We are developing MultiStem®, a patented and proprietary stem cell product for the treatment of multiple distinct disease indications including myocardial infarction, oncology treatment support (such as leukemia or lymphoma patients that receive a bone marrow transplant, and are at risk for developing GvHD), ischemic stroke, and a range of conditons involving immune system function, such as inflammatory bowel disease or other autoimmune diseases. MultiStem is a biologic product that is manufactured from human stem cells obtained from adult bone marrow or other nonembryonic tissue sources. Unlike other cell types, after isolation from a qualified donor MultiStem may be expanded on a large scale for future clinical use and stored in frozen form until needed. Cells obtained from a single donor require no genetic modification and may be used to produce banks yielding hundreds of thousands to millions of doses of MultiStem – an amount far greater than other stem cell types can achieve. Each bank is extensively characterized to ensure product consistency and safety.
MultiStem 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.
During several years of preclinical work, MultiStem has demonstrated the potential to address each of the fundamental limitations observed with traditional bone marrow or hematopoietic stem cell transplants. These limitations include the historical requirement for tissue matching between donor and patient, the typical need for one donor for each patient (a reflection of the inability to expand cells in a controlled and reproducible manner), frequent use of immune suppressive drugs to avoid rejection or immune system complications, and a range of other potential safety issues.
We believe that MultiStem represents a potential best-in-class stem cell therapy because it exhibits each of the following characteristics based on research and development to date: (1) it may be produced on an industrial scale, in a well validated and reproducible manner; (2) it may be administered without tissue matching or the need for immune suppressive drugs, making it analogous to type O blood; (3) it exhibits a consistent safety profile; and (4) it appears capable of delivering a therapeutic benefit through more than one mechanism of action. Factors expressed by MultiStem are believed to reduce inflammation and regulate immune system function, protect damaged or injured cells and tissue, promote formation of new blood vessels, and augment tissue repair and healing in other ways. Based upon work that we and independent collaborators have conducted over the past several years, we believe that MultiStem has the potential to treat a range of disease indications, including ischemic injury and cardiovascular disease, certain neurological diseases, autoimmune disease, transplant support (including in oncology patients), and a range of orphan disease indications
Though the cells have the potential to differentiate into a variety of cell types, in certain indications the primary mechanism of MultiStem appears to be the production of a physiologically relevant and complex set of therapeutic molecules in response to the local environment. In the initial indications Athersys is pursuing, the cells appear to minimize the inflammatory reaction that occurs in response to ischemic events (such as myocardial infarction or stroke) or the anti-host immune reaction seen in graft vs. host disease (GvHD), and promote healing and recovery. Unlike traditional pharmaceuticals, MultiStem cells are dynamically regulated, and have the potential to respond to signals of inflammation or tissue damage in multiple ways. Potential mechanisms of benefit include protection of damaged or injured cells, reduction of inflammation, stimulation of new blood vessels, and the recruitment of other cell types to promote tissue repair and healing.
MultiStem's ability to be delivered “off the shelf” like a traditional pharmaceutical is based on:
- Scalability - The product can be produced efficiently and consistently in large quantities and stored (frozen) for subsequent use
- "Off-the-shelf" administration - In contrast to conventional bone marrow or hematopoietic stem cell transplants and most other cell types, extensive preclinical studies have shown that MultiStem may be used without tissue matching or immunosuppression, making it analogous to type “O” blood
- Multiple approaches to delivery - MultiStem may be delivered systemically (e.g. intravenously) or via local administration (e.g. using a catheter based delivery system), depending on the disease indication and the specific needs of the patient
MultiStem allows us to efficiently pursue multiple, high-value commercial and therapeutic opportunities from a single product platform. We are focused on developing MultiStem broadly for the treatment of ischemic injury (e.g myocardial infarction, stroke and other indications) and for conditions involving the immune system, such as autoimmune disease or other indications. |