At ALS TDI, we’re dedicated to transforming the lives of everyone with ALS by advancing science and developing targeted treatments. We’re tackling ALS from every angle through a comprehensive, multi-pronged approach. Here’s how we’ve made an impact in ALS research in 2024:


Revolutionizing Biomarker Discovery

In 2024, ALS TDI achieved major advancements in biomarker research.

  • Study of Blood Neurofilaments (NfL): Using ARC Study samples, we completed one of the largest NfL studies in ALS history, confirming its potential to track disease progression and defining its limits, paving the way to guide its use for ALS clinical trials and care.
  • Optimizing Accelerometry Data: In 2023, we published a study in Nature showing that wearable accelerometers can accurately track ALS progression. In 2024, this breakthrough biomarker became poised to transform research, with ALS TDI housing the largest accelerometer data repository and collaborating globally to optimize its clinical use.
  • Proteomics Breakthroughs: Through our partnership with LifeArc, we’ve now identified several new blood-based biomarkers that may predict ALS progression and potentially provide better ways to monitor treatments.


Rapid Testing of New Ideas

In 2024, ALS TDI researchers swiftly tested IGFBP7, a potential biomarker and therapeutic target linked to ALS reversals, proposed by Dr. Rick Bedlack. While results didn’t warrant further study at ALS TDI, the project showcased our unmatched ability to rapidly evaluate new ideas. Learn more here.


Expanding Models to Drive Tailored Therapies

Because ALS Takes many forms, diverse animal models are essential for research. ALS TDI is a global leader in this critical work. In 2024, we advanced two new mouse models, Profilin1 and TDP43, for use in testing potential therapies. By integrating them with our existing models, we evaluated dozens of therapeutic approaches tailored to different forms of ALS. Learn more about ALS TDI animal models here.


Trailblazing mRNA Therapeutics 

ALS TDI is pioneering the use of mRNA in ALS research. Having initiated a partnership with investigators at UPenn, we’re using mRNA technology and lipid nanoparticles to deliver targeted therapies to the brain and spinal cord, advancing transformative treatments for ALS and other neurological diseases.


Advancing Promising ALS Therapies

In 2024, our researchers advanced two of our most promising treatments closer to human trials.         

  • PRMT Inhibitors: Discovered by our team in 2020, PRMT inhibitors show promise for treating specific forms of ALS. In 2024, we advanced multiple treatments into SOD1 and TDP43 mouse models. Though initially targeting C9orf72-related ALS, early findings suggest potential benefits for other forms, offering hope for broader impact.
  • Copper Complexes: ALS TDI is advancing copper complexes as a promising approach for ALS therapies, with a focus on developing targeted treatments. From this innovative program, TDI1831 has emerged as the lead candidate, with an initial focus on addressing an ultra-rare form of ALS.


Identifying ALS Risk Factors            

With a major grant from the CDC, ALS TDI is leading a groundbreaking study to uncover how lifestyle and environmental factors contribute to ALS. In 2024, our data science team streamlined extensive ARC Study survey data and integrated electronic health records, paving the way for an unprecedented evaluation of ALS risk factors. Learn more about the study here.


ARC Study Expansion

We made significant strides in expanding the ARC study in 2024, aiming for a total of 2,000 participants. This year's milestones included record annual enrollment and the highest number of blood samples collected in a single year. This invaluable data drives critical research at ALS TDI and beyond. We are profoundly grateful to our more than 1,000 participants for their contributions! To learn more about the ARC study and how you can share your data to advance ALS research, visit www.als.net/arc.


A United Front Against ALS                  

At ALS TDI, every milestone brings us closer to our mission to end ALS. Whether it’s advancing biomarkers, refining animal models, pioneering new therapies, or understanding risk factors, we’re leaving no stone unturned. With your ongoing support, we will develop treatments for everyone with ALS.


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