Funded Consortia

A Snapshot of our Innovative Research Consortia

Target ALS-funded collaborative consortia are at the forefront of driving discovery, validation and development of new ALS drug targets and clinical biomarkers. Here is a brief overview of our funded consortia.

Landmark, Precompetitive Biomarker Initiative Launched 

The ability to diagnose ALS early, track disease progression, or to group patients with similar forms of the disease requires reliable biomarkers. The barriers to discovery and development of biomarkers are especially intractable due to the higher costs, difficulty in accessing optimal biospecimens and requirement of multi-disciplinary expertise. To address these challenges, Target ALS launched a precompetitive biomarker initiative that connects pharma/biotech industry, academic experts and foundations to collaboratively discover and develop new biomarkers for ALS.


New Consortia Funded to Examine Disease Mechanisms, Targets and Biomarkers

Target ALS sought to build on the momentum generated by earlier funding calls by supporting multi-disciplinary teams working on ALS research themes, targets or biomarkers.

  • Validating poly(GP) and pNFH as biomarkers to detect symptomatic conversion in c9ALS: Leonard Petrucelli (Mayo Clinic, Project Leader), Tania Gendron (Mayo Clinic), Andreas Jeromin (Mayo Clinic), Yongjie Zhang (Mayo Clinic), Michael Benatar (University of Miami), Katharine Nicholson (MGH), Nathan Staff (Mayo Clinic).
  • Regulatory and Therapeutic role of microRNAs in ALS: Samuel Pfaff (Salk Institute for Biological Studies, Project Leader), Eran Hornstein (The Weizmann Institute), John Ravits (University of California, San Diego).
  • Therapeutic potential of CRISPR-Cas9 in C90RF72 – repeat deletion and transcriptional repression strategies in C90RF72 BAC transgenic mice: Laura Ranum (University of Florida, Project Leader), Eric Wang (University of Florida), Chad Cowan (CRISPR Therapeutics).
  • Mechanisms of cortical hyperexcitability in ALS: Solange Brown (Johns Hopkins University, Co-Project Leader), Clifford Woolf (Children’s Hospital Boston, Co-Project Leader), Paola Arlotta (Harvard University), Dwight Bergles (Johns Hopkins University), Loyal Goff (Johns Hopkins University).


Ground-breaking Industry-led and Young Investigator-led Consortia Funded to Validate Candidate Therapeutic Targets and Disease Mechanisms 

For the first time in the history of ALS research and in partnership with ALS Finding a Cure, Target ALS designed its 2016 funding call to encourage ALS research ideation and active involvement from the pharma/biotech industry. A pharma/biotech scientist was required to lead or co-lead the consortium.

  • Targeting Stress Granule Dynamics for Familial and Sporadic ALS: Joe Lewcock (Denali Therapeutics, Project Leader), Gene Yeo (University of California, San Diego), Steve Finkbeiner (Gladstone Institutes).
  • Gene Therapeutic Modulation of NMD for Treatment of ALS: Ben Shykind (Meira GTx, Co-Project Leader), Neil Shneider (Columbia University, Co-Project Leader), Gregory Petsko (Cornell University).
  • Developing human-derived antibodies to target dipeptide-repeat protein toxicity in C9ORF72 disease: Fabio Montrasio  (Neurimmune, Project Leader) Laura Ranum (University of Florida), Magdalini Polymenidou (University of Zurich), Clotilde Lagier-Tourenne (Massachusetts General Hospital).
  • Nuclear Export Inhibitor KPT-350 for C9orf72 and Sporadic ALS: Sharon Tamir (Karyopharm Pharmaceuticals; Co-Project Leader), Jeff Rothstein (Johns Hopkins University, Co-Project Leader), Thomas Lloyd (Johns Hopkins University), Laura Ranum (University of Florida).
  • Exploiting yeast to discover small-molecule drugs for ALS caused by aberrant TDP-43, FUS, and c9orf72 dipeptide repeat protein homeostasis: Dean Brown & Nick Brandon (AstraZeneca, Co-Project Leaders), Jim Shorter (University of Pennsylvania), Steve Finkbeiner (Gladstone Institutes), Aaron Gitler (Stanford University).

Target ALS sought to encourage ideas from newly independent investigators and enrich the future of ALS research. A newly independent investigator was required to lead or co-lead the consortium.

  • Critical Assessment of the Dying Back ALS Hypothesis Using Novel iPSC and Mouse Models: Brian Wainger (Massachusetts General Hospital, Project Leader), Robert Brown Jr. (University of Massachusetts), Jean Livet (Inserm).
  • Vesicles in the Transmission of ALS: Stephanie Duguez (Ulster University, Project Leader), Susan Knoblach (Georgetown University Medical Center), Cédric Raoul (Inserm), Pierre-Francois Pradat (Pitie-Salpetriere Hospital).
  • Identification of RAN Translation Regulators as Therapeutic Targets in C9ORF72-Related ALS: Shuying Sun (Johns Hopkins University, Project Leader), Michael Bassik (Stanford University), Aaron Gitler (Stanford University), Fen-Biao Gao (University of Massachusetts), Joel Richter (University of Massachusetts).
  • The Role of NK and NKT Cells in ALS: Benjamin Murdock (University of Michigan, Project Leader), Stephen Goutman (University of Michigan).


Inaugural Target Discovery and Validation Consortia 

During its first three years, Target ALS funded collaborative research consortia to examine ALS disease mechanisms and candidate therapeutic targets – the first critical step in developing a novel therapeutic. With the aim of casting a wide net, these funding calls supported ideas from, and successfully engaged involvement of investigators from within and outside ALS research community representing academia and pharma/biotech industry.

  • Oligodendrocyte dysfunction in ALS: Dwight Bergles (Johns Hopkins University, Project Leader), Jonah Chan (University of California, San Francisco), Tom McCown (University of North Carolina, Chapel Hill), Jeff Rothstein (Johns Hopkins University), Jude Samulski (University of North Carolina, Chapel Hill), Neil Shneider (Columbia University).
  • Unfolded protein response and endoplasmic reticulum stress in ALS: Hynek Wichterle (Columbia University, Project Leader), Hugo Bellen (Baylor College of Medicine), Kevin Eggan (Harvard University), Serge Przedborski (Columbia University), Brent Stockwell (Columbia University).
  • Motor neuron excitability and ALS: Clifford Woolf (Boston Children’s Hospital, Co-Project Leader), Solange Brown (Johns Hopkins University, Co-Project Leader), Paola Arlotta (Harvard University), Dwight Bergles (Johns Hopkins University), George Mentis (Columbia University), Neil Shneider (Columbia University), Brian Wainger (Massachusetts General Hospital), Daniel Zytnicki (University of Paris Descartes).
  • C9ORF72: Testing targets and developing models: Jeff Rothstein (Johns Hopkins University, Project Leader), Clotilde Lagier-Tourenne (University of California, San Diego), John Ravits (University of California, San Diego), Paul Taylor (St. Jude Children’s Hospital), Phil Wong (Johns Hopkins University), Kevin Eggan (Harvard University), Laura Ranum (University of Florida), Fen-Biao Gao (University of Massachusetts).
  • Unraveling mechanisms in RNA-binding protein-mediated ALS:Aaron Gitler (Stanford University, Project Leader), Michael Bassik (Stanford University), Don Cleveland (Ludwig Institute for Cancer Research), Steve Finkbeiner (Gladstone Institutes), Clotilde Lagier-Tourenne (University of California, San Diego), Jim Shorter (University of Pennsylvania), Paul Taylor (St. Jude Children’s Hospital).
  • A potassium channel and hyperexcitability screen for novel ALS therapeutics: Clifford Woolf (Boston Children’s Hospital, Project Leader), Bruce Bean (Harvard University), Kevin Eggan (Harvard University), Pharma/Biotech Partner.
  • Targeting miR-155 with antisense oligonucleotides as a therapy for ALS: Tim Miller (Washington University, Project Leader), Pharma/Biotech Partner.
  • Discovery of small molecules targeting C9ORF72 repeat RNA: Leonard Petrucelli (Mayo Clinic, Project Leader), Matthew Disney (The Scripps Research Institute), Jeff Rothstein (Johns Hopkins University), Pharma/Biotech partner.
  • Receptor-interacting protein -1 and -3 as potential targets for ALSSerge Przedborski (Columbia University, Project Leader), Pharma/Biotech partner.
  • Exploiting yeast to uncover small molecule drugs for ALS caused by aberrant TDP-43 and FUS proteostasis: Jim Shorter (University Pennsylvania, Project Leader), Aaron Gitler (Stanford University), Pharma/Biotech partner.
  • Validating ataxin 2 as a therapeutic target in ALS: Aaron Gitler (Stanford University, Project Leader), Robert Baloh (Cedars–Sinai Medical Center), Nancy Bonini (University Pennsylvania), Stefan Pulst (University of Utah), Pharma/Biotech partner.
  • Targeting the UPR for the treatment of ALS: Ray Roos (University of Chicago, Project Leader), Brian Popko (University of Chicago).
  • Preclinical foundation of urate-elevating therapy for ALS:Michael Schwarzschild (Harvard University, Project Leader), Evangelos Kiskinis (Northwestern University), Gazaleh Sadri-Vakili (Harvard University).
  • Drug efflux alterations in ALS: from mechanisms to therapy: Piera Pasinelli (Thomas Jefferson University, Project Leader), David Miller (National Institutes of Health), Davide Trotti (Thomas Jefferson University).
  • Targeting glucosyl-ceramide metabolism as a new approach to strengthen the motor units in ALS: Jean-Philippe Loeffler (INSERM, Project Leader), Frances Platt (University of Oxford).
  • Identification of therapeutic targets for cytoskeletal defects in ALS: John Landers (University of Massachusetts, Project Leader), Daryl Bosco (University of Massachusetts), Bruce Goode (Brandeis University), Steve Finkbeiner (Gladstone Institutes).
  • Enhancing proteosome function as a potential treatment for amyotrophic lateral sclerosis: Alfred Goldberg (Harvard University, Project Leader), Daniel Finley (Harvard University).
  • Astrocyte mGluR5 as a modulator of neurodegeneration in ALS: Dwight Bergles (Johns Hopkins University, Project Leader), Serge Przedborski (Columbia University), Pharma/Biotech partner.
  • Identification of small molecules that block RNA-mediated toxicity in c9ALS/FTD: Leonard Petrucelli (Mayo Clinic, Project Leader), Fen-Biao Gao (University of Massachusetts), Matthew Disney (The Scripps Research Institute).