First Patient to Get Stem Cell Treatment for Crohn's in Remission

Medically Reviewed by Gary D. Vogin, MD

Aug. 10, 2001 -- Joy Weiss treated herself to a Big Mac for lunch on Friday and then considered whether she should top off the meal with a salad, some fruit, or both. For most 20-somethings, that doesn't sound like an extraordinary lunch choice, but for Weiss it's a miracle meal.


The miracle in this case is a controversial, experimental medical procedure that involves stem cells harvested from a patient's own bone marrow.


Ten weeks ago, Weiss became the first person to undergo the stem cell infusion for treatment of Crohn's disease, a condition in which the body's immune system attacks the patient's digestive tract. On Monday, researchers at Chicago's Northwestern Memorial Hospital completed a second such treatment in another Crohn's patient. The second patient, reportedly a 16-year old male, has requested anonymity, says Richard Burt, MD, lead researcher in the pilot study. Burt says, however, that the second patient is doing well.


For years, Weiss did not know a single day without pain, the gut-wrenching pain caused by Crohn's disease. "Until I was 19 I could never get my weight up above 90 pounds," says the 22-year-old Weiss. Dairy foods, salad, fruit, nuts, fried foods -- all were dietary no-no's for Weiss, who was diagnosed with Crohn's disease when she was 11.


Over the years of treatment for the condition, Weiss suffered through as many as 10 daily attacks of painful diarrhea characterized by watery, bloody stools.


Treating the Crohn's symptoms required the powerful steroid prednisone, which helped quiet the inflammation caused by the disease but also weakened other tissues in her body. Moreover, years of intestinal disease plus steroid therapy impaired her body's ability to absorb calcium, so she has developed osteoporosis, the bone-wasting disease normally associated with old age.


Two years ago, Weiss' doctor began using IV tubes to deliver "night feeds so that I could get some nutrients." Her gastroenterologist recommended her for a colostomy, a procedure in which a large part of the colon is removed and the patient wears an external bag for waste. But after examining her, "my surgeon said that although my body was ready for a colostomy, I wasn't ready psychologically, so he said he would look for other alternatives."


The surgeon turned to the Internet, and there he found an article about Burt's proposal to treat Crohn's disease with an experimental procedure that required a stem cell transplant, using cells harvested from the patient's own bone marrow. This type of transplant is used to treat leukemia and other cancers.


Burt and his co-investigator Robert Craig, MD, had been waiting for about three years for the "right patient for this procedure," says Craig, a professor of medicine at Northwestern University Medical School.


The pilot study in which Weiss was the first patient will eventually include 10 Crohn's patients who have "failed all other accepted therapies," says Burt. Craig tells WebMD the patients not only "will have failed all other therapies, but they also must convince me that they are willing to take the risks associated with stem cell transplant."


Stem cell transplant is an experimental procedure that definitely carries its own risks. First, the cells are harvested from the patient's bone marrow, and then the patient is treated with powerful chemotherapy drugs, which are used to destroy the patient's immune system. After the immune system is destroyed, the patient's stem cells are injected back into the body and the patient is kept in a sterile environment for two weeks so that the "new" immune system can develop. During this time, any infection can pose fatal risks.


Because Crohn's disease is usually not fatal, some researchers are questioning the advisability of treating the disease with such a risky procedure.


In a statement released Thursday, the Crohn's and Colitis Foundation of America said "We are not certain that the benefits of stem cell transplants in Crohn's disease patients outweigh the risks. ... Scientists have yet to determine whether stem cell transplant can initiate a long-term remission in people with Crohn's disease. In addition, the potential benefits of this therapy must be weighed against the risk of infection. While Crohn's patients have an altered immune system, researchers have not yet determined whether Crohn's can be qualified solely as an autoimmune disease. Until those questions are answered through carefully monitored, long-term clinical studies, stem cell transplant in Crohn's disease patients remains an investigational therapy."


Craig tells WebMD, "I agree with the CCFA. Believe me, a patient has to convince me that this is the absolute right thing for him or her." He says that he worries "about the possibility that I will lose a patient to this therapy."


The type of caution expressed by Craig is well placed, says Richard MacDermott, MD, head of gastroenterology and immunology at Albany Medical College in New York. "This is obviously a truly investigational procedure at the very beginning of the investigational ladder. It has a long, long way to go," MacDermott tells WebMD.


"I don't personally know the [rate of sickness and death] associated with stem cell transplant, but it has got to be significant," says MacDermott, who is a trustee of the Crohn's and Colitis Foundation of America.


Burt says the procedure "wasn't done in a cavalier or relaxed manner -- the procedure was approved by the FDA." He says, too, that the chemotherapy used in his stem cell protocol is not as toxic as earlier stem cell transplant experiments. "The only complication that we had was a two-day fever," says Burt, who adds that tests done during that two-day period turned up no evidence of infection.


From her perspective, Weiss says she underwent two cycles of chemotherapy and neither was "as bad as my worst days with Crohn's." She says that she started feeling better "almost right away. All the pain didn't leave but it started getting better right away. This is the first time I have had a Crohn's remission in 11 years."


Weiss spent about two and half months in Chicago undergoing pretreatment screening, treatment, and immediate follow-up. She is expected back in Chicago on Aug. 18 for a follow-up exam and then will return again at six months, nine months, and 12 months for follow-up. After 12 months, "I'll go back every year for five years," says Weiss. Craig says that it will take at least five years to confirm a true remission of disease.


Meanwhile, at her home in Mariaville, Maine, population 500, Weiss is enjoying the "first summer of my life." Always interested in horses, Weiss is anticipating applying to college to study "equine science. I have a mare here and now I am able to go out and walk the mare. It is a miracle."