Fundación Occident, BioBizkaia andDalecandELA, together against ALS

Fundación Occident teams up with BioBizkaia and DalecandELA in a research project on amyotrophic lateral sclerosis (ALS)

Amyotrophic lateral sclerosis (ALS) is a serious neurodegenerative disease. In the absence of effective treatment, there is a pressing need to develop therapies which halt or significantly slow the progression of the disease. Breakthroughs in basic research are making it possible to identify therapeutic targets in preclinical ALS models. However, the effectiveness of these therapies has not been translated into successful clinical trials. This means that bridging methods are essential to effectively move therapeutic targets from preclinical models to ALS patients.

The average lifespan of patients is 4-5 years from the onset of the disease. From the onset of the disease, half of people with ALS die in less than 3 years, 80% in less than 5 years, and the majority (95%) in less than 10 years (Spanish Society of Neurology, 2022).

The DalecandELA association, founded in 2019, works to increase awareness of this disease among society, governments and institutions, raise funds for research and help improve the quality of life of people with ALS. 

Fundación Occident is teaming up with the health research institute BioBizkaia and DalecandELA to fund a research project on amyotrophic lateral sclerosis (ALS). This project, called “TranslationALStress”, is led by Dr Rosario Sánchez Perraute and Dr Montse Arrasate Iragui. Its aim is to develop individualised therapies, through either gene or pharmacological therapy, which modulate the response of the cells to stress (ISR). The ultimate goal is to speed up the process so that we can move on to the next clinical trial quickly, safely and effectively.

This study might help to treat at least 60% of familial ALS cases and potentially a high percentage of sporadic ALS cases.It also addresses tapping genomic information to devise a personalised therapy for ALS. It pursues two parallel strategies for ALS to precisely target affected cells with either adeno-associated virus-based gene therapy or pharmacological therapy.

Amyotrophic lateral sclerosis (ALS) is a degenerative neuromuscular disease in which the neurons controlling voluntary movement become affected and die and this leads to progressive paralysis. The disease affects motor neurons in the cerebral cortex (upper motor neurons), brainstem and spinal cord (lower motor neurons). The initial muscle weakness progresses to paralysis, spreading from one area of the body to another and compromising motor autonomy, oral communication, swallowing and breathing. Death is most often due to complications arising from respiratory failure as a result of involvement of muscles such as the diaphragm and intercostal muscles which are needed for the respiratory pump to operate. Although cognitive impairment has been recognised in a subset of patients, most are fully aware of their decline.