cells will be used in more and more labs. And human embryo stem cell research will be abandoned by more and more labs."

As the world’s largest research-based pharmaceutical and biomedical company, Pfizer ranks number one in sales in the world, having reported $48.4 billion in revenue in 2007, from which the company invested $8.1 billion into their own research and development. In January of 2009, Pfizer announced its agreement to buy the pharmaceutical giant Wyeth for $68 billion. Pfizer was founded in 1849 and today employs approximately 81,900 people in more than 150 countries. Pfizer’s launched their Regenerative Medicine unit in November of 2008. (Please see the related news article on this website, entitled, "Business is Booming as Pfizer Targets the Aging Process With New Adult Stem Cell Research", dated November 14, 2008, as originally reported by Bloomberg Press).

Founded in 1848, the University of Wisconsin at Madison today has a staff of over 16,000 employees, more than 2,000 of whom are faculty, and a student body of just under 50,000.

(Please see the related news article on this website entitled, "Pfizer and University College London Form Licensing Agreement", dated April 29, 2009).

Patient Enrollment Beginning for Adult Stem Cell Trial

The Stem Cell Center at the Texas Heart Institute at St. Luke’s Episcopal Hospital in Houston has announced the commencement of FDA-approved Phase I clinical trials in which the Hospital is currently enrolling patients who have experienced any of three cardiovascular conditions, namely, a heart attack, heart failure or peripheral vascular disease. While patients will not be given monetary compensation for enrollment in the trials, they also will not be charged for any of the therapy or post-therapy monitoring that is conducted in relation to the trials.

The Stem Cell Center currently has several concomitant trials in which patients are being enrolled for heart failure. Qualifying patients must be on maximal medical therapy, exhibit a low ejection fraction, and have exhausted all conventional medical therapy such as bypass surgery and angioplasty. For the heart attack trials, qualifying patients must recently have been treated with angioplasty following a heart attack. For the peripheral vascular disease trials, in which adult stem cells will be injected directly into the affected areas of the leg, qualifying patients must exhibit claudication (blocked arteries in the legs) but no active infections in the leg, and must be refractive to conventional therapy.

Patients who wish to be considered for enrollment, and physicians who would like to refer a patient for consideration, should contact the Texas Heart Institute directly.

The Texas Heart Institute was founded in 1962 by the cardiac surgeon and pioneer, Dr. Denton Cooley, and today the Institute ranks as one of the largest cardiovascular centers in the world. As of 2008, doctors at the Texas Heart Institute had performed over 200,000 cardiac catheterizations, 100,000 open heart surgeries, and 1,000 heart transplants. Additionally, the Texas Heart Institute was one of only five centers selected by NIH (the National Institutes of Health) to study stem cell therapy as a treatment for cardiovascular disease, and the Stem Cell Center at the Texas Heart Institute was the first organization within the U.S. to receive FDA approval for a clinical trial in which advanced heart failure is treated with adult stem cells.

Pfizer and University College London Form Licensing Agreement

Pfizer’s Regenerative Medicine division and the University College London (UCL) have announced the formation of a partnership for the development of stem cell-based therapies in the treatment of ophthalmic conditions. In particular, the collaboration will focus on the differentiation of human embryonic stem cells (hESCs) into retinal pigment epithelium (RPE) for the treatment of age-related macular degeneration (AMD).

According to Dr. Ruth McKernan, chief scientific officer of Pfizer Regenerative Medicine, "We are excited to be working with pioneers in the field of stem cell ophthalmology from UCL. While we have much to learn about how [embryonic] stem cells can be used therapeutically, we are confident that this relationship will increase that understanding and help us advance to a time when our work may benefit patients worldwide."

Indeed, the world has everything yet to learn, about how, or if, embryonic stem cells could be used therapeutically, since thus far embryonic stem cells have never been used therapeutically, for anything. The only types of stem cells that have ever been used therapeutically are adult stem cells, which are already being used in clinics throughout the world for the treatment of a wide variety of diseases and injuries.

In fact, many scientists are already working on stem cell therapies for eye diseases which do not involve embryonic stem cells, but which instead involve either adult stem cells or iPS (induced pluripotent stem) cells, the latter of which are not naturally occurring stem cells, per se, but instead have been "induced", as the name implies, to exhibit a pluripotency that resembles that of embryonic stem cells. Just last month, for example, in April of 2009, it was reported in the Proceedings of the National Academy of Sciences that cells which had been isolated in the eye and which scientists had previously presumed, as early as 2000, to be retinal stem cells, are instead just ordinary adult stem cells, without any particular predilection toward differentiating into retinal cells. Such a discovery prompted a number of specialists within the ophthalmology field to turn their attention to the re-engineering of iPS cells as the best form of treatment for eye diseases, especially retinal diseases in which the light-sensitive photoreceptor cells degenerate, since these cells could be re-differentiated from iPS cells. Many other eye diseases also involve damage to the layer of ciliary epithelial cells that line the inside of the eye and which contains retinal cells, and iPS cells could be differentiated into these tissue types to treat such diseases. The search for a specific neural stem cell or retinal progenitor cell is therefore no longer as important as it was once thought to be, since iPS can perform the same function. Furthermore, since iPS cells can be developed from cells that are derived from a specific patient who has a specific disease, the iPS cells would already be genetically and immunologically matched to the patient, thereby eliminating any threat of immune rejection. Embryonic stem cells, by sharp contrast, are by their very nature a foreign, heterologous material which for which the obvious and predictable problems of immune rejection have yet to be resolved.

In reference to iPS cells, Dr. Michael Dyer, a member of the St. Jude Children’s Research Hospital Department of Developmental Neurobiology, explains, "This approach would solve many problems of developing cell-based therapy for blindness. First, these cells are immortal, so they can be grown indefinitely to produce large amounts of cells for treatment. And secondly, they would be immunologically matched to the patient, so there would be no danger of rejection. And thanks to some excellent research during the past 15 years, we know a lot about how to reprogram such stem cells to make them into photoreceptors."

Nevertheless, this new partnership between Pfizer and the University College London no doubt represents one of the largest of its kind. According to the terms of the agreement, Pfizer will provide an unspecified amount of funding to UCL, where research will be conducted on the differentiation of embryonic stem cells into retinal pigment epithelium cells, hopefully, for the development of therapeutic applications to both wet and dry age-related macular degeneration as well as other retinal diseases. Pfizer will retain exclusive worldwide rights to commercialize the therapies that result from the research, although it is unclear who exactly will retain the rights to related discoveries that cannot strictly be classified as ophthalmologic in nature.

Age-related macular degeneration affects more than half a million people in Britain alone. According to Professor Pete Coffey of the UCL’s Institute for Ophthalmology, who will direct the project, "We have not only the benefit of Pfizer’s experience of the regulatory process and their expertise in stem cell technology, but the ability, if this works, to produce on a much larger scale. It has huge implications, not only for our project, but for the field of regenerative medicine as a whole."

As the world’s largest research-based pharmaceutical and biomedical company, Pfizer ranks number one in sales in the world, having reported $48.4 billion in revenue in 2007, from which the company invested $8.1 billion into their own research and development. In January of 2009, Pfizer announced its agreement to buy the pharmaceutical giant Wyeth for $68 billion, thereby increasing the scope of its resources even further. Pfizer was founded in 1849 and today employs approximately 81,900 people in more than 150 countries. Pfizer’s launched their Regenerative Medicine unit in November of 2008. (Please see the related news article on this website, entitled, "Business is Booming as Pfizer Targets the Aging Process With New Adult Stem Cell Research", dated November 14, 2008, as originally reported by Bloomberg Press).

Located in central London, University College London was founded in 1826 and today boasts a staff of 8,000 and a student body of over 22,000.

(Please see the related news article on this website entitled, "Pfizer and the University of Wisconsin Form Licensing Agreement", dated May 5, 2009).

South Korea Conditionally Lifts Embryonic Stem Cell Research Ban

Still cautious almost exactly 3 years after the international fraud scandal involving Dr. Hwang Woo-suk, at which time human embryonic stem cell research was halted in South Korea, the country is slowly allowing a return to such research, provided that certain conditions are met.

The National Bioethics Committee has approved a request from scientists at Seoul’s Cha General Hospital to conduct human fetal stem cell research on aborted human fetuses, as long as the scientists meet the following 4 conditions: 1/ the scientists must secure written consent from the donors of the fetuses, 2/ the main focus of the hospital’s research must be conducted on laboratory animals, in an attempt to minimize research on human tissue, 3/ the hospital must establish an internal screening group to check for possible abuse, and 4/ the scientists must remove words and phrasing from the project title that would give people false hope. As an example of the fourth condition, the current title of the research project includes the phrase, "stem cell research which can cure diseases such as Parkinson’s", which should be changed, according to the conditions of the authorization.

As Dr. Roh Jae-kyung, chief of the National Bioethics Committee, explained to the scientists at Cha General Hospital, "This research that we have conditionally approved today is really just the very first step towards a new academic study. Though we hope this will eventually become a ray of hope for those patients with an incurable disease, we want you to refrain from premature or excessive expectations."

It was in May of 2006 that Dr. Hwang Woo-suk was indicted on embezzlement and violations of bioethics laws committed in 2005, after confessing to having falsified his claims that he had cloned the first human embryonic stem cells. As a result of the ensuing scandal, the South Korean government has been cautious not to allow such fiascos to happen again.

According to Dr. Chung Hyung-min, a leading researcher at Cha Hospital, "The decision will help reactivate stem cell research in South Korea. Stem cell research has been done by scientists in Britain and other countries. But there has been no successful case yet, using human eggs."

The Bioethics Committee has rejected a similar request from Dr. Hwang himself, who has also formally petitioned the Committee to be allowed to begin new human stem cell research from aborted human fetuses. Dr. Hwang is still on trial, however, for a number of charges that include fraud, embezzlement and ethical breaches.

(This article is a continuation of the story and related news article on this website, entitled, "South Korea Delays Decision on Cloning", dated February 7, 2009).

Cornell Conducts Adult Stem Cell Study on Horses

Researchers at the College of Veterinary Medicine at Cornell University in Ithaca, New York have reported the effective treatment with an autolgous adult stem cell therapy of tendonitis in horses.

Led by Dr. Alan J. Nixon, the study was not so much a "clinical trial" in the ordinary sense of the term, but was perhaps more correctly an "experimental study", since the researchers sacrificed the horses at the end of the evaluation period in order to conduct thorough histological and mechanical analysis of the tendons.

In the study, the researchers artificially induced tendonitis in the superficial digital flexor tendons (SDFT) of both forelimbs of an undisclosed number of horses. Six days later, autologous mesenchymal stem cells (MSCs) were then derived from the bone marrow of each horse, expanded and injected into one of the SDFT lesions, while the other untreated, "control" limb received an injection of 1 milliliter of saline. The MSCs were also gene-enhanced with IGF-1 (insulin-like growth factor 1), which has been demonstrated to stimulate cellular proliferation and tendon fiber deposition in the core lesion of tendonitis. Ultrasound examination of the tendons was performed at the start of the trial as well as at 2, 4, 6 and 8 weeks after administration of the therapy. According to an online abstract, "the horses were euthanized at 8 weeks and SDFTs were mechanically tested to failure and evaluated for biochemical composition and histologic characteristics."

As Dr. Nixon explains, "The biochemical composition of the treated and untreated tendons were similar 8 weeks after treatment. However, tendons injected with the stem cells had significantly improved histology scores, indicating a more normal microscopic appearance in treated tendons than untreated tendons. Nonetheless, more research is needed regarding the optimal dose of stem cells and the use of gene enhancement techniques to augment the observed benefit before making this technology clinically available."

The researchers conclude, "These findings indicate a benefit to the use of MSCs and AdIGF-MSCs for the treatment of tendonitis."

The results of the trial will be published in an upcoming edition of the Journal of Orthopedic Research in an article entitled, "Mesenchymal stem cells and insulin-like growth factor-1 gene-enhanced mesenchymal stem cells improve structural aspects of healing in equine flexor digitorum superficialis tendons."

Tendon injuries are often catastrophic and fatal in horses, while even minor tendon injuries can end the career of a race horse. According to Dr. Nixon, "At present, few successful treatment options exist for horses with tendon injuries. While stem cell therapy has become a hot topic in equine medicine, there are few controlled studies clearly documenting the safety or efficacy of this treatment modality for tendonitis in horses."

If killing the horse is a requirement for conducting a "controlled" study, then most people would probably conclude it’s a good thing that there aren’t many controlled studies that have been conducted.

In fact, a number of companies throughout the world are already treating horses, as well as other animals, with autolgous adult stem cell therapies, and not only do the animals improve as a result of the therapy but the animals are also allowed to continue living and enjoy the remainder of their lives. Perhaps the most renowned of these companies is the U.S.-based Vet-Stem, which has treated over 3,000 horses and 2,000 dogs with autologous adult stem cell therapy for a variety of joint injuries and degenerative conditions that include tendonitis as well as ligament injuries and age-related osteoarthritis. None of the animals treated by Vet-Stem’s autolgous adult stem cell therapy ever had to be euthanized, but instead were allowed to live out their natural lives.

Additrionally, in September of 2007 Vet-Stem licensed their proprietary adult stem cell technology to the Central Veterinary Research Laboratory (CVRL) of Dubai in the United Arab Emirates, thereby allowing the CVRL to offer the same adipose-derived adult stem cell animal therapies throughout the Middle East. Sheik Mohammed bin Rashid al-Maktoum, the ruler of Dubai and the Prime Minister of the UAE, is an avid thoroughbred owner and a sponsor of the Dubai World Cup, the world’s most highly-prized horse race. As Vet-Stem founding CEO Dr. Harman described in 2007, "The Central Veterinary Research Laboratory will be an excellent partner in bringing this technology from the U.S. to the Middle East as they are already the most respected reference lab in the region." CVRL now provides stem cell services for the treatment of injuries not only in thoroughbred race horses and Arabian endurance horses, but also in racing camels, among other species, throughout the Middle East.

It is actually incorrect, therefore, to claim that "there are few controlled studies clearly documenting the safety or efficacy of this treatment modality for tendonitis in horses", since more than 3,000 case studies have been documented by Vet-Stem alone, not counting the other case studies conducted around the world by other companies that are performing similar therapies in other countries. It is also incorrect to claim that "more research is needed regarding the optimal dose of stem cells and the use of gene enhancement techniques to augment the observed benefit before making this technology clinically available", since, actually, such technology is, in fact, already clinicallly available.

Given the consistent success of such therapies as those used by Vet-Stem, one might seriously question the wisdom and necessity of conducting "controlled studies" in which the horses must be killed at the end of the study in order to determine the efficacy of the medical therapy. Horses are not generally bred and maintained in the same manner as laboratory mice, although in this Cornell study, one might easily get the impression that the two animals are interchangeable.

Ordinarily, veterinarians, like all physicians, measure the efficacy of a therapy by the ability of the patient to recover – not by killing the patient in order to examine the mechanical, biochemical and histological properties of the tissue that was treated. Such an approach to medical therapy would be considered entirely unacceptable in humans, and even in horses the concept seems to lack basic horse sense.