The objective remains one: financially support research projects that contribute to learning more about Amyotrophic Lateral Sclerosis (ALS) in order to improve early diagnosis and develop new treatments that prevent or, at least, slow down the progress of the disease and improve the quality of life of patients.
This is, once again, the reason why the ”la Caixa” Foundation and the Francisco Luzón Foundation have signed a second collaboration for the next five years, a period in which they will support five research projects on ALS with between 500,000 and 1 million of euros each. The ”la Caixa” Foundation will contribute 75% of the amount through the CaixaResearch call for Health Research, and the Francisco Luzón Foundation, 25% through the ELA Talent program. The budget will be dedicated to promoting one project per year, selected by an independent scientific committee.
ALS causes a progressive loss of motor neurons, which leads to muscle weakness and atrophy, leading to complete immobility in the affected person. Life expectancy is variable: 80% of patients die in the first five years. Therefore, it is necessary to continue providing resources for research into this disease. In the last decade, important advances have been made in understanding its causes and mechanisms thanks to basic and therapeutic research. However, ALS continues to be a rare disease, currently incurable and of unknown cause that affects approximately 200,000 people in the world, 4,000 of them in Spain.
This new agreement between the ”la Caixa” Foundation and the Francisco Luzón Foundation adds to the one they already signed in 2017 to support five first research projects on ALS from very different perspectives, but with a common purpose: to advance in the understanding of the causes of the neurodegeneration process and thus be able to improve the diagnosis and treatment of the disease. Since then, both institutions have allocated close to 2.5 million euros to five studies on the disease, led by researchers from the Autonomous University of Barcelona, the CEU San Pablo University, the Miguel Servet-Navarrabiomed Foundation, the Biological Research Center Margarita Salas of the CSIC and the National Center for Oncological Research (CNIO).
The first of the projects to be completed is developing a treatment against neuroinflammation based on the oral administration of a lipid derived from omega-3 fatty acids. Although the causes of the disease are still unknown, it is known that neuroinflammation is one of the pathogenic mechanisms that contribute to its appearance and progression, but, until now, anti-inflammatory drugs have been ineffective in treating the symptoms and slowing down its evolution.
Rubén López Vales, a researcher at the Universitat Autònoma de Barcelona, and his team have shown that oral administration of a lipid derived from omega-3 fatty acids, maresin, has greater therapeutic effects than riluzole, the only drug approved for ALS in Europe. The project has successfully tested the efficacy of maresin in animal models in reducing inflammation and slowing disease progression. After this preclinical stage, toxicity and pharmacokinetic studies are now being carried out as a step prior to the start of a clinical study in patients with the ultimate goal that these results end up being transformed into a treatment that reaches people affected by ALS.
The second of the projects, led by the researcher Carmen María Fernández-Martos at the CEU San Pablo University, studies the neuroprotective role of leptin, a hormone related to obesity, and that scientific evidence shows that it is associated with a lower risk of developing ALS, conferring a survival advantage on patients. Already at the Miguel Servet-Navarrabiomed Foundation, Maite Mendioroz and Ivonne Jericó are leading a third project that works to develop a non-invasive detection test for the disease. In it, a technique recently developed in the field of oncology is being used to apply it to patients with ALS, known as "liquid biopsy", which makes it possible to isolate DNA fragments in patients with ALS and to identify new genes that could be used as diagnostic biomarkers. and disease progression.
The fourth of the research projects, led by Ana Martínez, from the CSIC's Margarita Salas Biological Research Center, focuses on a new compound that recovers the functionality of the TDP-43 protein, which ALS patients have modified and which could prevent the death of motor neurons, whose death is responsible for the functional paralysis that characterizes patients affected by ALS.
Exploring the role of nucleolar stress in ALS to identify new therapeutic targets that make it possible to treat the disease is the subject of the latest study, headed by Óscar Fernández-Capetillo, from the National Center for Oncological Research. With the help of the “la Caixa” Foundation, the laboratory will continue trying to understand how mutations in RNA-related genes lead to ALS, and trying to exploit this knowledge to develop new treatments.
