Stem cell transplantation does not provide significant improvement for Crohn's diseaseDecember 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.
University of Nottingham
Related Clinical Trial Articles:
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.
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.
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.
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.
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.
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.
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.
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.
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.
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)
This is the fifth edition of a very successful textbook on clinical trials methodology, written by recognized leaders who have long and extensive experience in all areas of clinical trials. The three authors of the first four editions have been joined by two others who add great expertise. A chapter on regulatory issues has been included and the chapter on data monitoring has been split into two and expanded. Many contemporary clinical trial examples have been added. There is much new material on adverse events, adherence, issues in analysis, electronic data, data sharing and... View Details
Clinical Trials, Second Edition: Study Design, Endpoints and Biomarkers, Drug Safety, and FDA and ICH Guidelines
by Tom Brody PhD (Author)
Clinical Trials, Second Edition, offers those engaged in clinical trial design a valuable and practical guide. This book takes an integrated approach to incorporate biomedical science, laboratory data of human study, endpoint specification, legal and regulatory aspects and much more with the fundamentals of clinical trial design. It provides an overview of the design options along with the specific details of trial design and offers guidance on how to make appropriate choices. Full of numerous examples and now containing actual decisions from FDA reviewers to better inform trial... View Details
Clinical Trials: A Methodologic Perspective (Wiley Series in Probability and Statistics)
by Steven Piantadosi (Author)
Presents elements of clinical trial methods that are essential in planning, designing, conducting, analyzing, and interpreting clinical trials with the goal of improving the evidence derived from these important studies
This Third Edition builds on the text’s reputation as a straightforward, detailed, and authoritative presentation of quantitative methods for clinical trials. Readers will encounter the principles of design for various types of clinical trials, and are then skillfully guided through the complete process of planning the experiment, assembling a... View Details
A Concise Guide to Clinical Trials
by J. Rick Turner PhD (Author)
Biopharmaceutical drugs improve the health and well-being of people across the globe on a scale that is unrivaled by any other medical intervention. Before these drugs can be prescribed for patients by their doctors, they have to be approved for marketing by a regulatory agency. To gain marketing approval, drugs must go through an extremely rigorous process that investigates their safety and efficacy, the process of New Drug Development. The last stage of this long, complex, and expensive process involves conducting clinical trials, the topic of this book. Successfully conducting clinical... View Details
Clinical Trials: What Patients and Healthy Volunteers Need to Know
by Lorna Speid Ph.D (Author)
Every year, hundreds of thousands of healthy volunteers and patients worldwide undertake the journey through the maze that can be clinical trials. Research participants take part in clinical trials for a variety of reasons. The healthy volunteers may be seeking extra money to pay off college tuition, or they may know someone who is suffering and would potentially benefit from the results of the trial. The patient who is terminally ill might participate in a clinical trial simply as a last hope for a cure. Whatever the goals, though, most participants will experience the same sense of... View Details
A Clinical Trials Manual From The Duke Clinical Research Institute: Lessons from a Horse Named Jim
by Margaret Liu (Author), Kate Davis (Author)
"The publication of the second edition of this manual comes at an important juncture in the history of clinical research. As advances in information technology make it possible to link individuals and groups in diverse locations in jointly seeking the answers to pressing global health problems, it is critically important to remain vigilant about moral and ethical safeguards for every patient enrolled in a trial. Those who study this manual will be well aware of how to ensure patient safety along with fiscal responsibility, trial efficiency, and research integrity."
—Robert... View Details
The Four Villains of Clinical Trial Agreement Delays and How to Defeat Them: Addressing CTA Delays Comprehensively
by Débora S. Araujo (Author)
Fictional superheroes are often charged with the task of saving the world from villains such as the Joker, Lex Luthor, Doctor Doom, and the Green Goblin. But superheroes cannot be effective if they only take out one of their villains and let the others roam free. In the same way, the pharmaceutical industry cannot effectively tackle the issue of delays in the execution of clinical trial agreements (CTAs) that leave patients’ lives hanging in the balance by only addressing one or two of the villains contributing to this broad challenge.
Débora Araujo relies on seasoned experience... View Details
Oncology Clinical Trials: Successful Design, Conduct, and Analysis
by William Kevin Kelly DO (Editor), Susan Halabi PhD (Editor)
The second edition of Oncology Clinical Trials has been thoroughly revised and updated and now contains the latest designs and methods of conducting and analyzing cancer clinical trials in the era of precision medicine with biologic agents―including trials investigating the safety and efficacy of targeted therapies, immunotherapies, and combination therapies as well as novel radiation therapy modalities. Now divided into six sections this revamped book provides the necessary background and expert guidance from the principles governing oncology clinical trials to the innovative... View Details
Clinical Trials Handbook: Design and Conduct
by Curtis L. Meinert (Author)
A systematic approach to all aspects of designing and conducting clinical trials
The success or failure of clinical trials hinges on hundreds of details that need to be developed, often under less than ideal conditions. Written by one of the world's leading trialists, Clinical Trials Handbook: Design and Conduct provides clinicians with a complete guide to designing, conducting, and evaluating clinical trials—teaching them how to simplify the process and avoid costly mistakes.
The author draws on his extensive clinical trials experience to outline all steps... View Details
Clinical Trials in Neurology: Design, Conduct, Analysis
by Bernard Ravina MD MSCE (Editor), Jeffrey Cummings (Editor), Michael McDermott (Editor), R. Michael Poole (Editor)
Translating laboratory discoveries into successful therapeutics can be difficult. Clinical Trials in Neurology aims to improve the efficiency of clinical trials and the development of interventions in order to enhance the development of new treatments for neurologic diseases. It introduces the reader to the key concepts underpinning trials in the neurosciences. This volume tackles the challenges of developing therapies for neurologic disorders from measurement of agents in the nervous system to the progression of clinical signs and symptoms through illustrating specific study designs and... View Details