Clinical Research

Applications of Stem Cells

Our research group carries out stem cell clinical research with the main focus on hematological and autoimmune diseases such as aplastic anemia, acute promyelocitic leukemia, acute myeloid leukemia, multiple sclerosis, systemic sclerosis, type 1 diabetes, and graft versus host diseases. Hematopoietic stem cells (HSC) and mesenchymal stromal cells (MSC) are enrolled in those studies and the main topics of clinical research are illustrated in the figure below. We also developed protocols for large-scale production of MSCs.


Schematic representation of the main diseases and clinical conditions that are being considered for cell-based therapy. HSC: Hematopoietic stem cells; MSC mesenchymal stromal cells: IC-AL= International Consortium – Acute Promyelocytic Leukemia.

Clinical Researches list

1. To recruit and follow 10 patients with refractory/relapsed aplastic anemia for treatment with immunosuppression and mesenchymal stromal cells and to observe safety, hematologic response and MSC homing to the bone marrow niche.

2. To investigate novel biomarkers in Acute Promyelocytic Leukemia (APL) and to determine the impact of networking on the outcome of patients with acute promyelocytic leukemia in developing countries.

3. To determine the relevance of p73 isoforms in the response to treatment of Acute Promyelocytic Leukemia (APL).

4 .Treatments of Acute Myeloid Leukemia.

4.1. Establishment of an international multicenter collaborative group.

4.2. Use of Genome-wide DNA methylation mapping of Leukemic Stem Cells (LSCs) to identify novel biomarkers of daunorubicin and cytarabine response in Acute Myeloid Leukemia (AML).

5. Treatment of patients with inflammatory multiple sclerosis with high dose immunosuppression followed by autologous hematopoietic stem cell transplantation (HDI/AHSCT).

6. Treatment of patients with early-diagnosed type 1 diabetes with HDI/AHSCT.

7. Treatment of patients with systemic sclerosis with HDI/AHSCT.

8. Evaluation of the immune mechanisms of action of the HDI/AHSCT in type 1 diabetes, multiple sclerosis and systemic sclerosis patients.

9. Treatment of patients with graft-versus-host disease, in the setting of haploidentical allogeneic HSCT, with allogeneic bone marrow-mesenchymal stromal cell infusions.

10. and 11. Development of allogeneic mesenchymal stromal cells from umbilical cord (MSCs-UC) banks for “off-the-shelf” clinical use. Treatment of patients with acute graft-versus-host disease, refractory to corticosteroids, with allogeneic umbilical cord-mesenchymal stromal cells infusions.

12. Allogeneic hematopoietic stem cell transplantation for sickle cell anemia: evaluation of clinical response and inflammatory markers.

For more detailed information on clinical research download our activity reports.

Activities Report of 2014