Message in a package: characterizing amyotrophic lateral sclerosis through extracellular vesicles
Amyotrophic lateral sclerosis (ALS) is one of the most devastating neurodegenerative diseases. Most current therapeutic strategies aim to prolong the survival of the patients by a few months rather than targeting the cause of the disease. Extracellular vesicles (EVs) released by cells can transport cargo such as proteins, RNA and lipids. EVs can also deliver their pathogenic cargo to healthy cells and spread the disease. We will investigate ALS-secreted EVs as biomarkers and their roles as pathological seeds.
The majority of ALS patients is between the age of 40 and 70. ALS has a huge impact not only on these patients but equally on the beloved ones of patients and caregivers. The social impact of ALS includes emotional strain on patients and their families, as well as challenges in daily activities due to physical limitations. Economically, there are financial burdens associated with medical expenses, caregiving costs, and potential loss of employment for both patients and caregivers. The direct economic burden is significant as this devastating disease comes with a high loss of productive years.
Our results may deliver biomarkers that will help in more optimal monitoring of the disease process and improved clinical trials. These biomarkers will eventually enable the early diagnosis of ALS, before symptoms manifest, facilitating timely intervention and potentially improving treatment outcomes. Moreover, biomarkers in EVs provide a non-invasive and accessible means to monitor disease progression and response to therapy, allowing for personalized treatment therapy.
We will generate 1. Therapeutic target prioritization. We will prioritize proteins within EVs as potential therapeutic targets based on their proteomics data and functional validation studies. 2. Develop diagnostic assays utilizing EV proteins identified throughout this study. 3. Develop therapeutic intervention strategies targeting EV-mediated communication in ALS. These mechanisms can act as future therapeutic targets.
