Small molecule for treatment of Acute Myelogenous Leukemia


Acute myeloid leukemia (AML), also known as acute myelogenous leukemia, is a fast-growing form of cancer of the blood and bone marrow. There are about 30,000 new cases of AML diagnosed per year in the USA, and the incidence is rising given the aging of the population. The current standard-of–care repertoire of therapies is often able to achieve remission or partial remission, but disease relapse and progression occur in the majority of cases. Most newly developed targeted therapies such as bcl-2 or IDH1/2 inhibitors do not induce durable remissions when used in isolation or combination, and many older patients are not able to undergo intensive chemotherapy or stem cell transplantation due to higher morbidity and mortality in older individuals. Therefore, therapies that can result in better disease control through leukemia stem and progenitor cell inhibition are required if therapeutic progress is to be made. 

Technology Overview

We developed a small molecule inhibitor of IQGAP1, a scaffolding protein, for treatment of AML. IQGAP1 is involved in multiple signaling pathways and participates in diverse cellular activities. IQGAP1 showed increased expression of IQGAP-1 mRNA in primary human AML cells across cytogenetic subtypes compared to normal cell controls. In addition to AML, it is overexpressed in colon cancer, glioma, non-small cell lung cancer and gastric cancer. We demonstrate that 1) IQGAP1 is critical to the survival of AML, 2) IQGAP1 silencing reduced proliferation and colony forming potential of leukemia cells, 3) our inhibitor suppressed the viability, caused cell cycle arrest and reduced IQGAP1 expression in AML cell lines. All these evidences point out that this molecule could be developed as a novel therapeutic in AML.


First systematic effort in the disclosed literature to develop a small molecule inhibitor against IQGAP1. Potentially stimulate interest in developing therapies against this and other scaffolding proteins which could yield a new therapeutic class of drugs in AML as well as solid tumors.


AML, colon cancer, glioma, non-small cell lung cancer and gastric cancer.

URV Reference Number: 6-20098
Patent Information:
For Information, Contact:
Blaze Pharoah
Licensing Manager
University of Rochester
Rakesh Singh
Deepak Sahasrabudhe