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Mesenchymal stromal cells (MSCs) have emerged as a potential therapy option in patients with steroid-refractory graft-versus-host disease (SR-GvHD).1 MSCs do not express human leukocyte antigen class II and can therefore be administered without the need for matching, giving them a distinct advantage over other cellular therapies.1

The GvHD Hub spoke to Robert Zeiser, Medical Center – University of Freiberg, Freiberg, DE, about his key insights into MSCs for aGvHD, and the current clinical trial landscape.

The MSC clinical trial landscape

• The first patient treated with MSCs, in 2004, experienced improvements in gastrointestinal and hepatic manifestations of severe GvHD after infusion of haploidentical MSCs. Since then, there have been multiple studies investigating MSCs as a potential treatment in aGvHD, although not all trials have met their primary endpoint.
• In general, MSCs are well tolerated by patients, with promising efficacy demonstrated in clinical trials; however, there is some variation in response rates.
• The mechanism of action of MSCs is still unclear; current evidence suggests that intravascular MSC destruction is followed by monocyte-mediated immune modulation, with monocytes playing a key role in the efficacy of the treatment.

Recent developments

• Remestemcel-L-rknd, an allogeneic bone marrow-derived MSC therapy, was approved by the U.S. Food and Drug Administration (FDA) on December 18, 2024, for the treatment of steroid-refractory acute graft-versus-host disease (aGvHD) in pediatric patients (aged ≥2 months).
• Another allogeneic MSC product, Mesenchymal Stromal Cells-Frankfurt am Main (MSC-FFM), is currently being investigated in the phase III IDUNN trial (NCT04629833) across multiple centers in Europe. The trial will compare MSC-FFM to the best available therapy in patients with SR-aGvHD. This therapy differs from other MSC products, as it is made from pooling cells from multiple donors rather than a single donor, and is already available for use in Germany under Hospital Exemption.

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