Pioneers in Regenerative Medicine Reshaping the Landscape of Biotechnology
Every industry or sector has pioneers that worked at the forefront of the field to shepherd new technologies into the mainstream. Oncology, for example, took huge steps forward as a result of the efforts of Dr. Sidney Farber, earning him the titles of “the father of the modern era of chemotherapy” and “father of modern pediatric pathology”. What Dr. Farber was doing seventy years ago changed the landscape of cancer therapies. Fast forwarding to today, there are leaders in the regenerative medicine (sometimes called “tissue engineering”) field that one day may be referenced in the same manner as Farber. Henri Termeer, who retired from his post as CEO at Genzyme last year when the biotech leader was bought by Sanofi-Aventis (NYSE: SNY) for $20.1 billion; Geoff MacKay, CEO at privately-held Organogenesis, Inc.; and Gil Van Bokkelen, CEO at Athersys, Inc. (NASDAQ: ATHX) are each recognized as innovators in regenerative medicines that demonstrate the skill, tenacity and vision needed to reshape the way that doctors will treat patients in the future.
There is no road map to maneuver through the political, medical and regulatory challenges that confront an emerging and potentially transformational field like regenerative medicine. Helping guide the industry as a whole, Henri Termeer is recognized as a living legend in biotechnology and a leading advocate for cell therapy and tissue engineering. He built a legacy while convincing the world that cell therapy should be taken seriously as a field. During 26 years under Termeer’s tutelage, Genzyme established a business model using biological processes and developing products targeting rare genetic disorders that could not be easily copied by generic drug makers. This innovative strategy, which many predicted could never succeeed, took Genzyme from being a modest upstart into the third largest biotech in the world.
Following the blueprint of most great innovators, Termeer ignored naysayers early in his career and brought multiple products to market, including the first drug to treat Gaucher disease, which affects only about 10,000 people worldwide. Many successful drugs followed, including the FDA approving a Biologic License Application for Myozyme (alglucosidase alfa, rhGAA), the first treatment for patients with Pompe disease, another rare orphan disease, in 2006. A true visionary in many respects, Termeer also focused Genzyme on becoming the first biotechnology company to actually commit serious resources and figure out how to get a cell therapy to patients, including solving the complex logistical issues including storage, shipping and handling of these new types of products. He successfully pursued cartilage repair through cell therapy, and established a separate business unit within Genzyme devoted to the area of regenerative medicine and tissue engineering approaches. All the while, Termeer was known for focusing on the needs of patients while also displaying an intense commitment to creating shareholder value.
Termeer’s impact extended well beyond the walls of Genzyme, however, as he was a longstanding leader in the Biotechnology Industry Organization (BIO), which has been at the forefront of the industry for years and has been critical in helping to create or refine policies that impact the industry’s ability to develop of novel ways to treat disease. BIO’s role has been to promote the field of biotechnology, and to help pave the way for new medicines to become a reality.
Geoff MacKay, chief executive at Organogenesis, relies upon many of the same, patient-first characteristics of Termeer to build intrinsic value for the company. Still a “young gun,” MacKay has exemplified spirit and commitment to advance regenerative medicines with the bulk of his career in leadership positions at Novartis AG (NYSE: NVS), including roles in tissue engineering and immunology, before coming to Organogenesis in 2003.
The Organogenesis chief has successfully advanced two cell therapy products through the complex regulatory process and into the clinic where they now serve the needs of patients. Apligraf®, a living cell based product indicated for the treatment of venous leg ulcers and diabetic foot ulcers, became the first bio-engineered cell based product to receive FDA approval. GINTUIT™, a cellular sheet that has been shown to predictably generate new and aesthetically appealing oral soft tissue, has been approved by the FDA for the treatment of mucogingival conditions in adults and is slated for release this summer. The company is also developing other forms of regenerative medicine, including bio-engineered skin for the treatment of burns and other clinical indications. Two approved cell technologies and a third in the pipeline certainly proves MacKay’s abilities and devotion to regenerative medicines.
In addition to his role at Organogenesis, MacKay currently serves as a member of the Board of Directors of the Canadian Stem Cell Network and as Vice Chairman of the Board of the Massachusetts Biotechnology Council, a not-for-profit organization that represents and provides services and support for the Massachusetts biotechnology industry. MassBio is the nation’s oldest biotechnology trade association.
Dr. Gil Van Bokkelen is another prescient leader in the regenerative medicine space that that has stood head and shoulders above others for his exhaustive efforts for both Athersys and the industry on the whole. Under his guidance, Athersys has developed a robust portfolio of therapeutic product candidates centered on therapeutic stem cells and other tissue engineering to promote healing or replacement of diseased and damaged tissue.
Athersys is in the midst of four clinical trials, including two Phase II clinical trials. Their leading Phase II trial is being conducted in partnership with Pfizer (NYSE: PFE) targeting Inflammatory Bowel Disease utilizing Athersys’ proprietary MultiStem® stem cell technology. Results are expected early in 2013 from this research. A second Phase II trial using MultiStem® is being independently conducted by Athersys targeting ischemic stroke.
MultiStem® is viewed as a very significant technology because of its potential relevance for an assortment of diseases and complications that are in grave need of new therapies. Similar to Termeer’s approach, Van Bokkelen has Athersys targeting some smaller markets, in addition to larger areas of unmet need, like stroke, heart disease and inflammatory conditions. One orphan area the company is focused on involves providing treatment support to patients receiving a traditional bone marrow or hematopoietic stem cell transplants as part of treatment for cancer. Such transplants can provide life-saving help to patients being treated for leukemia or related conditions, but can also come at a cost – as treatment is frequently complicated by Graft Versus Host Disease (GVHD), an occurrence in which the newly transplanted material attacks the transplant recipient’s body. Athersys has focused on solving this issue, by using applying MultiStem to treat patients at significant risk of GVHD, intervening before the disease becomes a life threatening condition. With initial clinical testing successfully completed and showing promising results, Athersys recently met with the FDA to discuss advancement of MultiStem® into later stage clinical studies, to evaluate its potential as a new therapy to combat GVHD.
Other uses for MultiStem include repairing damage from acute myocardial infarction and other cardiovascular disease, diabetes and neurological damage (i.e. multiple sclerosis, ischemic stroke, and traumatic brain injury). The company’s vision and capabilities extend into other areas as well, such as developing a novel treatment for obesity, an area that represents a sizzling, hot button topic today.
Van Bokkelen’s leadership and role in the field of regenerative medicine extends beyond the programs at Athersys however. His resume includes serving as the current Chairman of the Alliance for Regenerative Medicine, as well as Chairman of the Board of Governors for the National Center for Regenerative Medicine. He also sits on a number of other boards, including the Biotechnology Industry Organization, the McGowan Institute for Regenerative Medicine and the Regenerative Medicine Foundation.
Work with stem cells has certainly faced a great many obstacles in the last two decades, but that worm is starting to turn as strong bi-partisan support is being demonstrated to enact new laws to expedite development of drugs and therapies to meet unmet medical needs. It seems likely that a broadened Accelerated Approval Pathway proposal will be enacted into law as part of the PDUFA renewal by the end of summer 2012. This should provide the FDA and innovators greater latitude in developing new therapies to treat serious and life threatening conditions that are beyond the current standards of care. While this could benefit the biotechnology industry across the board, regenerative medicine companies may stand to see the greatest rewards because of the large number of new therapies that can fall under the classification of “breakthrough medicines”, which the accelerated approval framework is designed to promote. Through their innovative nature and targeting of diseases that have proven resistant to traditional medicines, regenerative medicine approaches could help address some of the biggest and most serious long term challenges facing our healthcare system.
While there are many great leaders in biotechnology, the aforementioned CEO’s have consistently demonstrated vision, empathy and responsibility to bring a wave of novel therapies to the market that can substantially enhance the quality of life of patients and an unwavering dedication to reshape the options allotted to physicians in patient care. Their devotion to their respective companies is only paralleled by their unselfish efforts to help the industry overcome the many challenges it faces regarding not only inherent development, but also regulatory and political obstacles.
Whether it is in a bottle or a vial, the day of cell-based therapies is nearly upon us with a debt of gratitude being owed to pioneers such as Termeer, MacKay and Van Bokkelen.
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