Understanding environmental and biological causes of ALS.
The search for effective ALS therapies begins with a deep dive into the biology of the disease. Understanding how ALS disrupts key cellular and molecular processes is essential to uncovering new drug targets. These biological pressure points, if addressed, could slow or stop disease progression.
While motor neurons and the central nervous system remain central to ALS research, Target ALS is expanding the field’s focus by supporting studies that explore the broader biological landscape, including the immune system, metabolic pathways, and even peripheral tissues. This broader perspective is helping uncover previously overlooked mechanisms that may contribute to disease onset or progression.
At the 2025 Annual Meeting, consortia funded by Target ALS shared bold new discoveries made possible by advanced techniques in proteomics, transcriptomics, and single-cell analysis. These projects are charting new territory in ALS pathology and laying the groundwork for tomorrow’s therapies by targeting the roots of the disease, not just its symptoms.
Read more about the projects below:
Cracking the Code: How Cutting-Edge Biology Is Uncovering New Drug Targets for ALS
For decades, ALS has been viewed as a disease that begins and ends in the motor neurons. But today’s scientists are challenging that narrow view, and thanks to new research funded by Target ALS, they’re revealing a far more complex picture.
Unlocking ALS: How Cell Biology and Genetics Are Guiding New Treatment Pathways
A powerful team of scientists from the University of Sheffield, the University of Pennsylvania, Stanford, and the Weizmann Institute is working together to better understand what causes ALS, and how to stop it. They’re using advanced tools like artificial intelligence, high-speed genetic screening, and detailed cell imaging to find out how small changes in our DNA may lead to this devastating disease.
Neurons Fight Back: Innate Immunity and Cell Death in ALS
A research consortium led by MGH and Harvard is reshaping how we think about inflammation in ALS, not just as a reaction to dying nerve cells, but as a neuron-driven immune response that may actually be fueling disease progression.
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