Hematopoietic stem cell transplant for treating severe and refractory Crohn's disease patients

News  El País: 8 July 2016

 

The stem cell research undertaken, and ongoing, at the the Inflammatory Bowel Disease (IBD Unit), Barcelona has been profiled at "El Pais", Spain's highest-circulation daily newspaper.

 

The article (in Spanish only) can be viewed by clicking on the photo at left.

Former Crohn's patient Javier Casado beside Dr. Elena Ricart of Hospital Clinic Barcelona (on right) and Investigator Azucena Salas, Ph.D. of IDIBAPS (on left). / P.E.

Pharmaceutical treatment of Crohn’s disease is based upon suppression of the immune system using non-specific drugs and blockade of inflammatory processes by biological therapy. However, a significant percentage of patients remain refractory or intolerant to these therapies and require repeated surgeries to manage disease complications. Surgery, however, does not provide long-lasting resolution of the inflammatory process, and recurrence after surgery occurs in the majority of patients with CD.

Nurse Anna Ramirez meeting with a patient.

The use of autologous hematopoietic (bone marrow) stem cell (HSCT) transplantation as rescue therapy in patients with refractory Crohn’s disease or who are intolerant to conventional therapies, including biological therapy, was recently evaluated by the European Group for Blood and Marrow Transplantation (ASTIC Trial; paper under review). Despite the dramatic benefits of this stem-cell therapy, it is associated with a mortality rate of 2-10%, which makes it an acceptable option only for a reduced set of severe, highly refractory patients.

 

We propose that HSCT exerts a beneficial effect and halts the progression of CD by inducing an immune reset. During the HSCT procedure, chemotherapy may restore tolerance towards components of the intestinal flora as it existed before development of the disease.

Funding:

- Spanish Ministerio de Ciencia e Innovación; SAF 2012-33560; PI: Julian Panés

- Leona and Harry Helmsley Trust; PI: Azucena Salas

- R+D National Fund from Spanish Ministry of Economy and Innovation (I+D SAF2015-66379)

  PI's: Azucena Salas & Julian Panés

Publications:

Differences in peripheral and tissue immune cell populations following hematopoietic stem cell transplantation in Crohn's disease patients.

Corraliza AM, Ricart E, López-García A, Masamunt MC, Veny M, Esteller M, Mayorgas A, Le Bourhis L, Allez M, Planell N, Visvanathan S, Baum P, España C, Cabezón-Cabello R, Benítez-Ribas D, Rovira M, Panés J, Salas A.

 

J Crohns Colitis. 2018 Dec 6.

doi: 10.1093/ecco-jcc/jjy203

 

Autologous Haematopoietic Stem Cell Transplantation for Refractory Crohn's Disease: Efficacy in a Single-Centre Cohort.

López-García A, Rovira M, Jauregui-Amezaga A, Marín P, Barastegui R, Salas A, Ribas V, Feu F, Elizalde JI, Fernández-Avilés F, Martínez C, Gutiérrez G, Rosiñol L, Carreras E, Urbano A, Lozano M, Cid J, Suárez-Lledó M, Masamunt MC, Comas D, Giner A, Gallego M, Alfaro I, Ordás I, Panés J, Ricart E.

J Crohns Colitis. 2017 Oct 1;11(10):1161-1168.

doi: 10.1093/ecco-jcc/jjx054.

Autologous Hematopoetic Stem Cell Transplantation for Refractory Crohn Disease: A Randomized Clinical Trial.

Hawkey CJ, Allez M, Clark MM, Labopin M, Lindsay JO, Ricart E, Rogler G, Rovira M, Satsangi J, Danese S, Russell N, Gribben J, Johnson P, Larghero J, Thieblemont C, Ardizzone S, Dierickx D, Ibatici A, Littlewood T, Onida F, Schanz U, Vermeire S, Colombel JF, Jouet JP, Clark E, Saccardi R, Tyndall A, Travis S, Farge D.

 

JAMA. 2015 Dec 15;314(23):2524-34.

doi: 10.1001/jama.2015.16700.

Improving safety of autologous haematopoietic stem cell transplantation in patients with Crohn's disease.

Jauregui-Amezaga A, Rovira M, Marín P, Salas A, Pinó-Donnay S, Feu F, Elizalde JI, Fernández-Avilés F, Martínez C, Gutiérrez G, Rosiñol L, Carreras E, Urbano A, Lozano M, Cid J, Suárez-Lledó M, Mensa J, Rimola J, Rodríguez S, Masamunt MC, Comas D, Ruíz I, Ramírez-Morros A, Gallego M, Ordás I, Panés J, Ricart E.

 

Gut. 2015 Nov 19. pii: gutjnl-2015-309836.

doi: 10.1136/gutjnl-2015-309836.

 

 

Cell therapies for IBD: what works?

Ricart E, Jauregui-Amezaga A, Ordás I, Pinó S, Ramírez AM, Panés J.

Curr Drug Targets. 2013 Nov;14(12):1453-9. Review.

PMID: 24160439

 

 

Cell therapies for inflammatory bowel diseases.

Salas A, Ricart E and Panés J

 

J. Exp Rev Gastro 3(4):321-4; 2009.

doi: 10.1586/egh.09.26. / PMID: 19673617

 

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