A new cell therapy has been approved by the US Food and Drug Administration to treat paediatric patients from two months of age who experience complications from a stem cell transplant for blood cancer or an immune system disorder. The treatment, Ryoncil (remestemcell-L rknd), is an allogeneic therapy made up from mesenchymal stromal cells and isolated from the bone marrow of healthy adult donors. It is indicated for the treatment of acute graft-versus-host disease in patients undergoing transplants who do not respond to corticosteroids. “Steroid-refractory acute graft-versus-host disease can have significant, wide-ranging health consequences, including damage to multiple organs, reduced quality of life and risk of death in affected patients,” Nicole Verdun of the FDA’s Center for Biologics Evaluation and Research, said in a statement on 18 December 2024. Mesenchymal stem cells, the source of the therapy, are stromal cells that can be isolated from a variety of tissues and have the ability to self-renew.
Ryoncil was tested in a single-arm study of 54 paediatric patients with graft-versus-host disease undergoing transplants. Effectiveness was based on the rate and duration of response to treatment. The results showed that 30% of participants had a complete response to treatment, while 41% had a partial response. Ryoncil was developed by Mesoblast Inc whose parent company is Mesoblast Ltd of Australia.
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