Target ALS recently participated in the Northeast ALS Consortium (NEALS) Meeting, which took place virtually from October 21 – 24, 2024, where we presented two Target ALS-led research initiatives that improve access to critical research tools and resources for ALS researchers across the globe, advancing our understanding of ALS and the discovery of biomarkers and effective treatments.
The NEALS Meeting is an annual event aimed at unveiling scientific breakthroughs in ALS, providing updates on evolving clinical trials and showcasing exceptional research contributions.
Here’s a breakdown of what we shared, focusing on our Global Natural History & Longitudinal Biofluid Study and our Multicenter Human Postmortem Tissue Core.
1. Global Natural History & Longitudinal Biofluid Study
Understanding the Need for Diversity
Historically, ALS research has primarily included patients of European descent, which limits our understanding of the disease across different races and ethnicities. Our Global Natural History Study aims to enhance diversity by enrolling a broader participant cohort, targeting 800 symptomatic ALS participants and 200 healthy controls from various backgrounds. This is crucial for identifying genetic risk factors and developing effective treatments that work for everyone.
Study Design and Methods
Currently, our study spans 14 international sites, with more joining soon. Participants undergo thorough clinical assessments and cognitive evaluations every four months. We collect and analyze various biofluids—blood, urine, and cerebrospinal fluid (CSF)—to create a comprehensive biorepository and datasets. Notably, we also gather data through digital platforms, tracking speech and respiratory function. All data is made available to ALS researchers at no cost through the Target ALS Data Engine.
Early Results
So far, we’ve enrolled 159 participants, including 92 with ALS and 67 healthy controls. While 87% of participants are Caucasian, our efforts to increase diversity are showing promise, and we are actively expanding to new sites across the globe. We now have a biorepository with over 15,000 biofluid samples, which researchers can access to identify critical biomarkers for clinical use.
Conclusion
Our Global Natural History Study is set to provide valuable insights into the heterogeneous nature of ALS, paving the way for innovative biomarkers that can predict and track disease progression. Engaging populations outside the U.S. will significantly enrich our understanding of ALS.
Since presenting at the NEALS Conference, Target ALS has had additional participants enroll in the study, demonstrating the positive progression of this research in 2024. Follow us on LinkedIn and Instagram for updates. You can also follow our Longitudinal Biofluids page for updates.
2. Multicenter Human Postmortem Tissue Core
Accelerating ALS Research
The Target ALS Postmortem Tissue Core is dedicated to facilitating ALS research by providing high-quality postmortem tissues and associated data to researchers worldwide. Our initiative aims to optimize the use of every case, ensuring that these valuable resources are responsibly utilized for advancing biomarker and drug discovery and development for ALS.
Methodology
We conduct autopsies and collect tissues from ALS cases, following strict quality control measures. This includes detailed metadata collection and genomic analyses, ensuring that researchers have access to robust data alongside the tissue samples. Our repository now holds samples from 292 ALS cases, 39 Frontotemporal Dementia (FTD) cases, and 110 non-neurological controls.
The Target ALS postmortem tissue core comprises six geographically distributed sites.
Collaborations and Data Sharing
With support from key partners, we’ve expanded our resources, including curated samples with specific pathological features. We make tissue samples and genomic data available to researchers without any intellectual property concerns, fostering collaboration and accelerating discoveries in ALS research.
Impact of Our Research
Postmortem tissue analyses have revealed novel potential drug targets and key biological pathways impacted in ALS. The new multi-modal datasets generated from each case support well-powered statistical analyses and sophisticated applications such as AI algorithms.
Moving Forward
Both of these research initiatives underscore our commitment to understanding ALS in a comprehensive manner. By enhancing diversity in our studies and providing critical resources for research, Target ALS is working diligently to make strides in ALS biomarker and drug development. We look forward to sharing more updates and insights as our research progresses.
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