Mesenchymal Stem Cells: A Therapeutic Treatment For Severe And Acute Graft-Versus-Host Disease

Main Category: Stem Cell Research
Article Date: 23 Jun 2008 - 13:00 PDT

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A phase II multicenter study performed within the European Group for Blood and Marrow Transplantation (EBMT) Mesenchymal Stem Cell Expansion Consortium, shows that mesenchymal stem cells provide a therapeutic potential for the treatment of acute steroid-refractory GvHD (graft-versus-host disease).

Allogeneic stem-cell transplantation is the treatment of choice for many malignant and non-malignant disorders. Severe graft-versus-host disease (GvHD) is a life-threatening complication which could arise following this treatment. Especially if patients with GvHD do not respond to steroids, therapeutic options are limited and the success uncertain. This publication in one of the leading scientific journals opens new exciting possibilities for patients with GvHD.

The study was launched to assess whether mesenchymal stem cells could reduce the risk of GvHD after stem cell transplantation.

Between October 2001 and January 2007, 55 patients were treated. From this, 30 patients had a complete response and nine showed improvement. No patients had side effects during or immediately after infusions of mesenchymal stem cells. This response was not related to donor HLA-match. Three patients had recurrent malignant disease and one developed de-novo acute myeloid leukaemia of recipient origin.

This phase II study shows that the infusion of mesenchymal stem cells expanded in vitro, irrespective of donor, might be an effective therapy for patients with steroid-resistant, acute GvHD. Most interestingly, there was no difference in the response rates or side effects between patients receiving mesenchymal stem cells from third-party mismatched donors compared with those patients receiving cells from HLA-identical siblings or from haploidentical family members.

This finding makes the logistical requirements for this approach more convenient, because the establishment of local banks of mesenchymal stem cells would enable unproblematic and rapid availability of mesenchymal stem cells without the need of HLA typing.

Katarina Leblanc, who led the study, commented: "We find the results to be encouraging and hope to explore the immunosuppressive capacity of mesenchymal stem cells further in future trials, both as treatment of GvHD and in other inflammatory conditions."

This phase II study opens perspectives for a safe and effective treatment for patients with severe, acute GvHD who do not respond to corticosteroids and other immune-suppressive therapies, but further investigations are needed to determine the number of infusions, the best dose of cells in each infusion and the possible interactions of cells with other drugs for acute GvHD.

The full study has been published on May 10, 2008 in The Lancet (Vol 371, p.1579)

About the European Group for Blood and Marrow Transplantation

Bone marrow or stem cell transplantation is often the only curative treatment for different malignant diseases and is currently performed on more that 50,000 patients worldwide each year. The European Group for Blood and Marrow Transplantation (EBMT) as the leading non-profit, scientific society representing 530 transplant centres in and outside Europe, promotes all activity aiming to improve stem cell transplantation or cellular therapy. This includes registering all the activity relating to stem cell transplants with a view to improving treatment outcomes for patients. EBMT has set standards for indication and treatment for malignant and non-malignant diseases, along with running training programmes for continual professional development. These are continually audited and updated. EBMT is also responsible for accrediting the transplant centres based on their performance and data reporting.

European Group for Blood and Marrow Transplantation