Science on Target: Q3 Research We Enable

Read on for updates across the Research we Enable

Postmortem Tissue (PMT) Core

The Target ALS Postmortem Core has now collected brain and spinal cord tissue from over 650 cases, making it the largest and most comprehensive ALS tissue repository worldwide that is accessible with no-strings-attached to the global research community. This robust and expanding collection provides researchers with access to high-quality, well-annotated biospecimens essential for advancing understanding of ALS biology. The PMT Core is led by new co-director Dr. Kathryn Schwetye, Associate Professor of Pathology and Immunology at Washington University and Neurology, Georgetown University, and Dr. Bob Bowser, CSO, Barrow Neurological Institute (BNI), and supported by expert neurologists and pathologists from Columbia University, Washington University, UCSD, Georgetown University, BNI, and Edinburgh University.

This quarter, we continue to see strong interest in preconsenting tissue donations from participants in the Global Natural History Study. Through a coordinated network of five sites participating in both PMTC and GNHS, Target ALS is building a globally significant longitudinal resource that integrates premortem clinical and biomarker data with postmortem molecular and neuropathological analyses, creating a powerful platform for translational ALS research. To date, 70 Global Natural History Study participants have either pre-enrolled or expressed interest in both studies, forming a growing cohort that links data collected during life with molecular and neuropathological findings at autopsy.  

Building on our extensive multi-omic collection from postmortem tissue, we’re excited to release the first batch of single-nucleus sequencing data from the frontal cortex from 200 ALS, ALS-FTD, and non-neurological postmortem cases. This is the largest single-cell profiling study in ALS tissue to date, with additional data from motor cortex and spinal cord to follow in early 2027. We’re grateful to Dr. Panos Roussos and his entire lab at Mount Sinai for generating and sharing high-quality transcriptomic data that will deepen our understanding of the cell biology of ALS.

To learn more about the Postmortem Tissue Core resource, follow Marina Selenica, Core Project Manager, and other Postmortem Tissue Core members presenting at the upcoming NEALS conference.

Longitudinal Biofluid Core: Sustaining Discovery Over Time

Launched in 2024, the Longitudinal Biofluid Core has been fulfilling regular requests for the broader research community, fueling critical biomarker research needed to support on-going and future clinical trials. We have now distributed more than 7,400 samples to investigators to enable the development of biomarkers and support a growing body of longitudinal ALS research. Notably, many investigators returned for follow-on sample sets to extend promising initial findings, underscoring the value of repeated measures for biomarker validation. The longitudinal collection continues to expand, driven by improved patient adherence to the study protocol and sustained participant engagement. 

NEW! You can easily search our biofluid catalogue to see what is in our repository. Submit your request online through our Longitudinal Biofluid Core webpage. 

At Target ALS, no sample sits on a shelf. Every biofluid sample from our GNHS participants is being put to work generating data. Thank you to our colleagues at the University of Gothenburg and our partners at Psomagen for delivering high-quality WGS (short and long read), NfL and proteomics datasets (TMT-MS and Olink). New longitudinal plasma and CSF data will be available in the Data Engine later this month, so stay tuned.

New Data Engine Release Now Live

The Target ALS Data Engine reached a major milestone in Q3, with its largest Postmortem Tissue Core expansion to date. The release introduces a new single-nucleus transcriptomics modality and the largest aggregated collection of pTDP43 histopathology images from ALS cases. All new data arrive in the harmonized column structures and standardized values introduced earlier this year, so they integrate directly with existing tables across collections.

Key updates included:

• New Postmortem Tissue Core (PTC) datasets have now been incorporated into the Data Engine:
  • New bulk RNA-Seq data from 323 tissue samples across 67 subjects , incorporated into the RNA-Seq Metadata, RNA-Seq Counts, and Sequencing Data Files tables
  • New short-read whole genome sequencing (SR-WGS) data from 90 samples across 89 subjects incorporated into the WGS Metadata and Sequencing Data Files tables
  • New long-read whole genome sequencing (LR-WGS) data from 19 new subjects, incorporated into the WGS Metadata and Sequencing Data Files tables
  • 992 pTDP-43 histopathology images across 976 tissue samples from 111 new subjects, incorporated into the Histopathology Images table
  • A new Single-Nucleus RNA-Seq (snRNA-Seq) modality, launching with data from 193 frontal cortex samples across 193 subjects, incorporated into a new snRNA-Seq Metadata table as well as the Sequencing Data Files table. Matched snRNA-Seq data from motor cortex and spinal cord samples will follow soon.
• New Stem Cell Core (SCC) data have been added to the Data Engine:
  • New LR-WGS data for two patient-derived stem cell lines from C9orf72 repeat expansion carriers, together with their isogenic control lines
  • New SR-WGS data for patient-derived stem cell lines from two SOD1 carriers, one FUS carrier, and one TARDBP carrier, together with their isogenic control lines

You can log in and explore the updated datasets through the Target ALS Data Engine at any time.

Upcoming Webinar: A Novel ALS Whole-Genome Discovery Pipeline

Join us for “A Novel ALS Whole-Genome Discovery Pipeline: Leveraging the Target ALS Data Engine with Verily Workbench,” a webinar featuring guided walkthroughs of the Target ALS Data Portal and Verily Workbench, from locating and requesting data through to launching an analysis environment. The session will also feature a talk from Dr. Paul Sampognaro, neurologist and researcher at UCSF, on how his team is using these platforms together to train a promising new predictive model for ALS. Whether you are new to the Data Engine or already analyzing Target ALS data in Workbench, this is a great opportunity to see the full pipeline in action and bring your questions. Register for the webinar here.

For hands-on help in the meantime, Target ALS Data Engine / Verily Workbench office hours will run weekly throughout October, every Tuesday from 9:30 to 10:00 AM PDT, beginning Tuesday, October 6. Drop in with questions about finding data, setting up your Workbench environment, or getting an analysis off the ground. Join office hours via Google Meet, or dial in at (US) +1 475-329-8772, PIN: 822 864 400#.

A Growing Global Community of Data Engine Users

The Target ALS Data Engine has now welcomed more than 700 approved researchers since the portal opened in early 2024.  Data access is made easy with a simple clickable data use agreement and access within 48 hrs of applying. New users joining at an average pace of more than 20 per month and accelerating this year to roughly 26 per month. Those researchers come from 35 countries spanning North America, Europe, the British Isles, Asia, the Middle East, Africa, Oceania, and South America, and from across academia, biotech and pharma, hospitals and clinics, and nonprofit organizations. Every new user represents another lab bringing fresh questions to Target ALS data, and we are grateful to this community for making the Data Engine a shared resource for the global ALS research effort.

Reagents Core

HDGFL2-CE Antibody Availability

Cryptic exons (CEs), have played a central role in contemporary ALS research since their discovery. TDP-43 loss of function leads to the inclusion of CEs, among other transcriptional changes, which can lead to cryptic peptides and proteoforms that impact cellular homeostasis and function. CEs and their cryptic protein products of interest in the ALS community have included HDGFL2-CE, IgLon5-CE, and UNC13A-CE, and have been targeted in basic biology, biomarker, and drug discovery research.

Target ALS is excited to announce that the Reagent Core will soon be offering an HDGLF2-CE peptide-recognizing antibody. This antibody was produced in partnership with Len Petrucelli (Miami), whose monoclonal pTDP-43 antibody has been in high demand through the Target ALS Reagent Core. Target ALS expects that the new HDGLF2-CE peptide-recognizing antibody will be available for distribution through Sampled in Q4 2026.  

Target ALS also offers several other antibodies through in-kind funding, including

• C9orf72 Poly(GP) monoclonal antibody
• VAChT polyclonal antibody
• TDP-43 polyclonal antibody
• pTDP-43 monoclonal antibody

Scientists interested in requesting antibodies from Target ALS can apply through our Grantee Portal.

Related News

Science on Target: Q3 Research We Conduct

October 7, 2026

Science on Target: Q3 Research We Fund

October 7, 2026

Science on Target: Q3 Publication Spotlight

October 7, 2026

Brooke Eby | 1988-2026

October 2, 2026