The Lawrence and Isabel Barnett Drug Development Program Grant Recipients
There is an urgent need for new and improved therapies for ALS, as there is still no cure. To help expand the drug development pipeline, we are proud to support the preclinical assessment of emerging ALS therapies like these through The Lawrence and Isabel Barnett Drug Development Program.
2023 Recipients
John Blackwood, Ph.D., Samsara Therapeutics
A Novel Small Molecule that Enhances Autophagy for the Treatment of ALS
Julien Spatazza, Ph.D., Coave Therapeutics
Preclinical Proof of Concept of TFEB in ALS Disease
Frederik Rombouts, Ph.D., Augustine Therapeutics
Development of a Novel Selective HDAC6 Inhibitor for the Treatment of ALS
Howard Weiner, M.D., Brigham & Women’s Hospital
Modulation of Neurodegenerative Microglia by Nasal Anti-CD3 Monoclonal Antibody
Jordi Xaus, Ph.D., Oryzon Genomics
Efficacy and Preclinical Characterization of a HDAC6 Inhibitor (ORY-4001) in ALS
Michael Young, Ph.D., Myrobalan Therapeutics
Targeting the Neuroinflammatory Component of ALS Using CSF1R Inhibitors
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2022 Recipients
Sapan Gandhi, Ph.D., Drishti Discoveries Ltd.
Gene Therapy for ALS
Martin Gill, Ph.D., Libra Therapeutics, Inc.
TRPML1 Small Molecule Agonists for the Treatment of ALS
Justin Ichida, Ph.D., University of Southern California
Development of an Antisense Oligonucleotide Targeting SYF2 for ALS
Kim Staats, Ph.D., Raya Therapeutic, Inc.
A Novel Combination Approach to ALS
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2021 Recipients
Daniel Elbaum, Ph.D., QurAlis Corporation
GLP Studies on Antisense Oligonucleotide Therapies
Akinori Hishiya, Ph.D., Sola Biosciences
Novel Protein Folding Gene Therapy for ALS
Virginia Man-Yee Lee, Ph.D., University of Pennsylvania
Preclinical Evaluation of Bemcentinib as a Novel Therapeutic for ALS
Jeffrey Rothstein, M.D., Ph.D., Johns Hopkins University
ASO-Mediated Knockdown of CHMP7 as a Novel Therapeutic
Strategy for ALS/FTD
Ann Marie Schmidt, M.D., New York University Grossman School of Medicine
Antagonism of RAGE/DIAPH1 and Therapies for ALS
Irving Sucholeiki, Ph.D., Aquilus Pharmaceuticals, Inc.
Development of a Matrix Metalloproteinase Inhibitor for the Treatment of ALS
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2020 Recipients
Christopher Barnum, Ph.D., INmuneBio Inc.
Targeting soITNF-Dependent Inflammation in ALS with XPro1595
Kuti Baruch, Ph.D., ImmunoBrain Checkpoint, Inc.
Targeting Immune Checkpoint Pathways: A Novel Therapeutic Approach in ALS
Peter Crouch, Ph.D., University of Melbourne, Australia
Copper-ATSM and Follistatin: A Nerve-Muscle Combination Therapy Strategy
Diana Price, Ph.D., Neuropore Therapies, Inc.
Preclinical Evaluations of a Novel Small Molecule Toll-Like 2 Receptor (TLR2) Antagonist as a Putative ALS Therapeutic
Christopher Reyes, Ph.D., Bloom Science
Preclinical Development of a Live Biotherapeutic Product Used in the Treatment of ALS
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2019 Recipients
Steven Burden, Ph.D., New York University Grossman School of Medicine
Reducing Leg Muscle Denervation in SOD1 G93A Mice with an Agonist Antibody to MuSK
Don Cleveland, Ph.D., University of California San Diego
Antisense Oligonucleotide Therapy in ALS by Restoring Expression of Stathmin-2
Samar Hasnain, Ph.D., University of Liverpool, UK
Development of Ebselen and Its Derivatives as a Potential Drug for ALS
Justin Ichida, Ph.D., University of Southern California
Development of Novel PIKfyve Inhibitors for C9orf72 ALS
Clotilde Lagier-Tourenne, M.D., Ph.D., Massachusetts General Hospital*
Developing Immunotherapy Approaches Targeting Pathological Forms of TDP-43 in ALS
Glenn Larsen, Ph.D., Aquinnah Pharmaceuticals
Validation of Efficacy for an Inhibitor of TDP-43 Pathology in the DNLS Mouse Model of ALS
Giovanni Manfredi, M.D., Ph.D., Weill Cornell Medicine
Exploring the Disease-Modifying Effects of EH301 in Mouse Models of ALS
Magdalini Polymenidou, Ph.D., University of Zurich, Switzerland*
Developing Immunotherapy Approaches Targeting Pathological Forms of TDP-43 in ALS
Brigitte van Zundert, Ph.D., Universidad Andrés Bello, Chile
Synergistic Dual Target Approach to ALS Therapy: Simultaneous Activation of Two Complementary Targets – Neuronal Kv7.2/3 Channels and Glial TSPO
*Joint project
2018 Recipients
Daniel Elbaum, Ph.D., QurAlis Corporation
Development of a Clinical Candidate Targeting the TBK1 Branch of the Autophagy Pathway for the Treatment of ALS
Eliahu Heldman, Ph.D., Lauren Sciences LLC
Treatment of ALS by Targeted Delivery of GDNF to Motoneurons Using Novel V-Smart Nanovesicles
John Ravits, M.D., University of California San Diego
Ataxin-2 Neuropathology in Sporadic ALS
2017 Recipients
Antonius Bunt, M.D., Ph.D., Izumi Biosciences Inc.
Complement DoD Grant W81XWH-16-1-0072 for PK Enhancement of cART in HERV-K + ALS-FTD Patients to Reverse Neuroinflammation, Multidrug Resistance and Improve Outcomes
John Landers, Ph.D., University of Massachusetts Chan Medical School
Investigating Pridopidine, a Sigma-1 Receptor Activator, as a Novel Therapeutic Treatment for ALS
Nicholas Maragakis, M.D., Johns Hopkins University
NLY01: A GLP-1 Analog for the Treatment of ALS
Timothy Miller, M.D., Ph.D., Washington University in St. Louis*
Pathological Analysis of Selective Autophagy in Sporadic and Familial ALS
John Ravits, M.D., University of California San Diego*
Pathological Analysis of Selective Autophagy in Sporadic and Familial ALS
Raymond Roos, M.D., The University of Chicago
Combating ER Stress in G85R ALS Mice
Karolina Rygiel, Ph.D., University of Oxford, UK
Generation and Validation of Open Access Recombinant Monoclonal Antibodies for Reproducible ALS Research
*Joint project
2016 Recipients
Richard Keenan, Ph.D., OptiKira, LLC
Validating Inhibition of IRE1alpha Kinase/RNase as a Therapeutic Approach to ALS
Thomas Lloyd, M.D., Ph.D., Johns Hopkins University
Preclinical Development of KPT-350 for C9-ALS
Jiou Wang, M.D., Ph.D., Johns Hopkins University
Targeting Proteotoxicity as the Common Denominator in ALS
