Graft-versus-host disease is an alloimmune syndrome in which donor immune cells attack recipient tissues after allogeneic transplantation. It is classically divided into acute and chronic forms and can involve skin, liver, gastrointestinal tract, and other organs. GVHD remains a major source of morbidity and mortality after stem-cell transplantation.
A phase II trial of TAK-007 CAR-NK therapy in R/R B-cell NHL showed a 60.9% overall response rate. Preclinical CAR-NKT therapy eliminated endometrial tumors in models. Optimized costimulatory domains (2B4-DAP12) boosted CAR-NK tumor killing.
CRISPR Therapeutics said CASGEVY launch momentum is building and multiple pipeline assets could generate data in the next 12 to 18 months. The company also highlighted zugo-cel progress in oncology and autoimmune disease.
Mesoblast received FDA clearance for a registrational DMD trial of remestemcel-L-rknd, while Satellos began dosing boys in the Phase 2 BASECAMP study of SAT-3247. The studies are enrolling children ages 5-9 and 7-9, respectively.
The 2026 Tandem Meetings featured new data on CAR-T, allogeneic transplantation, and supportive care. Highlights included early efficacy signals for EB-103, KITE-753, Orca-T, and NXC-201, plus comparative cardiovascular safety data for lisocabtagene maraleucel.
Transformative therapies including stem cell transplant and gene therapy offer sickle cell disease patients potential freedom from the SCD phenotype with reduced morbidities. About 90% of patients successfully switch to donor blood cell production after transplant, while gene therapies work by editing the patient's own stem cells. These treatments require careful consideration of disease severity and potential risks including major adverse events.
Interim results from the ABC phase 1/2b trial show a calcineurin inhibitor-free GVHD prevention regimen combining posttransplant cyclophosphamide, bortezomib, and abatacept achieved low GVHD rates and favorable survival outcomes. The approach enables earlier introduction of posttransplant maintenance therapies while reducing toxicity associated with conventional immunosuppressants.
A Phase 1 trial of azer-cel, a CAR T-cell therapy using donor T-cells to target B-cells, is recruiting patients with progressive multiple sclerosis at eight U.S. sites. Separately, multiple randomized trials are comparing hematopoietic stem cell transplantation against high-efficacy disease-modifying therapies.
Orca-T, an investigational T-cell therapy under FDA priority review, is being studied to reduce graft-versus-host disease in patients undergoing allogeneic stem cell transplant for acute leukemias and high-risk blood disorders, with a decision expected in April 2026.
Faron Pharmaceuticals announces Phase II BEAM-X trial evaluating bexmarilimab plus azacitidine in MRD-positive AML patients after stem cell transplantation. The Nordic AML Group-led trial will enroll 24 patients with first results expected 12-15 months after enrollment begins in Q3 2026.
Research shows TET2-driven clonal hematopoiesis improves immune checkpoint blockade efficacy in solid tumors, while separate studies identify new immune pathways in childhood brain cancers and mechanisms behind immunotherapy resistance.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07356245 |
Ruxolitinib Maintenance Post-Hematopoietic Stem Cell Transplant T-Cell Lymphoma |
RECRUITING | PHASE2 |
| NCT07319000 |
Efficacy of Low-dose PT-Cy for Prevention of GVHD in Ambulatory Allogeneic HSCT |
COMPLETED | PHASE2 |
| NCT07235501 |
Kinetic Analysis of Immune Cells in Blood and Chronic Graft-Versus-Host Disease-Affected Tissues After Allogeneic Hematopoietic Cell Transplantation |
NOT_YET_RECRUITING | |
| NCT07006506 |
A Study of Belumosudil in People at Risk of Developing Graft-Versus-Host Disease After a Stem Cell Transplant |
RECRUITING | PHASE2 |
| NCT06996119 |
Emapalumab With Post-Transplant Cyclophosphamide, Tacrolimus and Mycophenolate Mofetil for the Prevention of Graft-versus-Host Disease After Donor Reduced-Intensity Hematopoietic Cell Transplant |
RECRUITING | PHASE1 |
| NCT06815003 |
Vedolizumab Plus Post-transplant Cyclophosphamide and Short Course Tacrolimus for the Prevention of Graft Versus Host Disease in Patients Undergoing Allogeneic Hematopoietic Cell Transplantation After Reduced Intensity Conditioning |
RECRUITING | PHASE2 |
| NCT06705062 |
Study Comparing Reduced Versus Standard Dose Post-transplantation Cyclophosphamide in Combination With Post-engraftment Anti-thymoglobin as Graft Versus Host Disease Prophylaxis in Alternative Donor Peripheral Stem Cell Transplantation |
RECRUITING | PHASE3 |
| NCT06698211 |
Posaconazole Tablet As Primary Prophylaxis of HSCT Patients with Gastrointestinal GVHD |
ENROLLING_BY_INVITATION | |
| NCT06304025 |
Sequential Administration of WJ-MSCs for the Treatment of GvHD Refractory to Second Line Treatment |
NOT_YET_RECRUITING | PHASE1/PHASE2 |
| NCT06252870 |
Study Testing Two Conditioning Regimen With a Single Prophylaxis of GVHD by Cyclophosphamide and Methotrexate Post-transplant in Patients Eligible for Matched-donor Allograft Transplantation |
RECRUITING | PHASE2 |
