Enhancing Stem Cell Mobilization

Description: This invention provides a potentially novel approach to enhancing the efficiency of stem cell mobilization, to improve the effectiveness of stem cell transplantation therapies. Hematopoietic stem cells (HSCs) can functionally regenerate an ablated hematopoietic system, and bone marrow and peripheral blood progenitor cell transplantation are therapeutic approaches that are increasingly used for the treatment of leukemia, lymphoma, multiple myeloma, breast and ovarian cancers, and genetic disorders affecting hematopoietic cell function. Mobilizing agents such as Amgen’s Neupogen (GC-SF) or Neulasta (pegylated GC-SF) are often given to transplant donors to enhance the number of HSCs which can be obtained for transplantation. A newer, drug, Mozobil, is in Phase III trials for this indication, and with worldwide sales estimates for the drug as high as $200-300 million, its prospects recently led to the acquisition of its developer AnorMed by Genzyme, following a protracted bidding war. The present invention is based on the inventors’ discovery that a particular zinc-finger transcription factor regulates both HSC expansion and mobilization processes. In particular, in transgenic mice lacking one or both copies of the gene for this factor, LT-HSCs were found in significantly greater frequency in both bone marrow and peripheral blood. This discovery leads to several potential strategies for enhancing stem cell mobilization by modulating this transcription factor. Clinical & Commercial Utility: The invention comprises methods and compositions for modulating HSC expansion and mobilization by targeting this zinc-finger factor. For example, the invention includes a method of increasing the number of hematopoietic stem cells in the peripheral blood of a subject, by administering to the subject an effective amount of an inhibitor of this particular zinc finger transcription factor. A number of small molecule drugs as well as nucleotide analogs are known to be able to inhibit zinc finger transcription factors, and such molecules are expected to be useful in the methods of the invention. A U.S. patent application claiming this invention has been filed.

Type of Offer: Licensing

Next Patent »
« More Medical Patents

Share on      

CrowdSell Your Patent