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

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.

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.


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

Beth Hinshaw Hall
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The Van Andel Research Institute–Stand Up To Cancer (VARI–SU2C) Epigenetics Dream Team fosters collaboration between several of the world’s most respected research and clinical organizations in an effort to translate scientific discoveries into new standards of patient care. The goal is simple—get new and more effective cancer therapies to patients faster.

The VARI–SU2C Epigenetics Dream Team was established in 2014 and builds on the foundations laid by the first iteration of the SU2C Epigenetics Dream Team, which was founded in 2009. Today’s team is based at Van Andel Research Institute in Grand Rapids, Michigan, and is led by the Institute’s Chief Scientific Officer Peter Jones, Ph.D., D.Sc., and Stephen Baylin, M.D., VARI Director’s Scholar and co-head of Cancer Biology at Johns Hopkins University’s Sidney Kimmel Comprehensive Cancer Center. The team includes leading scientists and clinicians with vast experience in translating basic science and promising therapies from the lab to the clinic.

The team is honored to be affiliated with Stand Up To Cancer, a program of the Entertainment Industry Foundation. Launched in 2008, SU2C draws on the resources of the entire entertainment industry to encourage the public to support research conducted by teams of scientists, as well as by individual investigators. To date, more than 1,200 researchers from more than 140 institutions in seven countries have collaborated across SU2C’s 20 Dream Teams, six Translational Research Teams and 46 Innovative Research Grants.

Rigorous and objective scientific oversight and review is provided by SU2C’s scientific partner, the American Association for Cancer Research (AACR), the world’s first and largest professional organization dedicated to advancing cancer research and its mission to prevent and cure cancer.

Van Andel Institute (VAI) is an independent nonprofit biomedical research and science education organization committed to improving the health and enhancing the lives of current and future generations. Established by Jay and Betty Van Andel in 1996 in Grand Rapids, Michigan, VAI has grown into a premier research and educational institution that supports the work of more than 360 scientists, educators and staff. Van Andel Research Institute (VARI), VAI’s research division, is dedicated to determining the epigenetic, genetic, molecular and cellular origins of cancer, Parkinson’s and other diseases and translating those findings into effective therapies. The Institute’s scientists work in onsite laboratories and participate in collaborative partnerships that span the globe. Learn more about Van Andel Institute or donate by visiting www.vai.org. 100% To Research, Discovery & Hope®

Stand Up To Cancer®  (SU2C) raises funds to accelerate the pace of research to get new therapies to patients quickly and save lives now. SU2C, a program of the Entertainment Industry Foundation (EIF), a 501(c)(3) charitable organization, was established in 2008 by film and media leaders who utilize the industry’s resources to engage the public in supporting a new, collaborative model of cancer research, and to increase awareness about cancer prevention as well as progress being made in the fight against the disease. As SU2C’s scientific partner, the American Association for Cancer Research (AACR) and a Scientific Advisory Committee led by Nobel Laureate Phillip A. Sharp, PhD, conduct rigorous, competitive review processes to identify the best research proposals to recommend for funding, oversee grants administration, and provide expert review of research progress.

Current members of the SU2C Council of Founders and Advisors (CFA) include Katie Couric, Sherry Lansing, Lisa Paulsen, Rusty Robertson, Sue Schwartz, Pamela Oas Williams, Ellen Ziffren, and Kathleen Lobb. Noreen Fraser and the late Laura Ziskin are also co-founders. Sung Poblete, PhD, RN, has served as SU2C’s president since 2011.

For more information on Stand Up To Cancer, visit www.standup2cancer.org.