Lay Summary

Proposal No. IBD-0170R
Principal Investigator:  Robert M. Craig, M.D.
Applicant Organization:  Northwestern University (Chicago, Illinois, U.S.A.)
Project Title: Immune ablation and hematopoietic stem cell support in patients with refractory Crohn’s. A randomized, controlled study.
Period of Award:  June 1, 2006 – September 30, 2008

Treatment for Crohn’s disease (CD) has mostly been:

• directed at opposing inflammation with aspirin-like medicines or other anti-inflammatory drugs like steroids;
• through using antibiotics to influence the bowel bacteria; or
• by administering agents that oppose the actions of these white cells (azathioprine, 6-mercaptopurine) or the chemicals (cytokines) released by these cells (infliximab).

Some patients do poorly on these conventional medications and require surgery to remove the involved segments. However, surgery does not cure the disease, as the disease usually returns, and surgery leaves the patient with less functional bowel, which may eventuate in the patient having insufficient bowel for nutrient absorption.

We have performed a new treatment in 15 patients who have failed conventional therapy for CD. Stem cells were collected from the blood of patients and re-administered after the patients underwent chemotherapy that eliminated the patients’ bone marrow and offending white blood cells. The chemotherapy is the same as is used for bone marrow transplantation for leukemia or for cancer, and carries the risk of infection and bleeding during the time before the stem cells repopulate the bone marrow and allow for recovery (about ten days). Most of the patients with Crohn’s disease did very well with this therapy, although there have been some relapses.

The purpose of the current investigation is to compare this new therapy in this group of patients to conventional therapy. At the end of one year, the experimental therapy will be offered to the patients receiving conventional therapy if their disease remains refractory. The disease activity will be followed with x-rays of their bowel, colonoscopic examinations, blood tests and physical examinations.