Fecal transplants pose challenge for FDA regulators

WASHINGTON – Imagine a low-cost treatment for a life-threatening infection that could cure up to 90 percent of patients with minimal side effects, often in a few days.

It may sound like a miracle drug, but this cutting-edge treatment is profoundly simple – though somewhat icky: take the stool of healthy patients to cure those with hard-to-treat intestinal infections. A small but growing number of physicians have begun using these so-called fecal transplants to treat Clostridium difficile, commonly referred to as C-diff, a bacterial infection that causes nausea, cramping and diarrhea. The germ afflicts a half-million Americans annually and kills about 15,000 of them.

But fecal transplants pose a challenge for the Food and Drug Administration, which has decided to regulate the treatment as an experimental drug. Stool transplants don’t fit neatly into the agency’s standard framework. And while regulators have shown flexibility in their approach, some critics say the mere presence of government oversight is discouraging many doctors from offering transplants. That’s led some patients to seek out questionable “do-it-yourself” websites, forums and videos.

Most researchers agree that the FDA’s concerns are warranted. Patients can contract HIV, hepatitis and other viruses and parasites from fecal matter that is not properly screened.

FDA officials declined to be interviewed for this story but said in a written response that the fecal transplantation “shows promise in treating C. difficile infection that has not been responsive to other therapies.”

Indeed, with many patients no longer responding to potent antibiotics, fecal transplants have emerged as an effective therapy against drug-resistant strains of the C-diff superbug. The procedure works because the healthy bacteria found in donors’ feces can help fight off foreign infections.

“We’re dealing with something that is pretty close to miraculous,” says Dr. Lawrence Brandt of New York’s Montefiore Medical Center, who has performed over 200 fecal transplants.

The FDA announced last May that it would regulate stool transplants as an experimental drug, meaning doctors could only perform transplants under an FDA-approved research application.

“FDA and some others are concerned about the long-term effects,” Brandt said. “But my point was these people are getting ready to die now. They are not going to survive long enough to develop the diseases you’re afraid they’re going to get.”

A few weeks later, the FDA revised its position, saying it would not enforce the requirement for doctors treating patients with drug-resistant C-diff – provided donors are properly screened and patients are informed that fecal transplants are still experimental.

But regulating stool samples as a drug presents other challenges. While it’s easy to limit access to experimental drugs, everyone has access to stool. And with detailed instructions available online, there’s nothing to stop patients from trying the procedure at home.

“Some of these patients are very desperate and they’re not going to take no for an answer,” says Dr. Michael Edmond of Virginia Commonwealth University, who has performed fecal transplants for patients who travel from as far away as Ohio.

According to a list maintained by the Fecal Transplant Foundation, only about 100 physicians offer fecal transplants in the U.S.

Another big hurdle for patients is the high cost of screening a stool sample, which can run up to $1,500 per sample. Insurance typically doesn’t cover testing because donors are usually healthy without signs of sickness.