NHGRI’s New Targeted Therapy for Acute Myeloid Leukemia and Acute Lymphoblastic Leukemia

NHGRI’s New Targeted Therapy for Acute Myeloid Leukemia and Acute Lymphoblastic Leukemia

Agency: 
Dept. of Health and Human Services

Laboratory: National Human Genome Research Institute (NHGRI)

Technology: New Targeted Therapy for Acute Myeloid Leukemia and Acute Lymphoblastic Leukemia

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 to treat core binding factor (CBF) leukemia, acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), and/or other RUNX-related disorders.

Details: The invention describes the use of benzodiazepine compounds for the treatment of AML and ALL. Specifically, the compounds can be used to treat 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 has been implicated in the development of other diseases, including (but not limited to) platelet disorders, solid tumors (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.

Benefits:

  • Proof of concept demonstrated in a mouse model.
  • Compounds have been previously tested in clinical studies for anti-HIV drugs.

Applications:

  • Targeted drug therapies for AML and ALL.
  • Combination chemotherapies for AML and ALL.
  • Therapies for other RUNX-related disorders, including platelet disorders, solid tumors (e.g., lymphoma, breast cancer, osteosarcoma), and bone diseases (e.g., osteoporosis, cleidocranial dysplasia and intervertebral disk degeneration).

Contact: For more information about this technology, contact Claire T. Driscoll, Director of NHGRI Technology Transfer Office, at cdriscoll@mail.nih.gov or 301-594-2235.

To view the original technology listing, visit https://www.ott.nih.gov/technology/e-060-2011.

Region: 
Mid-Atlantic