Fecal Pills: a New Niche

When NYU Langone Medical Center gastroenterologist Lee Ann Chen, MD needs frozen stool, she puts in an order with OpenBiome, a two-year-old Massachusetts non-profit whose sole mission is to provide them.

Chen is among a growing number of physicians who use fecal microbiota transplantation (FMT) as a way of treating Clostridium difficile (C. diff).

With nearly half a million cases of C. diff reported annually in the US, and FMT’s cure rates of about 90%, the therapy has also created a new niche market: companies that collect, prepare, and sell stool.

Initially FMT patients were left to their own devices when identifying their own donors.

OpenBiome, a Massachusetts nonprofit company, organizes a pool of healthy donors from whom to collect rigorously screened stool.

The materials are all filtered, frozen, and shipped to their myriad of clients — physicians, hospitals, and researchers.

In a video interview with HCPLive, Chen said she uses OpenBiome and commented on her team’s highly stringent set of criteria for donor stool. Those include screening donors for infectious diseases in their stool and blood, asking detailed questions about risky behaviors, travel history, and essentially requiring their donors not to have GI conditions of their own.

“We want our donors to be, more or less, completely healthy,” Chen said.

OpenBiome is one of several US and Canada enterprises focused on easing the FMT process.

Carolyn Edelstein, Director of Global Partnerships at OpenBiome explained her organization’s 2012 origin.

“When our friend Johnny fell ill with C. difficile, it made life very, very difficult for him,” she said, “He tried many rounds of antibiotics, but the infection kept coming back.” C. diff cure rates with antibiotics are about 30% to 50%.

Edelstein continued, “When Mark Smith, one of our co-founders, heard about Johnny’s story, he thought it was completely crazy that patients couldn’t get access to the therapy from medical providers, so were doing risky at-home treatments,”

Smith, a PhD microbiologist from MIT, did research that showed that frozen stool was just as effective as fresh stool for treating recurrent C. diff, she said. “That lent itself quite nicely to a stool-banking service, which could screen a suite of universal donors much more rigorously, and collect and prepare the material ahead of time,”

The development made it easier for doctors to provide the treatment.

“Patients like our friend could get the help they needed,” Edelstein said.

The OpenBiome donor screening process is pretty extensive — only about 4% of people pass the full set of screens. First, a prospective donor meets with a physician or nurse for a clinical assessment that follows a 109 item questionnaire.

If they pass this round, they are screened using 27 serological and stool-based assays, (for conditions that include HIV, C. diff, Hepatitis A, B, and C, rotavirus, ova, and parasites.) Once they pass the screens, they officially become qualified donors, and donate stool over a maximum 60-day collection window. All OpenBiome donors live or work very close to the lab, so that they can come in easily to make donations — they earn $40 per donation.

Chen said having prescreened stool makes the FMT process easier for her and patients—including explaining the details of FMT procedures. Pills make FMT far less invasive, she said.

In the video interview she discussed the latest developments in FMT, including the government’s regulation of the treatment.

Initially, the US Food and Drug Administration (FDA) had not been regulating FMT.

The agency only entered into the area within the last year. Classifying FMT as a biologic therapy, the FDA required any potential user to complete an investigational new drug (IND) application— paperwork pharma companies are familiar with, not clinicians. The requirement stopped FMT in the US for some time.

Then a meeting at the National Institutes of Health (NIH) spurred conversation on utilizing the life-saving method of FMT, leading to the FDA agreeing to “exercise enforcement discretion if the indication is for C. diff, while other indications will require IND paperwork,” said Chen.

Pills should make the whole process even easier for clinicians and patients.

Receiving frozen, ready-to-use fecal microbiota preparations can eliminate many of the logistical and administrative hurdles that made offering FMT so difficult. It will be far simpler and less invasive for patients to receive the therapy orally rather than through an ND tube, colonoscopy, or enema.

Competition in the FMT business appears to be leading to better products for patients and the healthcare system as a whole.

Among the companies working to do that:

Pureflora, Inc., San Francisco, CA, a medical device company, sponsored a Dartmouth College engineering team, which particpated in an NIH competition. Their invention, called a “Microflora Refinement System” took first place. It automated the preparation of donor feces, ultimately streamlining the process.

The resulting product can be used to deliver FMT orally by capsules as well as by the older methods of enema and colonoscopy.

Though a website isn’t developed yet, the system does have a patent number, WO2013090825, described under the patent title “Device for the collection, refinement, and administration of gastrointestinal microflora.”

Also on the rise is Repoopulate. This product with the clever moniker was developed at the University of Guelph, Ontario, Canada. It is artificial fecal product that includes 33 species of the bacteria found in the colon. Though it’s not yet commercially available, the product has twice been successfully used to treat C. diff.

That is a welcome development, said Edelstein.

“We’ve also said quite often that whole stool preparation is likely just a bridge solution to synthetic microbiota preparations (cultures of microbial communities, grown in the lab), technology that we think will be even better for patients because of the possibility for even better safety guarantees,” she said.

On the horizon, FMT may be used for other indications. Those could include inflammatory bowel disease, ulcerative colitis, and Crohn’s disease, though as yet there are no clear existing data on efficacy.

EDITOR'S NOTE: The fecal matter NYU orders is not in pill form as stated in an earlier version of this article.