Funded Research Projects

• Phase 3 Clinical Trial

Randomised double-blind placebo-controlled Phase 3 trial of Lithium Carbonate in MND, a sub-study of a Multi-arm, Adaptive, Groupsequential trial NETwork to evaluate drug efficacy in patients with MND (MAGNET).

Professor Matthew Kiernan
The University of Sydney, NSW

• Phase 2 Clinical Trial

A placebo-controlled safety and efficacy of ambroxol in individuals with MND,

Associate Professor Bradley Turner
The Florey, The University of Melbourne, VIC

• ORAL MONEPATEL

PHASE 1

Dr Susan Mathers

• TECFIDERA IN ALS (TEALS) STUDY

• IC14 TRIAL

• LIGHTHOUSE TRIAL

• COPPER-ATSM TRIAL

• NANOCRYSTALLINE-GOLD TRIALS (RESCUE-ALS)

• PHASE 1 CLINICAL TRIAL – TRIMETAZIDINE TRIAL (Dr Shyuan Ngo)

• GENE THERAPY/DRUG DELIVERY

Enhanced neuronal delivery, gene targeting and neuroprotection: development of a multimodal drug against MND

Dr Loren Flynn, Murdoch University, WA

• GENE THERAPIES

Targeted degradation of misfolded TDP-43 as a therapy for MND

Dr Luke McAlary, The University of Wollongong, NSW

• DISEASE MODELS

Advanced modelling of upper motor neuron MND pathology using human pluripotent stem cells

Professor Clare Parish, The University of Melbourne, VIC

• DISEASE MODELS

Evaluation of a novel inducible muscle specific TDP-43 mouse model of MND

Professor Aaron Russell, Deakin University, VIC

• GENE THERAPIES

Therapeutic targeting of TDP-43 through selective reduction of ataxin-2 expression with peptide-conjugated antisense oligonucleotides

Dr Fazel Shabanpoor, The University of Melbourne, VIC

• PAUL FISHER IMPACT GRANT – DISEASE HETEROGENEITY

RNA-binding proteins involved in the pathogenesis and disease heterogeneity of sporadic MND

Dr Rachel Tan, The University of Sydney, NSW

• DISEASE MODELS

Developing a validated C9orf72 mouse model of ALS/FTD using genome editing MND

Associate Professor Bradley Turner, The University of Melbourne, VIC

• DISEASE MODELS

New viral-mediated TDP-43 mouse models of MND

Dr Adam Walker, The University of Queensland, QLD

• DISEASE MODELS / DRUG DELIVERY

Development of a human MND Neurovascular Unit model to improve therapeutic translation in drug testing

Associate Professor Anthony White, QIMR Berghofer Medical Research Institute, QLD

• DISEASE HETEROGENEITY / DISEASE BIOMARKERS

Profiling monocytes in MND to assess disease progression and heterogeneity

Professor Trent Woodruff, The University of Queensland, QLD

• GENERATING A PROTEOMICS-BASED BIOMARKER FOR MND

Dr Allan McRae

• EC-FUS – A NOVEL BIOMARKER FOR MND?

Prof Julie Atkin

• THE GLYMPHATIC SYSTEM: NOVEL BIOMARKER OF DISEASE SEVERITY AND THERAPEUTIC TARGET

Prof David Wright

• EXPLOITING CRYPTIC RELATEDNESS IN GLOBAL FAMILIAL AND SPORADIC MND TO UNCOVER DISEASE-AND-PHENOTYPE-LINKED GENES

Dr Kelly Williams

• A HIGH-THROUGHPUT SYSTEM TO IDENTIFY MND RISK GENES FROM GENOME-WIDE ASSOCIATION STUDIES

Dr Fleur Garton

• IDENTIFICATION OF NOVEL STRUCTURAL VARIATIONS IN ALS GENES TO IMPROVE OUTCOMES OF CLINICAL TRIALS

Prof P. Anthony Akkari

RESTORING AUTOREGULATION OF TDP43 IN SPORADIC MND USING SPLICE-SWITCHING ANTISENSE OLIGONUCLEOTIDES

Prof Lezanne Ooi

ENHANCING DELIVERY OF GENE THERAPY TO MOTOR NEURONS AND GLIAL CELLS USING FOCUSED ULTRASOUND

Dr Kara Vine

SUBPIAL SPINAL CORD DELIVERY AS A STEM CELL-BASED TREATMENT FOR MND

Prof Lachlan Thompson

HARNESSING PHASE SEPARATION AS A PRECLINICAL STRATEGY FOR THE TREATMENT OF MND

Dr Marco Morsch

• DEVELOPMENT OF BLOOD-BRAIN BARRIER PENETRATING PEPTIDES AS A CNS DRUG DELIVERY PLATFORM

Dr Fazel Shabanpoor

• USING PATIENT-DERIVED CELLS TO DEFINE AN ELECTRICAL SIGNATURE OF SPORADIC ALS AND TO DEVELOP A HIGH THROUGHPUT DRUG SCREENING TECHNOLOGY AND NOVEL THERAPY

Prof Lezanne Ooi

• IDENTIFYING BIOMARKERS FROM EXTRACELLULAR VESCICLES FOR EARLY DETECTION, DISEASE PROGRESSION AND THERAPEUTIC EFFICIENCY IN MND

Prof Aaron Russell

• THE USE OF BIOMARKERS TO ADDRESS MND HETEROGENEITY AND IMPROVE DETECTION OF BENEFIT IN CLINICAL TRIALS

Dr Mary-Louise Rogers

• PRION-LIKE STRAINS OF TDP-43 AGGREGATES IN MND AND FTLD

Prof Justin Yerbury

• LIPIDOMIC SIGNATURES IN BLOOD AS A NOVEL BIOMARKER FOR MND

Dr Sophia Luikinga

• USING 3D HUMAN iPSC-DERIVED SPINAL CORD ORGANOIDS TO MODEL OLIGODENDROCYTE NEUROTOXICITY IN MND

Dr Samantha Barton

• DEVELOPMENT OF A DOSE-ESCALATABLE AAV DELIVERY SYSTEM FOR ALS GENE THERAPIES

Prof Roger Chung

• CHARACTERISING BLOOD-BRAIN BARRIER DYNAMICS TO ENHANCE CNS DRUG DELIVERY AND OPTIMISE MEDICINE USE IN MND

Prof Joseph Nicolazzo

• PATIENT MONOCUTE-DERIVED MICROGLIA AS A CLINICALLY APPLICABLE MODEL FOR PRECISION TREATMENT OF NEUROINFLAMMATION IN MND

Prof Anthony White

• POINT OF CARE ASSESSMENT OF VENOUS ACID-BASE BALANCE AND CREATININE AS MARKERS FOR DISEASE PROGRESSION IN PATIENTS WITH MND

Prof Frederik Steyn

• A NOVEL BIOMARKER FOR ALS

Dr Fleur Garton

• ESTABLISHING A NOVEL MOUSE MODEL OF SPORADIC MND USING A CHEMOGENETIC APPROACH

Dr Mouna Haidar

• DISEASE SUSCEPTIBILITY, PRECISION MEDICINE AND ALS

Prof Julie Atkin

• ESSENTIAL PRE-CLINICAL STEPS TOWARDS A STEM CELL TRAIL FOR MND

Prof Lachlan Thompson

• THERAPIES TARGETING MUSCLE

Intramuscular allosteric agonism of purinergic P2X7 receptor as a pharmacological approach to enhance skeletal muscle regeneration in MND

Dr Giovanni Nardo
Mario Negri Institute for Pharmacological
Research, Italy

• TREATMENTS TARGETING MULTIPLE CAUSES OF MND

Validation of the clinical-stage drug candidate RRx-001 as a novel disease modifying therapeutic for MND.

Dr Tony Reid
EpicentRx, Inc., California, USA

• GENETIC AND ENVIRONMENTAL INTERACTIONS

Epidemiology in a dish: using human iPSC to discover common and genotype specific molecular signatures of the multistep hypothesis of MND.

Associate Professor Anthony Cook, University of Tasmania, TAS

• RESCUING THE BLOCKAGE OF CRITICAL FUNCTIONS IN MOTOR NEURONS CAUSED BY GENE DEFECTS

Trouble at the ribosome in C9ORF-72-driven MND

Dr Danny Hatters, The University of Melbourne, VIC

• CHANGES IN THE FUNCTION OF CELLS IN THE BRAIN AND SPINAL CORD THAT NORMALLY SUPPORT MOTOR NEURON HEALTH

Multiomic interrogation of patient-derived neurotoxic glia

Dr Jeffrey Liddell, The University of Melbourne, VIC

• MND BIOMARKER PROGRAM

Pre-familial and early MND biomarker program

Associate Professor Mary-Louise Rogers, Flinders University, SA

• MND BRAIN IMAGING INITIATIVE

AMII: Asia-pacific MND Imaging Initiative

Dr Sicong Tu, The University of Sydney, NSW

• BILL GUEST MID-CAREER RESEARCH FELLOWSHIP

The Bill Guest Mid-Career Research Fellowship is named in recognition of the extraordinary contribution of Bill Guest AM, the inaugural Chairman at FightMND.

PROJECT: Clearing TDP-43 pathology for MND therapy

Dr Adam Walker – Bill Guest Mid-Career Research Fellow, The University of Queensland, QLD

• MID-CAREER RESEARCH FELLOWSHIP

PROJECT: Reversing TDP-43 pathology and neuronal loss in sporadic MND

Dr Rachel Tan, The University of Sydney, NSW

• EARLY CAREER RESEARCH FELLOWSHIP

PROJECT: Therapeutic targeting of ferroptotic cell death in MND

Dr Taide Wang, The University of Melbourne, VIC

Dr Taide Wang was also the inaugural recipient of the Angie Cunningham Scholarship.