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.

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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