Remestemcel-L

Remestemcel-L is a third-party, off-the-shelf suspension of ex-vivo cultured adult human mesenchymal stem cells intended for intravenous infusion. The mesenchymal stem cells are derived from the bone marrow of unrelated and human leukocyte antigen (HLA)–unmatched healthy adult donors and have the ability to differentiate into different tissue cells. It was approved for use in Canada in May 2012 as Prochymal for the management of refractory acute Graft versus Host Disease (aGvHD) in children who are unresponsive to systemic steroid therapies, with the approval conditional upon further trials being conducted.

aGvHD is a T-cell mediated disease that occurs after allogeneic hematopoietic stem cell transplant and is a reaction of donor immune cells against host tissues developed by 30-80% of the recipients . aGvHD is often characterized by the presence of inflammatory cascades through signalling of activated T cells. While systemic corticosteroids and other immunosuppressive agents are commonly used as first-line treatments to manage aGvHD, about 30-50% of patients with aGvHD experience inadequately control of disease with first-line therapies, putting them in a risk for poor outcomes and creating a significant clinical challenge . It is estimated that the patients with the most severe forms of refractory aGvHD that do not respond to steroid therapy have expected one-year survival rates of only 5% to 30% . Human mesenchymal stem cells (hMSCs) serve to down-regulate inflammatory responses and produce anti-inflammatory cytokines and growth factors to promote tissue repair . In clinical studies, patients treated with remestemcel-L demonstrated an improvement in their aGvHD and improved survival rates at subsequent days following intravenous infusion . Based on its tolerability and safety profile, remestemcel-L is a promising alternative to second-line immunosuppressive agents .

Indicated for in the management of acute Graft versus Host Disease (aGvHD) in pediatric patients. Acute GvHD should be refractory to treatment with systemic corticosteroid therapy and/or other immunosuppressive agents. Remestemcel-L may be used for Grades C and D of the disease in any organ. Remestemcel-L may also be used in the management of Grade B aGvHD involving any visceral organ, including the GI tract and the liver, but excluding skin.