Investigating a Rare Disease Drug to Treat AML and MDS Patients with Post-Stem Cell Transplant Relapse

Principal Investigator: Dr. Sawa Ito​

Disease: Acute Myeloid Leukemia and Myelodysplastic Syndrome

Research Description: Allogeneic hematopoietic stem cell transplantation (alloSCT) can be a curative therapy for patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). However, some AML and MDS patients relapse after alloSCT, and these patients face a dismal prognosis.  No FDA-approved therapy effectively treats or prevents post-alloSCT relapse. Interferon-gamma is approved to treat rare inherited disorders, chronic granulomatous disease and malignant osteopetrosis. Previous animal studies suggest that interferon-gamma could help treat AML after alloSCT through the immune-mediated killing of leukemia cells. The research team has already treated 4 AML/MDS patients who experienced a post-alloSCT relapse with interferon-gamma, and they will now expand this Phase I pilot trial by enrolling additional patients. Data from additional patients could confirm and strengthen existing results on the safety and efficacy of this repurposed treatment and support a subsequent multicenter clinical trial, potentially improving outcomes for AML/MDS patients who relapse post-alloSCT.

Funding Partners: Goldman Philanthropic Partnerships and others

Initial CWR Funding Role: Supplemental

Completed: 2025