Project BriefOpen Competition 1 - BiotechnologyTransdifferentiation of Adult Somatic CellsDevelop technology to efficiently transform a patient's own mature cells in culture to various differentiated neuronal cells without going through a stem-cell-like stage, without creating embryos, and without the need for fetal tissue, for eventual use in autologous cell transplantation therapies for degenerative diseases such as Parkinson's, Alzheimer's or multiple sclerosis. Sponsor: ACT Group, Inc.One Innovation DriveWorchester, MA 01605
Although much of the work is still in its early stages, cell replacement therapies are expected to provide important, effective treatment -- and perhaps cures -- for a wide range of intractable diseases, including Parkinson's, Alzheimer's, Huntington's, multiple sclerosis, strokes, heart disease, diabetes and many others, as well as spinal-cord injuries. Three main routes have been proposed to obtain the necessary cells. Potentially one of the best sources is embryonic stem cells, which have the potential to develop into any cell in the body, but there are significant ethical issues involved in the use of human embryonic cells. Another possibility is to harvest and culture tissue-specific progenitor cells -- a sort of secondary-level stem cell -- from adults, but the complex clinical and laboratory procedures required to produce sufficient quantities of these cells have not been fully established. A third possibility is to use embryonic stem cells from animals, particularly pigs, but this incurs significant problems, including overcoming the human immune response. Advanced Cell Technology proposes a novel alternative -- a technique to take the patient's cells, from skin or blood, and changes them into the desired neuronal cell for transplantation without regressing them to stem-like cells. This cell "transdifferentiation" occurs naturally during development, and has been found in some adult organs. However, controlling the process in vitro without causing the cells to revert to stem-like cells, prematurely aging them, or introducing unwanted side effects, while still retaining full functionality and the ability to reproduce, is expected to be very challenging. If fully successful, the technology could have a major impact on the treatment of a host of diseases and injuries that, combined, cost the nation several hundreds of billions of dollars annually. Because it starts with relatively large quantities of easily obtained cells, transdifferentiation would significantly shorten the time required to generate therapeutic quantities of desired neuronal cells. It involves no issues in the use of embryonic cells, and because it starts with the patient's own cells, it is expected to circumvent most problems in patient immune response and histocompatibility.
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