Stem cell transplantation does not lead to sustained improvement in Crohn's disease

Researchers, including CGEM’s Head of Gastroenterology Section Professor Jack Satsangi, have recently published results from the Autologous Stem Cell Transplantation International Crohn’s Disease (ASTIC): December 2015

Researchers, including CGEM’s Head of Gastroenterology Section Professor Jack Satsangi, have recently published results from the Autologous Stem Cell Transplantation International Crohn’s Disease (ASTIC) trial in the Journal of the American Medical Association (JAMA).

This study shows that bone marrow stem cell therapy (HSCT) was not significantly better than conventional therapy at treating a particular form of Crohn's disease (refractory Crohn’s disease). Despite some patients showing improvement and clinical remission, there was no statistical significance to the findings.

Patients with Crohn’s disease have abdominal pain and associated diarrhoea every day, with the problem starting at a young age, persisting on a lifelong basis and accompanied by malnutrition, fatigue and ill health. About one in 200 people in developed countries suffer from Crohn disease or ulcerative colitis. Crohn disease is one of life’s most challenging chronic diseases. The prevalence in children in Scotland is amongst the highest in the world.

Although there have been major advances in understanding and treatment of Crohn’s disease recently, some patients are resistant to all treatments and lead lives that are severely curtailed and of poor quality. ASTIC is a unique ground-breaking collaborative project conducted by leading researchers in the field aimed at addressing the needs of these patients.

The ASTIC study showed that HSCT lead to improvements in some individual measures of disease severity, and symptoms disappeared in about a quarter of patients tested. The main outcome was sustained disease remission without traditional treatment for the disease for at least the last 3 months and no physical evidence of active disease anywhere in the gut after one year. Despite the potential for HSCT in the treatment of refactory Crohn’s disease, it is also toxic, leading to severe damage of healthy cells as well as the diseased tissue.

Trial Chief Investigator Prof Chris Hawkey, Professor of Gastroenterology in Nottingham said “Haematopoietic stem cell transplantation is probably the most effective treatment for Crohn’s disease but also the most toxic. It cannot be recommended for widespread use at the present time but may be a risk worth taking for a small number of patients that have run out of treatment options”.

CGEM’s Prof Jack Satsangi added that although the study did not reach it’s primary end-point at one year, several patients in the trial in Scotland have had a very satisfactory long-term response. We need to address issues of safety, and patient selection in a future study.

The ASTIC team are now turning their attention to identifying those most likely to benefit, with minimal toxicity.

The full press release is on the University of Nottingham’s website.