The Impact of Regulatory Policies on the Future of Fecal Microbiota Transplantation

2019 ◽  
Vol 47 (4) ◽  
pp. 482-504 ◽  
Author(s):  
Alexander Khoruts ◽  
Diane E. Hoffmann ◽  
Francis B. Palumbo
Keyword(s):  
Fecal Microbiota Transplantation ◽  
Drug Approval ◽  
Fecal Microbiota ◽  
Regulatory Decision ◽  
Regulatory Policies ◽  
Research And Innovation ◽  
Recurrent Clostridium Difficile Infection ◽  
Potential Impact ◽  
Approved Drugs ◽  
The Impact

In this article, the authors explore the impact of a potential future regulatory decision by FDA whether or not to continue its enforcement discretion policy allowing physicians to perform, and stool banks to sell, stool product for fecal microbiota transplantation as a treatment for recurrent Clostridium Difficile infection without an Investigative New Drug (IND) application. The paper looks at the Agency's regulatory options in light of the current gut microbiota based products that are in the FDA pipeline for drug approval and the potential impact and repercussions of their approval on FDA action. In laying out FDA's options we consider the implications of market exclusivity and off-label use of newly approved drugs. Ultimately, we explore the potential impact of FDA's decision on patients, research, and innovation.

scholarly journals Gut microbiota density influences host physiology and is shaped by host and microbial factors

eLife ◽  
2019 ◽  
Vol 8 ◽  
Author(s):  
Eduardo J Contijoch ◽  
Graham J Britton ◽  
Chao Yang ◽  
Ilaria Mogno ◽  
Zhihua Li ◽  
...  
Keyword(s):  
Gut Microbiota ◽  
Carrying Capacity ◽  
Ileal Pouch ◽  
Fecal Microbiota Transplantation ◽  
Fecal Microbiota ◽  
Therapeutic Interventions ◽  
Editorial Note ◽  
Targeted Therapeutics ◽  
Recurrent Clostridium Difficile Infection ◽  
The Impact

To identify factors that regulate gut microbiota density and the impact of varied microbiota density on health, we assayed this fundamental ecosystem property in fecal samples across mammals, human disease, and therapeutic interventions. Physiologic features of the host (carrying capacity) and the fitness of the gut microbiota shape microbiota density. Therapeutic manipulation of microbiota density in mice altered host metabolic and immune homeostasis. In humans, gut microbiota density was reduced in Crohn’s disease, ulcerative colitis, and ileal pouch-anal anastomosis. The gut microbiota in recurrent Clostridium difficile infection had lower density and reduced fitness that were restored by fecal microbiota transplantation. Understanding the interplay between microbiota and disease in terms of microbiota density, host carrying capacity, and microbiota fitness provide new insights into microbiome structure and microbiome targeted therapeutics.Editorial note: This article has been through an editorial process in which the authors decide how to respond to the issues raised during peer review. The Reviewing Editor's assessment is that all the issues have been addressed (<xref ref-type="decision-letter" rid="SA1">see decision letter</xref>).

scholarly journals Prospective, Open-label Trial to Evaluate Efficacy of Lyophilized Fecal Microbiota Transplantation for Treatment of Recurrent C. difficile Infection

2017 ◽  
Vol 4 (suppl_1) ◽  
pp. S383-S384
Author(s):  
Peyman Goldeh ◽  
Peter Kim ◽  
Salaheddin Abouanaser ◽  
Eric Partlow ◽  
Patricia Beckett ◽  
...  
Keyword(s):  
Fecal Microbiota Transplantation ◽  
Controlled Trial ◽  
Similar Efficacy ◽  
Fecal Microbiota ◽  
Efficacy And Safety ◽  
Open Label ◽  
Retention Enema ◽  
Recurrent Clostridium Difficile Infection ◽  
Laboratory Facility ◽  
History Of

Abstract Background Fecal microbiota transplantation (FMT) has shown to be effective for recurrent Clostridium difficile infection (rCDI). However, significant laboratory costs for donor screening and a lack of suitable donors and laboratory facility have restricted the availability of the treatment. In order to expand access to FMT, we have investigated the efficacy of lyophilized FMT, comparing it to the published historical efficacy of frozen FMT in preventing further episodes of CDI in patients with a history of rCDI. This study was designed to be open-labeled to expedite and minimize costs associated with conducting a two-armed randomized controlled trial, given that the efficacy of frozen FMT is known to be 85%. Additionally, using lyophilized FMT offers two major advantages: 1) its prolonged shelf life reduces cost because fewer donors need to be screened; and 2) it can be transported without freezing. Methods This is an open-labeled, prospective study involving 50 patients with a history of 2 or more rCDI who have failed at least 1 course of tapered vancomycin therapy. Eligible patients received 2 lyophilized FMT via retention enema within 8 days of each treatment and were followed for 13 weeks post last FMT to determine efficacy and safety of FMT. Results The efficacy of lyophilized FMTs in preventing further episodes of CDI in patients with rCDI was 80%. The adverse events associated with lyophilized FMT were similar to frozen FMT. Conclusion Lyophilized FMT in treating rCDI showed similar efficacy and safety to frozen FMT. Lyophilized FMT appears to be promising in preventing further episode of CDI and increasing accessibility for patients with rCDI. Disclosures All authors: No reported disclosures.

scholarly journals Insights into the Impact of Microbiota in the Treatment of NAFLD/NASH and Its Potential as a Biomarker for Prognosis and Diagnosis

Biomedicines ◽  
2021 ◽  
Vol 9 (2) ◽  
pp. 145
Author(s):  
Julio Plaza-Díaz ◽  
Patricio Solis-Urra ◽  
Jerónimo Aragón-Vela ◽  
Fernando Rodríguez-Rodríguez ◽  
Jorge Olivares-Arancibia ◽  
...  
Keyword(s):  
Gut Microbiota ◽  
Fecal Microbiota Transplantation ◽  
Liver Steatosis ◽  
Intestinal Barrier ◽  
Fecal Microbiota ◽  
Current Treatment ◽  
Immune Defense ◽  
Alcoholic Fatty Liver ◽  
The Impact

Non-alcoholic fatty liver disease (NAFLD) is an increasing cause of chronic liver illness associated with obesity and metabolic disorders, such as hypertension, dyslipidemia, or type 2 diabetes mellitus. A more severe type of NAFLD, non-alcoholic steatohepatitis (NASH), is considered an ongoing global health threat and dramatically increases the risks of cirrhosis, liver failure, and hepatocellular carcinoma. Several reports have demonstrated that liver steatosis is associated with the elevation of certain clinical and biochemical markers but with low predictive potential. In addition, current imaging methods are inaccurate and inadequate for quantification of liver steatosis and do not distinguish clearly between the microvesicular and the macrovesicular types. On the other hand, an unhealthy status usually presents an altered gut microbiota, associated with the loss of its functions. Indeed, NAFLD pathophysiology has been linked to lower microbial diversity and a weakened intestinal barrier, exposing the host to bacterial components and stimulating pathways of immune defense and inflammation via toll-like receptor signaling. Moreover, this activation of inflammation in hepatocytes induces progression from simple steatosis to NASH. In the present review, we aim to: (a) summarize studies on both human and animals addressed to determine the impact of alterations in gut microbiota in NASH; (b) evaluate the potential role of such alterations as biomarkers for prognosis and diagnosis of this disorder; and (c) discuss the involvement of microbiota in the current treatment for NAFLD/NASH (i.e., bariatric surgery, physical exercise and lifestyle, diet, probiotics and prebiotics, and fecal microbiota transplantation).

Irritable bowel syndrome and transplantation of fecal microbiota. Review

2021 ◽  
Author(s):  
G. D. Fadieienko ◽  
O. Y. Gridnyev
Keyword(s):  
Irritable Bowel Syndrome ◽  
Fecal Microbiota Transplantation ◽  
Positive Outcome ◽  
Fecal Microbiota ◽  
Cochrane Library ◽  
Placebo Control ◽  
Global Improvement ◽  
Recurrent Clostridium Difficile Infection ◽  
Significant Difference ◽  
Irritable Bowel

Objective — to perform analysis of the literature data as regards the efficacy of the use of fecal microbiota transplantation (FMT) in patients with the irritable bowel syndrome (IBS). Materials and methods. The systematic review of the literature (PubMed, MEDLINE, Cochrane Library) has been performed to assess the results of investigation on the use of FMT in adult IBS patients. Results. Currently, FMT is a safe and highly successful method of treatment for recurrent Clostridium difficile infection, and possibility to use FMT in the treatment of other diseases is under investigation. The results of FMT treatment of patients with IBS are contradictory. A number of authors outline fairly high FMT efficacy when used for the treatment of patients with IBS, refractory to the standard methods: it varies from 36 % to 70 % with a duration of adequate symptoms’ relief up to 1 year. However, other authors indicate that the positive FMT clinical effect lasted from 8 weeks to 3 months, after which there was a gradual recovery of symptoms. At the same time, the researchers noted that mostly often the positive outcome was reached after FMT from donors with a higher number of streptococci in the feces. Still others point out that there was no sustained, at least 50‑point reduction in the severity of IBS symptoms from baseline. Some authors did not reveal the advantages of FMT over placebo control and even reported about the higher placebo efficacy. After data generalization no significant difference was revealed in the global improvement in IBS symptoms in patients receiving donor FM versus placebo. However, the attention is drawn to the relationship between the results of FMT with methodology. Indeed, several studies have shown that placebo capsules were more effective than capsules containing donor feces, whereas FMT from donor stool delivered by colonoscopy was superior to FMF from autologous stool. This may be due to the excipients, containing in the capsulated FMT. A number of researchers also indicated that the use of a super donor is necessary for successful FMT and that the response to FMT is dose dependent. Conclusions. To resolve the issue of the expediency of using FMT in routine clinical practice in patients with IBS, more qualitative controlled studies, involving large cohorts of IBS patients and long‑term follow‑up, are required.  

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