Science on Target: Q2 Research Cores Update
July 8, 2026 Amy Easton
Use the below jump links to navigate to each Research Core update.
Postmortem Tissue (PMT) Core
The Target ALS Postmortem Core has now collected 650 cases, making it the 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 co-directors Dr. Brent Harris, Adjunct Prof. of Neurology, Georgetown University, and Director of Neuropathology, U. Colorado, and Dr. Bob Bowser, CSO, Barrow Neurological Institute, and supported by expert neurologists and pathologists from Columbia University, Washington University, UCSD, Georgetown University, BNI, and Edinburgh University.
This quarter, we have placed strong emphasis on dual-enrollment participants across both the Global Natural History Study and the Postmortem 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, 44 biofluid 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.
The Core remains a highly sought-after resource, with 46 tissue requests filled in 2025 and 20 requests in the first half of 2026. Samples are used by researchers in academia and industry to directly examine disease-associated changes in cellular architecture, molecular pathways, and gene expression, yielding critical insights into the mechanisms that drive neurodegeneration. A good subset of the 19 publications from this quarter alone acknowledged the postmortem core and datasets generated from the tissue.
Longitudinal Biofluid Core: Sustaining Discovery Over Time
In 2025, the Longitudinal Biofluid Core fulfilled 30 biofluid requests, distributing more than 5,900 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. Submit your request online through our Longitudinal Biofluid Core webpage.
New Data Engine Release Now Live
Two major updates were deployed to the Target ALS Data Engine in May and June. The Postmortem Tissue Core, Global Natural History Study, Community-Based Pop-Up Outreach Study, and Stem Cell Core collections now feature fully harmonized column names and standardized column values across tables. This work is designed to make it easier to integrate data across collections and modalities while reducing friction in downstream analysis.
Key updates included:
- New GNHS data from 129 participants
- New CBOS data from 52 participants
- New Environmental Exposures Data table has been added to GNHS
- New, comprehensive Variant Annotations tables and ALS-associated genomic variant summary columns within the WGS Metadata tables have now been added across all four collections within the Data Engine.
- In addition, the following new Postmortem Tissue Core datasets have now been incorporated into the Data Engine:
- New Clinical Data from 81 subjects
- New RNA-Seq data from samples from 274 samples from 62 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 104 subjects, incorporated into the WGS Metadata and Sequencing Data Files tables.
- New long-read whole genome sequencing (LR-WGS) data from 54 subjects, incorporated into the WGS Metadata and Sequencing Data Files tables.
- New Single Cell Nuc-seq data are planned for an August ingestion
By aligning column structures and enumerated values across studies, we are laying the groundwork for more seamless cross-cohort analyses and improved reproducibility. In addition, the inclusion of ingestion date information in each table makes it easier to identify, filter for, and track newly added data over time. You can log in and access the updated datasets through the Target ALS Data Engine at any time. Users are provided access to Verily Workbench, where they can explore and analyze Target ALS data using Python (JupyterLab) or R (RStudio) across a range of configurable compute environments, including standard virtual machines, Spark-based clusters, and Nvidia-GPU-enabled platforms. To learn more about working in Verily Workbench, check in with Rachel Linker for Workbench office hours on Fridays between 11:30 and 12 noon PT. To watch a video recording of the workshop from April 30th, please contact daniel.weatherill@targetals.org
“Before the internet, there was ARPA-net. A new network for hybrid intelligence is emerging, and the Intelligent Generator of Research (IGoR) is ARPA-H’s bet on a new kind of research infrastructure: shared mechanistic models of disease, a common protocol layer that lets any qualified lab run the same experiment identically, and a distributed marketplace of trusted labs that execute those protocols and return gold-standard data. This networked intelligence uses AI to accelerate scientists toward the highest-value experiments, not to drive them.
In that world, the institutions that have carefully curated their data and certified its provenance become the critical ‘nodes of nodes’, and through the Data Engine, Target ALS has curated and maintains a trusted information supply chain ready for the age of networked intelligence. Our team believes this approach can meaningfully accelerate ALS research, and that Target ALS is uniquely positioned as a data custodian to coordinate and accelerate discovery in this new world.“
Peter Lund, Founder and CEO of Somos
Stem Cell Core: Building Robust Human Models for ALS
At the 2026 Target ALS Annual Meeting, we held a workshop where topics converged on a common theme: building better, more standardized iPSC-based cell models to overcome the variability and inconsistency that have long hampered ALS disease modeling and phenotype detection.
Bill Skarnes, Sami Barmada, and Joao Pereira described the creation of a new suite of patient-derived lines, pairing C9, SOD1, FUS, and TARDBP mutants with NGN2 knock-ins or isogenic controls; characterization in 2D and 3D organoid formats by the Target ALS Stem Cell Consortium. Initial data revealed that some lines reliably reproduce ALS phenotypes while others remain difficult to characterize. These lines, along with accompanying transcriptomic and whole-genome sequencing data, are set for staged distribution through Jackson Labs in July, and metadata will be made accessible via the Data Engine.
Joseph Klim, Director of Neuroscience for NuCyRna, extended this push toward standardization and scalability by demonstrating rapid, reliable motor neuron differentiation via NGN2 overexpression paired with developmental small-molecule cues, positioning these lines as a dependable platform for disease modeling and drug discovery. Johnathon Cooper-Knock, University of Sheffield, also presented work on developing high-throughput phenotyping methods using a “Village in a Dish” model.
Annalisa Pawlosky introduced a complementary, AI-driven angle, describing how Google Research’s AI Co-scientist is being used to generate novel disease hypotheses and inform the development of new ALS cell models—pointing toward a future where computational tools and improved cell systems work in tandem to accelerate ALS research.
To learn more about Target ALS stem cell lines, see our Stem Cell Core page and the Jackson Laboratories iPSC catalogue
Despite the progress in contemporary ALS research using iPSC-based methods, there remain several needs and gaps in the iPSC-community. Research gaps include:
- More lines from sporadic ALS patients are still needed due to experimental conditions requiring high sample numbers, accessibility issues, and licensing fees.
- Improved TDP-43 loss-of-function models with mislocalization and aggregation
- Inducible lines with TARDBP knock-out or knock-down
- Researchers brought forward both reservations and potential advantages of transdifferentiation methods and agreed more study is needed
