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ERC

CorticALS Report Summary

Project ID: 639737
Funded under: H2020-EU.1.1.

Periodic Reporting for period 1 - CorticALS (Amyotrophic Lateral Sclerosis from a cortical perspective: towards alternative therapeutic strategies)

Reporting period: 2015-04-01 to 2016-09-30

Summary of the context and overall objectives of the project

Amyotrophic Lateral Sclerosis (ALS) is the most common adult-onset neurodegenerative disease of the motor system, with a prevalence of 2-3/100 000. In spite of intensive research efforts, ALS remains an incurable disease and presents with a very severe prognosis, leading to death within 2 to 5 years following diagnosis.
At the cellular level, ALS is characterized by the combined degeneration of both upper motor neurons in the cerebral cortex (UMN), and lower motor neurons (LMN) in the medulla and spinal cord. This dual impairment allows discriminating ALS from other, less severe diseases affecting either UMN or LMN. Despite this precise clinical description, preclinical studies have so far mostly concentrated on LMN, leaving aside the role of UMN in ALS.
This project aims at shedding light on the contribution of the dysfunction and/or the loss of UMN in ALS, in order to design and test new therapeutic strategies based on the protection and/or the replacement of this exact neuronal type.

Work performed from the beginning of the project to the end of the period covered by the report and main results achieved so far

Work is in progress to understand the contribution of UMN to the onset and progression of ALS. In addition, we have developed new technical approaches to decipher the molecular mechanism behind UMN degeneration in the context of ALS. In turn, this will enable us to develop and test new therapeutic strategies to counteract UMN degeneration during the course of the disease. Finally, we are developing approaches that aim at replacing the UMN that have already been lost at the time of diagnosis.

Progress beyond the state of the art and expected potential impact (including the socio-economic impact and the wider societal implications of the project so far)

The project that started recently has the ambition, in the long run, to provide clinicians and patients with new answers and new therapeutic assets.

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