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Updated: Nov 26, 2025
Therapeutic Evaluation of Fecal Microbiota Transplantation in an Interleukin 10-Deficient Mouse Model
Published on: April 6, 2022
Fecal microbiota transplant promotes response in immunotherapy-refractory melanoma patients
Erez N Baruch1,2, Ilan Youngster3,4, Guy Ben-Betzalel5
1The Ella Lemelbaum Institute for Immuno-Oncology, Sheba Medical Center, Tel-HaShomer, Israel. erezbaruch@mail.tau.ac.il gal.markel@sheba.health.gov.il.
Abstract:
The gut microbiome has been shown to influence the response of tumors to anti-PD-1 (programmed cell death-1) immunotherapy in preclinical mouse models and observational patient cohorts. However, modulation of gut microbiota in cancer patients has not been investigated in clinical trials. In this study, we performed a phase 1 clinical trial to assess the safety and feasibility of fecal microbiota transplantation (FMT) and reinduction of anti-PD-1 immunotherapy in 10 patients with anti-PD-1-refractory metastatic melanoma. We observed clinical responses in three patients, including two partial responses and one complete response. Notably, treatment with FMT was associated with favorable changes in immune cell infiltrates and gene expression profiles in both the gut lamina propria and the tumor microenvironment. These early findings have implications for modulating the gut microbiota in cancer treatment.
Insights
Fecal microbiota transplantation (FMT) combined with anti-programmed cell death-1 (PD-1) immunotherapy showed promising results in patients with advanced melanoma. This approach modulated the gut microbiome and immune response, offering new hope for cancer treatment.
Area of Science:
- Oncology
- Immunology
- Microbiology
Background:
- The gut microbiome influences responses to anti-programmed cell death-1 (PD-1) immunotherapy in preclinical and observational studies.
- Clinical trials investigating gut microbiota modulation in cancer patients receiving immunotherapy are lacking.
Purpose of the Study:
- To assess the safety and feasibility of fecal microbiota transplantation (FMT) combined with reinduction of anti-PD-1 immunotherapy.
- To evaluate clinical responses and immune microenvironment changes in patients with anti-PD-1-refractory metastatic melanoma.
Main Methods:
- Phase 1 clinical trial involving 10 patients with anti-PD-1-refractory metastatic melanoma.
- Administration of fecal microbiota transplantation (FMT) followed by reinduction of anti-PD-1 immunotherapy.
Main Results:
- Clinical responses observed in 3 out of 10 patients (2 partial responses, 1 complete response).
- FMT treatment led to favorable changes in immune cell infiltrates and gene expression in the gut and tumor microenvironment.
Conclusions:
- Fecal microbiota transplantation (FMT) is a feasible approach to modulate the gut microbiome in cancer patients.
- This strategy shows potential for enhancing anti-PD-1 immunotherapy efficacy in refractory melanoma.
