Stem Cell Transplant for Blood Cancer
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new combination of chemotherapy and low-dose radiation to determine its safety and effectiveness for treating certain blood cancers. The aim is to develop a less intense treatment that still effectively targets cancer cells. It specifically examines a stem cell transplant method called Allogeneic Hematopoietic Stem Cell Transplantation (AlloHCT), which the FDA has not yet approved for this purpose. This trial may suit individuals with certain blood cancers, such as acute myeloid leukemia or severe aplastic anemia, who have already tried other treatments. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants a chance to be among the first to receive this new therapy.
Do I need to stop my current medications for the trial?
The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
What prior data suggests that the CliniMACS device is safe for T cell depletion?
Research has shown that allogeneic hematopoietic stem cell transplantation, like the one in this study, is generally well-tolerated by patients with blood disorders. In a study of patients with sickle cell disease, the overall survival rate reached 94%, which is very promising. This study also reported low rates of acute and chronic graft-versus-host disease, where donor cells attack the patient's body.
However, up to 30% of patients might experience a relapse after the transplant, and 2% to 5% could develop new cancers from the donor cells. While these outcomes highlight potential risks, they also indicate that many patients handle the treatment well.
As this is a Phase 1 study, the main goal is to determine safety. This phase usually involves fewer participants and aims to assess if the treatment is safe for humans. Past research suggests that while there are risks, the treatment has a manageable safety profile.12345Why are researchers excited about this trial's treatment?
Researchers are excited about this treatment because it uses allogeneic hematopoietic stem cell transplantation in a novel way, particularly for patients with myeloid malignancies and aplastic anemia. Unlike standard treatments that often involve chemotherapy or autologous stem cell transplants, this method incorporates a unique conditioning regimen with ATG, fludarabine, and total body irradiation (TBI), followed by post-transplant cyclophosphamide to help reduce complications like graft-versus-host disease. Additionally, a dose of Rituxan is included to lower the risk of viral infections, and donor stem cells are sourced from peripheral blood, aiming for a high target cell infusion to enhance recovery and improve outcomes. Researchers are hopeful that these innovations will lead to better immune function and disease control, potentially setting a new standard in treatment for these challenging conditions.
What evidence suggests that this trial's treatments could be effective for blood cancer?
Research has shown that using healthy donor stem cells to replace damaged bone marrow, as in the allogeneic hematopoietic stem cell transplantation studied in this trial, can effectively treat severe aplastic anemia and some blood cancers. One study found that 55 patients who received this treatment had a 3-year survival rate of 71.8%, meaning about 72 out of every 100 people were still alive three years after the transplant. Another study found that early transplantation for severe aplastic anemia improved survival chances. Although risks exist, these studies suggest that this treatment can potentially cure certain blood cancers and severe bone marrow diseases.16789
Who Is on the Research Team?
Roni Tamari, MD
Principal Investigator
Memorial Sloan Kettering Cancer Center
Are You a Good Fit for This Trial?
This trial is for adults with certain high-risk blood cancers or severe aplastic anemia who haven't had a previous stem cell transplant. They should be responding to current therapy, have adequate organ function, and not be pregnant or HIV positive. A detailed consent form must be signed indicating willingness to participate.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Conditioning
Participants undergo transplant conditioning with ATG, fludarabine, and TBI, followed by high dose cyclophosphamide post stem cell infusion
Transplantation
Stem cell infusion and administration of Rituxan to reduce EBV viremia risk
Post-Transplant Monitoring
Participants receive G-CSF and undergo chimerism studies and immune function assessments
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Allogeneic Hematopoietic Stem Cell Transplantation
Allogeneic Hematopoietic Stem Cell Transplantation is already approved in European Union, United States, Canada, Japan for the following indications:
- Primary immunodeficiency disorders
- Immune dysregulatory disorders
- Hemophagocytic lymphohistiocytosis
- Bone marrow failure syndromes
- Hemoglobinopathies
- Primary immunodeficiency disorders
- Immune dysregulatory disorders
- Hemophagocytic lymphohistiocytosis
- Bone marrow failure syndromes
- Hemoglobinopathies
- Primary immunodeficiency disorders
- Immune dysregulatory disorders
- Hemophagocytic lymphohistiocytosis
- Bone marrow failure syndromes
- Hemoglobinopathies
- Primary immunodeficiency disorders
- Immune dysregulatory disorders
- Hemophagocytic lymphohistiocytosis
- Bone marrow failure syndromes
- Hemoglobinopathies
Find a Clinic Near You
Who Is Running the Clinical Trial?
Memorial Sloan Kettering Cancer Center
Lead Sponsor