Nav: Home

Stem cell transplantation does not provide significant improvement for Crohn's disease

December 17, 2015

A clinical trial to test the effectiveness of a stem cell therapy among adults with difficult to treat Crohn's disease has found it is not significantly better than conventional treatment in producing sustained disease remission after one year.

Crohn's disease is a chronic inflammatory condition of the gastrointestinal tract causing pain and diarrhoea that can mean life-long ill health, impaired quality of life and reduced life expectancy. About one in 200 people in developed countries suffer from Crohn disease or ulcerative colitis. Immunosuppressive drugs are the standard treatment for Crohn's but some patients do not respond to it, or lose response to it over time.

Patient case reports had suggested that blood stem cell transplantation (HSCT) may help some patients so an international randomised clinical trial was set up by the Nottingham Digestive Diseases Centre (NDDC) to test this hypothesis. The results are published in the Journal of the American Medical Association.

The researchers randomly assigned 45 patients with Crohn's disease who were not responsive to treatment or suitable for surgery to autologous (the use of one's own cells) blood stem cell treatment or a control treatment in groups of 23 and 22. Patients were aged between 18 and 50 years and all were given standard Crohn's treatment as needed. The trial involved 11 European transplant units from July 2007 to September 2011 with follow-up until March 2013.

Leading the trial, Professor Chris Hawkey of the Nottingham Digestive Diseases Centre, said: "We found there was no statistically significant difference between the groups in the proportion of patients who met our highly stringent definition of sustained disease remission after treatment -- only two in the stem cell transplant group (8.7 percent) and one in the control group (4.5 percent). However, there was a significant difference among patients able to discontinue active treatment in the last three months of participation in the trial -- 61 percent in the stem cell group and 23 per cent in the control, and objective signs of disease disappeared in about a quarter of patients.

"There were 76 serious adverse events in patients undergoing stem cell therapy and 38 in the control group. Because very few patients achieved sustained disease remission, we conclude that HSCT is unlikely to alter the natural history of Crohn disease, and our findings argue against extension of HSCT to a wider group of patients outside of future additional trials."

Professor Dominique Farge Bancel, Saint Louis Hospital in Paris and Chair of the European Blood and Marrow Transplantation Society, added: "Although autologous HSCT therapy showed more toxicity in early stages of the follow-up in Crohn's patients, long term results after HSCT treatment may shed new lights on the expected benefit, as observed in Scleroderma, another autoimmune disease where the proof of a HSCT superiority over conventional therapy was obtained only after long term follow-up at the price of higher early toxicity."

The research concludes that further study of HSCT in patients with Crohn's disease which does not respond to standard treatment is needed. It recommends that future trials should examine the benefit of maintenance immunosuppressive therapy after HSCT and also an investigation of whether patients may regain responsiveness to treatments which were previously ineffective for them. As toxicity is the most significant barrier to HSCT in patients with Crohn's, more work is also needed to identify factors that predict either the risk of adverse effects or response to treatment to enhance the use of stem cell therapy in clinical practice.
-end-
The trial was a collaborative project conducted by leading entities in the fields of Bone Marrow Transplantation and Gastroenterology: the European Society for Blood and Marrow Transplantation (EBMT) and the European Crohn's and Colitis Organisation (ECCO) and funded by the Broad Medical Foundation and the Nottingham Digestive Diseases Centre.

University of Nottingham

Related Clinical Trial Articles:

Landmark clinical trial to help juvenile idiopathic arthritis sufferers
A clinical trial funded by Arthritis Research UK and the National Institute for Health Research led by professors from the universities of Liverpool and Bristol has discovered a drug combination that could help thousands of children with arthritis.
Clinical trial for new innovative osteoarthritis drug
The University of Liverpool, in partnership with AKL Research and Development Ltd, is to lead on a clinical trial to test a potential new drug treatment for osteoarthritis.
New malaria vaccine effective in clinical trial
University of Tuebingen researchers in collaboration with the biotech company Sanaria Inc. have demonstrated in a clinical trial that a new vaccine for malaria called Sanaria® PfSPZ-CVac has been up to 100 percent effective when assessed at 10 weeks after last dose of vaccine.
Clinical trial of infection detecting bandages begins
A clinical trial of a smart bandage which changes color when it detects infections is beginning using samples from burns patients from four UK hospitals.
First human clinical trial for nicotinamide riboside
In the first controlled clinical trial of nicotinamide riboside (NR), a newly discovered form of vitamin B3, researchers have shown that the compound is safe for humans and increases levels of a cell metabolite that is critical for cellular energy production and protection against stress and DNA damage.
Tapping into behavioral economics to boost clinical trial participation
Behavioral economics may offer a powerful tool for improving patient enrollment in clinical trials, argue Eric VanEpps, Kevin Volpp, and Scott Halpern in this Focus.
Clinical trial examines treatment of complicated grief
A new study reports on the results of a randomized clinical trial that looked at whether the antidepressant citalopram would enhance complicated grief treatment psychotherapy, and if citalopram would be efficacious without it in an article published online by JAMA Psychiatry.
Crossref Publishers deliver win for clinical trial openness
At the click of a button, health professionals and researchers will now be able to view all of the clinical trials referenced in the paper they are reading, and any related publications such as the protocol, statistical analysis plan, and results articles.
New database for sharing MS clinical trial data
The Multiple Sclerosis Outcome Assessments Consortium (MSOAC), a global effort of the National MS Society and Critical Path Institute (C-Path), is launching a database, accessible to qualified researchers, containing trial data from nearly 2,500 patients from the placebo arms of nine MS clinical trials.
Clinical trial for experimental Ebola drug publishes results
Results of the Wellcome Trust funded trial of the experimental anti-Ebola drug TKM-130803 published in PLOS Medicine.

Related Clinical Trial Reading:

Fundamentals of Clinical Trials
by Lawrence M. Friedman (Author), Curt D. Furberg (Author), David L. DeMets (Author), David M. Reboussin (Author), Christopher B. Granger (Author)

An Overview of Clinical Trial Operation: Fundamentals of clinical trial planning and management in drug development
by Biswal and Jose (Author), Shibadas Biswal (Author), Vinu M Jose (Author)

Clinical Trials: Study Design, Endpoints and Biomarkers, Drug Safety, and FDA and ICH Guidelines
by Tom Brody PhD (Author)

A Concise Guide to Clinical Trials
by J. Rick Turner PhD (Author)

Clinical Trials: A Methodologic Perspective (Wiley Series in Probability and Statistics)
by Steven Piantadosi (Author)

A Comprehensive and Practical Guide to Clinical Trials
by Delva Shamley BSc(Hons) Physiotherapy BSc(Medicine) PhD(Medical Cell Biology) Cert Ed (Editor), Brenda Wright (Editor)

Statistical Design and Analysis of Clinical Trials: Principles and Methods (Chapman & Hall/CRC Biostatistics Series)
by Weichung Joe Shih (Author), Joseph Aisner (Author)

Clinical Trials: A Practical Approach
by Stuart J. Pocock (Author)

Fundamentals of Clinical Trials
by Lawrence M. Friedman (Author), Curt D. Furberg (Author), David DeMets (Author)

Clinical Trials: What Patients and Healthy Volunteers Need to Know
by Lorna Speid Ph.D (Author)

Best Science Podcasts 2018

We have hand picked the best science podcasts for 2018. Sit back and enjoy new science podcasts updated daily from your favorite science news services and scientists.
Now Playing: TED Radio Hour

Dying Well
Is there a way to talk about death candidly, without fear ... and even with humor? How can we best prepare for it with those we love? This hour, TED speakers explore the beauty of life ... and death. Guests include lawyer Jason Rosenthal, humorist Emily Levine, banker and travel blogger Michelle Knox, mortician Caitlin Doughty, and entrepreneur Lux Narayan.
Now Playing: Science for the People

#491 Frankenstein LIVES
Two hundred years ago, Mary Shelley gave us a legendary monster, shaping science fiction for good. Thanks to her, the name of Frankenstein is now famous world-wide. But who was the real monster here? The creation? Or the scientist that put him together? Tune in to a live show from Dragon Con 2018 in Atlanta, as we breakdown the science of Frankenstein, complete with grave robbing and rivers of maggots. Featuring Tina Saey, Lucas Hernandez, Travor Valle, and Nancy Miorelli. Moderated by our own Bethany Brookshire. Related links: Scientists successfully transplant lab-grown lungs into pigs, by Maria Temming on Science...