Impact

Clinical trial design

‍FFI, like all other prion diseases, lacks an effective treatment. Testing potential drugs is challenging because the disease is very rare and has a highly variable age of onset, making it difficult to decide when to start the treatment. At the moment the only way to establish the efficacy of a drug is to monitor the treated individuals for a very long time to see how many of them will develop the disease. ProFFIle is expected to identify disease biomarkers that may indicate when to start treatment and allow monitoring treatment efficacy in real time, enabling the design of more tractable clinical trials.

‍The establishment of reliable disease biomarkers will allow early diagnosis of FFI, and most likely other prion diseases which share the same disease mechanism.

Early diagnosis

Research resources

‍ProFFIle will generate a large collection of biological fluids from FFI individuals, which will be not only instrumental for biomarker discovery and validation, but will also constitute a unique resource for the whole scientific community.

‍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