Clinical
C3G/IC-MPGN lack effective treatment. A variety of anti-complement drugs targeting different molecules and steps of the complement cascade are on the horizon and other target could be developed soon. However, as a reflection of underlying disease complexity, no single treatment will be universally appropriate in IC-MPGN/C3G. Trials to test new therapeutics will be challenging and heavily influenced by the disease heterogeneity. Cluster grouping will be the basis of phase 2 clinical trials to evaluate cluster specific complement inhibitors or drugs targeting the identified biomarkers, to ensure that each patient will receive the right drug offering better chances of recovery.
Public health
The results of this project could have a great impact for National Health Systems. The classification and treatment approaches proposed will significantly improve diagnosis and revolutionize the clinical practice of C3G/IC-MPGN. The combination of hierarchical clustering and cluster-specific biomarker by omics could be a model for the implementation of personalized medicine in other rare and common heterogeneous diseases (i.e. cardiovascular diseases, diabetes, and cancer).
The results of this project could have a great impact for National Health Systems. The classification and treatment approaches proposed will significantly improve diagnosis and revolutionize the clinical practice of C3G/IC-MPGN. The combination of hierarchical clustering and cluster-specific biomarker by omics could be a model for the implementation of personalized medicine in other rare and common heterogeneous diseases (i.e. cardiovascular diseases, diabetes, and cancer).
Public health
Socio
economic
The innovative strategies toward personalized medicine targeted by DECODE will have major impact on the life of affected children and adults. About half of C3G/IC-MPGN patients, mostly children, develop end-stage renal diseases and need dialysis within 10 years from onset. An effective therapy would most likely delay or halt the progression of renal failure and dramatically improve the health status, life expectancy, and quality of life of these children. The advent of efficacious new drugs in these diseases will be highly cost-effective since every patient in whom renal replacement therapy can be prevented will provide substantial savings to the health systems (e.g. 40-50k € mean annual dialysis cost per patient). Finally, the identification of a cheaper alternative to the complement inibitor Eculizumab, one of the most expensive drugs, would save many millions to health care systems.
Validated cluster-specific biomarkers will promote the industrial development of diagnostic kits and therapeutic compounds targeting the biomarkers. Moreover, the proposal will provide a user friendly diagnostic platform for clinicians to rapidly stratify new patients into a cluster and to direct them toward a more PM personalised medicine. The Coordinator will commit on-site maintenance of the platform after the project end. We anticipate opportunities for academic maintenance and further development through new project applications. We will also appoint efforts aimed at transferring the tool into a certified solution with commercial exploitation.
Industrial
Industrial
Validated cluster-specific biomarkers will promote the industrial development of diagnostic kits and therapeutic compounds targeting the biomarkers. Moreover, the proposal will provide a user friendly diagnostic platform for clinicians to rapidly stratify new patients into a cluster and to direct them toward a more PM. The Coordinator will commit on-site maintenance of the platform after the project end. We anticipate opportunities for academic maintenance and further development through new project applications. We will also appoint efforts aimed at transferring the tool into a certified solution with commercial exploitation.
