The ALS Therapy Development Institute (ALS TDI) is the world's largest drug discovery lab focused solely on ALS. We believe the most impactful breakthroughs happen through collaboration, and we're actively seeking partners to join us.

ALS TDI’s work is driven by three core research groups: our Clinical Research, Cell Biology, and In-Vivo Pharmacology Teams. Each offers unique capabilities and opportunities for partnership to advance your research and collectively drive progress toward ending ALS.

CLINICAL RESEARCH: The ALS Research Collaborative

The ALS Research Collaborative (ARC Study) is an ongoing natural history study that collects comprehensive data from people living with ALS and those carrying an ALS-related gene mutation. Participants contribute data including:

  • Family, work, lifestyle, medical history surveys
  • Electronic Health Records (EHRs)
  • ALSFRS-r scores
  • Movement tracking
  • Voice recordings
  • Blood samples

The ARC Data Commons is a cloud-based platform that allows scientists to access the ARC Study’s comprehensive dataset to advance their own ALS research. Since 2014, over 1,100 people with ALS have contributed at least three months of longitudinal data, resulting in trillions of data points.

How we can work together:

  • Access the ARC Data Commons: Academic researchers and non-profit partners may request free access to this longitudinal dataset. Industry partners may access the data for a small fee. We can also provide a subset of the data in the OMOP common data model for those who prefer that format.
  • Collaborate on Data Analysis & Discovery: Partner to explore specific hypotheses, identify novel biomarkers, or uncover new insights from these extensive real-world patient data.
  • Joint Publications & Grant Applications: Our past partnerships have successfully led to numerous research publications and successful grant submissions.

CELL BIOLOGY

The Cell Biology team at ALS TDI is focused on leveraging induced pluripotent stem cells(iPSCs) to model various forms of ALS, including SOD1, C9orf72, TDP-43, and sporadic cases. Using a combination of cell lines derived from participants in the ARC Study and genome-edited lines, the team transforms these cells into cell types relevant for ALS, including motor neurons and astrocytes. These models are used to test potential therapies and for basic research to learn more about ALS biology.

How we can work together:

  • Access Patient-Derived iPSC Lines: A collection of well-characterized ALS patient-derived iPSC lines and differentiated cell types (e.g., motor neurons, astrocytes) will be made widely available to researchers through a new agreement with Axol Bioscience Ltd.
  • Collaborate on Model Development & Validation: Work with our Cell Biology experts to develop and validate new cellular models of ALS or to characterize specific disease mechanisms in existing models.
  • Pre-clinical Compound Screening: Utilize our advanced cellular platforms for high-throughput screening of novel compounds or genetic targets, accelerating your drug discovery efforts.
  • Joint Research on Disease Mechanisms: Partner with our team to unravel the complex cellular biology underlying different forms of ALS.

IN-VIVO PHARMACOLOGY

ALS TDI's in-vivo pharmacology team utilizes a portfolio of mouse and zebrafish ALS models as platforms for validating novel small molecule and biologic therapies, focusing on immunomodulation, redox metabolism, RNA modulation, protein aggregation, and others. Successful candidates, like tegoprubart, have reached human clinical trials, while others are in lead optimization phases. This team also focuses on identifying and characterizing emerging models of ALS.

How we can work together:

  • Preclinical Drug Validation & Efficacy Studies: Partner with us to rigorously test your novel small molecules, biologics, gene therapies, or other therapeutic modalities in established and emerging in-vivo ALS models.
  • Pharmacokinetic/Pharmacodynamic (PK/PD) Analysis: Leverage our capabilities to assess drug exposure and target engagement in relevant animal models, providing critical data for lead optimization and clinical translation.
  • New Model Characterization & Development: Collaborate on the characterization of novel ALS animal models, expanding the toolkit available for preclinical research and drug discovery.
  • Co-development & Translational Partnerships: Explore opportunities for co-development partnerships to advance promising therapeutic candidates through preclinical stages and toward human clinical trials.

Explore Our Full Portfolio of Active Research Programs

Discover the full scope of our current research initiatives and identify additional areas where your expertise can accelerate the development of effective ALS treatments: Active ALS TDI Research Programs

You can also connect with us via email (info@als.net) to explore how your organization can contribute to and benefit from these ongoing efforts.