A new treatment available in clinical trials may shine the light for certain people living with Crohn’s disease.
Just a year ago, Michael Miceli felt totally depleted from his Crohn’s disease. The 38-year-old dad of two little boys was diagnosed in 1997, when he was a sophomore in high school. He spent the next 20 years trying an array of medications and procedures to get relief from his symptoms. But all of them was a waste of time and money.
“My life was mostly just getting by. I knew nothing else,” he said. “It was visiting the restroom 20, 30 times a day, unforgiving pain everyday. When I would eat, if I did hold it down, nutrients would never be absorbed so it would just go right through me. I really didn’t have much of a quality life.”
Then Miceli realized he qualified for a clinical trial at the Icahn School of Medicine at Mount Sinai in New York City that uses stem cell therapy to treat Crohn’s disease.
Stem cells are cells that can develop into many different types of cells in the body. Stem cell therapy is a treatment that uses these cells to treat a condition by generating healthy cells to replace bad ones.
Doctors have performed stem cell transplants to treat blood-related diseases, including leukemia, lymphoma, and neuroblastoma. Researchers are also testing the therapy for other disorders, including Crohn’s disease.
“How we believe it works is that the stem cells travel to the area of inflammation and release proteins to so-called messengers that then recruit your body’s own cells to restore balance in the immune system,” says Amy Lightner, MD, a colorectal surgeon at Cleveland Clinic.
After going through the procedure, Miceli noticed a difference almost instantly. His symptoms subsided, and he was able to go back to the things he loved doing, like playing with his 9- and 11-year-old sons.
“Having the energy and zeal to do things, I feel like a totally different person,” he says. “It was almost like being reborn.”
Louis Cohen, MD, an assistant professor in the department of medicine at the division of gastroenterology at Mount Sinai, is overseeing the trial.
“In the last 2 decades, treatments for Crohn’s disease have expanded leaps and bounds, but the unfortunate truth is that there’s still more to do,” he says. “Despite all these drugs, somewhere around 20 percent of our patients remain quite symptomatic and debilitated.”
For these patients, he explains, stem cell therapy may be an option.
The History of Stem Cell Therapy to Treat Crohn’s
For decades, Dr. Cohen explains, doctors observed a unique phenomenon in patients who had both Crohn’s disease and cancer. “They would go through a stem cell transplant [for cancer], and then they would come out on the other side with their Crohn’s symptoms feeling a lot better,” he says.
Over the past 10 years, researchers in Europe began studying this phenomenon in a scientific way. In 2015, a study published in The Journal of the American Medical Association (JAMA) called the ASTIC trial considered the safety and effectiveness of stem cell therapy for Crohn’s.
For the study, 23 patients underwent stem cell transplantation to treat their disease and 22 received standard Crohn’s disease treatment for one year.
Sustained remission was achieved in two patients in the stem cell group versus one patient in the control group. Additionally, 14 patients who received stem cell therapy were able to stop taking medication to treat Crohn’s, while only five control patients were able to do the same.
The study “showed that stem cell transplants for Crohn’s disease could really be a life-saving endeavor,” Cohen says.
Since then, more trials have begun around the world, including the one at Mount Sinai.
How Does Stem Cell Therapy Work?
Essentially, stem cell therapy works by resetting the immune system so patients can restart medication that had stopped working as a result of the disease’s progression.
The first step is for patients to undergo an intense screening process to learn if the trial is right for them. For Crohn’s patients, doctors verify that they have tried the standard treatments and still have ongoing disease.
If patients are approved, the treatment begins with a two-week hospital stay, during which their own stem cells are collected. They then spend some time at home before returning for the transplant, which requires another four weeks in the hospital.
During this time, the patients also receive chemotherapy. “The goal is to eradicate all of the immune cells that are populated in the body that we think are causing the problem,” Cohen says. “So when we give them back their own stem cells to help their immune system to recover, what we think is happening in terms of the Crohn’s is that we’re giving them back immune cells that haven’t learned the bad behaviors of Crohn’s disease.”
In the Mount Sinai trial, patients are given the medication Entyvio (vedolizumab) as part of the protocol once the patient’s immune system has regenerated. It belongs to a class of drugs called monoclonal antibodies, which help reduce inflammation.
“One thing that we’ve come to learn from this type of transplant is that it’s not a cure. It can arrest the disease, and we hope what it’s doing is that it resets the disease to a time when it wasn’t so bad. But for most patients who go through the transplant, the disease does come back. We hope that by adding Entyvio to our protocol we can try to prevent that from happening and offer patients better transplant outcomes.”
What Are the Risks?
In the ASTIC trial published in 2015, one patient who underwent stem cell therapy for Crohn’s died.
“So one of the things we talk about with patients is the risk of death,” Cohen says. “It’s not a high risk. It’s a very, very low risk. We haven’t seen it in other data sets.”
However, he says, while many are unwilling to take that risk, for others, stem cell transplant may still be the right choice.
“When you take the time to talk to patients who agree to be in these trials, they will tell you how horrible and debilitating the disease is and the effect it has on their life,” Cohen says. “Their inability to go out and do things, their inability to be spontaneous, never mind the pain and the fatigue that they go through. For a lot of people, the response is that it’s a small risk but one that needs to be taken.”
Other risks include infections and the side effects of chemotherapy, including hair loss, nausea and vomiting, fatigue, and infertility.
For Miceli, the potential benefits of the treatment far outweighed the risks. And for him, it paid off.
“I joined an over-40 men’s soccer league. It’s the first time I’ve played soccer in over 10 years. I feel perfectly healthy to do that,” he says. “I play soccer with my kids all the time. It’s literally a night and day difference. I wouldn’t say I was dying before, but I was just kind of living, really just trying to get by.”