In 1957, The New England Journal of Medicine published results from the first clinical study of an allogeneic stem cell transplant. In the procedure — now commonly known as a bone marrow transplant — six patients with blood cancer received stem cells from a healthy donor. Only two patients achieved engraftment, a key treatment milestone in which donor cells begin producing healthy cells in a patient’s bone marrow. And all six patients died within 100 days of transplantation.1
In the more than 60 years since that study, the scientific community has made tremendous strides in improving bone marrow transplant. But there is still a significant need to make this potentially curative treatment option available to more patients around the world.
Each year, approximately 13,000 people in the U.S. who have been diagnosed with a blood cancer may need a bone marrow transplant. Yet only about 8,000 of these patients go on to receive a transplant.2 Therefore, nearly 40% of patients do not receive a bone marrow transplant for a variety of reasons, including the inability to find a matched donor.3
Even when patients do find a match, transplants using current graft sources may result in a long time to engraftment, potentially leading to serious infections and long hospitalizations that diminish quality of life. Patients also have a high risk of life-threatening complications, especially during the period when their blood counts are low and their immune system is suppressed.
Current graft sources for patients who do not have a fully matched family donor include matched unrelated donors, or those who are matched but not related to the patient, haploidentical, or half-matched related donors, and unrelated umbilical cord blood. Each of these sources comes with certain limitations. For example, grafts from both haploidentical and matched unrelated donors are not available for all patients and may be limited by donor features including age, while umbilical cord blood is limited by the relatively small number of stem cells.
Innovation in Bone Marrow Transplant
In light of these challenges, new approaches are needed to redefine standards of care and improve treatment outcomes for patients.
Gamida Cell is at the forefront of this wave of innovation in bone marrow transplant. Using its proprietary NAM-based cell expansion platform, the company is developing omidubicel, an investigational advanced cell therapy in Phase 3 development as a potential life-saving treatment option for patients in need of bone marrow transplant.4 Data from a Phase 1/2 clinical study published in the Journal of Clinical Oncology demonstrated that treatment with omidubicel resulted in rapid and durable engraftment and was generally well tolerated.5
Omidubicel has the potential to improve outcomes for patients like Stacey Khoury of Nashville, Tennessee. After being diagnosed with acute myeloid leukemia, Stacey needed a bone marrow transplant but did not have an available donor. She received omidubicel in 2011 as part of a clinical trial. Today, eight years post-transplant, she remains cancer-free and is back to working full-time while enjoying lifelong hobbies like traveling with her husband, Rick.
“After this experience, my outlook is certainly one of treasuring each and every day,” says Stacey. “When people say, ‘I don’t want to be having another birthday,’ I’m like, you’re so wrong. In fact, I took up the advice of someone who told me, ‘Now that you have a transplant, you have two birthdays.’ And I celebrate them both.”
To learn more about omidubicel and read Stacey’s story, visit www.gamida-cell.com.
This is one patient, and results may not be indicative as set forth above. Please note that omidubicel is investigational and safety and efficacy have not been established by any agency.
1 Henig I, Zuckerman T. Hematopoietic stem cell transplantation-50 years of evolution and future perspectives. Rambam Maimonides Med J. 2014;5(4):e0028. Published 2014 Oct 29. doi:10.5041/RMMJ.10162
2 D’Souza A., Fretham C. Current Uses and Outcomes of Hematopoietic Cell Transplantation (HCT): CIBMTR Summary Slides, 2018. Available at https://www.cibmtr.org.
3 Besse, K. et al. Estimating Demand and Unmet Need for Allogeneic Hematopoietic Cell Transplantation in the United States Using Geographic Information Systems. Journal of Oncology Practice Vol. 11, Issue 2. 2015.
4 ClinicalTrials.gov identifier NCT02730299
5 Phase I/II Study of Stem-Cell Transplantation Using a Single Cord Blood Unit Expanded Ex Vivo with Nicotinamide. Horwitz M.E., Wease S., Blackwell B., et al. Journal of Clinical Oncology. 2018 DOI: 10.1200/ JCO.18.00053.