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MSB-GVHD001 showed that donor bone marrow stromal cells, given twice a week for a month, helped about seven in ten children whose acute graft-versus-host disease had not settled on steroids, which supported the first US approval of a mesenchymal stromal cell therapy in 2024.
MSB-GVHD001 was an open-label, single-arm phase 3 study of remestemcel-L, culture-expanded mesenchymal stromal cells from the bone marrow of healthy adult donors, in children with primary steroid-refractory acute graft-versus-host disease after a donor stem-cell transplant who were naive to other immunosuppressant therapies for it. According to the US label, patients had grade B to D disease (excluding grade B skin disease alone), 67 percent had been transplanted for a blood cancer and 33 percent for a non-malignant disease. Remestemcel-L was infused at 2 million cells per kilogram twice a week for four weeks (eight infusions). The primary endpoint was overall response (complete plus partial) at day 28, tested against a prespecified historical control rate of 45 percent.
In the 54 treated children the day-28 overall response was 70.4 percent (P = .0003 against 45 percent) and was sustained through day 100, with complete responses rising from 29.6 percent at day 28 to 44.4 percent at day 100. Overall survival was 74.1 percent at day 100 and 68.5 percent at day 180, and children who had responded by day 28 were more likely to be alive at day 100 (86.8 against 47.1 percent) and day 180 (78.9 against 43.8 percent). No infusion-related toxicities were identified (Biology of Blood and Marrow Transplantation 2020).
The US label reports the same 54 children as an overall response of 70 percent (38 of 54, 95% CI 56.4 to 82.0) with a median response duration of 54 days. The registry counts 55 participants in its full analysis set, one more than received treatment, and records the study as completed in April 2018. The FDA approved remestemcel-L (Ryoncil) in December 2024 for steroid-refractory acute graft-versus-host disease in children aged two months and older. The trial treats a complication of transplant rather than a cancer, so it carries no cancer ids.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
55 enrolled.
Against a prespecified historical control of 45%, P = .0003; complete response 29.6%
SourceShares Graft-versus-host disease (GVHD) and graft-versus-leukaemia, Allogeneic stem cell transplant (allo-SCT), Allogeneic (off-the-shelf) cell therapy.
Shares Graft-versus-host disease (GVHD) and graft-versus-leukaemia, Allogeneic stem cell transplant (allo-SCT).
Shares Graft-versus-host disease (GVHD) and graft-versus-leukaemia, Allogeneic stem cell transplant (allo-SCT).
Shares Remestemcel-L, Graft-versus-host disease (GVHD) and graft-versus-leukaemia.
Shares Graft-versus-host disease (GVHD) and graft-versus-leukaemia, Allogeneic stem cell transplant (allo-SCT).
Shares Graft-versus-host disease (GVHD) and graft-versus-leukaemia, Allogeneic stem cell transplant (allo-SCT), Allogeneic (off-the-shelf) cell therapy.