Research consortia

Target ALS has funded and coordinated the research of a total of 19 preformed collaborative consortia, focused on key topics in target discovery and target validation, and involving investigators from 30 academic institutions. We have also funded 5 Springboard Fellowships to allow the best young ALS investigators to found their own laboratories in this field and to start work on their new projects without delay. This unprecedented support of nationwide and international collaborations lies at the heart of our thrust toward better target validation and discovery for ALS.

Year 2017

New Consortia

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)

Year 2016

Industry-led Consortia (in partnership with ALS Finding A Cure)

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)

Young investigator-led Consortia

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)

Years 2013-2016

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 ALS

Serge 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).

Nuclear pore dysfunction and disrupted cytonuclear trafficking in C9ORF72 and non-C9ORF72 ALS

Chris Donnelly (Johns Hopkins University; Target ALS Springboard Fellowship awardee)

Hsp104 mediated disaggregation of TDP-43, FUS, and SOD1 aggregates implicated in ALS

Meredith Jackrel (University of Pennsylvania; Target ALS Springboard Fellowship awardee)

Role of the ALS-linked genes in stress granule dynamics and ALS pathogenesis

Regina Kolaitis (St. Jude Children’s Hospital; Target ALS Springboard Fellowship awardee)

Spinal inhibition in ALS

Katharina Quinlan (Northwestern University; Target ALS Springboard Fellowship awardee)

Mechanisms of C9orf72 hexanucleotide repeat expansion caused neurodegeneration in ALS and FTD

Shuying Sun (Ludwig Institute for Cancer Research; Target ALS Springboard Fellowship awardee)