New Targeted Therapy for Acute Myeloid Leukemia and Acute Lymphoblastic Leukemia
Posted Sep 09 2012 8:00pm
Description of Invention: The invention describes the use of benzodiazepine compounds for the treatment of acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL). Specifically, the compounds can be used to treat core binding factor (CBF) leukemias, which are a subgroup of leukemia associated with the generation of fusion genes, arising from the binding between the transcription factors: core binding factor-beta (CBFbeta) and runt-related transcription factor 1 (RUNX1). The compounds described in this invention have been found to inhibit the binding of CBFbeta and RUNX1, resulting in selectively killing leukemia cells in culture and suppressing leukemia in a mouse model.
In addition, the binding of runt-related transcription factors from the RUNX family have been implicated in the development of other diseases, including (but not limited to): platelet disorders, solid tumours (e.g., lymphoma, breast cancer, osteosarcoma) and bone diseases (e.g., osteoporosis, cleidocranial dysplasia and intervertebral disk degeneration). Thus, the use of these compounds may represent new targeted therapies for AML and ALL as well as other RUNX-related disorders.
Targeted drug therapies for AML and ALL.
Combination chemotherapies for AML and ALL.
Therapies for other RUNX related disorders, including platelet disorders, solid tumours (e.g., lymphoma, breast cancer, osteosarcoma) and bone diseases (e.g., osteoporosis, cleidocranial dysplasia and intervertebral disk degeneration).
Proof of concept demonstrated in a mouse model.
Compounds have been previously tested in clinical studies for anti-HIV drugs.
In vitro data available
In vivo data available (animal)
Inventors: Pu Liu (NHGRI) Wei Zheng (NCATS) Juan J Marugan (NCATS) Noel T Southall (NCATS) Lea C Cunningham (NCI)
Collaborative Research Opportunity: The National Human Genome Research Institute (NHGRI), Oncogenesis and Development Section, is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate, or commercialize benzodiazepine compounds described above to treat CBF leukemia, AML, ALL, and/or other RUNX-related disorders. Please contact Claire T. Driscoll, Director of NHGRI Technology Transfer Office ( firstname.lastname@example.org ; 301-594-2235) for more information.