Announcing the 2026 Biomarker Consortia Awards

Target ALS is proud to announce that three consortia have been selected for awards through our Biomarker Consortia Program. As new therapeutics begin moving the needle for people with ALS, the field needs better tools to measure whether those treatments are working, and to do so faster than current trial designs allow. Biomarkers are critical to stratify people with ALS (ex/ fast versus slow progressors), understand response to therapeutics, and quantify disease progression.

Reinforcing our value of radical collaboration, each of these consortia brings together academic labs and an industry partner to advance a specific biomarker approach from early discovery toward real-world use in clinical trials.

We thank all the applicants for their dedication and hard work, and congratulate the selected groups who are advancing these promising programs.

Meet the Consortia

Redefining progression and treatment response in ALS

Alexander Thompson (University of Oxford), Johnathan Cooper-Knock (University of Sheffield),  Eran Hornstein (Weizmann Institute of Science), Carrie Rubel (Biogen)

Clinical trials evaluating new ALS therapeutics have faced challenges related to trial size and length, as well as their reliance on clinical endpoints. While these indirect measurements are critical, novel biomarkers are necessary to better track disease progression and treatment response. This consortium will first use existing proteomic datasets from Oxford, Sheffield, the Weizmann Institute of Science, and the Target ALS Data Engine built through the Target ALS Global Natural History Study (GHNS) to model signatures of disease progression. Then, in partnership with Biogen, the consortium will evaluate proteomic signatures from people with ALS in former and ongoing clinical studies that contained a treatment arm, such as VALOR and EXPERTS-ALS, to validate biomarkers of treatment effectiveness. The inclusion of VALOR samples provides the unique opportunity to measure the response of signatures to effective treatment, and was only made possible through collaboration with Biogen. This project could deliver a novel set of biomarkers for use in clinical studies as readouts of therapeutic effect.

Proteomic signatures for ALS stratification and progression across presymptomatic and symptomatic disease stages

Paul Lingor (Technical University of Munich), Johannes Dorst (University of Ulm), Michael Menden (University of Melbourne), and Alexander Brandt (Eli Lilly)

While ALS symptoms are shared between people with ALS, its underlying causes remain misunderstood. Although evidence is emerging that ALS has molecular subgroups, clinical trials have not changed their design, and the risk of trial failure is inflated without accounting for these subgroups. Paul Lingor and academic partners, Johannes Dorst and Michael Menden, will partner with Alexander Brandt and Eli Lilly to evaluate proteomic, metabolomic, and lipidomic signatures of ALS molecular subgroups using plasma and cerebrospinal fluid (CSF) from people with ALS, advanced machine learning, and artificial intelligence. While this project will utilize discovery cohorts from Europe, validation will be performed using samples from the Target ALS Biofluids Repository collected through the GNHS. This project could further characterize ALS disease heterogeneity and progression, and connect people with ALS with clinical trials and candidate therapeutics that best suit their molecular subtype.

Circular RNA Biomarkers of TDP-43 Dysfunction in ALS

Pietro Fratta (University College London), Michael Ward (University of California, Los Angeles), David Walt (Harvard University), Sanjay Chandriani (Trace Neuroscience)

TDP-43 pathology is present in nearly 97% of people with ALS and about half of people with frontotemporal dementia (FTD), yet a reliable biomarker of TDP-43 dysfunction has remained elusive. TDP-43 dysfunction leads to RNA-binding and -splicing errors, creating “cryptic” RNA species, such as cryptic exons (CEs) or cryptic circular RNAs (c-circRNAs). While recent efforts have aimed to detect CEs in plasma and CSF from people with ALS, their structure has imposed technical constraints on their use as biomarkers.  The unique structure of c-circRNAs increases their stability in plasma and CSF, which is expected to offset the drawbacks of CEs. Led by Pietro Fratta, this consortium will integrate samples from participants in an ongoing clinical trial, well-characterized cohorts at UCL, and the Target ALS Biofluids Repository with innovative technologies to detect, identify, and characterize c-circRNAs as a biomarker of TDP-43 dysfunction. This consortium has a strong history of synergy and leadership in ALS biomarker research and, through this funding, they are bringing innovative next generation technology into the field to continue pursuing novel ALS biomarkers.

Together, these three consortia are addressing the same problem from different angles, giving researchers and clinicians faster, more direct ways to see what ALS is doing in the body and whether a treatment is changing it. At Target ALS, our Independent Review Committee (IRC)  application receives a fair, unbiased evaluation. Thank you to all applicants for their dedication and hard work. For more information about this program, visit our Biomarker Consortia page. 

About Our Review Process

At Target ALS, we hold fairness and transparency paramount in our review process. The Target ALS Independent Review Committee (IRC) makes all research funding decisions without involvement from the organization’s staff or leadership, ensuring every application receives a fair evaluation. The IRC is comprised of experts across scientific disciplines from both industry and academia, reflecting the evolving nature of ALS research. To avoid any possible conflicts of interest, no member of the IRC can apply for or receive Target ALS funding for their own work. Members on the IRC abide by a comprehensive conflict of interest policy and are all under confidentiality agreements.  

Our Vision and Commitment 

At Target ALS, we are driven by a sense of urgency, knowing that every day counts for people living with ALS and their families. Currently approved treatments have limited benefit or only apply to a small subset of cases. ALS remains a fatal disease. We are committed to advancing cutting-edge research that moves faster than this disease. Together, we achieve our vision: a world where Everyone Lives. 

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