Inflammatory Myositis

This study primarily involves the use of BCT301, an anti-CD19 Chemically induced pluripotent stem cell (CiPSC)-derived CAR-iT cells, for the treatment of patients with refractory autoimmune diseases, aiming to evaluate its safety, tolerability, and dose-limiting toxicities(DLT), and to determine the recommended therapeutic dose for further investigation. Additionally, the study assesses the efficacy of BCT301 cell injection in refractory autoimmune diseases, as well as the pharmacokinetic (PK) and pharmacodynamic (PD) characteristics in study participants.

Myositis are rare diseases for which the development of a cohort associated with a bank of biological samples (biobank) will allow for the conduct of researches to better delineate the underlying pathophysiology and find cures. This prospective cohort of patients with myositis will allow for identification of factors favouring the occurrence of myositis, whether they are constitutional (genetic) or acquired (environmental or drug). Different subgroups of myositis used for prognostication will be identified based on clinico-demographical variables, the nature of the organs involved beyond peripheral muscles (cardiac, diaphragm) and biomarkers abnormalities

The rare autoimmune rheumatic diseases (RAIRDs) are life-long multi-system diseases that are life or organ threatening. RAIRDs can impair quality of life similar to chronic diseases such as heart failure. The aim of the study is to explore content and structure of a support programme for people with RAIRDs in focus groups and survey meetings.

Blood platelets, well known for their role in hemostasis, are abnormally activated in patients suffering from systemic lupus erythematosus (SLE), but also from other immunomediated diseases (scleroderma, vasculitis, myositis, Gougerot-Sjögren's and rheumatoid arthritis) in cases of high disease activity. Once activated, platelets express adhesion molecules such as P-selectin on their surface, enabling them to interact physically with immune cells. In a recent work, we identified that activated platelets from lupus patients interact with regulatory T cells and block their regulatory function, thus participating in the deregulated activation of the immune system in SLE. In addition, inhibition of platelet-immune cell interactions by an anti-P-selectin antibody improved LES symptoms in two mouse models. The aim of this work is to investigate other potential platelet-immune cell interactions in patients with SLE, in comparison with other autoimmune diseases (systemic scleroderma, ANCA vasculitides, inflammatory myositis, Gougerot-Sjögren syndrome and rheumatoid arthritis). This study could lead to a better understanding of the role of platelets in the pathophysiology of autoimmune diseases, identify new biomarkers of activity, and assess the potential of new therapeutic avenues in these diseases, such as platelet targeting.