A New Kind of Transplant Bank

 

By Peter Andrey Smith

Feb. 17, 2014

CAMBRIDGE, MASS. — Around noon on a recent Friday, Donor Five, a healthy 31-year-old, walked across M.I.T.’s frigid, wind-swept campus to a third-floor restroom to make a contribution to public health.

Less than two hours later, a technician blended the donor’s stool into preparations that looked like chocolate milk. The material was separated and stored in freezers at an M.I.T. microbiology lab, awaiting shipment to hospitals around the country. Each container was carefully labeled: Fecal Microbiota Preparation.

Nearly a year ago, Mark Smith, a 27-year-old doctoral candidate, and three colleagues launched OpenBiome, the nation’s first human stool bank. Its mission: to provide doctors with safe, inexpensive fecal material from screened donors to treat patients with Clostridium difficile, a gastrointestinal infection that kills at least 14,000 Americans a year.

“People are dying, and it’s crazy because we know what the solution is,” Mr. Smith said. “People are doing fecal transplants in their basements and may not be doing any of the right screening or sterile preparation. We need an intermediate solution until there are commercial products on the market.”

C. diff, as it’s known, resides among trillions of other bacteria in normal, healthy humans. When antibiotics wipe out the competition, the bacteria can produce toxins, causing persistent diarrhea.

The bacteria are increasingly resistant to conventional treatments. But researchers have discovered an alternative: A donor’s stool can be transplanted in the intestine or colon of a sick patient via an enema, colonoscopy or nasogastric tube. The healthy bacteria fight off C. diff and re-establish a normal community in the gut.

A study published last year in The New England Journal of Medicine found that fecal transplants were nearly twice as effective as antibiotics in treating patients with recurring C. difficile.

But where to get healthy donor stool? For doctors, it’s a tedious, time-consuming process, and some patients turn awkwardly to relatives or friends. Since September, OpenBiome has delivered more than 135 frozen, ready-to-use preparations to 13 hospitals. The nonprofit project fields dozens of requests from doctors, hospitals and patients every week. (The preparations are not sent directly to patients.)

Carol Capps, 75, a retired nurse in Clemmons, N.C., had been in and out of hospitals for months with a C. diff infection that was not going away despite multiple courses of antibiotics. After a recurrence, her doctor suggested OpenBiome, and she received a fecal transplant. By that afternoon, Ms. Capps said, she felt like a new person and has been healthy since.

“I am just thankful for whoever donates,” she said. “It’s such a miracle.”

Despite the promise, the Food and Drug Administration has grappled with the regulation of fecal transplants. In early 2013, the agency announced that it would treat them as biologic drugs requiring an Investigational New Drug application, which typically precedes a clinical trial.

Following outcry from patients and doctors, the F.D.A. announced it would “exercise enforcement discretion” — in effect, saying that it would not go after physicians performing fecal transplants for C. diff. Jennifer Rodriguez, an F.D.A. spokeswoman, said the agency planned to issue industry guidelines later this year.

Because of the legal ambiguity, some researchers are not preparing fecal microbiota for sale (usually at cost) — including Dr. Alexander Khoruts, a gastroenterologist at the University Minnesota, who first published the methodology for doing so.

Dr. Khoruts worries that approval of OpenBiome’s efforts will slow the development of next-generation therapeutics beyond the crude preparations available today. “We desperately need some clarity,” Dr. Khoruts said. “We’re going to be stuck with the OpenBiome model, and nothing better’s going to come along.”

At the same time, Mr. Smith and Eric J. Alm, an M.I.T. microbiologist and adviser to OpenBiome, said the F.D.A.’s classification of fecal transplants as drugs hinders research into their possible uses to treat inflammatory bowel diseases and obesity.

“It’s going to give a monopoly to whatever company gets the drug approved,” Mr. Smith said.

“We think it should be regulated, but unlike most products the F.D.A. oversees, there’s a real risk of the black market,” he said. “If you restrict access, there’s going to be lots of people doing it underground.”

Read the full piece here.