MultiStem Programs

MultiStem Cell Therapy: An "Off The Shelf" Biologic Product

MultiStem Product Profile

  • Cell therapy product based on patented (MAPC®) technology
  • Developing for "off-the-shelf" administration - no tissue matching needed
  • Large scale production potential (e.g., millions of doses from each donor)
  • Long storage life - can be kept frozen for years
  • Consistent safety profile
  • Promotes healing and tissue repair through multiple mechanisms of action
  • Not a permanent transplant - cells cleared from the body over time (like a drug)

We are developing MultiStem®, a proprietary stem cell product for the treatment of multiple distinct diseases and conditions in the cardiovascular, neurological, inflammatory and immune disease areas. 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 reducing inflammation, protecting damaged or injured tissue, and enhancing 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 multiple factors that regulate the immune system, protect damaged or injured cells, promote tissue repair and healing, and the cells are subsequently 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, making it analogous to type O blood; (3) it exhibits a consistent safety profile; and (4) it appears capable of delivering therapeutic benefit through multiple mechanisms of action. 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.


Multiple Potential Mechanisms of Benefit



Though MAPC’s have the potential to differentiate into a variety of cell types, the primary mechanisms of action of MultiStem appear to be achieved through 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 in other ways. 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.