Since Gateway for Cancer Research was founded 30 years ago, the organization has funded more than 190 clinical trials serving over 9,000 patients through a large spectrum of research grant programs. Their focus is always and only on the patient, with the vision of creating a world where a cancer diagnosis is no longer feared.
Gateway works with dedicated physicians, clinician-scientists, and renowned research institutions around the world, and we are honored that they chose to partner with the National Marrow Donor Program (NMDP)/Be The Match. Gateway’s $800,000 grant is funding a critically important, first-ever CAR T clinical trial for children and young adults with relapsed or refractory acute myeloid leukemia (AML), which means their leukemia either returned after remission or did not respond to previous treatment.
With CAR T-cell therapy, a patient’s own cells are removed from their body and modified in a lab to recognize and destroy their cancer cells. These cells are then infused back into the patient to eliminate the cancer. This study will first test how many CAR T-cells are safe to use—enough to kill the cancer but not cause life-threatening complications. Once the “maximum tolerated dose” is found, the trial will expand to include more pediatric patients and evaluate their response to this novel therapy.
These patients have exhausted currently available treatment options, but this trial provides hope. It could significantly extend their lives or offer a chance for a cure. Beyond that, it has the potential to transform cancer care and significantly advance the science of CAR T-cell therapy, saving countless more lives.
Delora Senft, Director of Strategic Partnerships at Gateway for Cancer Research, knows that “success” for a clinical trial comes in many different forms. “Not having the trial go as planned still provides key learnings that could shift the standard of care and implement new treatments,” she says. “As long as we recruit the number of patients needed to obtain valuable data, the study is a success.”
We recognize the courageous young patients whose participation in this clinical trial will lead to vital discoveries, and we thank Gateway for Cancer Research for their remarkable generosity and leadership in the pursuit of saving lives.
Ms. Senft says that watching our virtual Be The Mala Gala last year highlighted the synergy between our two organizations. “Both are passionate, intentional and relentless in the fight against cancer,” she shares—and we are even stronger together.
About Gateway for Cancer Research
Gateway for Cancer Research is one of the only nonprofits that exclusively funds innovative Phase I and Phase II cancer clinical trials for patients of all ages, regardless of cancer type. At the forefront of research and discovery, these early-phase trials are the first step in evaluating new cancer drugs, treatments, and therapies. Gateway funds transformational cancer research that helps patients feel better and live longer, seeking to put an end to cancer as we know it.
About the CD33 CAR T-Cell Clinical Trial
NMDP/Be The Match serves as the sponsor of the Investigational New Drug Application for the groundbreaking pediatric CD33 CAR T-cell clinical trial. This trial opened in January 2020 with Children’s Hospital of Philadelphia (CHOP) and the National Cancer Institute (NCI) as the first participating study sites. It will ultimately enroll patients at Children’s Hospital of Los Angeles, Children’s Hospital Colorado, Seattle Children’s Hospital, and Boston Children’s Hospital. The trial is sponsored by the Pediatric Transplantation and Cellular Therapy Consortium and is led by Drs. Nirali Shah (NCI) and Richard Aplenc (CHOP). The CD33 CAR T product is manufactured at NCI’s Biopharmaceutical Development Program at Frederick National Laboratory for Cancer Research.
To learn more about research initiatives at Be The Match, contact Amy Conner at AConner@NMDP.org.