Results from an early-phase clinical trial of a commercial preparation of urinary-derived human chorionic gonadotropin (uhCG) showed benefit as adjunctive treatment of patients with acute graft-versus-host disease (aGVHD). These findings were reported in Blood Advances.

aGVHD occurring after receipt of allogeneic hematopoietic cell transplantation (HCT) for the treatment of hematologic malignancies, such as acute leukemia, involves attack by activated donor T cells on host epithelial cells. This immune-mediated complication is common in the setting of allogeneic HCT, and has a high associated mortality rate.

Although corticosteroids are considered the standard-of-care treatment for aGVHD, only approximately half of patients respond to this approach. Most of those with corticosteroid-refractory aGVHD receive intensive immunosuppression as a second-line approach, although only a minority of these patients achieve a complete response (CR) with this treatment, and long-term survival is low in this setting.

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“This fact suggests that impaired healing of damaged tissues, not solely excessive immune activation, may also contribute to the poor outcomes after severe aGVHD,” the study authors posited, leading them to suggest that “interventions to facilitate healing might be beneficial in life-threatening aGVHD.”


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Toward that end, the study authors evaluated a relatively low-cost, commercial preparation of uhCG known to be contaminated with EGF as an approach to promote healing of tissue damage. Furthermore, uhCG is also known to facilitate development of maternal/fetal tolerance through promotion of regulatory T-cell (Treg) expansion.