Bone marrow transplantation replaces damaged or diseased marrow with healthy stem cells, either from the patient (autologous) or a donor (allogeneic). It's used to treat blood cancers like leukemia and lymphoma, sickle cell disease, immune deficiencies, and other conditions where the marrow fails. Outcomes have improved dramatically as doctors have learned to prevent rejection and infection.
What's actually going on in research
Trials are testing ways to make transplant safer and more accessible — expanding donor pools with haploidentical (half-matched) family donors, reducing graft-versus-host disease with drugs like ruxolitinib and abatacept, and using gene-edited cells to correct genetic diseases. Researchers are also studying reduced-intensity conditioning regimens that require less chemotherapy before transplant.
Haploidentical transplant
New techniques allow half-matched family members to donate, which expands options for patients without perfect matches. Studies show outcomes now approach those of matched donors.
GVHD prevention
Drugs like abatacept and post-transplant cyclophosphamide are reducing rates of graft-versus-host disease, where donor immune cells attack the recipient. This makes transplant tolerable for more people.
Gene-edited transplant
Trials are testing whether editing a patient's own stem cells can cure sickle cell disease and beta-thalassemia without needing a donor. Early results show people living free of transfusions and pain crises.
What to know before you search
Eligibility depends on the underlying disease, age, organ function, donor availability, and whether prior treatments have failed.
What types of trials are currently open
- Conditioning regimen trials — Testing lower doses of chemotherapy or radiation before transplant to reduce side effects while still clearing space for new marrow.
- GVHD prevention trials — Testing drugs and cell therapies given before or after transplant to prevent donor immune cells from attacking the recipient's body.
- Donor matching trials — Studies of transplants using half-matched family donors, cord blood, or other alternative sources when no perfect match exists.
- Gene therapy trials — Testing whether correcting a patient's own stem cells can replace the need for a donor transplant in genetic diseases.
- Supportive care studies — Research on preventing infections, managing side effects, and improving quality of life during and after transplant.
Recently added Bone Marrow Transplantation trials
Forced Oscillatory Technique for Bronchiolitis Obliterans Syndrome After Allogeneic Bone Marrow Transplantation
Bronchiolitis obliterans syndrome (BOS) is a severe pulmonary complication following allogeneic hematopoietic stem cell transplantation (HSCT). This prospective study evaluates the usefulness of the Forced Oscillation Technique (FOT) for the diagnosis, early detection, monitoring, and assessment of treatment response in patients undergoing allogeneic HSCT. FOT results will be compared with standard pulmonary function tests (spirometry, lung volumes, and DLCO).
The MedMemos Study: Promoting Adherence to Medications in Pediatric Bone Marrow Transplant Patients
The goal of this clinical trial is to test the MedMemos Intervention with parents and children who have received a bone marrow transplant. The main study questions are: * Is the MedMemos intervention acceptable to patients and families? * Is the MedMemos intervention feasible for patients, families, and healthcare providers? * Does MedMemos help patients and families take their medications better? Participants will be asked to complete surveys, attend 4 intervention sessions, and use an electronic box or pill bottle to store their medications in.
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