Menin-MLL Inhibitors: A Potential Treatment for a Rare and Deadly Leukemia

Oncology researchers Jolanta Grembecka and Tomasz Cierpicki began what they refer to as their “adventures in drug discovery” at the University of Virginia, where they focused on treatments for leukemia-related diseases. Their ultimate goal has always been to create new small molecules for protein targets neglected by industry—what are known as orphan diseases—and then license the compounds to commercial ventures for development into breakthrough medicines.

But academia-based drug development requires tremendous resources. In 2009, they accepted faculty positions at the University of Michigan. As Grembecka notes, “We knew the U-M had a great infrastructure and culture for drug discovery and technology transfer. Here, we would have every kind of support: access to core facilities and great scientists, the opportunity to interact with hematologists and other researchers in the field, and assistance with commercialization through U-M Tech Transfer.”

During their time at U-M, Grembecka and Cierpicki have leveraged all these resources. They have also received significant funding from the Leukemia & Lymphoma Society and its Therapy Acceleration Program, which helps advance discoveries from the lab to clinical trials.

By 2014, the two had successfully developed a number of first-in-class small molecule compounds targeting protein interactions involved in MLL fusion leukemia—a rare, aggressive and lethal disease. Each year, approximately 1,500 new cases of MLL leukemia are reported in this country, most of them among infants and children.

In 2015, with the ongoing assistance of Tech Transfer, the compounds were licensed to California-based
Kura Oncology, a clinical stage biopharmaceutical startup dedicated to developing precision medicines for the treatment of solid tumors and blood cancers. In addition, Kura Oncology has also agreed to support future research in the Grembecka and Cierpicki labs, where continued progress is being made in new therapeutics for acute leukemia and other cancers.

[source: U-M Tech Transfer 2015 Impact Report]