Blood Stem Cell and Bone Marrow Transplant Research

May 6, 2024 
This page was reviewed under our medical and editorial policy by Leslie Popplewell, M.D., Hematologist and Medical Director of Hematology and Blood and Bone Marrow Transplant, City of Hope Atlanta

City of Hope® has one of the nation’s largest and most successful blood stem cell and bone marrow transplant programs. Physicians at our cancer centers in Atlanta, Chicago, Duarte and Phoenix have performed nearly 20,000 bone marrow transplant procedures. Our outcomes consistently rank at the highest level among transplant centers nationwide. In fact, the Center for International Blood and Marrow Transplant Research recognizes City of Hope for our ability to deliver exceptional survivor rates to our patients.

Our researchers and clinicians continually explore new approaches to blood stem cell and bone marrow transplants to improve care and outcomes for patients even more. We work to develop and evaluate new therapies and ways to deliver them to improve survival and quality-of-life outcomes for all of our patients.

Why Is Stem Cell Research Important?

Researchers are actively engaged in studies to expand their understanding of stem cells and find potential uses for them in treating not only blood cancers — also called hematologic malignancies — but other diseases as well.

Patients who have stem cell transplants are at risk for relapse and transplant failure. The patient’s risk of relapse depends on several factors, including how advanced the disease was when the transplant was done. City of Hope researchers are constantly looking for innovative treatment strategies that may help prevent relapse after a donor stem cell transplant.

Graft-versus-host disease (GVHD), which may develop in patients who have an allogeneic stem cell transplant, occurs in about 60% of patients who have a bone marrow transplant, according to the National Organization for Rare Disorders (NORD). GVHD is when the donor cells that have been transplanted into the patient attack the recipient’s cells because they don’t recognize them and see them as foreign invaders. City of Hope researchers are committed to looking for appropriate ways to minimize GVHD and allow the transplanted cells to grow and survive.

Bone Marrow Transplant Clinical Trials

Clinical trials are research studies involving people and new ideas to tackle disease. Stem cell and bone marrow transplant (BMT) trials are important because they may help improve outcomes for patients who undergo a BMT. Clinical trials also help scientists make important discoveries that may help patients who need BMTs in the future.

Clinical trials may also be used to help find the most appropriate methods of delivering treatment and investigate the side effects and safety of new medications and approaches to BMT. Clinical trials may also help health care providers diagnose BMT complications sooner — and perhaps prevent them.

City of Hope Expertise in Bone Marrow and Stem Cell Research

At City of Hope, our researchers collaborate extensively to develop and evaluate new therapies to improve survival and quality of life for patients with blood cancers and other disorders. Our patients have the opportunity to participate in clinical trials that investigate:

  • New chemotherapy and targeted therapy options
  • Hormone therapies
  • Novel surgical techniques
  • Innovative ways to deliver radiation therapy
  • Strategies for preventing side effects

Patients who take part in clinical trials are given access to promising, leading-edge therapies, and their participation may help improve future care for patients around the world.

Some of our current research projects include those listed below.

Investigating "leukemia stem cells" that allow leukemia to relapse and grow after treatment for cancer: Understanding the biology behind these cancer stem cells may help scientists and health care providers develop more appropriate therapies that lead to lasting results.

Researching enhanced bone marrow/stem cell transplants to help reduce the risk of GVHD and potentially boost transplant outcomes: City of Hope is investigating new and inventive ways to classify and match stem cell transplant recipients and donors.

Studying the side effects and appropriate dose of total bone marrow and lymphoid irradiation when given with chemotherapy before a donor transplant in patients diagnosed with acute leukemia or myelodysplastic syndromes.

Studying how well the chemotherapy drugs clofarabine and melphalan given before a donor stem cell transplant help stop the growth of cancer cells and prevent cancer’s return after transplant in patients with acute leukemia in remission, myelodysplasia or chronic myelomonocytic leukemia.

Investigating how well vaccines made from a peptide reduce the frequency of severe infections in patients with blood cancer who are undergoing a donor stem cell transplant.

Studying whether combining the monoclonal antibody natalizumab with corticosteroids helps reduce the risk of GVHD.

References
References
  • Hong S, Rybicki L, Corrigan D, et al. (2021). Survival following relapse after allogeneic hematopoietic cell transplantation for acute leukemia and myelodysplastic syndromes in the contemporary era. Hematology/Oncology and Stem Cell Therapy. doi.org/10.1016/j.hemonc.2020.11.006. 
    https://www.sciencedirect.com/journal/hematology-oncology-and-stem-cell-therapy

  • StatPearls [Internet] (2022, October 10). Graft-Versus-Host Disease. 
    https://www.ncbi.nlm.nih.gov/books/NBK538235/

  • National Organization for Rare Disorders (2022, January 10). Graft versus Host Disease. 
    https://rarediseases.org/rare-diseases/graft-versus-host-disease/

  • Centenary Institute (2023, April 29). What are stem cells?. 
    https://www.centenary.org.au/news/what-are-stem-cells/

  • Be the Match. Clinical Trials Search and Support. 
    https://bethematch.org/patients-and-families/about-transplant/clinical-trials/

  • Clinicaltrials.gov (2023, September 11). Total Marrow and Lymphoid Irradiation and Chemotherapy Before Donor Transplant in Treating Patients With Myelodysplastic Syndrome or Acute Leukemia. 
    https://classic.clinicaltrials.gov/ct2/show/NCT02446964

  • National Cancer Institute. Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia. 
    https://www.cancer.gov/research/participate/clinical-trials-search/v?id=NCI-2013-01193&r=1

  • National Cancer Institute. Vaccine Therapy in Reducing the Frequency of Cytomegalovirus Events in Patients with Blood Cancer Undergoing Donor Stem Cell Transplant. 
    https://www.cancer.gov/research/participate/clinical-trials-search/v?id=NCI-2015-00283&r=1

  • Al Malki MM, London K, Baez J, et al. (2023, September 12). Phase 2 Study Of Natalizumab Plus Standard Corticosteroid Treatment For High-Risk Acute Graft-Versus-Host Disease. Blood Advances, 7(17), 5189–5198. 
    https://doi.org/10.1182/bloodadvances.2023009853

  • National Cancer Institute (2023, October 5). Stem Cell Transplants in Cancer Treatment. 
    https://www.cancer.gov/about-cancer/treatment/types/stem-cell-transplant

  • American Cancer Society (2020, March 20). How Stem Cell and Bone Marrow Transplants are Used to Treat Cancer. 
    https://www.cancer.org/cancer/managing-cancer/treatment-types/stem-cell-transplant/why-stem-cell-transplants-are-used.html