Grant Funds Research and Clinical Trial to Address Health Disparities in Bone Marrow Transplantation

July 21, 2016

Ethnic minorities who need bone marrow transplantation (BMT) have less than a 35 percent chance of finding a matched unrelated donor.

At the University of Arizona Health Sciences, physicians and researchers at  the UA Cancer Center and the UA Steele Children’s Research Center have been working together  to address this disturbing disparity.

Emmanuel Katsanis, MD, professor of pediatrics, medicine, pathology and immunobiology, and director of the UA Cancer Center Blood and Marrow Transplant Program, recently was awarded a three-year, $600,000 grant from the Leukemia & Lymphoma Society. The “LLS Translational Research Program” will advance his ongoing haplo-BMT research at the UA Steele Center, and fund a Phase I/II clinical trial at the UA Cancer Center.

For patients who need a bone marrow transplant but cannot find a donor match, a procedure called haploidentical bone marrow transplantation (haplo-BMT) could be lifesaving. The procedure provides an alternative source of stem cells for patients who need a bone marrow transplant but cannot find a related or unrelated donor match. This means the donor doesn’t have to be a perfect match—he or she can be a “haplo,” or “half-match.” Thus, a patient’s parent, child or sibling could be a suitable donor.

“Haplo-BMT is particularly important in Arizona where we have a large population of Hispanics, who have a very low chance of finding an unrelated bone marrow match through the national registry,” said Dr. Katsanis.

The UA Cancer Center’s BMT program has successfully treated 12 patients—both children and adults—with haplo-BMT at Banner – University Medical Center Tucson and Banner Children’s at Diamond Children’s Medical Center.

“However, the research we’ve conducted at the Steele Center has shown that we can improve upon what’s currently the standard of care, in regard to haplo-BMT treatment,” Dr. Katsanis said.

Dr. Katsanis’ research findings were published this month in the British Journal of Haematology. He and his research team (Jessica Stokes, BS; Emely Hoffman, BS; Yi Zeng, MD, PhD; Nicolas Larmonier, PhD) showed that replacing the drug cyclophosphamide with the novel drug bendamustine following haplo-BMT, improved survival rates in mice undergoing haploidentical BMT by controlling graft versus host disease (GvHD) while increasing graft versus leukemia effect which improves leukemia-free survival.

Based on these research findings, Dr. Katsanis has written a phase I/II clinical trial protocol to test the efficacy of bendamustine in decreasing relapse in patients who have received haplo-BMT. The clinical trial is expected to begin soon.

“With this funding from the Leukemia & Lymphoma Society, all the pieces are in place, and we now can move our research from ‘bench to bedside.’ We hope to start the clinical trial this fall,” said Dr. Katsanis.

Adds Fayez K. Ghishan, MD, director of the UA Steele Center, “This grant enables us to advance our basic science cancer research that ultimately will lead to improved outcomes for patients receiving haplo-BMT.”

About the UA Steele Children’s Research Center

The UA Steele Children’s Research Center is one of the prestigious Centers of Excellence at the UA College of Medicine – Tucson at the University of Arizona Health Sciences. It is the state’s only academic pediatric research center designated by the Arizona Board of Regents, and the only facility in Southern Arizona where researchers and physician-scientists are dedicated to advancing medical knowledge through basic and translational research to improve children’s health. As researchers, they seek to discover answers to children’s medical mysteries. As physician-scientists, they provide compassionate care to hospitalized patients at Banner Children’s at Diamond Children’s Medical Center and pediatric outpatient clinics throughout Tucson and the state. And, as faculty members with the UA Department of Pediatrics, they teach and train the next generation of pediatricians and researchers.

About the University of Arizona Cancer Center

The University of Arizona Cancer Center is the only National Cancer Institute-designated Comprehensive Cancer Center headquartered in Arizona. The UA Cancer Center is supported by NCI Cancer Center Support Grant number CA023074. With primary locations at the University of Arizona in Tucson and at St. Joseph’s Hospital and Medical Center in Phoenix, the Cancer Center has more than a dozen research and education offices in Phoenix and throughout the state and 300 physician and scientist members working together to prevent and cure cancer. For more information:

About the University of Arizona Health Sciences

The University of Arizona Health Sciences is the statewide leader in biomedical research and health professions training. The UA Health Sciences includes the UA Colleges of Medicine (Phoenix and Tucson), Nursing, Pharmacy and Mel and Enid Zuckerman College of Public Health, with main campus locations in Tucson and the growing Phoenix Biomedical Campus in downtown Phoenix. From these vantage points, the UA Health Sciences reaches across the state of Arizona and the greater Southwest to provide cutting-edge health education, research, patient care and community outreach services. A major economic engine, the UA Health Sciences employs almost 5,000 people, has nearly 1,000 faculty members and garners more than $126 million in research grants and contracts annually. For more information: