Researchers at Northwestern Memorial Hospital in Chicago have announced positive results from the use of autologous adult stem cells in the treatment of heart damage. Led by Dr. Douglas Losordo, the FDA-approved, Phase II clinical trial is testing both the efficacy and safety of mesenchymal stem cells (MSCs) that are derived from each patient’s own peripheral blood. Results of the study are expected to be presented in September.

In the study, treatment involves the use of CD34+ cells which are harvested from each patient’s own blood, purified, expanded and administered to the patient by injection directly into the damaged heart muscle. Since CD34+ cells are capable of angiogenesis, which is the formation of new blood vessels, the treatment is expected not only to regenerate new heart muscle but also to prevent the formation of scar tissue. Sponsored by Baxter, Inc., the study is currently the largest adult stem cell study for heart disease that is being conducted in the United States.

As Dr. Losordo explains, "It’s important to point out that this is a use of a patient’s own body repair capabilities." In other words, there is no risk of immune rejection, nor are there any ethical controversies surrounding the source of the stem cells, which are strictly autologous (in which the donor and recipient are the same person) adult stem cells, not embryonic stem cells.

GCSF (granulocyte colony-stimulating factor) is used in conjunction with the autologous adult stem cell therapy, to increase stem cell mobilization and migration from the bone marrow into the peripheral blood. Thus far, no side effects have been observed.

According to Dr. Jeffrey Karp of Harvard University, who is collaborating with Dr. Te Chung Lee at the State University of New York at Buffalo on similar studies, "Essentially if we know the zip code of vessels within a certain tissue, we can program the address on the surface of the cell." Speaking metaphorically, in reference to the "homing" ability of adult stem cells to target and repair damaged tissue, Dr. Karp is referring to the specific properties of mesenchymal stem cells (MSCs), which he and Dr. Lee have used in a number of related studies.

Dr. Losordo believes that the treatment could be made widely available in approximately 4 years, with a target patient population that consists of end-stage cardiac patients who have unsuccessfully exhausted all other available therapies – a population that has been estimated to consist of anywhere from one-third of a million to a million people. Dr. Lee, however, believes it could take as long as 5 years for such a treatment to become widely available, while Dr. Karp believes it could take as long as ten years. In any case, As Dr. Ronald Crystal, chief of the Division of Pulomary and Critical Care Medicine at New York Presbyterian Hospital/Weill Cornell Medical Center, adds, "This is a good idea, but patients and families should not expect immediate results."

An official FDA-approved clinical trial is currently underway at the School of Medicine and Public Health at the University of Wisconsin at Madison, for the use of adult stem cells in the treatment of Type 1 diabetes. Specifically, the Phase II study is testing both the safety and efficacy of Prochymal, a proprietary mesenchymal stem cell (MSC) formulation manufactured by the company Osiris Therapeutics.

The Phase II, randomized, double-blind, placebo controlled, parallel assignment, multi-center study is being concurrently conducted at 20 medical centers nationally. The study has a target enrollment of 60 newly diagnosed patients between the ages of 18 and 30. Patient monitoring will be conducted during a follow-up period lasting two years. The study is co-sponsored jointly by Osiris Therapeutics and the Juvenile Diabetes Research Foundation.

John Markwardt, a 20-year-old UW student who was just diagnosed with Type 1 diabetes in the spring of this year, became the first patient to be treated with Prochymal in the study. His first infusion took place in July, and the second infusion is scheduled for later this month. The study is specifically targetted at newly-diagnosed Type 1 diabetes patients, in whom the pancreatic beta cells have not yet been completely destroyed.

According to Dr. Melissa Meredith, associate professor of endocrinology at UW and the principal investigator of the study, "The reason we have focused on people recently diagnosed is they still have some beta cells capable of making insulin. If we can stop that immune destruction, they still have the ability to make some insulin. We also know beta cells have the ability to regenerate. Even if they aren’t totally off insulin, it’s a better way to control the disease when they aren’t reliant on injected insulin and are making more of it themselves. The reason I am excited about this is that it is the heart of what causes diabetes. The hope is it will preserve the ability that is left."

Also collaborating with Dr. Meredith in the study is Dr. Peiman Hermatti, assistant professor of hematology and oncology at UW, who has previously used bone-marrow-derived MSCs in the treatment of graft-versus-host disease.

According to the website of the National Institutes of Health (NIH), "The objective of the present study is to establish the safety and efficacy of multiple administrations of Prochymal in subjects recently diagnosed with Type 1 diabetes mellitus." As Dr. Paul Strumph, chief medical officer of the Juvenile Diabetes Research Foundation, adds, "This therapy is aimed at decreasing the immune response, which we know is involved in the progression of Type 1 diabetes. It’s very promising."

As further described on the website of NIH, this study utilizes an "intravenous infusion of ex vivo cultured adult human mesenchymal stem cells" (MSCs). As described on the website of Osiris Therapeutics, Prochymal consists of a proprietary formulation of human MSCs that "are derived from bone marrow aspirate obtained from qualified donors. … Upon arrival at Osiris, the MSCs are isolated and selectively removed from the bone marrow aspirate. These cells are then expanded, harvested, packaged and cryopreserved as an in-process intermediate, where a second series of tests ensure the highest level of quality and safety. … All manufacturing activities are performed in compliance with the Food and Drug Administration’s current Good Manufacturing Practices (cGMP) standards… Osiris Therapeutics, Inc., adheres to strict FDA regulations in the production and formulation of our stem cell treatments to ensure that our products are of the highest quality and safety."

Often featured in the news, Osiris Therapeutics is a prominent leader in the field of adult stem cell therapies and is focused on the development of products for the treatment of inflammatory, orthopedic and cardiovascular diseases. Osiris was founded in 1992 for the specific purpose of developing and commercializing the innovative technology for isolating and expanding bone-marrow-derived MSCs, a process originally pioneered by Dr. Arnold Caplan and his colleagues of Case Western Reserve University. Since MSCs are "immune privileged", "universal donor" cells, they do not need to be administered autologously (in which the donor and recipient are the same person), since even allogeneically (in which the donor and recipient are not the same person) these highly potent adult stem cells do not pose any risk of immune rejection. In addition to other products in its pipeline, Osiris currently has a number of clinical trials under way for both Prochymal and its other leading adult stem cell product, Chondrogen.

In November of last year, Osiris formed a strategic alliance with the biotech company Genzyme that was valued at over $1.3 billion. In 2007, the two companies were awarded a $224.7 million contract from the U.S. Department of Defense for the development of Prochymal in the treatment of radiation sickness.

As Dr. Hans Klingemann of Tufts Medical Center explains, "MSCs have the ability to interpret injury within the body and respond accordingly, giving them exceptional therapeutic potential to treat a variety of disorders. And, they can be administered quickly, where and when patients need them."

Formerly known as juvenile diabetes or insulin-dependent diabetes, Type 1 diabetes is a chronic, life-threatening condition for which conventional medicine has no known cure. Though not as common as Type 2 diabetes, Type 1 diabetes is characterized by the autoimmune destruction of the beta islet cells of the pancreas, which are required to produce insulin. Left untreated, both types of diabetes can lead to serious complications such as stroke, blindness, heart disease and peripheral neuropathy which in its most advanced form is treatable only by amputation. Together, Types 1 and 2 diabetes constitute the seventh most prevalent cause of death in the United States.

Now, for the first time, adult stem cell therapy may offer the first type of treatment which not only reverses the course of the disease but also regenerates damaged pancreatic tissue.

This FDA-approved clinical trial is currently still recruiting patients. Interested parties should contact Osiris Therapeutics directly for eligibility information.

The biopharmaceutical company Athersys is looking for commercial partners in the R&D of its various therapies, which include conventional drug therapies as well as some novel stem cell therapies.

According to Athersys chairman and CEO, Dr. Gil Van Bokkelen, who spoke to analysts during a company conference call, Athersys currently has the cash equivalents of $25.3 million, which is enough to keep the company operating through 2011. Nevertheless, the company would like to add to its capital base by partnering with other companies in the completion of the testing and development of therapeutic product lines.

The main product in the company’s stem cell line, known as MultiStem, is an off-the-shelf therapy that Athersys is currently testing for the treatment of heart attack, stroke, and graft-versus-host disease. As Dr. Van Bokkelan explained during the conference call, "We believe these and other capabilities will allow them to be relevant in a number of therapeutic areas, and we and our collaborators are excited about the potential utility of MultiStem for treating major conditions." Unlike many other types of stem cell therapies, which are designed "to achieve wholesale replacement of damaged tissue", Dr. Van Bokkelan described MultiStem as exhibiting "a more drug-like profile in which cells augment healing and exert a benefit in multiple ways, and then are cleared from the body over time." He added, however, that the wide range of applications for MultiStem "represent a development challenge, especially for an emerging biopharmaceutical company," which is why Athersys has decided to seek commercial partnerships. As Dr. Van Bokkelan further adds, "Accordingly, we believe that the optimal way to advance our MultiStem program is to find a significant commercial partner."

Under Dr. Van Bokkelan’s leadership, Athersys has been exploring partnership opportunities for months and is "greatly encouraged by the ongoing level of interest among potential partners." As he further describes, "We are confident that we will be able to secure strategic partnerships that will enable us to advance our MultiStem programs in a meaningful and substantial way."

Located in Cleveland, Ohio, Athersys is also currently developing two separate drug candidates for the treatment of obesisty and problems in cognitive attention, both of which deal with the chemical regulation and control of neuroreceptors. Even in this field, Dr. Van Bokkelan finds that, "Once again, we are greatly encouraged by the level of interest among potential partners in both areas, and believe we are in a strong position to construct relationships with leading companies that share our vision of developing best-in-class therapeutics."

As described on their website, "Athersys is a clinical stage biopharmaceutical company with a growing pipeline of highly differentiated, potential best-in-class therapeutics to treat significant and life-threatening diseases. … Our current product development portfolio consists of MultiStem, a patented and proprietary stem cell product that we are developing as a treatment for multiple disease indications, and that is currently being evaluated in two Phase I clinical trials. In addition, we are developing novel pharmaceuticals to treat indications such as obesity, as well as certain conditions that affect cognition, attention, and wakefulness. Our strategic approach to drug development builds on internally and externally generated knowledge to identify and develop proprietary and highly differentiated products, as well as enable the company to limit development risks and costs."

As further described on the website of Athersys, "MultiStem is a biologic product that is manufactured from human stem cells obtained from adult bone marrow or other nonembryonic tissue sources. The product consists of a special class of human stem cells that have the ability to express a range of therapeutically relevant proteins and other factors, as well as form multiple cell types. Factors expressed by MultiStem have the potential to deliver a therapeutic benefit in several ways, such as the reduction of inflammation, protection of damaged or injured tissue, and the formation of new blood vessels in regions of ischemic injury. These cells exhibit a drug-like profile in that they act primarily through the production of factors that regulate the immune system, protect damaged or injured cells, promote tissue repair and healing and most or all of the cells are cleared from the body over time."

One of the co-founders of Athersys, Dr. Van Bokkelan has served as CEO of the company since its founding, as well as the president of Athersys prior to 2006. Dr. Van Bokkelan holds a double B.A. in Economics and Molecular Biology from the University of California at Berkeley, and a Ph.D. in Genetics from Stanford.

Cord Blood America Inc. (CBAI), one of the largest providers in the world of umbilical cord blood stem cell preservation, reviews its accomplishments thus far in 2009. Even though the year is only half over, CBAI considers its achievements thus far in 2009 to be among the most significant in the company’s history. Some of its accomplishments in the first half of this year are described herein.

In January: The company launched its first social media campaign in the industry to start a public conversation about the potential usefulness of adult stem cells and the importance of storing umbilical cord blood. Additionally, the company established its "3 pillars of success" for 2009, which are, namely, organic growth, acquisitions, and debt reduction for an improvement in the company’s balance sheet.

In February: CBAI was listed on the ETF Innovators Stem Cell 40 company index. Additionally, CBAI announced $1 million in long-term debt reduction as part of its cash-flow restructuring.

In March: CBAI was noted as the top stock performer on ETF Innovators Stem Cell index, with a year-to-date stock price increase of 279% at that time.

In April: Congress introduced legislation to promote the "Family Banking of Umbilical Cord Blood" stem cells, which is a major boost to the entire cord blood banking industry. Additionally, CBAI received its first customer in Germany and announced a long-term debt reduction of over $2.5 million.

In May: CBAI announced that it had received $2.3 million for the purpose of building its own stem cell processing and storage laboratory, rather than continuing to outsource the services, which thereby resulted in a further debt reduction of $4.2 million.

In June: CBAI announced further expansion throughout Europe as well as independent contracting with affilates in the Caribbean and Central America.

In July: CBAI has further secured $7.5 in long-term equity financing for acquisition and growth opportunities, while also hiring a separate management team to direct its new stem cell laboratories in the U.S., which will be among the largest in the country, construction on which has already begun.

According to Matthew Schissler, chairman and CEO of CBAI, "On January 6, 2009, the closing CBAI stock price was $0.0017. On July 10, 2009, the closing stock price was $0.0033, a 94% increase in 6 months of trading. That’s not where we want to be, we are still very undervalued, and I understand that the day-to-day swings of our very liquid and volatile stock price cause some investor concern. I tell investors to take a snapshot of the last 6 months, not six hours. Micro cap stocks are perhaps not for the faint-of-heart. We believe we’re building a very valuable company and this snapshot of the past 6 months shows our progress."

Additionally, Mr. Schissler adds, "In January, CBAI stated its mission is to lay the foundation for a much stronger, healthier company by the end of 2009, which included organic growth, acquisition and debt reduction. As you can see by these highlights, we remain laser-focused on executing these strategies. Because of our focus, we are able to raise significant funding to build a lab with the future growth opportunities it affords us. We are also pleased by the current support of stem cell research in Washington, D.C. and Congressional and state legislative focus on family stem cell banking legislation."

CBAI is the parent company of Cord Partners, which was founded in 2002 and became operational on January 1, 2003. According to the company’s website, "Cord Partners, a Cord Blood America company, has established itself as a platinum standard national competitor in the umbilical cord blood storage industry… CBAI was founded on a simple principle: The families who seek stem cell preservation should never have to work to achieve this. By bundling such services as medical kit preparation, medical courier arrangement, 24/7 customer service, easy enrollment, and one price for all services, we feel an unparalleled commitment to our clinets, and have the foundation for providing the highest quality stem cell preservation available."

Umbilical cord blood is an extremely rich source of highly potent adult stem cells, such as mesenchymal stem cells and CD34+ cells, which have already been in use for decades in the clinical treatment of various diseases, safely and without side effects. Unlike embryonic stem cells, adult stem cells derived from umbilical cord blood are ethically noncontroversial, as umbilical cord blood is easily and harmlessly collectable.

Researchers at the Mayo Clinic in Rochester, Minnesota, have used induced pluripotent stem (iPS) cells to treat damaged cardiac tissue in a mouse model of heart attack. The iPS cells were found to repair the heart tissue both structurally and functionally.

According to Timothy Nelson, M.D., Ph.D., one of the lead investigators of the research, "This study establishes the real potential for using iPS cells in cardiac treatment. Bioengineered fibroblasts acquired the capacity to repair and regenerate infarcted hearts."

Previously, iPS cells had been tested in animal models for three other diseases, namely, Parkinson’s disease, sickle cell anemia, and hemophilia A. This new study is the first of its kind in which iPS cells were tested specifically for their ability to regenerate cardiac tissue.

As Dr. Nelson further explains, "We’re taking advantage of a diseased tissue environment that is sending out a distress signal that is asking the tissue to repair itself. When we put these iPS cells in, they are able to respond. They were able to respond to this damaged environment and spontaneously give rise to the appropriate tissues and create new tissues within that diseased heart. That is a key ‘wow factor’ of this paper. It was obvious to the observer which animals had been treated and which ones hadn’t."

The scientists used fibroblasts which they genetically reprogrammed to dedifferentiate into iPS cells which were then redifferentiated into heart tissue. When transplanted directly into the damaged hearts of the mice, the new cells were found to have engrafted within two weeks of transplantation, and by 4 weeks the transplanted cells were found to have contributed to improved structure and function of the hearts. Not only did the iPS cells halt the progression of structural damage within the heart muscle itself, but the iPS cells were also found to regenerate new cardiac tissue which included not only heart muscle but also blood vessels. Ultrasound also revealed significant improvement in the ability of the hearts to pump blood.

According to Adre Terzic, M.D., Ph.D., Mayo Clinic physician and senior author of the study, "This iPS innovation lays the groundwork for translational applications. Through advances in nuclear programming, we should be able to reverse the fate of adult cells and customize ‘on demand’ cardiovascular regenerative medicine."

However, the researchers still used dangerous viral vectors for dedifferentiation of the fibroblasts into iPS cells, even though other scientists have replaced such methods with less dangerous reprogramming techniques.

Nevertheless, according to Dr. Nelson, he believes that iPS technology will be able to be used in clinical therapies to help people, eventually, even though it might take "several years". Until then, iPS cells are still ineligible for clinical therapeutic use since there are still a number of biological hurdles remaining that have yet to be overcome.

Meanwhile, autologous adult stem cells have already been used therapeutically in the clinical treatment of heart and other conditions, and are already available in clinics around the world – except, that is, in the U.S., where outdated FDA regulations have been unable to keep pace with scientific progress.