Osiris Receives $5 Million Milestone Payment

Osiris Therapeutics announced today that it has received $5 million in payment for having reached the first production threshold of the Osteocel supply agreement with NuVasive. In a business deal that was valued at $85 million, Osiris sold Osteocel, its bone matrix product, to the San Diego-based spine specialization company NuVasive Inc. in July of 2008 for an initial payment of $35 million which is to be followed by further payments totalling an additional $50 million when milestones are reached.

According to NuVasive chairman and CEO Alex Lukianov, “Osteocel provides what we believe to be one of the more powerful biologic platforms on the market today.” Osiris CFO Richard Hunt adds, “It is through the hard work and dedication of our Osteocel team that we have achieved the first product supply milestone under the agreement and are solidly on track to hit the remaining milestones. With more than 15 years of rigorous stem cell research and development behind it, today Osteocel stands alone in orthopedics. Along with our excellent partners at NuVasive, we look forward to the product’s continued success.”

A leader in adult stem cell therapies, Osiris Therapeutics is focused on the development of products for the treatment of inflammatory, orthopedic and cardiovascular diseases. One of its proprietary adult stem cell products, Prochymal, is currently in Phase III clinical trials for the treatment of Graft-versus-host disease and Crohn’s disease, and is also being evaluated for the repair of heart attack damage, the protection of pancreatic beta islet cells in patients with type 1 diabetes, and for the treatment of chronic obstructive pulmonary disease. Another product, Chondrogen, is currently undergoing evaluation for the treatment of arthritis in the knee. With an extensive intellectual property portfolio containing 47 patents, Osiris has formed a partnership with Genzyme Corp. for the development and commercialization of Prochymal and Chondrogen in countries outside of the U.S. and Canada.

Scientists Regenerate Various Tissue Types by Stimulating the Body’s Own Stem Cells

In a series of experiments that are founded upon the success of Genzyme’s proprietary drug, Mozobil (see “Genzyme Receives FDA Approval for Mozobil”, December 15, 2008), scientists at the Imperial College of London have demonstrated the unsuspected regenerative potency of endogenous, autologous adult stem cells.

A novel, proprietary small-molecule CXCR4 chemokine receptor agonist, Mozobil is designed for use with G-CSF (granulocyte-colony stimulating factor) and has already received FDA marketing approval in the treatment of non-Hodgkin’s lymphoma and multiple myeloma. By stimulating the migration of hematopoietic stem cells from the bone marrow into the bloodstream for subsequent collection and autologous stem cell transplantation, Mozobil has already been shown to play an important role in the treatment of plasma and hematologic cancers. Now, however, researchers at the Imperial College of London have demonstrated for the first time that the regenerative power of autologous adult stem cells is not limited only to blood cells but also extends to the regeneration of other tissue, such as bone and blood vessels.

According to Dr. Sara Rankin, who led the study at the Imperial College of London, “It’s promoting self-healing. We’re simply boosting what’s going on naturally.”

The experiments were conducted with a mouse model in which Dr. Rankin and her colleagues selectively administered Mozobil in combination with either of the natural growth factors G-CSF or VEGF (vascular endothelial growth factor). When combined with VEGF, Mozobil was found to stimulate mesenchymal stem cells (MSCs) which differentiate into bone and other tissue types, as well as endothelial progenitor cells which differentiate into blood vessels. By contrast, G-CSF, which is known to stimulate the mobilization of hematopoietic stem cells, was found not to stimulate the production of MSCs. Similarly, VEGF was found not to stimulate hematopoietic stem cell mobilization. The degree to which stem cell mobilization increased was particularly dramatic, as mice that received VEGF in combination with Mozobil were found to release approximately 100 times as many endothelial and mesenchymal stem cells into the bloodsteram when compared to control mice who did not receive the treatment.

This is the first concrete evidence that different mechanisms of stimulation release different, specific types of stem cells. As Dr. Robert Lanza, chief scientist at Advanced Cell Technology in Worcestor, Massachusetts, explains, “The ability to selectively stimulate a patient’s own stem cells could be a powerful tool for treating disease, and in speeding up the repair of damaged and worn-out tissues.”

Genzyme, the manufacturer of Mozobil, received marketing approval last month from the FDA for the use of Mozobil in combination with G-CSF for the mobilization of hematopoietic stem cells in the treatment of non-Hodgkin’s lymphoma and multiple myeloma. According to John DiPersio, M.D., Ph.D., a professor at Washington University in St. Louis, “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.”

Professor Peter Weissberg, Medical Director at the British Heart Foundation, agrees. “It has long been known that the bone marrow contains cells that can replace lost or aged blood cells,” he explains. “It now seems increasingly likely that the bone marrow also contains cells that have the capacity to repair damaged internal organs, such as the heart and blood vessels, but that too few of them are released to be effective. This research has identified some important molecular pathways involved in mobilizing these cells. It may be possible to develop a drug that interacts with these pathways to encourage the right number and type of stem cells to enter the circulation and repair damage to the heart.”

Dr. Sara Rankin adds, “The body repairs itself all the time. However, when the damage is severe, there are limits to what it can do of its own accord. We hope that by releasing extra stem cells, as we were able to do in mice in our study, we could potentially call up extra numbers of whichever stem cells the body needs, in order to boost its ability to mend itself and accelerate the repair process. Mozobil is already out there on the market, so it’s feasible that trials could happen in the next five to ten years.”

Physical Exercise Stimulates Neural Stem Cells and Sharpens Cognitive Function

A familiar Latin adage attributed to the second century Roman poet Juvenal states: “Mens sana in corpore sano” (a healthy mind in a healthy body). Nearly 2 millennia later, scientists are still discovering new scientific proof of such timeless wisdom.

The leading Harvard Medical School associate clinical professor of psychiatry, Dr. John J. Ratey, would like you to know that physical exercise offers a number of neurological and even intellectual benefits. Of course, most people were probably already aware of such a claim, at least intuitively if not scientifically, although they may not have fully understood the precise mechanisms underlying such phenomena. In his new book, Dr. Ratey sheds light on the compelling science behind this important topic by elucidating the many ways in which physical activity stimulates various parts of the brain, including, among other components, the brain’s own endogenous stem cells. These stem cells, which naturally reside within the brain throughout life, even into the advanced decades of adulthood, are capable of being prompted and directed in their formation of new brain cells by external stimuli such as physical exercise. In “Spark: The Revolutionary New Science of Exercise and the Brain”, Dr. Ratey explores the connection not just between physical and mental health, but between specific types of physical exercise and cognitive acuity.

For years, neurophysiologists have already been studying the various factors, of both genetic and environmental origin, that influence the constant re-patterning of neural network connections which in turn provide the cellular basis upon which new information and experiences are processed. Now Dr. Ratey offers further depth and breadth of insight into the cellular and molecular processes of the brain, and into the essential role that physical exercise plays in catalyzing such mechanisms. As the title suggests, the resulting message is nothing short of “revolutionary” in conveying the absolutely critical importance of physical activity.

Among other benefits, Dr. Ratey explains, physical exercise improves the “fitness” of the neocortex, which in turn improves mental agility and mood as well as cognitive processes that require attention, alertness and motivation. But not all physical activity is created equal, and some types are more effective than others at accomplishing specific goals. For the greatest intellectual benefit, aerobic exercise in combination with “complex activity” has been found to maximize “brain power”. According to Dr. Ratey, “A fast-paced workout boosts the production of a protein called brain-derived neurotrophic factor. I call it Miracle-Gro for the brain, and physical activity is one of the best ways to release this brain-nourishing protein. A workout at the gym or a brisk walk also seems to build better connections between brain cells. Studies show that regular physical activity may increase the production of cells in the hippocampus, the region of the brain involved in learning and memory. The end result is a brain that’s better able to perform in school, at home or on the job.” Underlying the continuous replenishing of brain cells are none other than the brain’s own stem cells, which are stimulated into action by physical exercise and without which new brain cells and connections between neurons could not be formed.

Dr. Ratey adds, “Aside from elevating endorphins, exercise regulates all of the neurotransmitters targeted by antidepressants. It wakes up the brain and gets it going and improves self-esteem, which is one component of depression. Exercise also boosts dopamine, which improves mood and feelings of wellness. Studies have shown that chronic exercise increases dopamine storage in the brain. The process of getting fit is all about building up your aerobic base. The more you work your heart and lungs, the more efficient they become at delivering oxygen to your body and brain.”

As Dr. Ratey further explains, “We need to change the way we think about exercise. We really need to understand that exercise keeps the brain functioning well, and then realize that it also happens to be good for the body. We tend to think about it the other way around, but in fact it readies the cells in the brain to be optimal. We are made to move and people aren’t moving anymore.”

Devotees of the “mind-body medicine” that was so popular in the 1990s will be particularly interested in this book by Dr. Ratey, which advances the mind-body connection an order of magnitude further by examining with rigorous scientific objectivity the inseparability of physical health and cognitive performance. Even for those people who may not fall into the category of Olympic athletes, however, Dr. Ratey still offers hope by encouraging the reader to find motivation in the knowledge that even moderate exercise can sharpen memory and improve mental function.

Since the publication of Dr. Ratey’s book, Harvard Medical School has begun offering seminars on the subject through their Department of Continuing Education. Other universities and health conscious organizations are following the trend.

The book is coauthored with Eric Hagerman and published by Little, Brown & Company, 2008.

Genzyme on Track to Meet 2011 Financial Goal

With 2009 earnings that are estimated at $4.70 a share, and despite the current dismal global economic conditions, Genzyme has announced that it is on track to meet its goal of 20% compound earnings growth, or $7.00 growth per share, by 2011.

Unlike many companies whose stock has evaporated in recent months, Genzyme enjoyed a 13% growth in revenue which rose to $1.17 billion in the final 3 months of 2008. The company bases its projections of continued sustainable growth through 2011 upon strong sales of its proprietary drugs for rare and chronic diseases, for which Genzyme currently has more than a dozen novel therapies in its pipeline, several of which are expected to receive FDA approval in the coming year alone.

Speaking in Boston at the Goldman Sachs conference on healthcare, Genzyme’s CEO, Henri Termeer, expressed confidence that the company will continue to expand internationally, especially into the so-called “BRIC” countries, namely, Brazil, Russia, India and China. According to Termeer, “The key here is to bring other programs in to create growth drivers when we get there. I would suggest that any company that does not become competent in how to do business in those countries in a way that is sustainable will be missing an opportunity over the next 10 to 20 years.”

Genzyme’s stock closed today at $65.41 per share, up $1.29 from its previous close yesterday.

Cord Blood America Begins a Media Campaign via Social Networking Websites

One of the leaders in the growing field of the cryopreservation and banking of umbilical cord blood stem cells, Cord Blood America, is now poised to become a leader in the use of social networking websites. Realizing that the recent explosion in the popularity of such sites heralds the dawn of a new era of change and opportunity, not only in technology but also in business, Cord Blood America has announced its plans to seize such an opportunity. From now on, clients, shareholders and investors, both current and prospective, will be actively engaged with all the latest developments at Cord Blood America through social networking sites such as Twitter, Facebook and FriendFeed, among others. Cord Blood America intends to use the social networking sites to develop relationships and to build networks as well as to answer questions regarding their business and the basic science of stem cells.

In a statement issued by Cord Blood America, founding CEO Matthew Schissler offered the following invitation: “It’s time to start the conversation. The word ‘stem cells’ invokes many thoughts, yet we have found that the vast majority of the population still does not know what they are, where they come from or how they dramatically impact almost all aspects of life as we know it. To most, ‘stem cells’ is a scientific term that has no direct impact on the individual. It’s our belief that most people want to understand in a practical fashion how stem cells affect their family directly. This is the purpose of the social media, to deliver in a user-friendly fashion practical information about stem cells. We intend to look at the issue from multiple angles, including medically from the eyes of the patients and health care practitioners, fundamentally from the scientists, economically from the investor and business perspective, and socially from a public policy perspective. Social media is growing explosively and this trend, according to all authorities, is going to continue. We believe that building a network of clients and investors in two-way communication via social media will establish ourselves and our business as market leaders of the future. We invite you to join us in the conversation at the outlets previously mentioned. Additionally we have set up a social media newsroom at Intelligendo.com. We will leverage this newsroom and Intelligendo to further our relationship and network building efforts.”

Cord Blood America is the parent company of CorCell, an umbilical cord blood adult stem cell preservation company that services expectant parents and their children.

International Stem Cell Corporation Receives First Round of Funding

International Stem Cell Corporation (ISCO) announces the receipt of the first $1 million tranche of an anticipated $5 million in private equity financing that is to be spread out over the following months.

According to CEO Kenneth Aldrich, “This is a first major step toward the company’s goal of becoming financially self-sufficient.” Jeffry Janus, president and COO of ISCO, adds, “Although additional financing will be needed in 2009 to reach our goal, we believe our Lifeline subsidiary has the ability to bring the company to profitability within two years and support the clinical research of the company thereafter.” The company’s “Lifeline” subsidiary produces the particular stem cell lines in which the company specializes and from which the bulk of revenue is expected to be generated.

ISCO is the creator and developer of a new class of stem cells that are produced from its proprietary “parthenogenesis” technology, by which “parthenote cell lines” are obtained from unfertilized eggs that contain only the DNA of the donor and which therefore involve neither fertilized ova nor embryos. Parthenote cell lines are diploid and contain a full complement of genes and can be differentiated into all major cell types. As described on ISCO’s website, parthenote cell lines “display typical hESC (human embryonic stem cell) morphology, differentiate into all three embryonic germ layers and are immune matched (Major Histocompatability Complex matched) to the donors.”

ISCO’s parthenote cell lines have already been differentited into retinal cells, human corneal tissue, liver cells and heart cells, and work is currently in progress to differentiate the the cells into pancreatic beta islet cells.

Anthrogenesis Receives Another Patent for Placental Stem Cell Innovation

In a patent application that Anthrogenesis filed on June 22, 2004, materials and methods are detailed at length for the extraction, recovery, isolation, propagation, collection and utilization of “embryonic-like stem cells, including but not limited to pluripotent or multipotent stem cells from an exsanguinated human placenta”. Further described within the application is “an isolated human placental stem cell” with the ability to differentiate into cells of neural, osteogenic, and chondrogenic phenotypes, among others.

Although it is certainly not the first and will probably not be the last, this is the most recent patent to be awarded to Anthrogenesis Corporation for its placental stem cell technology.

Anthrogenesis was acquired by Celgene in 2003, and operates as a wholly owned subsidiary of Celgene. Founded in 1986, Celgene has grown to a multinational biopharmaceutical company that employs approximately 1,600 people around the world.

Located in New Jersey, Anthrogenesis Corporation is a recognized leader in pioneering the technological means and methods for the derivation and commercialization of large quantities of adult stem cells that are found in human placental tissue. According to John Jackson, chairman and CEO of Celgene, “Placental stem cells have the potential to transform the way physicians treat serious diseases and the way novel therapies are developed.”

Apparently, however, the news of this latest patent has not yet reached Wall Street, as Celgene’s stock closed at $53.48 today, down 24 cents from its close yesterday.

U.S. Doctor Plans Stem Cell Fact-Finding Mission to China

Ophthalmologist David Klein has accepted an invitation to travel to the People’s Republic of China in the hopes of learning first-hand about human stem cell therapies that are currently being conducted outside of U.S. borders. In particular, Dr. Klein will focus specifically on medical advances that have been made with adult stem cells that are derived from umbilical cord blood.

As Dr. Klein explains, “A lot of Americans assume that if it’s being done overseas, they’re experimenting on human beings. I’m going into it with an open mind.”

Dr. Klein first became interested in learning about stem cell therapies when several of his patients returned to the U.S. after having traveled overseas to receive stem cell treatments that have not yet received FDA approval to be conducted within the United States. Dr. Klein became especially interested after observing the progress of a 2-year-old patient who had suffered from optic nerve hypoplasia, which is a leading cause of blindness in children, and for which American doctors told the parents that no treatment exists. The family researched other options, however, and in 2007 they traveled to China where they visited a clinic that uses adult stem cells derived from umbilical cord blood. The therapy consisted of 4 infusions of the stem cells into the child’s arm and lumbar region of the spine, and by the third treatment the child was able to see well enough to stand on his own and crawl toward objects, which he had previously been unable to do. By the fourth treatment, the child could see objects at a distance of ten feet away and began attending school. According to his grandmother, the boy “has made huge improvements. There was nothing for him before this treatment. Now, his world is limitless.”

Stem cell “tourism” is a thriving industry, in which increasing numbers of people are flocking to foreign clinics overseas in search of therapies that are not yet legal within the United States. Precisely for this reason, one of the main criticisms against the U.S. FDA (Food and Drug Administration) is that its laws are outdated and no longer apply to new medical technologies such as those that require approval for the use of stem cell therapies. Before any new therapy can become legal within the U.S., it must undergo rigorous scientific and clinical scrutinization that can take a decade or longer and cost the company that is developing the therapy hundreds of millions of dollars. While no one questions the importance and necessity of such testing for safety and efficacy, many scientists do question the lethargic pace at which such testing is often conducted. Increasingly, there are many patients with life-threatening illnesses and injuries who cannot afford to wait a decade or longer to receive a treatment that could literally make the difference between life or death. When such people are forced to travel outside of their home countries in search of any therapy that offers any hope at all, even false hope, the dangers to which these patients are exposed are potentially greater than those that they would face without any treatment whatsoever. Although there do exist a number of scientifically reputable and legitimate stem cell clinics outside of the U.S., these clinics are often competing against the many disreputable and illigitimate “clinics” that offer only bogus, illicit “therapies”, and it is not always easy for the average person to distinguish between these two types of clinics. What is urgently needed now more than ever is an updated overhaul of the U.S. FDA regulatory system, so that those therapeutic stem cell methods which are safe and effective can be made legally available within the U.S. in as timely a manner as possible, while at the same time the laws must also be updated to protect patients from medical treatments that are not verifiable as safe and effective. If such a revision of the FDA approval process were to occur, the direction of stem cell “tourism” might be reversed, with many patients throughout the world traveling to the U.S. in order to benefit from sound medical treatment, rather than so many U.S. citizens having no other option but to travel outside of the U.S. in search of any glimmer of hope.

Meanwhile, Dr. Klein is looking forward to his fact-finding mission to China. Although he has described himself as “cautiously optimistic” about stem cell therapy, he wisely adds that “I want to make sure there’s good science behind it.”

His trip is planned for 2009.

Neuralstem Files FDA Application for ALS Adult Stem Cell Trial

Neuralstem Inc. announced this morning that it has filed an IND (investigational new drug) application with the U.S. FDA (Food and Drug Administration) to begin the first ever human clinical trials for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, with adult stem cells.

According to Richard Garr, J.D., president and CEO of Neuralstem, “Like all first human trials, this proposed trial is primarily designed to test the safety and feasibility of both our stem cells and our method of delivering the cells to the spinal cord in ALS patients. We are also proposing secondary endpoints which we hope will be able to measure a slowing down of the degenerative process.”

The treatment will consist of spinal injections of the adult stem cells using the company’s patented and proprietary technology. The clinical trials will take place at Emory University under the direction of Dr. Johnathan Glass, Director of the Emory Neuromuscular Laboratory and Director of the Emory ALS Center, pending offical FDA authorization.

Approximately 30,000 people in the U.S. alone are afflicted with ALS, which is a progressive neurodegenerative disease in which the deterioration of nerve cells and motor neurons progresses from a loss of muscular control to paralysis and ultimately death. Conventional medical modalities have thus far been unsuccessful in halting or reversing the course of the disease, which until now has been considered incurable. Adult stem cell therapies, however, constitute the first type of treatment that is actually capable of offering realistically achievable improvement.

According to Richard Garr, “The filing of this IND is an important event for Neuralstem, but it marks only the beginning of a process which includes working together with the FDA to approve the first human ALS stem cell trial, refining our understanding of how to optimize delivery of our cells into patients, and ultimately delivering a new treatment for patients with this currently incurable disease.”

As described on their website, “Neuralstem, Inc. is a publicly traded biotherapeutics company whose mission is to apply stem cell research and its patented human neural stem cell technology to treat diseases of the central nervous system including ischemic paraplegia, traumatic spinal cord injury, ALS and Parkinson’s disease. Our stem cell research has resulted in patent-protected technology that allows us to produce mature, commercial quantities of neural stem cells with the ability to control the differentiation of the cells into physiologically relevant human neurons and glia.”

Adult Bone Marrow Stem Cells Treat Heart Failure in an Infant

Doctors in Germany have reported improvement in the cardiac function of a 2-year-old child who was critically ill with severe heart failure caused by dilated cardiomyopathy (DCM), and who improved after receiving autologous adult stem cell therapy.

Led by Dr. Stefan Rupp of the Pediatric Heart Center at Justus-Liebig University in Giessen, Germany, the team of physicians conducted an intercoronary administration of autologous adult progenitor cells derived from the infant’s own bone marrow. As described in their article, “DCM is the most common cardiomyopathy in childhood. Effectiveness of anticongestive therapy is limited in most cases and about one-third of children diagnosed with DCM die or receive heart transplantation within the first year after diagnosis.”

This case documents the first instance of an autologous adult stem cell treatment administered to a patient this young, whose condition was progressively worsening despite maximal anticongestive therapy prior to receiving the autologous adult stem cells, and who has consequently shown measurable improvement as a result of the autologous adult stem cells.

As the authors conclude, “Cardiac stem cell therapy proved to be technically feasible, was associated with improvement in cardiac function, and might represent an option before heart transplantation in children with severe heart failure.”