Advanced Cell Technology provides therapies for a broad range of diseases, including retinal pigment epithelial cells for treating macular degeneration and other retinal degenerative disorders; myoblasts for treating heart failure; and hemangioblasts for treating blood disorders and cardiovascular disease. Stem cells can be used to generate healthy and functioning specific cell types, which can then replace diseased or dysfunctional cells. Replacing diseased cells with healthy cells, called cell therapy, is similar to the process of organ transplantation. Currently, researchers are investigating the use of
adult and embryonic stem cells as a resource for various specialized cell types such as nerve cells, muscle cells, blood cells and skin cells. ACT is using a combination of embryonic and adult stem cell technologies to develop novel therapies for indications with few or no alternatives.
The ability to produce embryonic stem cells that are immunologically compatible with the patient is one of the strengths of our embryonic stem cell technology platform. We believe our technology platform will enable the transformation of a patient’s cells into an embryonic state where those cells can be differentiated into specific therapeutically relevant cell types that are genetically identical to the patient. We believe our technology may also enable the production of stem cell lines, from sources external to the patient, that have a sufficiently high level of histo-compatibility to be useful in making cell therapies readily accessible to a large segment of the patient population, without the need for exact genetic matching of tissues. In August 2001, The President of the United States set guidelines for federal funding of research on embryonic stem cells from human embryos created by in-vitro fertilization (IVF). IVF-ES cells have the drawback that they are not genetically matched to the recipient patient. These ES cells are allogeneic. The word allogeneic literally means other DNA type. Therapies using allogeneic cell lines can result in immune system incompatibilities where the host immune system attacks and rejects the transplanted cells or the transplanted cells attack the host. These incompatibilities may be partially suppressed with powerful immunosuppressive drugs, but the side effects can be severe and result in life-threatening complications. As a result, these incompatibilities will generate significant inefficiencies in the application of cell therapies.
ACT is also focused on solving the problem of immune rejection and graft-versus-host disease by creating stem-cell therapeutics using donor tissues that are compatible, or that the immunologically active antigens on the donor cells are the same as those of the recipient so that they will not activate an immune response leading to rejection. This ability to produce tissue cells for transplantation that have the same genetic make-up as the patient may constitute a competitive advantage for Advanced Cell Technology.