Free Enterprise Staff  | June 8, 2015

The Key to a Stunning Medical Breakthrough? Your Poop

The Birth of an Idea

In 2012, James Burgess and Mark Smith learned that a friend of theirs had contracted Clostridium difficile—commonly known as C. difficile—a virulent bacterial infection that, somewhat paradoxically, results from the prolonged use of antibiotics. Once vibrant and healthy, their friend’s health deteriorated over the course of 18 months, as he suffered multiple recurrences. Fortuitously, Smith, Burgress, and a few other friends had previously learned about a revolutionary new medical treatment that could potentially do what standard care had failed to accomplish: cure their friend.

That solution? Poop.

“He was a bit of an unusual case in that, like us, he was a young guy in his mid-20s, just out of school and working in healthcare investment, so he was plugged into the healthcare world,” says Burgess. “He just wasn’t responding to standard treatments, and he actually came to us and said, ‘Hey, what do you know about fecal transplants? I know you’ve been researching them.’ And at that time, fecal transplants weren’t just really broadly available in the medical system.”

Though their friend was able to find a hospital in New York City willing to perform a fecal transplant—a procedure in which fecal matter from a healthy person is transplanted to someone suffering from a gastrointestinal malady such as C. difficile—he would have had to wait six months before undergoing treatment, Burgess says. Unwilling to do that, he ultimately got a donor sample from a friend and performed one himself in his own home. “It’s a pretty unpleasant thing to do at home, and from our view, it’s also risky because that donor is not getting the proper screening,” he says.

After watching their friend’s nearly two-year-long struggle with C. difficile, Burgess, Smith, and a group of friends that included Zain Kassam, a gastroenterologist and fecal transplant researcher, decided to act on their idea to create a stool bank for people suffering from the same condition. Their labor of love, Medford, Massachusetts-based OpenBiome, formalizes the process by which donors are screened and patients undergo fecal transplants. (Donors receive $40 per sample, with those who come in five times per week earning an additional $50, translating into $250 per week—and potentially $13,000 per year.)

Farfetched as it may sound, fecal transplants—we’ll get into the nitty gritty of them later on—are actually among the most promising new medical therapies to appear over the past half-century. The reason? A slew of research suggests that transplanting stool from a healthy person to someone suffering from C. difficile—a procedure formally known as fecal microbiota transplantation (FMT)— is more than 90% effective at eliminating the potentially deadly bacterial infection.

Solving the C. Difficile Problem

C. difficile is no laughing matter. According to the American Gastroenterological Association, nearly 350,000 people are hospitalized each year in the U.S. because of such infections, resulting in up to 30,000 deaths and costing the healthcare system billions of dollars each year. Though antibiotics like vancomycin and fidaxomicin have traditionally been used to treat patients sick with C. difficile, such medications cost thousands of dollars, have serious side effects, and are not always effective, according to the Centers for Disease Control and Prevention.

With U.S. healthcare expenditures totaling just shy of $3 trillion in 2013, innovative organizations like OpenBiome are needed more than ever to solve some of the most vexing problems affecting large swaths of the American patient population. Before opening its bathroom doors, so to speak, OpenBiome devised a thorough, long-form method for screening potential donors, explains Kassam.

Interested donors who e-mail the nonprofit are subsequently placed in a registry, Kassam says, before they’re put through a 109-point clinical assessment that’s performed by a nurse and a physician. Though arduous, the process is intended to effectively eliminate infectious disease risk factors or conditions that may alter the trillions of microbes living in and on the human body collectively known as the microbiome. “That process excludes a shockingly high number of people,” Kassam stresses.

“Anyone who passes then undergoes laboratory investigations, so we test their stool and their blood for 27 different conditions to ensure they’re in complete health,” he says. “Donors provide us with their poop for 60 days under medical monitoring, so if their health history changes they may be asked not to donate anymore. The fecal material that is donated goes into quarantine, and after the 60-day window, donors are required to pass the clinical, stool, and blood tests again. Only if they pass all those tests does the material get released to patients. Safety is OpenBiome’s number one priority, and we have the most comprehensive safety program of anyone in the space.”

The screening procedures are so extensive, Burgess says, that OpenBiome currently has only 16 active donors, representing roughly 4% of total prospective candidates. Yet screening candidates is only a part of what OpenBiome does. Before the organization sends stool samples to the 180 hospital providers it partners with in nearly 40 states, its lab technicians begin the process of preparing stool for transplantation.

Stool is first mixed with a buffer to help protect its bacteria, Kassam says, before it’s put through a homogenization and filtration process that removes fiber and bulk. The resulting sample is then stored at -80 degrees Celsius in a container that resembles a Nalgene bottle.

OpenBiome researchers have calculated how many people benefit from a single stool donation. (Chart courtesy of OpenBiome.)

OpenBiome researchers have calculated how many people benefit from a single stool donation. (Chart courtesy of OpenBiome.)

“We also take safety and research samples from each stool, so heaven forbid there’s an adverse event we can verify if the source was from the fecal transplant or not, and engage our robust safety protocol to trace where any sample has been sent in the U.S.,” Kassam says. “It’s shipped on dry ice to our hospital partners, who can use it immediately or store in freezers for later. The material is then thawed before it’s put to use.”

From there, FMTs can be performed in one of four discrete ways, Kassam explains. “The first and most common is by a colonoscopy,” he says.

“Another is an enema; then there’s also a way using a tube that travels from your nose to your stomach or small bowel; and the last approach, which we’re very excited about and are in the rolling out phase, is the capsule. We’ve developed a frozen, encapsulated pill—a poop pill, so to speak—and have had some resounding successes in early stage validations, so we’ll be rolling that out in the very near future.”

Are Fecal Transplants the Way of the Future?

What does all this mean for the healthcare system and for patients suffering from C. difficile infections? For starters, fecal transplants are vastly less expensive compared to standard antibiotic treatments, according to a study conducted at Massachusetts General Hospital, which pegged the cost of fecal transplants at one-sixth that of traditional antibiotics. For its part, OpenBiome charges only $250 for every treatment it sends to a partner organization, and it also runs a pro bono fund for patients that can’t afford that price.

Fecal transplants also lack the side effects often associated with long-term antibiotic use, the researchers determined, and they’re significantly faster, with only one treatment often needed. That astoundingly high success rate is rarely, if ever, observed in medical science. As Kassam told The New Yorker in an interview: “It’s the closest thing to a miracle I’ve seen in medicine.”

Yet for all its untapped potential, the future of both OpenBiome and fecal transplant technology are not necessarily assured. OpenBiome is currently operating under “enforcement discretion” from the Food and Drug Administration, whose regulation authority the procedure falls under. That regulatory level, Kassam explains, essentially amounts to a yellow light zone, somewhere between green and red.

Still, given the impact that C. difficile has on the medical system, Kassam, Burgress, and their OpenBiome co-founders are confident that their trailblazing path will both become a model for other organizations and ultimately attain full regulatory clearance from the government. Anecdotal evidence does suggest, after all, that fecal transplants may be an effective treatment for a host of other afflictions, including Crohn’s disease, ulcerative colitis, and irritable bowel syndrome.

With OpenBiome continuing to accrue partner organizations and attract interest from both researchers and the public, the nonprofit and its executive team are optimistic about its future. As the saying goes, the proof is in the pudding, and, given the context, the poop is, as well.