NIH Microbiota Transplantation Project
Microbiota Transplantation: Recommendations for a Regulatory Framework
Working Group Meeting #1
December 3-4, 2015
Microbiota Transplantation Project Final Report on Meeting #1 (December 3-4, 2015)
Working Group Members and Invited Speakers
Presentations
- Overview of Human Microbiome Project, Dr. L. Clifford McDonald, Senior Adviser for Science and Integrity, Division of Healthcare Quality Promotion, Centers for Disease Control and Prevention
- Emergence of Fecal Microbiota Transplantation and Microbiota Therapeutics, Dr. Alexander Khoruts, Department of Gastroenterology, University of Minnesota Medical School
- Vaginal Microbiota, Dr. Richard Cone, Thomas C. Jenkins Department of Biophysics, Johns Hopkins University
- Skin microbiota transplant overview, Dr. Elizabeth Grice, Penn Institute for Immunology, Perelman School of Medicine, University of Pennsylvania/li>
- Microbiota Transplantation Workshop: Oral Cavity, Dr. Floyd Dewhirst, Department of Microbiology, Forsyth Institute and Harvard School of Dentistry
- Nasal and Sinus Microbiome: the potential for microbiota-targeted therapeutics, Dr. Katherine Lemon, Department of Microbiology, Forsyth Institute and Harvard Medical School
- The Current Regulatory Framework for Fecal Microbiota Transplantation, Rachel Sachs, Academic Fellow, Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics, Harvard Law School
- Alternative Regulatory Framework for FMT: transplantation and implantation of human cells, tissues and cellular and tissue-based products (HCT/Ps), Scott A. Brubaker, CTBS, Senior Vice President of Policy, American Association of Tissue Banks
- Regulatory Schema for Blood Transfusion, Dr. John R. Hess, Professor of Laboratory Medicine and Hematology, University of Washington
Recommended Readings
- Johan S. Bakken [and others], Treating Clostridium difficile Infection with Fecal Microbiota Transplantation, 9 (12) Clinical Gastroenterology and Hepatology 1044-1049 (2011)
- Guidance for Industry: Enforcement Policy Regarding Investigational New Drug Requirements for Use of Fecal Microbiota for Transplantation to Treat Clostridium difficile Infection Not Responsive to Standard Therapies, Draft Guidance, U.S. Dept. of Health and Human Services, Food and Drug Administration, Center for Biologics Evaluation and Research (March 2014)
- Gail A. Hecht [and others], Editorial: What is the Value of a Food and Drug Administration Investigational New Drug Application for Fecal Microbiota Transplantation to Treat Clostridium difficile Infection? 12 Clinical Gastroenterology and Hepatology 289-291 (2014)
- Janis C. Kelly, Fecal Transplants Bring Hope to Patients, Challenge the FDA, Medscape.com (Dec. 15, 2014)
- Francis Megerlin [and others], Faecal Microbiota Transplantation: A Sui Generis Biological Drug, Not a Tissue, 72 (4) Annales Pharmaceutiques Francaises 217-220 (July 2014)
- Elaine O. Petrof & Alexander Khoruts, From Stool Transplants to Next-generation Microbiota Therapeutics, 146 (6) Gastroenterology 1573-1582 (May 2014)
- Margaret F. Riley & Bernat Olle, FDA’s Pathway for Regulation of FMT: Not So Fraught, 10 Journal of Law and the Biosciences 1-5 (2015)
- Rachel E. Sachs & Carolyn A. Edelstein, Ensuring the Safe and Effective FDA Regulation of Fecal Microbiota Transplantation, 10 J. of Law and the Biosciences 396-415 (2015)
- Jeffrey K. Shapiro & Brian J. Wesoloski, FDA’s Regulatory Scheme for Human Tissue: A Brief Overview, Update Magazine (Nov-Dec 2007)
- Mark B. Smith, Colleen Kelly & Eric J. Alm, How to Regulate Faecal Transplants for Medicinal Use, Human Stool Should Be Considered a Tissue, Not a Drug, 506 Nature 290 (Feb. 20, 2014)
- Tim Spector & Rob Knight, Faecal Transplants: Still Need Good Long-Term Trials and Monitoring, 351 BMJ 5149 (Oct 20, 2015)
- Pritish K. Tosh & L. Clifford McDonald, Infection Control in the Multidrug-Resistant Era: Tending the Human Microbiome, 54 Healthcare Epidemiology 707 (2012)
Microbiota Transplantation: Recommendations for a Regulatory Framework
Working Group Meeting #2
May 2-3, 2016
Presentations
- Oral Microbiome Case Study, Dr. Floyd Dewhirst, Department of Microbiology, Forsyth Institute and Harvard School of Dentistry
- Vaginal Microbiome Case Study, Dr. Laura Ensign, Women's Health Program, The Center for Nanomedicine, Johns Hopkins Medicine
- Microbiome Industry Perspective: Janssen Research & Development, Adam Hacker, Vice President, Regulatory Affairs, Janssen Human Microbiome Institute at Janssen Research & Development
- Stool Bank Perspective, Dr. Mark B. Smith, OpenBiome
- Microbiome Industry Perspective: Rebiotix, Lee Jones, Founder, President & CEO of Rebiotix, Inc.
- Legal Frameworks Relevant to Microbiota Transplantation, David Clissold, Hyman, Phelps, & McNamara
- Is FMT a Drug?, Lance Shea, BakerHostetler
Recommended Readings
- FDA Guidance re: use of FMT to treat C. diff, March 2016. [Open Access]
- Blogpost in Harvard Petrie-Flom Center’s Bill of Health Blog, FDA Announces Draft Guidance That Would Limit Enforcement Discretion for FMT, Rachel Sachs. [Open Access]
- Dominguez-Bello M et al, Partial restoration of the microbiota of cesarean-born infants via vaginal microbial transfer, Nature Medicine Volume: 22 (2016)
- Cunnington AJ, Editorial: Vaginal seeding” of infants born by caesarean section, BMJ 2016 (Published 23 February 2016)
- Pozhitkov AE et al, Towards microbiome transplant as a therapy for periodontitis, BMS Oral Health 2015 Oct 14: 15. [Open Access]
- Ravel, J et al, Translating the vaginal microbiome: gaps and challenges, Genome Medicine2016. [Open Access]
- Fourie, N et al, The microbiome of the oral mucosa in irritable bowel syndrome, Gut Microbes, Vol. 6, Issue 4, July/August 2015
- Lee C et al, Frozen vs. fresh fecal microbiota transplantation and clinical resolution of diarrhea in patients with recurrent C. diff, JAMA2016: 315(2). [Open Access]
- Moayyedi P et al, Fecal microbiota transplantation induces remission in patients with active ulcerative colitis in a randomized controlled trial, Gastroenterology, July 2015, Volume 149, Issue 1
- Blanton L et al, Gut bacteria that prevent growth impairments transmitted by microbiota from malnourished children, Science, 19 February 2016, Vol 351, Issue 6275. [Open Access]
- Schwarzer, M et al, Lactobacillus plantarum strain maintains growth of infant mice during chronic undernutrition, Science 19 February 2016, Vol 351, Issue 6275. [Open Access]
Microbiota Transplantation: Recommendations for a Regulatory Framework
Working Group Meeting #3
Dec. 12-13, 2016
Microbiota Transplantation Project Final Report on Meeting #3
Third Working Group Meeting Agenda
Third Working Group Meeting Participant List
Presentations
- Review of Public Comment to FDA March 2016 FMT Guidance
- FMT Registry Talk, Drs. Gary Wu and Colleen Kelly
- Review of RCTs involving FMT, Drs. Erik von Rosenvinge and Colleen Kelly
- International FMT Regulatory Frameworks, Carolyn Edelstein
- Hypothetical Skin MT Case Study Presentation, Dr. Elizabeth Grice
- Hypothetical Skin MT Case Study and questions, Dr. Elizabeth Grice
- Hypothetical Nasal MT Case Study Presentation, Dr. Katherine Lemon
- Hypothetical Nasal MT Case Study and questions, Dr. Katherine Lemon
- Vaginal Gauze Microbiota Transplantation Case Study, Dr. Jacques Ravel
- Microbiota Transplantation to Treat Malnutrition Case Study Presentation, Virginia Rowthorn
- Microbiota Transplantation to Treat Malnutrition Case Study and questions, Virginia Rowthorn
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