NCI Convenes Workshop on Reproducibility of Fecal Microbiota Transplants in Cancer Therapeutics

A 2016 NCI workshop on the “State of the Science: Cancer Complementary and Alternative Medicine Therapeutics Research” and the recent upsurge in the field of gut microbiota research suggest that the microbiome plays a role in cancer therapeutic outcomes. This may be especially important for patients receiving anti-PD-1 immunotherapy. To explore the potential role that fecal microbiota transplants (FMT) and pre/probiotics may play in cancer therapeutics and to address reproducibility and safety to inform translational human studies and clinical trials, NCI convened a “Strategic Workshop on Rigor and Reproducibility: Precision Fecal Microbiota Transplant and Microbiome Cancer Therapeutics” on September 5, 2019 (agenda). This timely meeting brought together scientists and FDA regulatory staff in the fields of FMT, pre/probiotics, nutrition, microbiome, synthetic biology, biomarkers, computation, immunology, gastroenterology, oncology, and infection.

Workshop Goals

• Assess the current state of clinical research and clinical trials involving FMT and microbiome-based cancer therapeutics
• Discuss the knowledge gaps and future opportunities in the field
• Provide feedback to NCI and NIH regarding future priority areas to enhance precision- and mechanism-based rigor and reproducibility of defined microbiome-based therapeutic clinical research for cancer and other diseases

Workshop Focus Areas

• Standardization (material manufacture and quality control), FMT donor selection, microbiome measurement, clinical protocols, procedures, and trial design
• Microbiome composition and function as biomarkers associated with cancer therapy, such as reproducibility, safety, and efficacy
• Confounding variables — host genetic and disease state, environment, antibiotics, diet and other lifestyle factors
• Clinical efficacy and safety of FMT and microbiome-based cancer therapy
• Clinically relevant models and human translational studies
• Data sharing
• FDA regulatory issues

Workshop Highlights

• Human and mouse preliminary data demonstrated possible immunotherapy benefits from providing FMT from anti-PD1 responders to some non-responders.
• In mice, a fiber-rich diet improved anti-PD-1 immunotherapy, and an association was observed in humans along with improvement in gut microbiome diversity.
• A collaboration with the FMT national registry was suggested to include cancer patients for long-term safety and outcome follow up.
• Coordinated, collaborative, multi-center cancer therapeutic FMT clinical trials were endorsed.
• A network to develop a Human Cancer Immunotherapy Fecal Microbiome Atlas or Biobank was proposed.

For more information on this workshop, contact Dan Xi, PhD (xida@mail.nih.gov).