EXECUTIVE SUMMARY February 19, 2013 | New York
Analysis and Market Forecasts 2013-2023
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STEM CELL SUMMIT EXECUTIVE SUMMARY
Robin R Young, CEO NEW YORK STEM CELL SUMMIT PUBLISHER: ORTHOPEDICS THIS WEEK CEO, PEARLDIVER TECHNOLOGIES INC. (A MEDICAL DATA MINING COMPANY)
Phone: 610-260-6449 Cell: 917-887-7376 robin@ryortho.com www.ryortho.com www.pearldiverinc.com
February 19, 2013
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T ABLE OF C ONTENTS INTRODUCTION ............................................................................................................................................................ 5 BACKGROUND .............................................................................................................................................................. 6 STEM CELL USE IN THE UNITED STATES ....................................................................................................................... 6 AUTOLOGOUS STEM CELL USE IN THE UNITED STATES ................................................................................................ 7 Limbal Stem Cells .................................................................................................................................................. 7 Bone Marrow Stem Cells ....................................................................................................................................... 8 Adipose (or Fat) Tissue Stem Cells........................................................................................................................ 9 ALLOGRAFT STEM CELL USE IN THE UNITED STATES ................................................................................................... 9 COMPANIES THAT SUPPLY SYSTEMS FOR AUTOLOGOUS STEM CELL HARVESTING OR STEM CELL RICH ALLOGRAFTS 11 U.S. GOVERNMENT AS PURCHASER OF STEM CELL THERAPIES .................................................................................. 12 STEM CELL MARKET OVERVIEW ................................................................................................................................. 12 Exhibit 1: Sales of Stem Cell Products in the U.S. (2010-2014) ......................................................................... 12 Exhibit 2: Stem Cell Companies Change in Value .............................................................................................. 14 Exhibit 3: Universe of Public Stem Cell Companies ........................................................................................... 15 MARKETS FOR STEM CELL PRODUCTS 2006-2020 ..................................................................................................... 16 Exhibit 4: Stem Cell Therapy Market Estimates (U.S., 2006-2020).................................................................... 17 Exhibit 5: Stem Cell Market Forecast, by Indication, (U.S., 2005-2020)............................................................ 18 ROBIN R. YOUNG – BACKGROUND ............................................................................................................................. 19 BIBLIOGRAPHY ........................................................................................................................................................... 77
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Company Profiles AllCells, LLC................................................................................................................23 Athersys, Inc. ................................................................................................................25 BioLife Solutions, Inc. ..................................................................................................27 Brainstorm Cell Therapeutics ........................................................................................29 CellGenix GmbH...........................................................................................................31 Celling Biosciences .......................................................................................................33 Cellular BioMedicine Group .........................................................................................35 CTI Clinical Trial and Consulting Services ...................................................................37 Gamida Cell Ltd. ...........................................................................................................39 Garnet BioTherapeutics, Inc. .........................................................................................41 INCELL Corp. LLC. .....................................................................................................43 ISTO Technologies, Inc.................................................................................................45 LifeNet Health...............................................................................................................47 LONZA Group Ltd. .......................................................................................................49 Mesoblast Limited.........................................................................................................51 NeoStem, Inc.................................................................................................................53 Neuralstem, Inc. ............................................................................................................55 NexGen Medical Systems, Inc.......................................................................................57 Orthopedic Stem Cell Institute ......................................................................................59 Osiris Therapeutics, Inc.................................................................................................61 Parcell Laboratories LLC ..............................................................................................63 Pluristem Therapeutics, Inc. ..........................................................................................65 Progenitor Cell Therapy LLC (PCT) .............................................................................67 Promethera Biosciences.................................................................................................69 Stem Cell Therapeutics Corp. ........................................................................................71 TiGenix NV...................................................................................................................73 Tissue Genesis, Inc........................................................................................................75
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INTRODUCTION Basic stem cell facts: •
There are thousands, perhaps tens of thousands of cell types which have in them some or all of the capabilities of stem cells.
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The qualities which distinguish stem cells from other cells are their ability to progress up a particular tissue lineage.
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Recently, scientists have discovered how to move cells in reverse direction down a particular tissue lineage and, in the process, restore many of the characteristics which define stem cells.
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When cells have some, not all, of the characteristics of a stem cell, they are a progenitor cell.
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The vast majority of therapeutic cells are progenitor cells and they exhibit most of the same characteristics of stem cells including the ability to express anti-inflammatory signals, growth factor proteins and to move along a particular tissue lineage to become specific tissue forms.
When 2012 began, an estimated 1 million U.S. patients had been treated with stem cells over the course of the previous 15 years. By the end of 2012, that number of patients treated rose by, we estimate, an astonishing 100,000! Physician users now number in the thousands. Indeed, in certain sectors of the U.S. healthcare industry, it is harder to find physicians who have NOT used stem cells than those that have. Who is using stem cells? Where are they getting the cells? For which indications are cells being used? What is appearing in peer review literature? Who is using stem cells? Where are they getting the cells? For which indications are cells being used? What is appearing in peer review literature? At this most recent edition of the New York Stem Cell Summit we will be answering those questions.
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BACKGROUND The primary source of stem cells for patient care currently are either autologous (harvesting stem cells from adult patients or then re-injecting them into the same patient) or allogeneic. In either form, stem cells have emerged as an increasingly routine therapy in U.S. medicine. More recently, as stem cell research has developed, the precise method of harvesting, processing and then re-injecting into patients has changed and improved. Roughly 1.1 million patients have been treated with their own stem cells since the mid-1980s in the United States. In this summary and as part of the curriculum for the New York Stem Cell Summit, we will describe the purpose of autologous and allograft stem cells in medicine, the history of autologous stem cell therapies in the United States, examples of companies that are supplying equipment to facilitate autologous stem cell therapies in the United States, the size of this as a commercial business and finally a ten-year forecast of these therapies.
STEM CELL USE IN THE UNITED STATES The most difficult element to understand about adult stem cells is their purpose in the human body. While embryonic stem cells are, in effect, the blank slate upon which all of the tissues, organs and structure of the human body is drawn, the adult stem cell has a different, though related, overall function. It is a repair cell. Sitting quiescently in bone marrow, tooth enamel, adipose (fat) tissues and other places in the body, these cells “wake up” upon receiving signals related to injury– inflammation. Their function is to begin the cascade of healing when the human body is injured. The number of stem cells in an adult human begins declining shortly after birth. It is, in fact, this decline in the number of stem cells that ultimately leads to wear, deterioration and, finally, death. So, when the human body is injured, the adult stem cells “wake up,” move through the body to the site of injury, express proteins to down regulate inflammation, express other proteins to stimulate new cellular growth, recruit new growth factor proteins and then, themselves, begin to change into the very cells that have been damaged. Throughout this process, these cells are giving and receiving signals with the surrounding environment. This is an extremely important aspect and one of the central discoveries regarding stem cells and the point that differentiates them from any other cell.
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The most common therapeutic use of stem cells by physicians in the United States is to harvest them from one part of the body where they exist in greater amounts and then re-implant them at the site of injury—where they are most needed but are in short supply. For adults with diminishing stores of stem cells, this is an effective way to stimulate healing at sites of injury. Frankly, this process of harvesting cells (be they bone marrow, skin, fat or any other cells) from one part of the body and then re-implanting them in another part has been an important aspect of medicine for, literally, hundreds if not thousands of years. What makes this discussion so relevant is that physicians are now specifically targeting stem cells (i.e., repair cells) for concentration, re-implanting and expansion before re-implanting. One problem with autologous use of adult stem cells is that the quantity of cells is only as good as the patient’s supply. Older patients with fewer stem cells will not likely have enough to overcome, say, osteoporosis when a bone fractures, or a tear in cartilage or a dead spot on the heart muscle, etc. In those cases, one strategy is to put the patient’s small number of stem cells into a culture media and allow them to expand. This is the next logical step in the development of modern autologous stem cell treatment.
AUTOLOGOUS STEM CELL USE IN THE UNITED STATES The discovery that a particular cell existed which was, in effect, a blank slate upon which all other cells can be developed stimulated an explosion of basic scientific research. Through the 1950s, ’60s and ’70s this research characterized these cells, began to unravel their secrets and began to provide clues as to their therapeutic potential. Almost immediately, scientists landed on the idea that physicians could harvest stem cells from one part of the human and re-implant them in another. The first widespread practice of doing exactly this therapy was among ophthalmologists.
Limbal Stem Cells Limbal stem cell transplants were the first autologous stem cell transplant and were first used in the 1970s. In this procedure limbal stem cells are taken from the healthy eye of the patient or a live donor (usually a sibling or a parent). During this outpatient procedure, which lasts about one hour, stem cells are harvested from the healthy eye or the donor's eye and then transplanted to the affected eye. If the transplant is successful, the stem cells will 2013 Stem Cell Summit
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