Clinical trial seeks to cure advanced Crohn's disease using bone marrow transplant

July 23, 2012

Researchers at Fred Hutchinson Cancer Research Center have opened a clinical trial to test the theory that giving a patient a new immune system can cure severe cases of Crohn's disease, a chronic inflammatory condition of the gastrointestinal tract.

Funded by an infrastructure grant from The Eli and Edythe Broad Foundation, the initial goal of the Crohn's Allogeneic Transplant Study (CATS) is to treat a small number of patients with treatment-resistant Crohn's disease by transplanting matched from a sibling or unrelated donor. Such a replaces a diseased or abnormal immune system with a healthy one.

The idea of swapping out the immune system is based on evidence that Crohn's is related to an abnormal immune response to and a loss of . There is strong evidence that in the immune regulatory system are linked to the disease, according to CATS principal investigator George McDonald, M.D., a transplant researcher and gastroenterologist in the Hutchinson Center's Clinical Research Division.

Although the CATS clinical trial represents a new direction for , the procedure has precedent. The Hutchinson Center, which pioneered bone marrow and hematopoietic to treat , has used allogeneic transplants to cure patients who suffered from both leukemia and Crohn's, with subsequent disappearance of the signs and symptoms of Crohn's. Similar experiences have been reported from studies done in Germany.

While autologous – in which the patient's own hematopoietic cells are removed and then returned after high-dose chemotherapy is given to suppress the immune system – have been used to treat Crohn's patients, the benefits have not always been permanent, probably because the risk genes for Crohn's are still present. "Autologous transplantation following chemotherapy beats the disease down but the Crohn's tends to come back," McDonald said.

More information about CATS can be found on the website www.cats-fhcrc.org, which includes a patient-eligibility questionnaire. In general, patients must be 18 to 60 years of age and have failed all existing conventional treatments but be healthy enough to undergo a bone marrow transplant. A matched donor of bone marrow must be found from either a sibling or an unrelated person who has volunteered to donate marrow. Private insurance must cover the cost of the transplant and related medical expenses.

Crohn's disease is usually discovered in adolescents and young adults but can occur from early childhood to older age. The incidence of Crohn's disease varies in different parts of the world with rates of four to nine persons per 100,000 people in North America. According to the Crohn's and Colitis Foundation of America, a leading advocacy organization, Crohn's may affect more than 700,000 Americans. Of those affected by Crohn's, about 10 percent suffer from the most severe form for which no treatment is completely effective.

Symptoms of Crohn's may include pain, fever, diarrhea and weight loss. Substantial progress has been made in medical treatment of Crohn's disease over the last 15 years. However, even with the best immunosuppressive therapy, less than half of patients with moderate to severe Crohn's achieve long-term relief. When patients stop taking their medicines, their intestinal inflammation returns. Some severe infections have been seen in patients who took prolonged courses of medicines that suppress the immune system.

"The burden of this disease lays heavily on those who don't respond to any therapy," McDonald said.

The CATS investigator team includes transplant physicians, gastroenterologists, pathologists and nurses from the Hutchinson Center, University of Washington, Seattle Children's and the Benaroya Research Institute. The transplant procedures will be conducted at the Seattle Cancer Care Alliance, the University of Washington Medical Center, and Seattle Children's Hospital.

Explore further: Antibodies from rabbits improve survival and relapse outcomes of leukemia and myelodysplasia

Related Stories

Antibodies from rabbits improve survival and relapse outcomes of leukemia and myelodysplasia

July 6, 2012
Researchers at Virginia Commonwealth University (VCU) Massey Cancer Center's Bone Marrow Transplant Program have demonstrated that the use of antibodies derived from rabbits can improve the survival and relapse outcomes of ...

Recommended for you

Newly discovered viral marker could help predict flu severity in infected patients

October 20, 2017
Flu viruses contain defective genetic material that may activate the immune system in infected patients, and new research published in PLOS Pathogens suggests that lower levels of these molecules could increase flu severity.

H7N9 influenza is both lethal and transmissible in animal model for flu

October 19, 2017
In 2013, an influenza virus that had never before been detected began circulating among poultry in China. It caused several waves of human infection and in late 2016, the number of people to become sick from the H7N9 virus ...

Flu simulations suggest pandemics more likely in spring, early summer

October 19, 2017
New statistical simulations suggest that Northern Hemisphere flu pandemics are most likely to emerge in late spring or early summer at the tail end of the normal flu season, according to a new study published in PLOS Computational ...

New insights into herpes virus could inform vaccine development

October 18, 2017
A team of scientists has discovered new insights into the mechanisms of Epstein-Barr virus (EBV) infection, as well as two antibodies that block the virus' entry into cells. The findings, published in Proceedings of the National ...

Pair of discoveries illuminate new paths to flu and anthrax treatments

October 17, 2017
Two recent studies led by biologists at the University of California San Diego have set the research groundwork for new avenues to treat influenza and anthrax poisoning.

Portable 3-D scanner assesses patients with elephantiasis

October 17, 2017
An estimated 120 million people worldwide are infected with lymphatic filariasis, a parasitic, mosquito-borne disease that can cause major swelling and deformity of the legs, a condition known as elephantiasis. Health-care ...

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.