HONOLULU -- Results from a clinical study from Northwestern University (NU) in Chicago suggest that stem cell transplantation (SCT) can halt the progression of Crohn’s disease, a chronic and sometimes severe autoimmune disease. The researchers, from the NU Feinberg School of Medicine, also are performing the technique to combat other autoimmune disorders for which there are no effective therapies, or for patients who have exhausted all other treatment options. The findings were presented recently at the annual blood and marrow (BMT) Tandem Meetings.
The study’s principal investigator Richard K. Burt, MD, associate professor and chief, division of immunotherapy and autoimmune diseases, NU Feinberg School of Medicine and Northwestern Memorial Hospital, where research into SCT and autoimmune disease was pioneered, reported successful clinical trial outcomes, and outlined plans for multicenter, randomized clinical trials to compare SCT with standard treatments for autoimmune disorders.
A total of 17 patients with severe, chronic Crohn's disease who did not respond to conventional treatments were enrolled in a Phase I study. Crohn's disease is an immune-mediated inflammation of the gastrointestinal tract. After transplant, symptoms and biological markers of the disease tended to improve quickly and dramatically, and X-ray and colonoscopy findings have shown gradual improvement over months and years, according to the researchers. Among 15 patients with follow up of more than six months (median 29, range 11-48 months), 12 have experienced sustained disease remission. Approximately 500,000 people in the U.S. have Crohn's disease, which affects men and women equally.
"It is important to remember that the effects of these and other autoimmune diseases vary widely -- from very mild cases that cause few symptoms, to moderate disease that can be controlled with conventional treatments, to acute, life-threatening or disabling diseases that do not respond to current treatments," Burt said. "It is this latter group of patients with severe disease who have exhausted all other treatment options for whom stem cell transplant may be indicated."
There are more than 80 autoimmune diseases, in which the immune system turns against the body it's supposed to protect, attacking and often damaging or destroying normal tissues and organ systems. They are estimated to affect up to 24 million Americans. Women are disproportionately affected, and account for nearly 80 percent of cases in the United States. Autoimmune disease is one of the top 10 leading causes of death in children and women age 65 and younger.
SCT is an established technique used to restore blood cells destroyed by treatments such as chemotherapy for some types of cancers. "Our research suggests that transplant also may be effective against autoimmune disease by reconstituting a healthy new immune system that functions normally," Burt said.
The BMT Tandem Meetings are the joint annual meetings of two leading professional organizations dedicated to research, education and patient care in the field of blood and marrow transplantation and cellular therapy. The Center for International Blood and Marrow Transplant Research (CIBMTR), based in Milwaukee, is an affiliation of the International Bone Marrow Transplant Registry (IBMTR) of the Medical College of Wisconsin and the research arm of the National Marrow Donor Program, which collects, analyses and disseminates clinical data on blood and marrow transplantation and conducts original research to determine the most effective BMT strategies. The American Society for Blood and Marrow Transplantation, based in Arlington Heights, Ill., is a professional association of clinicians, investigators and other health personnel promoting blood and marrow transplantation research, education, scholarly publication and clinical standards. For more information on the BMT Tandem Meetings, visit www.asbmt.org/annualmeet.
Source: Blood and Marrow Transplant Meetings
