MultiStem is a biologic product that consists of undifferentiated human stem cells obtained from adult bone marrow or other non-embryonic tissue sources. The cells may be produced on a large scale for future clinical use and stored in frozen form until needed. Material from a single qualified donor may be used to produce hundreds of thousand or even millions of clinical doses of MultiStem, which are extensively characterized to ensure product consistency and safety.
MultiStem has great potential as a “best-in-class” cell therapy based on its ability to deliver therapeutic benefit through multiple mechanisms of action, its ability to be delivered “off-the-shelf” like a pharmaceutical product and its safety profile.
For many types of cancer, such as leukemia or other blood-borne cancers, treatment typically involves radiation therapy or chemotherapy, alone or in combination. Such treatment can substantially deplete the cells of the blood and immune system, by reducing the number of stem cells in the bone marrow from which they arise. The more intense the radiation treatment or chemotherapy, the more severe the resulting depletion of the bone marrow, blood, and immune system. However, other tissues may also be affected, such as cells in the digestive tract and in the pulmonary system. The result may be severe anemia, immunodeficiency, substantial reduction in digestive capacity, and other problems, which may result in significant disability or death.
One strategy for treating the depletion of bone marrow is to perform a bone marrow transplant. This approach may augment the patient’s ability to form new blood and immune cells and provide a significant survival advantage. However, finding a closely matched donor is frequently difficult or even impossible. Even when such a donor is found, in many cases there are immunological complications, such as Graft Versus Host Disease (GVHD), which may result in death or serious disability. Working with leading experts in the stem cell and bone marrow transplantation field, we have studied MultiStem in animal models of radiation therapy and GVHD. In multiple animal models, MultiStem has been shown to be non-immunogenic, even when administered without the genetic matching that is typically required for conventional bone marrow or stem cell transplantation. Furthermore, in animal model systems testing immune reactivity of T-cells against unrelated donor tissue, MultiStem has been shown to suppress the undesired T-cell-mediated immune responses that are an important factor in causing GVHD. MultiStem-treated animals also displayed a significant increase in survival relative to controls. As a result, we believe that the administration of MultiStem in conjunction with standard bone marrow transplantation may have the potential to reduce the incidence or severity of complications and may enhance other important functions.
We plan to initiate a company-sponsored Phase I clinical trial with leading experts in the field of bone marrow transplantation to evaluate the safety of MultiStem administration in support of bone marrow transplantation for the treatment of certain cancers of the blood and immune system.
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