Pediatric Hematology Service, Timone Hospital
Our service treats children with serious blood disorders. Some of these diseases are fairly common such as leukemia and lymphoma, other are much rarer including: hemoglobin abnormality (sickle cell disease), bone marrow deficiency (idiopathic or constitutional medullary aplasia) and severe combined immunodeficiency (SCID). The service also incorporates a specialised unit for haematopoietic stem cell transplants. Thus, when the treatments fail to overcome severe forms of these diseases, we replace the diseased bone marrow of our patients with a healthy one from hematopoietic stem cells of a compatible donor, to allow them to "restart from scratch". Each year, we carry out on average 30 transplants, two thirds of these are in children with leukaemia, placing the service in third place after the hôpital Necker and hôpital Robert-Debré.
We also want out patients to quickly benefit from the latest advances in genetics: treating children with thalassaemia or anaemia using a "repair gene" or arming our patients’ lymphocytes so they can at long last destroy cancer cells that have evaded their immune system (CAR T-cell therapy).
Given the complexity of the diseases we treat, we also work in close collaboration with other specialised units and research centres, in France and internationally. Therefore, along with Claudine Shiff's laboratory at CIML, we have been able to shed light on part of the biological mechanisms involved in agammaglobulinaemia. More recently, with Eric Viver's team at the CIML and Alain Fisher's teams at the Institut Imagine and Hôpital Universitaire Necker-Enfants Malades, we have been investigating post-transplant reconstruction of different immune cell populations in patients affected by SCID. We have discovered that these patients have very few or no innate lymphoid cells (ILCs), and yet do not develop more cancers, infections or inflammatory diseases than the control group. Given the strategic immune function of these cells, this discovery is astonishing but at the same time it demonstrates the redundancy and robustness of our immune system, which is great news for our patients. The fact remains that these same cells could, in the near future, also allow us to prevent Graft Versus Host Disease (GDHD), which is the main complication in transplants at the present time. It’s at any rate the supposition that we are exploring with Fréderic Vely, researcher at CIML and manager of Marseille Immunopole immuno-profiling platform.
From our service’s perspective, MI is therefore already proving its worth. Thanks to this unprecedented alliance of researchers, clinicians, technological platforms and industrials we should be able to quickly do much better: increasing our transplant unit's capacity, leading to more research programs and also taking advantage of new tools such as Next Generation Sequencing, so that in the near future we will be able to simultaneously characterise up to 300 immunodeficiencies!Back
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