A collaboration between the University of Malta (UM) and Shanghai Jiao Tong University is focused on combating amyotrophic lateral sclerosis (ALS), a severe neurodegenerative disease.
The genetics of the Maltese population contribute to a higher incidence of ALS compared to the European average. It is projected that the ageing population will lead to a more than 20% increase in ALS cases in Europe and a 50% increase in China over the next two decades.
Project astrALS aims to identify common molecular targets for developing therapies that address the diverse forms of ALS, which has complicated the development of broadly effective treatments.
The research will harness the power of model organisms, including fruit flies (Drosophila), to understand how genetic alterations associated with ALS disrupt cellular processes. While simple, these organisms provide highly informative systems due to their conservation of key biological pathways that are also found in humans. Their fast generation times and suitability for genetic manipulation make them ideal for high-throughput investigation of disease mechanisms.
One of the project’s core objectives is to uncover therapeutic vulnerabilities that are shared across different genetic forms of ALS
By applying cutting-edge omic technologies, including transcriptomics and proteomics, the project will generate comprehensive datasets that map the impact of a global ALS risk factor on gene expression and protein function. These data will then be integrated to reveal common patterns and pathways affected by the disease across different biological models.
The collaboration combines UM’s long-standing expertise in motor neuron disease research with the technological capabilities of Shanghai Jiao Tong University, particularly in large-scale molecular profiling and data analysis. This partnership exemplifies how international scientific cooperation can enhance discovery and innovation.
One of the project’s core objectives is to uncover therapeutic vulnerabilities that are shared across different genetic forms of ALS. Targeting these shared mechanisms holds promise for the development of therapies that are not limited to specific patient subgroups, but rather have broad applicability.
This research is expected to generate novel insights into how ALS develops and progresses, and provide a robust scientific foundation for the development of future treatments. It also reflects Malta’s growing contribution to global neuroscience research and our commitment to improving outcomes for those living with ALS.
As the project moves forward, we remain optimistic that it will lead to discoveries that not only deepen our understanding of ALS but also pave the way for innovative therapies that can truly make a difference.
Project astrALS received funding from Xjenza Malta and the Ministry for Science and Technology of the People’s Republic of China (MOST) through the SINO-MALTA Fund 2024 Call (Science and Technology Cooperation).
Francesca Grech is a research support officer II for project astrALS at UM’s Faculty of Medicine and Surgery, while Ruben J. Cauchi is a professor of Neurogenetics and heads the Motor Neuron Disease Lab there.
Photo of the week
Photo: University of Malta ALS/MND Lab
Freshly collected blood samples on ice from a newly diagnosed ALS patient in Malta. Processed at the University of Malta’s Motor Neuron Disease Lab, the DNA (Deoxyribonucleic acid) in the samples will be stored at Malta’s National ALS Registry and Biobank to support research by Maltese scientists into the genetic factors driving ALS in the Maltese population. The most recent study using DNA from Maltese ALS patients revealed that the genetic factors driving the disease in Malta are distinct, highlighting a unique ALS genetic profile within the population of Malta.
Sound Bites
• In a major breakthrough, a new drug called tofersen has been shown to slow down a genetic form of ALS. It works by targeting the faulty SOD1 gene and reducing the production of toxic proteins in the body. People who started the treatment early showed slower disease progression and improved strength.
• Project MinE is a global research effort dedicated to uncovering the genetic causes of ALS. In their largest study to date, scientists, including those from the University of Malta, analysed DNA from over 29,000 people with ALS and 120,000 without. They discovered 15 new genetic risk patterns, shedding light on how the disease starts and offering new clues for future treatments.
For more soundbites, listen to Radio Mocha www.fb.com/RadioMochaMalta/.
DID YOU KNOW?
• Jean-Martin Charcot, a pioneering 19th-century French neurologist, often called the “father of modern neurology”, was the first to describe and name ALS in 1869.
• In 1993, scientists discovered that flaws in the SOD1 gene are linked to some inherited cases of ALS, marking a major milestone in ALS research.
• Riluzole, approved in 1995, was the first treatment to alter ALS’s course and remains the only broadly available option today, extending survival by just two to three months.
• In 2014, over 17 million people took part in the viral ALS Ice Bucket Challenge, raising over €185 million for ALS research in just a few months.
For more trivia, see: www.um.edu.mt/think.
