Stem Cell transplant Crohn's Disease - Stem Cell Research and Crohn's Articles

Long story short. I am now having what I think to be an allergic reaction to Humira. (I have had them to Remicade in the past). My doctor wants me to stop treatment and come in next week to discuss the possibility of a stem-cell transplant. I profess complete ignorance and in fact know nothing about it. Below are some articles that I have not even had time to read yet. In scanning them apparently they can cure Crohn's, something I did not understand to be possible. I meet with Dr. Shafran and his staff a week from Monday to discuss this. I plan on doing a whole lot of research in the meantime. It looks like it has only been done a few times (see below). Grasping at straws here, but does anyone have any insight?
-Scott

Stem Cell Research and Crohn's
Roanoke Teen to Head to Chicago in Pursuit of Crohn's Disease Cure (from July 2003)
An insurance company, hospital and federal regulators have approved a Roanoke youngster with a severe intestinal disease to have an experimental stem-cell transplant that could cure or kill him.
Thirteen-year-old Jordan Fifer heads to Children's Memorial Hospital in Chicago later this month.
People ask him if he is scared or afraid of dying. He tells them he's not.
"I don't know why I am not scared. I'm just not," he said. "I'm incredibly eager. And I just know when I come back, I will be able to do things that I haven't been able to do for so long and that means a lot to me."
Like eat a piece of pizza and have the energy to play soccer.
Jordan, who this fall will be a freshman at Patrick Henry High School and the Roanoke Valley Governor's School for Science and Technology, was stricken with Crohn's disease at about age 10. With Crohn's, the digestive system comes under attack by the immune system. When the disease is active, it causes stomach aches, diarrhea, rectal bleeding, weight loss, fatigue and fever.
About a million Americans have Crohn's or a similar condition, ulcerative colitis, according to the Crohn's and Colitis Foundation of America. There is no known cure.
Jordan had tried without success to get relief through a variety of prescription drugs and other traditional therapies. His mother, Hope Trachtenberg-Fifer, learned about a Chicago medical team that has treated about a dozen Crohn's patients, adults and children, with a transplant of blood stem cells.
Jordan's health insurer - he's insured through his dad Gary's health plan - this week approved paying part of the cost for Jordan to have a transplant. He will be the youngest person treated so far.
Blood stem cells are the building blocks of blood inside the bones. Already, blood stem cell transplants correct disorders of the blood and disease-fighting immune system and repair damage to such systems caused by some cancer treatments, such as chemotherapy.
But until the transplant has been proved to address Crohn's, it is classified by the federal government as an experimental treatment. The U.S. Food and Drug Administration supports the ongoing experiment in which Jordan will participate and has approved methods to be used.
So within weeks, Jordan will leave for a three- to four-month stay in the Chicago area. He will be hospitalized only part of that time but must remain near the hospital for follow-up care. His mother will stay with him at a home for transplant patients and their families, from which Jordan and his Mom may move to an apartment.
Here's how the procedure works: Medication will draw stem cells from Jordan's bone marrow into his bloodstream for collection (he'll be his own stem-cell donor). High-dose chemotherapy will destroy his immune system. Then doctors will reintroduce the stem cells to his bloodstream. If all goes well, the cells will create a new immune system without the malfunction that afflicts Jordan today.
The procedure carries a small risk of death. Jordan's immune system may not regenerate, placing his body at serious risk of infection. He also could have an allergic reaction.
The Crohn's and Colitis Foundation of America, dedicated to education, patient support and fund raising for research, sounded a note of caution. The organization released a statement in which a member of its scientific advisory panel, Dr. William Sandborn of the Mayo Clinic, said he's uncertain the benefits of stem-cell transplants in Crohn's disease patients outweigh the risks.
Jordan's mother said all of about a dozen people who have had the procedure have been helped. The average Crohn's patient does not experience severe symptoms and does not need a transplant, she said. However, Jordan's Crohn's, because it is uncontrolled, actually poses a much higher risk of death than the procedure, she said.
Jordan has been hospitalized numerous times. He takes 33 pills a day. He suffers from bouts of digestive system bleeding that sometimes make it necessary for him to have a blood transfusion. His disease has stunted his growth and kept him from school and social activities. Yet, the youngster is getting good grades, played xylophone in his middle-school band and volunteers.
"Patients like Jordan, like my child, who have volunteered for this study are very desperate patients who are looking for light at the end of the tunnel and, it seems, have found it," Trachtenberg- Fifer said.

First Patient to Get Stem Cell Treatment for Crohn's in Remission
By Peggy Peck WebMD Medical News
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."
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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."
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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."

Stem Cell Transplants Cure Crohn's Disease
Another advance in regenerative medicine is reported in the Reno Gazette-Journal. Ten sufferers of the deadly Crohn's disease have been cured by stem cell transplants that regenerate the damage to their intestines and immune system. The article focuses on the young man who will hopefully be number 11 and live to see a full life. This is the sort of amazing application of stem cell medicine, like recent advances in regenerating normally fatal heart damage, that we hope will become commonplace. Being able to regenerate any part of the body in this fashion will lead to large gains in healthy lifespan.

More about Stem Cell Research, Stem Cell Transplants and Crohn's Disease:
Stem Cell Transplant
What is a stem cell transplant?
The stem cells corresponding to each part of the body provide instructions for how to grow that part. For Jordan's kind of stem cell transplant, bone marrow was drawn from his hip bone using a long needle. The stem cells were separated out and frozen. Jordan's immune system was then partially destroyed by high-dose chemotherapy, and his stem cells were reintroduced to his body in the hopes that they will multiply and build a healthy immune system.
Most stem cell transplants of this type are autologous, meaning that the patient is both the donor and the recipient. Only cells are transplanted; the procedure does not involve the replacement of any organs or other body parts. Although the transplant itself does not involve surgery, Jordan has had many surgical procedures to implant and remove ports and catheters used to inject and draw fluids and drugs.
How often are stem cell transplants performed? What are the chances for success?
Although about 2.5 million people worldwide have had stem cell transplants for various diseases (usually certain cancers), only between 15 and 20 people have had stem cell transplants to treat Crohn's disease. Jordan was the youngest patient in the United States -- and the first at Duke University -- to undergo the procedure as a treatment for Crohn's disease.
Each patient who has previously had a stem cell transplant for Crohn's disease has experienced almost complete remission. Reports from the patients and their medical teams continue to be very encouraging. The patients have experienced significant improvements, and many have been able to return to lives essentially free of the symptoms of the disease. Many have been able to dramatically reduce or eliminate their dependence on daily medication, and the resulting side effects. The younger patients have experienced growth spurts, as their bodies are relieved of the stress of the disease and receive proper nutrition.
When and where was Jordan's transplant performed?
Jordan's transplant was performed on June 2, 2005, by a medical team at the Pediatic Bone Marrow and Stem Cell Transplant Program at Duke University Medical Center. This team includes hematology and oncology specialists, gastroenterology specialists, and other support staff. Jordan and a parent travelled to Duke on April 2, 2005, and returned home to Roanoke, Va., on July 31, 2005.
How long does the stem cell transplant take?
The transplant itself (the reintroduction of Jordan's previously harvested stem cells to his body) took only 30 minutes, but the entire transplant protocol took about four months. During that time, Jordan lived in the hospital and in an apartment near the hospital. As his health permitted, he participated in distance learning and received homebound tutoring to keep up his high school studies. It will take about one year for Jordan's immune system to completely rebuild itself. Jordan will also be monitored by doctors in Roanoke, and at the treatment site, for routine checkups.
What risks and side effects are associated with a stem cell transplant?
The primary side effects of the transplant come from the chemotherapy and other drugs involved in the protocol, and not the transplant itself. Jordan has experienced nausea, loss of appetite, and hair loss. Because Jordan's immune system was partially destroyed, he is at an increased risk of infection. To guard against this, he was hospitalized in a special room with a positive air pressure system and high efficiency particulate air (HEPA) filtration. Even after returning to Roanoke, his exposure to people and places is restricted. Although all medical procedures carry some risk, and stem cell transplants carry a higher risk of complications than some other procedures, the risk of death from an autologous stem cell transplant is still extremely low.
How much will the stem cell transplant cost?
For more information about the costs associated with the transplant, see About the Fund.
Aren't stem cell transplants very controversial?
The political controversy surrounding stem cells relates to the use of animal or fetal tissue to clone embryos for research. In Jordan's transplant, like in most stem cell transplants of this type, Jordan received his own stem cells.