Clinical trials began using stem cell transplants to develop a new immune system for people with Crohn's disease - a serious and chronic bowel disease affecting at least 115,000 people in the UK. The study, conducted at Queen Mary University of London and the Barts Health NHS Trust, is funded with £ 2 million from a Medical Research Council and the National Institute for Health Research and recruits patients from centers to Cambridge, Edinburgh, Liverpool, London, Nottingham, Oxford, and Sheffield. The test is coordinated by the Sheffield University Clinical Trial Unit.
Crohn's disease is a long-term condition which causes inflammation of the internal digestive system and affects patients suffering from symptoms of diarrhea, abdominal pain, and severe fatigue among others. Current treatments include drugs used to reduce inflammation, and surgery which often requires removal of the affected part of the intestine. In severe cases, after years of operation, patients may need a final surgery to get the colon out of the anus into a stomach opening, called a stoma, where the bowels are collected in a bag.
Chief investigator Professor James Lindsay of Queen Mary University of London and a consultant to the Barts Health NHS Trust stated: "Despite the introduction of new drugs, more patients will not respond gradually to all available treatments. Although surgery is an option in developing a stoma, others do not want to consider this technique.”
Helen Bartlett, a patient with Crohn's Disease who had cell therapy in the John Radcliffe Hospital, Oxford, said: "Living with Crohn's disease is an everyday battle, you often go to the bathroom, bleeding. There are times when there was no toilet and I had to go every now and then.
The use of stem cell transplantation has been proven to destroy patients' immune systems and has recently been proven successful in treating multiple sclerosis.
In the test, patients underwent chemotherapy and hormone treatments to transform their stem cells. When stem cells are reintroduced into the body, they create new immune cells rising to a whole new immune system. Theoretically, the new immune system does not negatively affect the patient's body to cause inflammation, nor does it affect the compound's medication to remove it from their intestines.
The NHS Regulatory Authority in England and Wales funding for TiGenix and Takeda Alofisel - the first allogeneic stem cell therapy to be approved for use in the European Union - has been approved for use in patients with Crohn's disease. The therapy (formerly referred to as Cx601) was approved in March for the treatment of complex perianal fistulas in patients with adult inactive / mildly active luminal Crohn's disease, when the fistula showed an inadequate response to at least one conventional or biological therapy. The Alofisel (darvadstrocel) is a local administration of allogeneic (donor derived) transplantation derived from expanded adipose-derived stem cells (eASCs).
TiGenix NV, a biopharmaceutical company based in Belgium, creates and lists therapeutics from its proprietary technology platform for stem cells. Its stem cell programs rely on allogeneic expanded stem cells targeting autoimmune, inflammatory and cardiac diseases. The company's lead product is Cx601, has successfully completed Phase III clinical trials in the management of complicated perianal fistulas in patients with Crohn's disease. Another focal point is the development of Cx611, a clinical product candidate stage based on adipose stem cells (derived from eASC), which will complete a phase I sepsis and phase Ib / II study on the treatment of persistent rheumatism; and Cx621, which completed a Phase I study to treat the intra-lymphatic administration of allogeneic eASCs. It also includes AlloCSC-01, a candidate-based phase I / II study of severe myocardial infarction. As of July 3, 2018, TiGenix NV is a subsidiary of Takeda Pharmaceutical Company Limited.
Patients receiving clinical trial drug treatment with a 44 percent greater likelihood of combined remission compared to placebo, follow-up (52 weeks and 104 weeks after treatment) were confirmed to to be safe and effective, according to the company. However, the National Institute for Health and Care Excellence states in its drafting rules that alofosel has shown only a small improvement in the proportion of individuals with complex perianal fistulas who have received a complete response in the clinical comparison compared to placebo.
"Reliable follow-up results are only available for up to a year, so it's unclear how long the treatment benefits will last," says the guidelines. As a result, cost-effectiveness estimates are "extremely uncertain" and the committee cannot identify exactly the ‘cost-effective’ measures, according to NICE. Takeda said that "Working with the IBD, NICE and NHS England communities is fully committed to identifying relevant data that can help answer and raise questions raised in the NICE Draft Guidelines to give a positive recommendation ".
Perianal fistula, a common complication of Crohn's disease, occurs when there is an abnormal path existing between the rectum and the outside of the body, possibly leading to incontinence and sepsis. Complicated fistulas are rarely more resistant than the usual simple fistulas.