An extraordinary study, the result of research conducted at the Bambino Gesù Children’s Hospital (Italy) in collaboration with the University of Erlangen (Germany), was recently published in Nature Medicine.

Eight paediatric patients, suffering from autoimmune diseases refractory to treatment, were able to completely discontinue immunosuppressive therapies following CAR-T cell treatment (a type of advanced therapy). These children were affected by particularly aggressive forms of paediatric-onset autoimmune diseases: four had systemic lupus erythematosus (SLE), three had dermatomyositis (a rare inflammatory and autoimmune pathology), and one had juvenile systemic sclerosis (a rare chronic autoimmune disease). All patients had complex clinical histories with previous partial or temporary responses to numerous immunosuppressive treatments.

Following the CAR-T treatment, seven of them are in clinical remission, while the eighth, who has systemic sclerosis, is showing a continuous and significant reduction in disease severity without progression.

The treatment uses CAR-T cells that target the CD19 antigen expressed by B lymphocytes in the immune system. In B-mediated autoimmune diseases, B lymphocytes play a crucial role; this treatment eliminates them in a targeted manner, leading to reduced inflammation and the restoration of immune system balance. This increases the likelihood of long-term remission without the need for chronic therapies. Data from a follow-up of over 24 months already demonstrate that clinical benefits are maintained over time.

This study is highly relevant for paediatric and rare autoimmune diseases, illustrating how advanced therapies, which have grown rapidly over the last decade, can offer a concrete treatment and cure.

To learn more about: Su Nature Medicine lo studio che dimostra l’efficacia delle cellule CAR-T nelle malattie autoimmuni dell’età pediatrica – Ospedale Pediatrico Bambino Gesù