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OUR APPROACH

Our vision is for a world without MND, sooner. To achieve this, we need to fund interdisciplinary partnerships, collaborate, and fast-track the research pathways most likely to translate to clinical benefits for people with MND.

THE MNDi Foundation and The Murdoch MND Research Partnership

The MNDi Foundation funds The Murdoch MND Research Partnership headed by Professor Sue Fletcher. The partnership is a multi-institutional collaboration that brings together research and clinical expertise to accelerate the development of genetic drugs that directly target pathways involved in MND pathology and motor neuron disruption.

Research we fund

The teams we are currently funding are exclusively focussed on understanding mechanisms in MND and developing RNA therapeutics for inherited and acquired MND. This means therapies are developed specifically to target pathways that are disrupted in motor neurons and supporting cells.

“We believe that missing heritability is currently one of the biggest barriers to finding a cure for MND.”

Professor Anthony Akkari

Find out more about the important work the The Racing For MNDi Foundation is funding:

1. Tackling The Effects Of The C9ORF72 Mutation:

C9ORF72 ‘expansions’ are responsible for approximately 40% of familial MND cases but can also be found in some of those diagnosed with sporadic MND. PhD Student Leon Larcher is working under the supervision of Sue Fletcher and Ianthe Pitout to understand the effects of genetic mutations in the C9ORF72 gene, and designing novel strategies to overcome these effects.

2. Understanding Genetic Risk Factors and Neurodegeneration.

Dr Sarah Rea’s research is focused on understanding the mechanisms underlying neurodegeneration. Dr Rea uses innovative analytical techniques to demonstrate changes to key biochemical, cellular and structural components of neurons in response to genetic risk factors and potential therapeutic molecules.

3. Pioneering Novel MND Cell Models

After experiencing success in researching oligonucleotides to treat inherited retinopathy, post-doctoral scientist Dr Ianthe Pitout is applying similar therapeutic strategies for MND. She has also been establishing MND cell models to evaluate novel antisense oligonucleotide therapeutics for MND.

4. Unearthing Hidden Heritability in MND

Professor Anthony Akkari is focussed on studies that reveal structural variants that influence severity or lead to MND. His studies include the important work of gene sequencing to identify the missing heritability.

5. Funding The Australian MND Registry

The MNDi Foundation provided seed funding to help establish the Australian MND registry. This is a clinical registry to record the data of all those diagnosed in the country but is also a self reporting archive so patients can log their symptoms and treatment. Critically, the database is allowing more people to participate in clinical trials nation-wide. The foundation will continue to support this important resource in the fight against MND.

6. Collecting and banking of patient cells

The Racing For MNDi Foundation funds the sampling and banking of patient cells where national funding is not provided. Western Australian samples were included in an Australia-wide study looking at screening potential treatments for MND. Some of those cells have been ‘programmed’ into stem cells that will eventually be returned to the research team as a resource for their studies.

Read more about our 2021 Goals

Our research team

Professor Sue Fletcher

Professor Sue Fletcher is a Senior Principal Research Fellow at Murdoch University and together with Professor Merrilee Needham leads the MND research collaboration. In addition to her role at Murdoch University, Professor Fletcher is also the Chief Scientific Officer at PYC Therapeutics, a drug-development company that specialises in RNA-targeted therapies, with a current focus on inherited retinal disease, the central nervous system and neurodegeneration.

Professor Fletcher together with Prof. Steve Wilton pioneered the development of novel therapies for muscular dystrophy, the first to be approved by the USA FDA.

“What we learn from inherited MND will benefit those with sporadic MND, and vice versa. A better understanding of the mechanisms underpinning MND will ultimately lead us to the answers that will help us beat this disease.”

Professor Sue Fletcher

Dr Ianthe Pitout

Dr Ianthe Pitout’s research is focused on the design of splice switching antisense oligonucleotides for treating amenable genetic diseases. Innovations from Ianthe’s PhD research have contributed to the development of a molecule that is currently undergoing early phase commercialisation to treat an inherited retinopathy.

As both a PhD student and postdoctoral scientist, Ianthe has more than 5 years practical experience in the development of antisense oligonucleotides for potential therapeutic application. Currently, Ianthe is committed to developing novel therapeutic strategies for neurodegenerative diseases, with a focus on motor neuron disease. She has also been establishing MND cell models to evaluate novel antisense oligonucleotide therapeutics for MND.

“The courage and hope of MND patients and their families as well as their commitment to furthering MND research inspires the work we do every day in pursuit of an effective treatment for this devastating disease.“

Dr Ianthe Pitout

Dr Sarah Rea - Group Head - Functional genomics of neurodegeneration

Dr Sarah Rea is a Senior Research Fellow and mid-career researcher with 10 years postdoctoral experience. Her research is focused on understanding the mechanisms underlying neurodegeneration. Dr Rea uses innovative analytical techniques to demonstrate changes to key biochemical, cellular and structural components of neurons in response to genetic risk factors and potential therapeutic molecules.

My work focuses on understanding how critical proteins malfunction in the motor neurons of MND patients. We have made great strides already and are in the process of testing pharmaceutical treatments and gene therapies to get these proteins doing the jobs they’re supposed to do. We wouldn't be able to do this important work without funding from the MNDi foundation.

Dr Sarah Rea

PhD student Mr Leon Larcher

Leon Larcher has a Bachelor of Science with Honours in Molecular Biology from Murdoch University. He is currently undertaking a PhD under the supervision of Professor Sue Fletcher, Professor Rakesh Veedu and Dr Ianthe Pitout at Murdoch University’s Centre for Molecular Medicine and Innovative Therapeutics. His PhD project is focussing on understanding the effects of genetic mutations in the C9ORF72 gene that causes an inherited form of MND, and designing novel strategies to overcome these effects.

“I am driven to do my PhD in MND research, to provide a therapy for those suffering from MND and for the scientific challenge it provides. I am passionate for this project because I feel that I can have a real impact on patients’ outcomes and can really provide a benefit to those who need it.”

Leon Larcher

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