Merck Licensing Harvard Cancer Drug Molecules

Matthew Shair (Stephanie Mitchell, Harvard University)

21 March 2016. The pharmaceutical company Merck is licensing small molecule research discoveries from a Harvard University lab as potential treatments for leukemia. The agreement brings the university $20 million immediately, with further payments expected as Merck develops the discoveries into products.

The deal gives Merck an exclusive license to research by biochemistry professor Matthew Shair, whose recent work highlights inhibitors of chemicals in the body that promote the growth of acute myeloid leukemia cells. Acute myeloid leukemia is a cancer of the blood and bone marrow that worsens quickly if left untreated. As the disease develops, bone marrow produces abnormal white blood stem cells called myeloblasts that do not mature into normal functioning white blood cells.

The excessive growth of abnormal myeloblasts crowds out healthy white, red, and platelet blood cells, and can spread to other parts of the body. Leukemia and Lymphoma Society says acute myeloid leukemia has a 5-year survival rate of 25 percent in the U.S., although for children and teens under the age of 15, about two-thirds (66%) survive for at least 5 years.

In a September 2015 paper, Shair and colleagues discovered a natural small-molecule steroid known as cortistatin A inhibits actions of the enzymes cyclin-dependent kinase 8 (CDK8) and 19. These enzymes encourage the growth of acute myeloid leukemia cells by limiting the transcription of genes feeding the growth of healthy cells, clearing the way for cancerous cells to develop. In lab tests, Shair’s team found cortistatin A promotes activity of genes that help suppress tumors, resulting in fewer leukemia cells. In addition, cortistatin A appears to work only with cell lines affected by CDK8 and CDK19 enzymes, which suggests it works selectively and may not affect other functions in the body.

The deal with Merck gives the company an exclusive license to develop Shair’s work into therapies for acute myeloid leukemia. Merck is paying Harvard a $20 million licensing fee, with the university eligible for progress milestones and royalties on sales of products developed from this technology, although amounts of further payments were not disclosed. Shair’s lab and Merck will also collaborate on further research into enzymes that regulate gene transcription.

Shair credits a Harvard program known as the Blavatnik Biomedical Accelerator that identifies early-stage basic research with commercial potential and funds its development to encourage licensing by industry. “Accelerator funding over the course of several years,” says Shair in a university statement, “has enabled my laboratory to advance some of our experimental compounds to a relatively late stage of preclinical development.”

In August 2015, Science & Enterprise reported on 12 new research projects added to Blavatnik Biomedical Accelerator’s portfolio. Those new projects included research from Shair’s lab.

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