Discovery Approach
The Company's stem cell discovery engine relies upon a library of monoclonal antibodies to human proteins and state of the art cell sorting capabilities. The approach begins with cells harvested from human brain, liver or pancreas. Using a library of known and proprietary monoclonal antibodies, StemCells identifies, purifies, and characterizes the human stem cell of interest. The stem cells are sorted and tested in rigorous in vitro or in vivo models. Purified populations of stem cells are then expanded, banked and retested in in vivo models to demonstrate that the expanded cells maintain the original stem cell properties. StemCells has a broad patent portfolio covering multiple aspects of this discovery process.
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The Need for Cell-based Therapeutics
Many diseases - such as Alzheimer's, Parkinson's, and other degenerative diseases of or injuries to the brain or nervous system - involve the failure of organs that cannot be transplanted. Other diseases, such as hepatitis and diabetes, involve organs such as the liver or pancreas that can be transplanted, but for which there are not enough organs available for transplant. As can be seen from the table, the annual direct expenditures in the U.S. associated with the treatment of such diseases are in excess of $200 billion dollars. The financial costs are in addition to the pain and distress to the patients and their families and friends.
Impaired cellular function is associated with the progressive decline common to many of these diseases. Cells maintain normal physiological function in healthy individuals by secreting or metabolizing substances, such as sugars, amino acids, neurotransmitters and hormones, which are essential to life. When cells are damaged or destroyed, they no longer produce, metabolize or accurately regulate these substances.
Advances in biotechnology have led to the identification of some of the specific substances or proteins that are deficient. While administering these substances or proteins as medication does overcome some of the limitations of traditional pharmaceutical products, such as lack of specificity, there is no existing technology that can deliver them to the precise sites of action under the appropriate physiological regulation and quantities, or for the duration required to cure the degenerative condition. Cells, however, do this naturally. As a result, investigators have considered supplementing the failing cells by implanting other cells capable of producing the needed substances necessary for organ or tissue repair. Transplantation of these stem or progenitor cells offers the possibility of generating new and healthy mature cells in cases of irreversible tissue damage or organ failure, thus potentially restoring the organ function and the patient's health.
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The Therapeutic Potential of The Company's Stem Cell-Based Therapy
The Company believes that human tissue-derived stem cell-based therapy — the use of stem or progenitor cells to treat diseases — has the potential to provide a broad therapeutic approach comparable in importance to traditional pharmaceuticals and genetically engineered biologics (such as Epogen and Neupogen). The therapeutic value of organ-derived stem cells lies in the fact that stem cells can give rise to cell types that are characteristic of the surrounding tissue (i.e., the organ from which they were derived and into which they are transplanted). Mature, functionally differentiated cells have lost the capacity for reproduction. Therefore, when such mature, specialized cells are lost due to disease or damage, other specialized cells cannot regenerate to fill the gap. Stem cells, in contrast, are cells at an early stage of development that have the ability to self-renew (that is, reproduce themselves, dividing into two cells at least one of which is also a stem cell) for indefinite periods and give rise to a number of different kinds of mature, functionally differentiated (specialized) cells. Implantation of these cells in damaged tissues can potentially lead to tissue and organ repair.
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