NeuroDegenerative Diseases such as Alzheimer disease (AD), Parkinson disease (PD) and Amyotrophic Lateral Sclerosis (ALS)are amongst the most crucialmedical challenges of our time. The economic costs of the NeuroDegenerative Diseases are large, and include not only treatment costs, but also the chronic loss of productivity of patients and their caregivers. In Europe, the total cost of brain disorders has been estimated at €386 billion per year in 2004 prices. The overall societal costs, taking into account the drastic effects on the quality of life of patients and their families, are clearly much higher.
POLARIS will focus on Amyotrophic Lateral Sclerosis (ALS),a rapidly fatal disease with no effective treatment, and still lacking a biomarker for early diagnosis. The annual cost in the USA has been estimated at US$ 69,475 per patient-year, or US$ 279-472 million per year in total. Costs associated with ALS are proportionally greater than for other NDD, highlighting the desperate need of financial investments not only for the care of patients and their families, but also to promote research-driven medical advances.
Up until now most ALS clinical trials have reported negative results, and no effective disease-modifying treatment has been introduced since Riluzole, whereas animal models like the SOD1 transgenic mice have so far failed to predict efficacy of novel therapeutic approaches in humans. Thus the development of alternative preclinical drug-screening platforms, including human validation assays, is mandatory.
POLARIS aims to fulfil this objective by implementing novel cutting-edge technologies and multidisciplinary approaches. In particular, POLARIS will combine two emerging technologies, namely hiPSC-derived cell lines and miniaturized Organ-on-Chip (OoC) cell culture systems, to create human-relevant, real-time accessible, standardized and cheaper disease models. The implementation and use of OoC models based on human iPSCs in the NeuroDegenerative Diseases is expected to revolutionize both basic research and preclinical experimentation. By providing a system that may be tailored to the individual patient, it will enable the investigation of specific pathogenetic mechanisms. In parallel, its application to drug development will make drug discovery faster and cheaper compared to conventional processes. This approach is widely predicted to become a crucial tool of Personalized (Precision) Medicine spanning from diagnoses to monitoring of disease progression and treatment, and a major disruptive technology leading to a radical improvement of clinical care standards.
POLARIS will use induced pluripotent stem cells (iPSCs) derived from ALS patients that carry defined disease-causing mutations as well as from sporadic patients. Neural lineage cells derived from these iPSCs will be used to generate OrganOnChip cultures replicating the human CNS (BrainOnChip). The devices will be applied to identify leads that prevent the death of ALS motor neuron and that will be further validated in subsequent clinical trials. The approaches pursued and the results obtained by POLARIS in the context of ALS will also be applicable to other neurodegenerative diseases such as PD and AD.
POLARIS partners (CNR Nanotec, the Institute of Experimental Neurology (INSPE), San Raffaele Scientific Institute (Milano and Lecce), the Department of Clinical Research in Neurology, University of Bari and the University of Salento) include basic and clinical scientists representing the cutting edge of Italian research on NeuroDegenerative Diseases and nanotechnology. Each participant has specific expertise in one or more key aspects of the research plan, so that considerable interdisciplinary added value is achieved. The combination of both basic science and clinical research expertise is central for the success of this translational project.
POLARIS will also actively explore aspects related to the industrialization and commercialization of the technology, with an aim to secure its wide availability at reasonable costs to the benefit of patients, and to promote the creation of new companies/jobs in a hi-tech sector with an explosive future ahead.
Finally, POLARIS will offer exciting job opportunities to young talented researchers in a leading neurobiology/nanotechnology center in the South of Italy, with a prospect to translate their research via start-up companies.
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