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VARI-SU2C Epigenetics Dream Team launches new clinical trial to combat blood cancers

International collaboration pairs immunotherapy with an epigenetic drug to stop progression of chronic myelomonocytic leukemia and myelodysplastic syndrome into acute myeloid leukemi

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GRAND RAPIDS, Mich. (June 13, 2017)—A new clinical trial spanning three cancer centers in California, Maryland and Pennsylvania is investigating a promising treatment for chronic myelomonocytic leukemia (CMML) and myelodysplastic syndrome (MDS).

“Stopping these diseases, particularly in patients who have relapsed or developed resistance to first-line therapies, is absolutely critical,” says trial co-leader Casey O’Connell, M.D., associate professor of clinical medicine at the Keck School of Medicine at the University of Southern California and member of the
Van Andel Research Institute–Stand Up To Cancer (VARI-SU2C) Epigenetics Dream Team. “As the global population ages, we expect to see the incidence of these cancers rise, which further underscores the importance of finding new, more effective treatments.”

An estimated 13,000 people in the U.S. are diagnosed every year with MDS and an additional 1,100 are diagnosed with CMML. Both conditions are incurable with current drugs, and both can progress to acute myeloid leukemia (AML), a lethal blood cancer that accounts for more than 10,000 deaths every year. Stopping these precursor conditions early-on has the potential to save thousands of lives.

Currently, there are only two FDA-approved drugs to treat aggressive MDS and CMML. The options for patients whose cancers do not respond to or became resistant to these therapies are very limited. Bone marrow transplants may be curative but are associated with significant risks, particularly for patients with other illnesses.

The new trial, facilitated by the VARI-SU2C Epigenetics Dream Team, studies the combination of guadecitabine, an investigational new drug from Astex Pharmaceuticals that modulates epigenetics (sometimes called a DNA methyltransferase  inhibitor or hypomethylating agent), with atezolizumab, a cancer immunotherapy medicine developed by Genentech, a member of the Roche Group. Guadecitabine also is being studied in colorectal cancer as part of another VARI–SU2C Epigenetics Dream Team trial. Both companies have partnered with the team to supply the drugs for clinical study.

“Our laboratory models show these drugs synergize well in helping the body’s immune system recognize and destroy cancer, even in patients who no longer respond to standard treatments,” says Kirsten Grønbæk, M.D., DMSc., trial co-leader and professor and chief hematologist at University of Copenhagen’s Rigshospitalet, a member of the VARI–SU2C Epigenetics Dream Team.

“The immune system has an outstanding ability to fight back against cancer, but sometimes it needs help,” says O’Connell. “We believe the complementary effects of these drugs could be key to giving people with MDS and CMML more time.”

The trial is currently enrolling patients at University of Southern California in Los Angeles and is expected to open soon at Fox Chase Cancer Center/Temple University Hospital in Philadelphia, and University of Maryland Greenebaum Comprehensive Cancer Center in Baltimore, all collaborators on the VARI–SU2C Epigenetics Dream Team. Results are expected sometime in 2019.


Learn more about the Van Andel Institute–Stand Up To Cancer® (SU2C) Epigenetics Dream Team ➔


Collaboration between research organizations, medical centers, philanthropy and pharmaceutical companies is at the core of the VARI–SU2C Epigenetics Dream Team’s mission, which follows Stand Up To Cancer’s groundbreaking and impactful paradigm of competing against cancer rather than against each other. Since its establishment in 2014, the team has launched a total of four clinical trials with several others in various stages of development.

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Trial information
Trial name:
Guadecitabine and atezolizumab in treating patients with advanced myelodysplastic syndrome or chronic myelomonocytic leukemia that is refractory or relapsed
ClinicalTrials.gov identifier: NCT02935361
Lead investigator: Casey O’Connell, M.D., University of Southern California
Co-lead investigator: Kirsten Grønbaek, M.D., DMSc, University of Copenhagen/Rigshospitalet
Number of patients: 42

Media Contact

Beth Hinshaw Van Andel Institute [email protected] 616.234.5519