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Small Companies Hubs of Future Profit in Regenerative Medicine

Companies / BioTech Mar 03, 2013 - 05:16 PM GMT

By: TLSReport



Gil Van Bokkelen has been in the stem cell industry for more than a decade. In recent years, the co-founder, chairman and chief executive officer of Athersys Inc. has stepped into regenerative medicine’s forefront, acting as a spokesman for the industry as well as demonstrating his love of science and passionately advocating for patients. In this interview with The Life Sciences Report, Van Bokkelen explores the past, present and future of the cell therapy and regenerative medicine industry, and outlines the investment possibilities in small companies on the sector's cutting edge.


The Life Sciences Report: Gil, you serve on numerous boards, and you are the former chairman of the Alliance for Regenerative Medicine (ARM). You're also current chairman of the board of governors of the National Center for Regenerative Medicine (NCRM). Are these lobbying organizations? What do they do?


Gil Van Bokkelen: The National Center for Regenerative Medicine, which is based in Ohio, is not a lobbying organization. It is a collection of institutions focused on translational research in regenerative medicine. Put simply, these institutions, like the Cleveland Clinic, University Hospitals Case Western Reserve University, Ohio State University and member affiliates including Athersys Inc. (ATHX:NASDAQ), Juventas Therapeutics Inc. (private) and others, are focused on trying to advance the science and clinical practice of regenerative medicine therapies. Originally founded in 2003 as the Center for Stem Cell and Regenerative Medicine with a substantial grant from the state of Ohio, the organization was designated the NCRM in 2005 by Congress.


When the effort was first initiated more than a decade ago, we recognized that concerted cooperation and joint effort was needed to address some of the bottlenecks that impede translational activity. You can imagine that the field of cell therapy and regenerative medicine had some unique complexities associated with it, including conducting preclinical studies where investigators used human cells and evaluated their safety and efficacy in animal models, as well as actual clinical application of various types of therapies in human patient populations. It is not as simple as giving somebody a pill. The investigators dealing with these types of studies have to be aware of the complexities. The organization was originally formed to help satisfy that need.


The Alliance for Regenerative Medicine was formed in 2009. It is a lobbying group, but really it is much more than that. Both large and small companies, and a couple of large pharmas, were among the founding group of members—Pfizer Inc. (PFE:NYSE), Johnson & Johnson (JNJ:NYSE). Leading disease foundations and patient groups were also among the founders, as were some clinical institutions—the Cleveland Clinic, again, was involved from the very beginning, as well as the Juvenile Diabetes Research Foundation. In contrast to the broader biotechnology industry, which is well represented by the Biotechnology Industry Organization (BIO), where I have served on the board for a number of years, and the pharmaceutical industry (which is represented by the Pharmaceutical Research and Manufacturers of America [PhRMA]), when the cell therapy and regenerative medicine industry started up, nobody directly represented us or oversaw how we interacted with the U.S. Food and Drug Administration (FDA) and other regulators and parts of the federal government.


TLSR: So then, you are saying that ARM is an advocacy organization?


GVB: Yes, that's exactly right. ARM is about advocacy.


TLSR: If we compare cell technologies to small molecules, which represent a 7,000–10,000-year-old industry, or to antibodies, still brand new as a 20-year old industry, where do regenerative medicine and cell technologies fall on the timeline of development?


GVB: I see a lot of parallels, as your question suggests, in terms of what is going on in cell therapy now and what was going on a few years ago in the field of therapeutic antibody development. Small molecules, as you correctly noted, have been around for a long, long time. The interesting thing about the traditional small molecule, pharmaceutical-based approach is that those drugs bind to a particular protein/receptor and may have one specific activity. It is the same with antibodies, which also bind to a specific target protein/antigen to perform a specific function. Cell therapy is more complex, because cells can do multiple things in parallel that act in concert to enhance healing and repair.


In terms of the timeline, I would say that the cell therapy industry is where the field of antibodies was when it emerged from the dark, and the real value creation began. The parallels continue, as there was much excitement about antibodies in the early days, when researchers thought they could use mouse cells to produce antibodies that could be used in human patients to cure cancer or some other disease. What they learned early on was that it is just not that simple. Investigators became aware of the fact that if they used a mouse-derived antibody in a human patient, it might elicit an immune response, or what researchers refer to as a HAMA (human anti-mouse antibody) response.


The field of antibodies started to take off when researchers began to make humanized forms of antibodies that were much more reflective of what the body needs, and could have a dramatic, therapeutic effect. In fact, if you look at some of the most successful therapies on the market today, a meaningful number are therapeutic antibodies.


Cell therapy has undergone the same kind of iterative learning process. We are showing tremendous progress with various forms of cell therapy versus a decade ago, but that hasn't yet fully translated into the type of clinical experience and results that most of us have been anticipating for a long time. However, now we have a growing number of programs in mid- and late-stage clinical development that cut across a broad range of therapeutic areas. And, perhaps even more to the point, we have seen a few cell therapies progress all the way through the clinical development process: They are on the market and doing good for patients who really need help. An example would be a product like Apligraf, which has been developed by Organogenesis Inc. (private) to treat chronic diabetic or venous leg ulcers. Advanced BioHealing, which was acquired by Shire Plc (SHPGY:NASDAQ; SHP:LSE), has a very similar platform with its product Dermagraft. Other products or therapies have been developed that are providing relief and help to patients in orthopedic indications and a growing number of other settings. But I think we've really just scratched the surface.


TLSR: So we are close, is that what you are saying?


GVB: I believe, over the next three to five years, we are going to see enormous growth and progress in treatments that will deliver on the promise of cell therapy, and these will do a lot of good for patients. Right now, the field of cell therapy generates roughly $1 billion ($1B) in revenue annually. We see more product approvals, and that's basically because there are a limited number of them. But frankly, I think this number is going to grow substantially, and as it does I believe we are going to see rapid and explosive growth. More therapies will be validated and move forward through the regulatory pathway, get approval, get reimbursement and ultimately reach patients.


TLSR: The popular model of cell therapies is that of rebuilding tissues and organs. But there's much more to it, isn't there?


GVB: The interesting thing about cells is that they are multimodal. A decade ago, researchers thought that we would simply use cells to replace what has already been lost. In fact, there is an enormous amount of evidence suggesting that different cell therapy approaches can express factors that promote healing and tissue repair, and enhance patient recovery in very powerful ways. These cells can be delivery systems that are finely tuned to the needs of the body. They actually accomplish some amazing things, such as interacting with the body and regulating other cell types by expressing multiple different factors. It is not always about directly replacing what has been lost.


TLSR: You seem to be making the case for regenerative therapy not building new tissues with stem cells as building blocks, but rather utilizing a paracrine-signaling effect, for lack of a better term, where the cells react dynamically with the patient. Do you believe that this druglike model is going to be the biggest area of cell therapy?


GVB: I think it is going to play a big role. Let me be clear. I am not saying that cell therapy can't have a profound role in helping to replace damaged or injured tissue. We are already seeing some pretty exciting examples of that, which could benefit patients who are suffering from spinal cord injury. For instance, StemCells Inc. (STEM:NASDAQ) is starting to generate some very encouraging but still very early data using its human neural stem cell candidate in patients with spinal cord injuries. If the company successfully extends that work with additional trials, then investors and patients could see pretty exciting things come to fruition.


But I do believe, to answer your question, that the paracrine effects of stem cells, meaning their ability to express factors directly in response to significant tissue damage and resulting inflammation, has emerged over the past decade as a major component of the therapeutic effectiveness of cell-based therapies.


There is the potential to accomplish both endpoints. Administering cells can be a dynamic, druglike event, where the cells don't permanently engraft but are around for days to weeks to help with tissue repair and healing, then clear the body as would a drug or traditional biologic. Then there is the potential to actually augment, replace or regenerate certain types of tissue. These are an incredibly powerful set of capabilities that, frankly, you could never reasonably expect to achieve using traditional, pharmaceutical-based approaches in most areas.


TLSR: You mentioned StemCells Inc., which has demonstrated durable engraftment of adult or differentiated stem cells of embryonic origin in the brains of patients with lysosomal storage disorders. This has been proven on autopsy by determination that an engrafted donor neuron was of a different genotype than that of the recipient patient. However, with all this exciting technology, StemCells Inc. has a $75M market cap. Neuralstem Inc. (CUR:NYSE.MKT), with an $81M market cap also has a validated technology, as does Athersys, your company, which has a $91M market cap. Why has this industry been so slow to take off with investors?


GVB: Part of it is that the industry is complicated. I think a lot of investors, frankly, don't really understand it. Institutional investors are asking, "Show me the evidence. How does it work? Show me that it's safe, and show me that there are big pharmaceutical companies that might be interested in this area." Historically, this is what institutional investors need to know about a drug.


Of course, now there is that evidence. We at Athersys have a partnership with Pfizer for inflammatory bowel disease. Other big companies have declared their commitments to the space and made meaningful investments. What you've seen over the past several years, in particular, is more evidence that this is a legitimate area of opportunity that big companies are interested in. Many investors tend to shy away from the things that they perceive as unproven or risky until that point when the data becomes overwhelming.


Another issue is that investors feel it is going to be a long haul. There is a lot of work to do between early-stage development and actually showing that a therapy works, and then getting the therapy into patients in a scalable and reproducible way. I've seen a lot of signs over the past year—and going back even earlier—that a certain segment of the investment community is beginning to see the potential and recognize what the upside is, and that it may not be that far off. That's important because investor capital is what drives advancement of the field.


Getting to your question, the stem cell field is unquestionably undervalued right now. But I think that will change as more evidence comes to light. It's going to be based on clinical data and tangible partnerships, which are what I think will be the biggest drivers.


TLSR: This cell therapy and regenerative medicine industry is still brand new. Isn't it hard to expect investors to shoulder the burden of developing the first therapies in a brand new industry where development can take decades?


GVB: It takes patience. One of the things I always explain to investors is that stem cell therapy development is a marathon. It's not a sprint. It takes time. We need to take a step back and look at it from a macro perspective. There are stumbles and disappointments that happen along the way. We've experienced some of those ourselves at Athersys, but I have seen some pretty amazing things happen too.


There are some historical examples. For example, look what Amgen Inc. (AMGN:NASDAQ) went through before it finally got its first product, Epogen, through development and approval, and things really began to happen for the company. Another example would be Celgene Corp. (CELG:NASDAQ), which spent many years focused on and pursuing therapies that people were incredibly skeptical of. And yet finally it broke through and was able to show that a drug that basically had been written off by everyone else as a disaster, thalidomide, could provide meaningful benefit to patients being treated for certain types of cancer. Frankly, I could give you a whole laundry list of companies like that.


TLSR: Let me switch to another side of regenerative medicine. There are many services and tools needed, such as instrumentation, to administer cells to patients. There are also adjunctive areas, such as processing, management, handling and testing of cells. On the regenerative side there would be the creation of tissue, such as tubes and organs, with ink jet-style or three-dimensional (3-D) printers. My question is, does each cell technology company have to develop its own platforms and systems for these necessary steps, or are there other industries popping up around cell technologies?


GVB: All you have to do is look at the membership of ARM to answer that question. There are many different companies involved in regenerative medicine and, as you correctly noted, there are subsectors and distinct industry segments that have arisen to develop solutions for some of the challenges. Contract service organizations (CSOs), like Lonza Group AG (LONN:SIX; LO3:FSE; LZAGF:OTCPK), specialize in scalable manufacturing of cell therapy-based products, while others, like EMD Millipore (a division of Merck KGaA [MKGAY:OTCPK]) and Terumo Medical Corp. focus on development, validation and commercialization of novel bioreactor technologies. Still others, like Life Technologies Corp. (LIFE:NASDAQ), focus on providing the tools and reagents that enable the cutting-edge research to occur. These are just a few examples.


But, as you also noted, there are emerging specialties in the sector—for instance, companies developing 3-D printer systems for tissue engineering and other applications. An exciting example of that would be a company like Organovo Holdings Inc. (ONVO:OTCPK).


The field of regenerative medicine is giving birth to not just companies focused on developing the cell therapy, but also to a whole interrelated group of companies and industries that are leveraging their respective strengths, expertise and capabilities to move the field forward. That is really exciting to see.


TLSR: That brings to mind another important question that investors are concerned with. Can current good manufacturing practice (cGMP) be developed in an efficient way so that payers can afford the therapies and developers can make a decent bottom line? I ask that question because investors have not been happy with margins at Dendreon Corp. (DNDN:NASDAQ), which has developed Provenge (sipuleucel-T), a therapeutic vaccine for the treatment of advanced prostate cancer, using autologous (the patient's own) cells.


GVB: I see room for both autologous therapies and allogeneic (same species donor-derived) therapies. In the long run, those technologies that are the most scalable, the most standardized and that work the best are going to have the greatest impact. Based on what we know how to do today with cGMP product manufacturing, the answer to your question is clearly that we can deliver. In fact, there are already companies doing it—Shire's FDA-approved regenerative medicine therapy, Dermagraft, acquired through Advanced BioHealing, and Organogenesis with its state-of-the-art GMP manufacturing capabilities for Apligraf, its FDA-approved product for diabetic foot ulcers and venous leg ulcers, and its more recently approved Gintuit product for oral tissue regeneration. I think your question has, at least to a certain degree, been answered by the industry.


TLSR: My impression is that the allogeneic or allograft therapies are going to offer a better margin for the developer because they can be taken off the shelf, while autologous cells have to be harvested and processed individually and then administered back to the patient. Am I exaggerating that effect?


GVB: There are some challenges that apply to autologous therapies that truly scalable allogeneic therapies can overcome. I think that's why more interest from some of the bigger companies is focused on the allogeneic therapies.


But that being said, I also think there will be opportunity for autologous-based therapy that can address areas of unmet medical need. One example is Aastrom Biosciences Inc. (ASTM:NASDAQ), which is focused on treating conditions like dilated cardiomyopathy and critical limb ischemia. In the latter case, patients are looking at potential limb amputation. Aastrom's autologous therapy can make a difference in the clinical outcomes for these patients, using their own cells. Other examples might be Cytori Therapeutics Inc. (CYTX:NASDAQ), which has developed innovative technology focused on the processing of stem cells from autologous adipose cells, or NeoStem Inc. (NBS:NYSA.A), which has programs focused on the processing and administration of autologously derived hematopoietic stem cells. Each of these are showing promise. Ultimately, the clinical data will show the real winners. The best approaches will be embraced by patients and clinicians alike because traditional therapies and interventions have utterly failed in many areas where cell therapies are poised to have an impact.


TLSR: Gil, do you foresee patients being routinely treated with stem cell therapies, such as patients who have had strokes, patients who have had spinal injuries, and patients undergoing coronary artery bypass surgeries who simultaneously receive cells delivered to ischemic myocardial tissues? Do you see this in our lifetime?


GVB: Absolutely. In fact, I see evidence that some of those things are going to happen in the next few years. Some will take longer. If you look at the complexity of healing with a condition like spinal cord injury, or with some of the other conditions you've mentioned, they are definitely not the easiest indications to go after. They may take time, but in cardiovascular disease, stroke and other neurological conditions, and a broad range of other areas, I absolutely see cell therapy making a difference. It is already happening, and it will have a big impact in the next several years. You will start to see some of the really exciting things coming to the fore.


TLSR: It is difficult for a small company to enter many different specialties of medicine on its own. Do you see the small cell technology company as the hub, feeding many different therapies to different large pharmas for many different disease indications?


GVB: We now have more than 30 years of experience in the biotechnology industry, and we can see many different instances of that. But we also know that the bigger companies, the global companies, have capabilities and expertise in areas that smaller companies don't. They will help accelerate the process in a lot of different ways. If smaller companies are developing innovative new therapies that address substantial areas of need, that have substantial clinical and value-generation potential, they are producing exactly what the bigger companies want as they look to refresh and rebuild their pipelines.


I think that hub model is how it's going to work in a lot of situations. The smaller companies are doing high-risk research and, of course, some projects are going to pan out and some won't. If you take a step back and look at the history of the industry, it's the smaller, entrepreneurial, innovative, unafraid organizations that say, "Yes, we're going to do that, even if logic or historical data say maybe we can't." These small companies are comprised of the people who fearlessly believe in the science, the technology, and the impact it can have. Nothing is going to stop them from pursuing it. They are going to develop ways to address the challenges, the roadblocks and the hurdles.


That's not to say that everyone will be successful in that regard. But I think the smaller companies have historically embraced that risk and then turned that gamble into medical reality.


TLSR: Gil, this has been the greatest of pleasures. Thank you.


GVB: George, thank you. I really appreciate your time.


Gil Van Bokkelen has served as Athersys Inc.'s chief executive officer and chairman since August 2000. Van Bokkelen co-founded Athersys in October 1995 and has served as CEO and director since then. Prior to May 2006, he served as Athersys' president. From 2010 through 2012 Van Bokkelen served as chairman of the Alliance for Regenerative Medicine, a Washington D.C.-based consortium of companies, patient advocacy groups, disease foundations and clinical and research institutions committed to the advancement of the field of regenerative medicine. He is also chairman of the board of governors for the National Center for Regenerative Medicine, and serves on a number of other boards, including the Biotechnology Industry Organization's ECS board of directors (from 2001 to 2004, and from 2008 to present), the McGowan Institute for Regenerative Medicine and the Regenerative Medicine Foundation. He received a doctorate in genetics from Stanford University and bachelor's degrees in economics and molecular biology from the University of California at Berkeley.


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1) George S. Mack conducted this interview for The Life Sciences Report and provides services to The Life Sciences Report as an employee or as an independent contractor. He or his family own shares of the following companies mentioned in this interview: None.
2) The following companies mentioned in the interview are sponsors of The Life Sciences Report: Athersys Inc., Neuralstem Inc. Streetwise Reports does not accept stock in exchange for its services or as sponsorship payment. Johnson & Johnson is not affiliated with Streetwise Reports.
3) Gil Van Bokkelen: I or my family own shares of the following companies mentioned in this interview: Athersys Inc. I personally or my family am paid by the following companies mentioned in this interview: Athersys Inc. My company has a financial relationship with the following companies mentioned in this interview: Pfizer Inc., Lonza Group Ltd. I was not paid by Streetwise Reports for participating in this interview. Comments and opinions expressed are my own comments and opinions. I had the opportunity to review the interview for accuracy as of the date of the interview and am responsible for the content of the interview.
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