Michigan investigators improve on a new approach to acute leukemia

A pair of researchers at the University of Michigan Comprehensive Cancer Center say they have been making fresh progress on a new therapeutic approach to a relatively rare form of acute leukemia. And the biotech that in-licensed the work heralded the latest advance as another indication of the promise it holds for the cancer field.

Jolanta Grembecka and Tomasz Cierpicki have been improving on small molecules that can interfere with the protein-protein interactions of menin and MLL fusion proteins, scrambling a driver behind MLL fusion leukemia, which accounts for about 10% of all acute leukemia cases in adults and 70% of the incidents among infants.

Currently, that patient population has a high mortality rate, with all but about a third of patients dying in 5 years. 

Working in cell lines and mouse models, the investigators report that MI-463 and MI-503 blocked MLL-menin interactions without inflicting damage on normal blood cells. And they've been amping up the drug's potency in an effort to improve the likelihood of success.

"Against all odds, we decided to explore finding a way to block the MLL-menin interaction with small molecules. From nothing, we have been able to identify and greatly improve a compound and show that it's got valuable potential in blocking MLL fusion leukemia in animal models," says Cierpicki, assistant professor of pathology at the University of Michigan Medical School.

That's good news for Kura Oncology, a newly unveiled biotech that has set out to build a pipeline of new cancer treatments. Kura in-licensed the technology late last year.

Troy Wilson

"Jolanta Grembecka and Tomasz Cierpicki are scientific leaders in targeting the menin-MLL interaction, and it's a privilege to work with them to advance this promising class of compounds," said Troy Wilson, the CEO of Kura Oncology. "We share with them the goal of translating this cutting-edge science as quickly as possible into breakthrough medicines for patients with acute leukemias and other cancers."

In the meantime, other Michigan researchers say that the same approach could have an effect on androgen receptor signaling, a driver of castration-resistant prostate cancer. 

- here's the release from Kura
- and the release from Michigan

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