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Refractory Celiac Patient Successfully Treated With Mesenchymal Stem Cell Infusions


For the first time, stem cells have been used to successfully treat refractory celiac disease. Photo: CC--Reza Ahmed

Celiac.com 05/24/2016 - People with type II refractory celiac disease (RCD), suffer from severe malabsorption syndrome and face a poor prognosis, as there is currently no effective treatment.

Prompted by the regenerative and immune-influencing properties of mesenchymal stem cells (MSCs), a research team recently set out to assess the viability, safety, and efficacy of a series of infusions of autologous bone marrow-derived MSCs in a 51-year-old woman with type II RCD.

The research team included R Ciccocioppo, A Gallia, MA Avanzini, E Betti, C Picone, A Vanoli, C Paganini, F Biagi, R Maccario, and GR Corazza. They are variously affiliated with the Department of Internal Medicine, Fondazione IRCCS Policlinico San Matteo and Università degli Studi di Pavia, the Department of Internal Medicine, Fondazione IRCCS Policlinico San Matteo and Università degli Studi di Pavia, the Cell Factory and Research Laboratory, Department of Pediatrics, Fondazione IRCCS Policlinico San Matteo and Università degli Studi di Pavia, the Clinic Cytometry Laboratory, Department of Hematology, Fondazione IRCCS Policlinico San Matteo and Università degli Studi di Pavia, Department of Molecular Medicine, Fondazione IRCCS Policlinico San Matteo and Università degli Studi di Pavia, all in Pavia, Italy.

The team began by isolating, expanding, and characterizing mesenchymal stem cells using standard clinical protocols. For each patient, the team arranged to monitor malabsorption indexes, mucosal architecture, and percentage of aberrant intraepithelial lymphocytes at the time of enrollment, at each infusion, and after 6 months.

The also arranged to assess mucosal expression of interleukin (IL)-15 and its receptor. Once the team determined that the expansion of MSCs was feasible, they provided the patient with four systemic infusions of 2 × 106 MSCs per kg body weight 4 months apart, with no adverse effects.

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Over the course of the treatment, the patient experienced gradual and durable improvement of her condition, including normalized stool frequency, body mass index, laboratory test results, and mucosal architecture. Most impressively, the expression of IL-15 and its receptor almost completely vanished.

Based on this clinical case, treatment of RCD with serial MSC infusions seems to offer a path to recovery from this life-threatening condition, while blocking the IL-15 pathogenic pathway.

This is the first successful treatment of refractory celiac disease. Stay tuned for further developments regarding the use of stem cell infusions to treat refractory celiac disease.

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4 Responses:

 
Adam Liebi
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said this on
24 May 2016 6:15:42 PM PDT
Glad to know research is advancing in this arena. Good synopsis!

 
Matthew
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said this on
24 May 2016 11:14:30 PM PDT
It appears this treatment requires collecting a patient's bone marrow.
I would like to make you aware of a company called Cynata (Cynata.com) and its unique Cymerus technology which addresses many of the complexities and challenges of manufacturing MSCs at commercial scale.
They can manufacture robust and consistent MSCs in an economically viable process – all under Good Manufacturing Practice requirements, providing a starting material with unlimited expansion potential, which means that they and their commercial partners can source all the cells you will ever need from a single blood donation.
I would encourage you to check them out.

 
Pippy
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said this on
03 Jun 2016 11:13:40 PM PDT
Matthew, fascinating, thank you for sharing.

 
Fran LaChance
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said this on
31 May 2016 3:39:49 AM PDT
Great research, great article! So necessary, for those of us who have ongoing issues with CD. Thanks, Jefferson!




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