Our program is engaged in ongoing clinical research aimed at better understanding autologous stem cell transplantation for refractory Crohn’s disease. This research focuses on identifying the biological mechanisms underlying stem cell transplantation, as well as the factors that lead some patients to develop treatment-resistant disease, with the goal of improving future therapeutic outcomes.

Clinical Outcomes

To maximize patient outcomes, we conduct research on post-transplant therapies to evaluate their safety and potential to help maintain clinical and endoscopic remission. Additional areas of investigation include assessing the long-term effects of stem cell transplantation, exploring ways to refine transplant protocols to improve safety and effectiveness, and developing strategies to optimize the stem cell graft.

Scientific Outcomes

Our research has suggested that stem cell transplantation may act as a cellular therapy, helping to restore normal function to the immune system by eradicating inflammatory immune cells and restoring normal immune cell functions from the hematopoietic stem cells (i.e., an immune “reset”). To explore this hypothesis further, we take samples from patients throughout the transplant process and study the patient’s hematopoietic stem cells. Cell lines have been established for future research, and samples have been analyzed through multiple methods, including single-cell RNA sequencing and mass cytometry with datasets available for use.

The process of stem cell transplantation involves three phases: a pre-transplant evaluation, the transplant, and post-transplant monitoring.

Pre-transplant Evaluation

This phase begins with a review of the patient’s Crohn’s disease history and studies to ensure stem cell transplantation may be effective and can be done safely. For patients who do not live near The Mount Sinai Hospital, this pre-transplant evaluation can be completed in collaboration with our team and local providers. After the initial pre-transplant evaluation, our team works with the patient’s insurance for authorization, after which the patient undergoes a final pre-transplant review by Mount Sinai’s Crohn’s Disease Stem Cell Transplant Steering Committee.

The Transplant

The patient is then admitted to the hospital for stem cell mobilization. This admission lasts 10 days on average, after which they can choose to remain in the hospital for the transplant or return home. The patient is then readmitted to the hospital for stem cell transplantation, which takes an average of 20 days. During the first week, patients undergo conditioning chemotherapy to eradicate their inflammatory immune cells. They are then given their hematopoietic stem cells, after which they are observed until it is clear their immune system has recovered adequately, and the stem cells are working. This takes place on a floor of The Mount Sinai Hospital dedicated exclusively to stem cell transplantation, where patients are cared for by a multidisciplinary team of doctors with specialization in stem cell transplantation, inflammatory bowel disease, infectious disease, palliative care, psychology, and nutrition.

Post-transplant Monitoring

After stem cell transplantation, patients return home, where they are monitored by our team. Patients may also follow up with their local inflammatory bowel disease and stem cell transplantation team if they do not live close to Mount Sinai. Clinical evaluations and laboratory testing are performed monthly until six months post stem cell transplant, when they return to Mount Sinai for endoscopic evaluation. Patients then continue to be followed by our team until one year after their transplant, after which they can transition back to their usual care teams.