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In June, after one patient died and another patient from a fecal transplant containing a drug-resistant bacterium, another patient fell ill, the Food and Drug Administration intervened and fixed new guidelines for the procedure.
The guidelines specified that donors and their stools should be screened for the presence of "multidrug-resistant organisms". They were included in an alert issued by the agency stating that the two patients who became ill had a weakened immune system and that the stools they received had not been tested for the specific superbug that had made them sick.
But no additional information about the cases was provided, such as how the stool was treated, how it was distributed to patients or what it was used to treat.
The announcement raised more questions than she answered. Chief among them: what happened exactly in both cases? And, given the growing threat posed by drug-resistant bacteria, why are these guidelines not already in place?
To the frustration of many doctors, the FDA was silent on the details of the cases. Beyond the short security alert, the agency did not provide additional information, probably because the patients were part of a clinical trial, which meant that this information was confidential.
As to the question of why there were no guidelines, we have to go back to the long history of the procedure.
History of fecal transplants
Until several years ago, fecal microbiota transplants, or FMTs, were completely unregulated. The procedures were considered to be part of the "practice of medicine", a rubric allowing doctors to use unapproved treatments that do not even have long experience.
But the FMTs have a long record. As early as the 1950s, American doctors began transplanting the stools of healthy donors into patients with life-threatening diarrhea caused by infection with a nasty bacterium called C. difficile. The transplants worked and the patients improved.
The reason for the success of the procedure is what first led the patients to become ill: all had a history of extensive antibiotic treatment.
"The doctors explained that the problem was that antibiotics had killed normal microbes in the gut and somehow disrupted the normal ecosystem, allowing bad bacteria to thrive," said Dr. Alexander Khoruts, gastroenterologist and professor of medicine at the University. from Minnesota. "They thought, maybe it could be reversed by adding [gut bacteria] healthy people through the enemas. It worked spectacularly well. "
The success of the treatment began to be talked about and the country's doctors began to use this technique, even though no clinical trial has proved its value.
This changed in January 2013 when a study published in the New England Journal of Medicine showed that the FMT was so effective at treating C. diff that the researchers had to stop recruiting new patients for their clinical trial because it would be unethical to have a control group did not receive the treatment.
In the meantime, specialists have begun to modify the treatment of the stool given. "Essentially, all of the food, fiber, and other materials are filtered and only the fluid containing the bacteria remains," said Dr. Daniel Uslan, Clinical Responsible for Infectious Diseases at UCLA Health.
Nowadays, FMT can also be administered orally: doctors have refined the process so that bacteria can enter a capsule that patients can swallow.
In 2012, Open Biome, a not-for-profit company, began collecting and treating the stool of donors, selling their "product" to gastroenterologists who did not want to do it themselves.
However, the FDA still had not established guidelines on what doctors should look for in donors and their stools before performing transplants. While many claimants verified some pathogens, it was also assumed that if the donor was in good health, his stool probably did not contain any dangerous bacteria.
Drug designation
After the publication of the New England Journal of Medicine article, the FDA intervened.
In February 2013, the agency organized a workshop that ultimately concluded that the best way to proceed would be to designate the FMT as an experimental drug, which could only be used in clinical trials. (Until a drug is approved by the FDA, it can only be used as part of clinical trials.)
The decision provoked a brutal reaction from specialists who knew that many patients requiring treatment could not participate in these trials.
In response, the FDA sold slightly. The agency has decided to allow, through so-called "enforcement discretion", the use of the FMT outside of clinical trials for a specific group of patients: people with C. diff infections that have not disappeared with standard antibiotic therapy. Doctors, for their part, had to explain the potential risks of the procedure.
At that time, no one, including the FDA, knew exactly what these risks might be – in fact, unproven, unproven treatment is still a concern – but, according to Khoruts, the main concern of the FDA was the risk of infection.
Despite the discretionary application, many specialists were unhappy to see the treatment referred to as a drug because it meant that it was possible that in the future, companies would develop products marketed as substitutes for the microbiome and charge such a burden that some patients would not. able to afford it. (Nevertheless, the current treatment may be expensive: Open Biome, for example, charges between $ 1,595 and $ 1,950 for transformed stool.)
According to Khoruts, another solution would be to approach FMTs like organ transplants. This means that doctors would continue to treat patients, some even doing their own studies, but without FDA regulation and with a lower likelihood that a pharmaceutical company will enter the table.
From the FDA's perspective, however, the drug designation was just one way to keep an eye on this growing field.
"We determined that it made more sense to regulate it as a biologic drug," said Dr. Peter Marks, director of the FDA's Center for Biologics Evaluation and Research. "And in many ways, it looks like a drug. Is it the perfect analogy? I will be the first to admit that it is not perfect. "
With the drug designation, the therapy was more likely to be tested as part of rigorous clinical trials, said Marks. In these trials, its efficacy and safety should be evaluated, and scientists could study the best ways to administer the treatment.
"There are a lot of things in the history of medicine where the preliminary data seemed good, but when there were randomized controlled trials, they turned out not to help people and potentially hurt them," he said. Marks told NBC News. "We want to protect public health while encouraging people to get the treatments they need."
There is no doubt that the popularity of FMT has risen sharply in recent years. On clinicaltrials.gov, there are more than 300 recorded trials on the FMT in a wide variety of conditions, many of which go far beyond the intended use, including transplant rejection, obesity and the cancer, as well as some of the most anticipated gastrointestinal reactions. disorders such as irritable bowel syndrome and ulcerative colitis.
For Dr. Ari Grinspan, increased surveillance is welcome, especially after a death involving an FMT.
"What I remember is that a lot of us in the field have been a bit cavalier with our use," said Grinspan, an assistant professor of medicine at the Gastroenterology Division of the University of Toronto. Icahn Medical School of Mount Sinai. "It's a bit like blood donation in the 1980s when we did not know we should be screened for hepatitis B or HIV."
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