Crohn’s Disease Stem Cell Transplantation Program

Despite advances in the surgical and medical management of Crohn’s disease, there remain many patients who do not respond to any therapy (i.e., refractory Crohn’s disease). For patients with refractory Crohn’s disease, hematopoietic stem cell transplantation has emerged as an effective therapy in cases where standard medications and surgery have failed.

Hematopoietic stem cell transplantation involves two parts:

  1. Mobilization of a patient’s hematopoietic stem cells from the bone marrow into the blood to collect and store the stem cells.

  2. Transplantation where patients are given medications to eradicate inflammatory immune cells (i.e., conditioning chemotherapy) followed by infusion of their own hematopoietic stem cells (i.e., autologous stem cells). During stem cell transplantation patients are in the hospital, and afterwards they are monitored by the stem cell transplant team. The current stem cell transplant protocol at Mount Sinai has demonstrated in clinical studies helping patients achieve improvement in their symptoms, quality of life, and bowel healing.

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

Pre-transplant evaluation: This process begins with a review of the patient’s Crohn’s disease history and studies to assess a patient’s health to ensure stem cell transplantation would be effective and can be done safely. For patients who do not live near The Mount Sinai Hospital, this pre-transplant evaluation can be done in collaboration between the Mount Sinai Crohn’s Disease Stem Cell Transplant Team and local providers. After the initial pre-transplant evaluation, the Mount Sinai team works with the patient’s insurance provider for authorization, after which all patients undergo final pre-transplant review by the Mount Sinai Crohn’s Disease Stem Cell Transplant Steering Committee to ensure that no other medical or surgical options are available to the patient and there are no specific safety concerns.

The transplant: After insurance and steering committee approval, patients are admitted to the hospital for stem cell mobilization. Patients are admitted on average for 10 days to collect their stem cells, after which they can choose to remain in the hospital for the transplant or return home. Patients are then admitted to the hospital again for the stem cell transplantation, which takes on average 20 days. During the first week, patients are given conditioning chemotherapy to eradicate their inflammatory immune cells. Then, they are given their hematopoietic stem cells, after which they are observed until it is clear their immune system has recovered and the stem cells are working. The stem cell transplant takes place on a floor of The Mount Sinai Hospital dedicated to stem cell transplantation, where patients are taken care of by a unique 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 will be monitored by the Mount Sinai Crohn’s Disease Stem Cell Transplantation 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. Patients are monitored monthly with clinical evaluations and laboratory testing until six months post stem cell transplant, when they return to Mount Sinai for endoscopic evaluation. Patients will continue to be followed by the Mount Sinai stem cell transplantation team until one year after their transplant, after which they can transition back to their usual care teams.

For health care providers interested in learning more about stem cell translation for refractory Crohn’s disease, please contact: louis.cohen@mssm.edu or aaron.etra@mountsinai.org

The Autologous Stem Cell Transplantation Program for Crohn’s disease is also part of an active clinical trial at The Mount Sinai Hospital (clinicaltrials.gov NCT03219359). The aims of this trial are:

  • Improve stem cell transplant clinical outcomes
  • Understand the mechanisms of autologous stem cell transplant for refractory Crohn’s disease and why patients develop refractory Crohn’s disease

Clinical outcomes: To improve clinical outcomes of patients with Crohn’s disease after stem cell transplantation, we are conducting a phase 2a clinical trial of vedolizumab after stem cell transplantation. The purpose of this trial is to assess the safety of vedolizumab after stem cell transplantation and the ability for vedolizumab to maintain patients in clinical and endoscopic remission. Other active areas of clinical research include understanding the long-term effects of stem cell transplantation, consideration of new stem cell transplant protocols to improve safety and efficacy, and strategies to therapeutically manipulate the stem cell graft.

Scientific outcomes: It is thought that stem cell transplantation may act as a cellular therapy helping to restore normal function to the immune system by eradicating inflammatory immune cells with chemotherapy and restoring normal immune cell functions from the hematopoietic stem cells (i.e., an immune “reset”). To explore this hypothesis, we take samples from patients throughout the transplant process (intestine, blood) 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. Active areas of research include understanding hematopoietic stem cells in Crohn’s disease, molecular pathways associated with the development of medically refractory Crohn’s disease, and the role of myeloid cells in Crohn’s disease pathophysiology.

Investigators interested in collaboration or accessing data/samples may contact louis.cohen@mssm.edu.

The Crohn’s Disease Stem Cell Transplantation Program is actively involved in research work conducted by Louis Cohen, MD and Judy Cho, MD.

Please visit:

The Autologous Stem Cell Transplantation Program for Crohn’s disease integrates clinical programs across the Icahn School of Medicine at Mount Sinai led by faculty from the Feinstein IBD Center and the Bone Marrow and Stem Cell Transplant Program. The Autologous Stem Cell Transplantation Program also utilizes faculty from the Transplant Infectious Disease Program and the Brookdale Department of Geriatrics and Palliative Medicine to safely and comfortably navigate patients through the transplant process.

The Autologous Stem Cell Transplantation Program for Crohn’s disease integrates research faculty from multiple institutes at the Icahn School of Medicine to understand why patients with Crohn’s develop refractory disease, how stem cell transplantation treats Crohn’s disease and to develop the next generation of cellular and regenerative therapies.

Stem cell transplants for patients with Crohn’s disease have become increasingly popular as a treatment option for many patients in the United States and in Europe. Read the articles and watch the video clips below to gain understanding as to why this treatment option can be a gamechanger in the way we treat Crohn’s disease.

Our multidisciplinary team brings together specialists from diverse fields to provide comprehensive insight and expertise, ensuring the best possible patient outcomes.

Program Director
Louis Cohen, MD

Clinical Director
Aaron Etra, MD

Gastroenterology/Inflammatory Bowel Disease
Judy Cho, MD
Jean-Frederic Colombel, MD
Alexander Greenstein, MD
Daniela Guisado, MD
Maia Kayal, MD
Saurabh Mehandru, MD
Bruce Sands, MD
Ryan Ungaro, MD

Stem Cell Transplantation
Jacques M. Azzi, MD
James Ferrara, MD
Uroosa Ibrahim, MD
Luis M. Isola, MD
Alla Keyzner, MD
John Levine, MD
Adriana K. Malone, MD
Keren Osman, MD

Hematology-Oncology 
Ronald Hoffman, MD
Bridget K. Marcellino, MD, PhD

Psychology
Laurie Keefer, PhD
Claire Raizen, LCSW

Nutrition
Laura Manning, RDN, MPH

Infectious Diseases
Samantha Jacobs, MD

Palliative Care
Ayla Pelleg, MD 

Regulatory and Research
Jonathan Lagdameo
Katha Makwana 

Patient Insurance Navigation
Naghma Ali
Erica Baez
Alema Gonzalez