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Crohn's disease: New trial tests transplantation for cure

Fred Hutchinson Cancer Research Center study represents new use of bone marrow transplantation

July 23, 2012

A new clinical trial testing bone marrow transplantation to cure severe cases of Crohn’s disease may provide a new treatment option to those who suffer from the chronic inflammatory condition of the gastrointestinal tract.

Dr. George McDonald, a transplant researcher and gastroenterologist in the Hutchinson Center’s Clinical Research Division, leads the study, which is funded by an infrastructure grant from the Los Angeles-based 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 bone marrow cells from a sibling or unrelated donor. The transplant 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 intestinal bacteria and a loss of immune tolerance. There is strong evidence that genetic abnormalities in the immune regulatory system are linked to the disease, McDonald said.

Although the CATS clinical trial represents a new direction for bone marrow transplantation, the procedure has precedent. The Hutchinson Center 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 stem cell transplants – 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.

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.

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

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 bone marrow transplant procedures will be conducted at Seattle Cancer Care Alliance, the University of Washington Medical Center and Seattle Children’s Hospital.

Fred Hutchinson Cancer Research Center is a world leader in research to prevent, detect and treat cancer and other life-threatening diseases.