Dimethyl Fasudil for Inducing Polyploidization of Megakaryocytes and for Treating Blood and Bone Marrow Diseases and Disorders

Acute Megakaryocytic Leukemia (AMKL) Treatment NU 2008-077 Inventors John Crispino* Qiang Wen Zan Huang Robert Gould Andrew Stern Short Description New approach for treating AMKL by inducing polyploidization to revert cancerous cells into normal cells #therapeutics #cancer # smallmolecules Abstract A unique therapeutic approach for acute megakaryocytic leukemia (AMKL) has been developed at Northwestern University. AMKL is an aggressive form of blood cancer for which there are few promising drug options. Based upon their identification of a unique feature of AMKL cells, Northwestern scientists developed an innovative treatment strategy. Cancerous megakaryocytic cells in AMKL sidestep a normal differentiation process called polyploidization. The screening method Prof. Crispino and colleagues designed may be used to search for compounds that would induce this process and render the cancerous cells normal. They believe this approach to chemotherapy may be less toxic to patients and would produce fewer side effects than standard chemotherapies, which eliminate both cancerous and healthy cells. Applications o Treatment of AMKL and other megakaryopoetic diseases o Research reagents for study of megakaryocytes Advantages o Low chance of side-effects o Applicable to various megakaryopoetic disorders Publications Wen Q, Goldenson B, Silver SJ, Schenone M, Dancik V, Huang Z, Wang L-Z, Thioller C, Diebold L, Gilles L, Vokes MS, Moore CB, Bliss-Moreau M, VerPlank L, Tolliday NJ, Mishra R, Vemula S, Shi J, Wei L, Kapur R, Lopez CK, Gerby B, Ballerini P, Pflumio F, Gilliland DG, Golberg L, Birger Y, Izraeli S, Gamis AS, Smith FO, Woods WG, Taub J, Scherer CA, Bradner J, Goh B-C, Mercher T, Carpenter AE, Gould RJ, Clemons PA, Carr SA, Root DE, Schreiber SL, Stern AM, Crispino JD (2012) Integrative Screening Approach Identifies Regulators of Polyploidization and Targets for Acute Megakaryocytic Leukemia, Cell, 150: 575-589. IP Status Issued US patent 8,993,071 Marketing Contact Michael Moore, PhD Invention Manager (p) 847.491.4645 (e) [email protected] Tags THERAPEUTICS: cancer, THERAPEUTICS: small molecule