$1 million from Neale Daniher’s FightMND campaign for Perth research project

A young Perth researcher has secured a $1million grant from Neale Daniher’s FightMND campaign to develop a drug to treat the deadly disease - the first time the footy legend’s foundation has funded a major project in WA. Loren Flynn and her team hopes to use pioneering WA-developed genetic technology to create a drug to treat motor neurone disease, which attacks the body’s nerve cells causing paralysis and death. Footy champion Daniher founded FightMND, which has now raised $48.45 million for Australian research and treatment of the disease, after he was diagnosed with it seven years ago. Dr Flynn, a 32-year-old postdoctoral researcher at Perth’s Perron Institute and Murdoch University, drove the investigation to use antisense oligonucleotide (AO) technology to develop a drug to target a damaged protein called SOD1, which helps to kill motor neurons in people with the disease. “Preclinical models are showing that if you can reduce the build-up of the SOD1 protein, that will delay the motor neurone death and should delay the progression of the disease for patients,” she said. Dr Flynn and her colleagues will take skin samples from people with MND and use them to grow motor neurone cells in the laboratory. They will then use AO technology to suppress production of the SOD1 protein to stop it damaging the motor neurone. In preclinical studies, the team has already achieved 93 per cent suppression of SOD1 which affects two per cent of MND sufferers who have SOD1 genetic mutation. The FightMND grant will enable Dr Flynn and her team to test their compound on MND sufferers who do not have the genetic mutation. Recent research has found that SOD1 proteins are toxic to the majority of people with MND. AO technology was previously used by the study’s lead investigator Steve Wilton, who is director of the Perron Institute, and his team to invent an effective treatment for Duchenne muscular dystrophy. The severe muscle-wasting disease mostly affects boys and usually first affects them by the age of five and ends their lives in their 20s. The breakthrough treatment was given fast-track approval by the US Food and Drug Administration in 2016. Dr Flynn, who completed her PhD only three years ago, said they hoped to have preliminary results within the next year and have a drug to test on patients in clinical trials within three years. “This is definitely the biggest project I've worked on. To be looking at a potential drug in the clinic in three years, that's huge,” she said. The project is a collaboration with a US company called Black Swan Pharmaceuticals, which would see clinical trials take place in the US. FightMND has announced $10.68 million of funding for 16 projects nationwide today. Despite the cancellation of the sixth Big Freeze event at the MCG because of COVID-19 this year, a fundraising campaign, including the sale of the popular blue beanies, raised a record $11.9 million. Daniher told The West Australian that the significant and positive impact the funding would have on MND research and fostering hope for future generations could not be underestimated. "The Australian community, through the most challenging of times, have answered our call for support again by donating generously and I extend my gratitude for the contribution they've made, they are the true heroes of the fight,” he said.