Final Report Summary - TARGET GLOMDIS (Glomerulonephritis and Focal Segmental Glomerulosclerosis as a Model to Investigate the Link between Inflammation and Kidney Disease: From Basic Mechanisms to Clinical Application)
Rapidly Progressing Crescentic Glomerulonephritis (RPGN) and Focal Segmental Glomerulosclerosis (FSGS) are severe kidney diseases responsible for irreversible renal failure, which is a major risk factor for cardiovascular mortality. RPGN can even result in a loss of kidney function within days or weeks. For this reason, these diseases are a significant issue in terms of public healthcare expenses. In France, the annual cost for maintenance of dialysis reaches 2.1 billion €, of which an estimated 12% (250 M€) is related to care for patients suffering from RPGN. Similar expenses are generated for treatment of end-stage renal disease (ESRD) subjects diagnosed with FSGS.
Most importantly, these pathologies greatly impact the quality of life of patients surviving with disease, due to the side effects of harsh and non-specific treatments applied, the need for continued dialysis, and related personal expenses. Despite the aggressiveness of immunosuppressive protocols applied, treatments against RPGN have limited effectiveness, and the prognosis for retaining kidney function in RPGN patients is only 72% after 5 years. Similarly, there is no approved, specific treatment for FSGS, and current therapies only aim to control the disease consequences (e.g. blood pressure, proteinuria).
The ambition of the TARGET GLOMDIS project has been to make progress in order to indentify therapeutic targets for Rapidly Progressing Crescentic Glomerulonephritis (RPGN) and Focal Segmental Glomerulosclerosis (FSGS) that
I- would be specific to the pathophysiological mechanism,
II- would improve the renal outcome that still remains dramatically poor,
III- would be well tolerated,
IV- would be pharmacologically compatible with current standard therapies, which may be then alleviated.
The TARGET GLOMDIS project has mobilised multidisciplinary expertise in order to shed light on the key mechanisms and progression of RPGN and FSGS development, based on exploitation of biomarkers and the conduction of observational trials in a preclinical set-up. Ultimately, the results of the project is being exploited for the preparation of clinical trials aiming to improve clinical practice in the management of patients suffering from acute or chronic renal insufficiency.
Most importantly, these pathologies greatly impact the quality of life of patients surviving with disease, due to the side effects of harsh and non-specific treatments applied, the need for continued dialysis, and related personal expenses. Despite the aggressiveness of immunosuppressive protocols applied, treatments against RPGN have limited effectiveness, and the prognosis for retaining kidney function in RPGN patients is only 72% after 5 years. Similarly, there is no approved, specific treatment for FSGS, and current therapies only aim to control the disease consequences (e.g. blood pressure, proteinuria).
The ambition of the TARGET GLOMDIS project has been to make progress in order to indentify therapeutic targets for Rapidly Progressing Crescentic Glomerulonephritis (RPGN) and Focal Segmental Glomerulosclerosis (FSGS) that
I- would be specific to the pathophysiological mechanism,
II- would improve the renal outcome that still remains dramatically poor,
III- would be well tolerated,
IV- would be pharmacologically compatible with current standard therapies, which may be then alleviated.
The TARGET GLOMDIS project has mobilised multidisciplinary expertise in order to shed light on the key mechanisms and progression of RPGN and FSGS development, based on exploitation of biomarkers and the conduction of observational trials in a preclinical set-up. Ultimately, the results of the project is being exploited for the preparation of clinical trials aiming to improve clinical practice in the management of patients suffering from acute or chronic renal insufficiency.