Trial will test stem cell transplant for Crohn’s disease

A new trial led by researchers from Queen Mary University of London and Barts Health NHS Trust, as well as other institutions in the United Kingdom, will investigate a therapy that attempts to restart the immune systems of patients with Crohn’s disease using stem cells.

Previous studies found that using stem cells to wipe out and replace patients’ immune systems was successful in treating patients with multiple sclerosis, and the new trial will test a similar process in patients with CD who have not responded to currently available therapies, according to a press release

“Although drug therapy for [CD] has evolved dramatically over the last decade with the introduction of targeted biological therapies, there are a substantial number of patients for whom these drugs do not work, or whom initially respond and then loose this response,” James Lindsay, PhD, FRCP, from Queen Mary University of London and chief investigator of the trial told Healio Gastroenterology and Liver Disease. “We hope that stem cell transplant may allow these patients with chronic active disease who may be suffering a considerable burden of symptoms from the disease to achieve a good quality of life, by resetting the immune system.”

The study comprises 99 patients with CD who will undergo either the stem cell therapy (n = 66) or current best standard of care (n = 33).

The patients in the stem cell arm will undergo chemotherapy and hormone treatment to activate their stem cells that are then collected from their blood. When reintroduced, the stem cells turn into new immune cells, giving the patient an entirely new immune system.

Theoretically, the new immune system will not attack the gut to cause inflammation and will not adversely affect drugs that target the inflammation, according to the press release.

The primary endpoint of the trial is mucosal healing defined using a Simple Endoscopic Score for CD (SES-CD) ulcer subscore.

“We are all looking at clinical response and remission, quality of life and safety,” Lindsay said.

The study follows a previous trial conducted in 2015 that found that some patients saw a benefit from the stem cell treatment. Because investigators observed some serious side effects in the previous study, the upcoming trial will use a lower dose of the therapy to minimize the risks due to toxicity.

“[Stem cell therapy] does not ‘cure’ [CD],” Lindsay said. “Because patients are receiving their own stem cells, a percentage do redevelop [CD] after the transplant.”

To address this, investigators are following up with patients at six months after the transplant and restarting targeted therapy when appropriate.

Researchers from the University of Manchester, University of Nottingham, University of Sheffield, Nottingham Trent University, University of Edinburgh, University of Oxford, and King’s College London will also take part in the study, which is funded by the Medical Research Council and the National Institute for Health Research.

Disclosures: Lindsay reports no relevant financial disclosures.

A new trial led by researchers from Queen Mary University of London and Barts Health NHS Trust, as well as other institutions in the United Kingdom, will investigate a therapy that attempts to restart the immune systems of patients with Crohn’s disease using stem cells.

Previous studies found that using stem cells to wipe out and replace patients’ immune systems was successful in treating patients with multiple sclerosis, and the new trial will test a similar process in patients with CD who have not responded to currently available therapies, according to a press release

“Although drug therapy for [CD] has evolved dramatically over the last decade with the introduction of targeted biological therapies, there are a substantial number of patients for whom these drugs do not work, or whom initially respond and then loose this response,” James Lindsay, PhD, FRCP, from Queen Mary University of London and chief investigator of the trial told Healio Gastroenterology and Liver Disease. “We hope that stem cell transplant may allow these patients with chronic active disease who may be suffering a considerable burden of symptoms from the disease to achieve a good quality of life, by resetting the immune system.”

The study comprises 99 patients with CD who will undergo either the stem cell therapy (n = 66) or current best standard of care (n = 33).

The patients in the stem cell arm will undergo chemotherapy and hormone treatment to activate their stem cells that are then collected from their blood. When reintroduced, the stem cells turn into new immune cells, giving the patient an entirely new immune system.

Theoretically, the new immune system will not attack the gut to cause inflammation and will not adversely affect drugs that target the inflammation, according to the press release.

The primary endpoint of the trial is mucosal healing defined using a Simple Endoscopic Score for CD (SES-CD) ulcer subscore.

“We are all looking at clinical response and remission, quality of life and safety,” Lindsay said.

The study follows a previous trial conducted in 2015 that found that some patients saw a benefit from the stem cell treatment. Because investigators observed some serious side effects in the previous study, the upcoming trial will use a lower dose of the therapy to minimize the risks due to toxicity.

“[Stem cell therapy] does not ‘cure’ [CD],” Lindsay said. “Because patients are receiving their own stem cells, a percentage do redevelop [CD] after the transplant.”

To address this, investigators are following up with patients at six months after the transplant and restarting targeted therapy when appropriate.

Researchers from the University of Manchester, University of Nottingham, University of Sheffield, Nottingham Trent University, University of Edinburgh, University of Oxford, and King’s College London will also take part in the study, which is funded by the Medical Research Council and the National Institute for Health Research.

Disclosures: Lindsay reports no relevant financial disclosures.