Driving Innovation in ALS: Key Takeaways from the 4th Annual ALS Drug Development Summit thumbnail image

Driving Innovation in ALS: Key Takeaways from the 4th Annual ALS Drug Development Summit

The ALS Drug Development Summit 2025 in Boston brought together over 150 stakeholders across pharma, biotech, academia, and patient advocacy to accelerate therapeutic breakthroughs for ALS and FTD. Quanterix was proud to be at the forefront of this dialogue, showcasing how blood-based biomarkers can transform clinical research and care delivery in ALS. 

From Discovery to Clinical Impact: Biomarkers Reshaping ALS Research 

Kicking off the event, Quanterix’s Chief Scientific & Collaboration Officer, Dr. Mark Roskey, joined Dr. Tiziana Petroziello from Harvard Medical School to present “Unlocking the Role of Tau in  Amyotrophic Lateral Sclerosis” Their session highlighted how ultra-sensitive Simoa® technology is enabling quantification of Tau in blood, unlocking new possibilities for monitoring disease progression and treatment efficacy in a minimally invasive way. 

The talk also addressed how Tau is being used in clinical trials for patient stratification, therapeutic monitoring, and endpoint development, offering researchers a powerful toolset for advancing precision medicine in ALS. 

Expanding Beyond NfL: The Next Generation of ALS Biomarkers 

While NfL remains a foundational biomarker in ALS research, regulatory interest in its use is growing. Several summit presentations addressed the development of NfL assays under IVD and IVDR standards, critical for advancing these tools toward clinical adoption. At the same time, researchers explored the role of tau and phospho-tau biomarkers, especially the potential of tau/p-tau ratios as pharmacodynamic markers in trials. 

There was a clear consensus that more work is urgently needed to validate tau-based biomarkers in ALS. While tau levels in CSF appear to increase over time in patients, plasma pTau-181 remains less well-characterized. One study suggested it may also increase longitudinally, but more rigorous studies are required to confirm its trajectory and clinical relevance. Calls for deeper research into these markers reflect the field’s drive to expand beyond traditional targets and better capture disease biology. 

ALL ALS: A National Push Toward Harmonization and Scale 

A standout presentation from Dr. James Berry (Mass General) introduced the ALL ALS initiative, a sweeping, NIH-funded effort to enroll nearly all ALS patients in the U.S. and integrate data across major initiatives like Target ALS, Answer ALS, and AMP-ALS. The goal: create a harmonized data ecosystem that bridges clinical data, biosamples, and digital health metrics to accelerate biomarker discovery and therapeutic validation. 

Quanterix supports these efforts by enabling sensitive, scalable biomarker measurements that can unify global research workflows. 

Looking Ahead: A Neuro-Metabolic Future for ALS 

In one compelling session, 2N Pharma’s John Nieland advocated for a neuro-metabolic lens on ALS, pushing for biomarker panels that reflect underlying metabolic dysfunction alongside neurodegeneration. This vision aligns with a growing industry trend to incorporate broader pathophysiological insights into therapeutic and diagnostic strategies. 

Quanterix: Empowering ALS Research with Precision Tools 

From early detection to clinical decision-making, Simoa® assays are transforming the biomarker landscape in ALS. With up to 1000x greater sensitivity than conventional immunoassays, our platform enables researchers to measure low-abundance proteins in blood that previously required invasive CSF collection. As ALS research moves toward more scalable, data-driven approaches, Quanterix remains committed to powering discovery, improving trial outcomes, and advancing patient care. 

Explore our Full Neurology Assay Menu 

Our neurology portfolio includes validated assays for NfL, GFAP, Tau, BD-Tau, p-Tau 181, and more, supporting both preclinical and clinical studies. Learn how we’re helping researchers unlock new biomarkers for neurodegenerative disease in our Blood Markers of Brain Health Brochure