Funded research projects

Clinical trials

Clinical trials test and evaluate promising new treatments to find better ways to prevent, detect or treat MND.

2021

PHASE 1B – EPHRIN RECEPTOR A4 FC (new drug) (Prof Perry Bartlett, AO)

2020

• ORAL MONEPATEL – PHASE 1 (Dr Susan Mathers)

2016-19

• 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) 

Drug Development Projects

Drug Development projects are focused on advancing promising new drugs or therapies through the final stages of testing in preparation for their assessment in clinical trials with MND patients.

2021

• PREVENTING NEUROINFLAMMATION IN MND BY INHIBITING THE mPTP (Prof Seth Masters)
• A MONOCLONAL ANTIBODY TARGETING ALPHA 5 BETA Q INTEGRIN AS A POTENTIAL TREATMENT FOR MND (Prof Larence Steinman)
• PRECLINICAL DEVELOPMENT OF A MONOCLONAL ANTIBODY MODULATING CD38 AGAINST MND (Dr Damien Toulorge)
• TARGETING MISFOLDED PROTEINS WITH MISFOLDUbLs AS A THERAPEUTIC STRATEGY FOR MND (Prof Justin Yerbury)
• TARGETING CK1e-MEDITATED TDP-43 PHOSPHORYLATION IN MND (Dr John Ravits)

2020

• A PHASE 1 TOLERABILITY, SAFETY, PHARMACOKINETICS AND PRELIMINARY EFFICACY STUDY OF ORAL MONEPANTEL IN INDVIDUALS WITH MND (Dr Susan Mathers)
• MODULATING ACTIN DYNAMICS IN ALS/MND AS A NOVEL THERAPEUTIC APPROACH IN ALS (Prof Julie Atkin)
• DEVELOPMENT OF M102 FOR THE TREATMENT OF ALS (Dr Ning Shan)
• DEVELOPMENT OF TDP43-TARGETING GENE THERAPY FOR ALS (Prof Roger Chung)
• COMBINATION THERAPY TO IMPROVE ON CuATSM OUTCOMES IN ALS (Prof Justin Yerbury)
• PRE-CLINICAL DEVELOPMENT OF A SOD1 GENETIC THERAPY IN SPORADIC ALS (Prof Steve Wilton)
• PRE-CLINICAL DEVELOPMENT OF CENTRALLY-ACTIVE COMPLEMENT C3a RECEPTOR MODULATORS AS DISEASE-MODIFYING DRUGS FOR MND (Prof Trent Woodruff)
• SMN2 SPLICE-SWITCHING OLIGONUCLEOTIDE THERAPY DEVELOPMENT FOR MND (Prof Bradley Turner)

2016-19

• PROGRESSION OF C5a ANTAGONIST PMX05 TO CLINICAL TRIALS (Prof Trent Woodruff)
• TARGETING A DEREGULATED COPPER-IRON AXIS TO TREAT SPORADIC MND (Dr Peter Crouch)
• PRECLINICAL DEVELOPMENT OF NEUROPEPTIDE Y FOR THE TREATMENT OF MND (Prof Tracey Dickson)
• A NOVEL EPHRIN RECEPTOR A4-Fc FUSION PROTEIN FOR THE TREATMENT OF SPORADIC MND (Prof Perry Bartlett)
• THERAPEUTICS BASED ON THE PROTECTIVE ACTIVITY OF PROTEIN DISULPHIDE IN MND (PROJECT 1) AND NOVEL THERAPEUTICS TARGETING NEURONAL TRANSPORT PATHWAYS IN MND (PROJECT 2) (Prof Julie Atkin)
• TARGETING THE METABOLISM OF GLYCOSPHINGOLIPIDS AS A NOVEL THERAPEUTIC (Prof Bradley Turner)
• V-SMART® NANOMEDICINE (LAUR-301) (Prof Bradley Bartlett)
• TARGETING METABOLIC FLEXIBILITY IN ALS (MetFlex) (Dr Shyuan Ngo)
• HDAC6 INHIBITION TO RESCUE AXON DEGENERATION IN ALS (Prof Anna King)

IMPACT grants

To accelerate the development of effective therapies for MND, FightMND IMProving and ACcelerating Translation (IMPACT) grants support projects focused on overcoming one or more key barriers preventing the advancement of potential treatments through to clinical trial.

2021

• 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)

2020

• 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)

2016-19

• 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)

Angie Cunningham PhD Scholarship and Grant-in-Aid

Honouring the life of Angie Cunningham, FightMND’s Angie Cunningham PhD Scholarship aims to support future leaders in MND research.

FightMND supports biomedical research in the field of Motor Neurone Disease (MND), encompassing all relevant research disciplines. The objectives of the Angie Cunningham Scholarship Scholarship and Project Grant-in-Aid Award are to:

• Support research aimed at understanding the causes of sporadic MND,
  elucidating disease mechanisms and facilitating the translation of therapeutic strategies from the laboratory to the clinic;
• Encourage new interest within the field of MND research from exceptional new postgraduates;
• Help develop PhD graduates who demonstrate academic leadership,
  independence, creativity and innovation in their work and foster a passion for future MND research;
• Honour the memory and legacy of Angie Cunningham

https://www.facebook.com/plugins/video.php?height=314&href=https%3A%2F%2Fwww.facebook.com%2FFightMND%2Fvideos%2F2115138615291252%2F&show_text=false&width=560&t=0

Angie Cunningham Scholarship recipients:

• Ted Wang 

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Drug Screening Platform

In 2017, with support from the Andrews Labor Government, FightMND partnered with the Balcon Group Pty Ltd to establish the FightMND Drug Screening Program. The four-year, $5M program aims to establish a high-throughput drug screening platform to screen approved and experimental treatments on motor neurons grown from skin cells from Australians living with MND.

The FightMND Drug Screening Program has drawn together an expert team of stem cell scientists and MND clinicians and made significant progress over the last four years. The major research outcomes for the study include:

• the generation of the world’s second-largest MND iPSC bank available for drug screening and a vital tool for MND researchers globally
• the development of a robust motor neuron generation protocol that will be published and shared within the research community
• an MND specific drug screening platform and pipeline for screening approved and novel promising therapeutics
• the foundation for comprehensive single-centre multidimensional characterisation.