高清福利片

Judy Featherstone never met fellow Motor Neurone Disease patient Dr Angela Worthington before she passed away in February 2020.

But the doctor鈥檚 enduring legacy is now offering Judy a new chance to 鈥渂eat the beast鈥� head on.

A respected Australian cardiologist, Dr Worthington鈥檚 dying wish was to establish a new research position for MND, the devastating and fatal disease that impacts听.

With her body rapidly unravelling, unable to walk or talk and confined to a wheelchair, Dr Worthington used eye-gaze tracking technology to convey her wish to her loved ones, to donate her brain to scientific research and fund a new research position for MND.

Medical colleagues, friends, family and her fellow cardiologists rallied to raise nearly $70,000 in just four weeks. This was matched with $30,000 from MND Research Institute Australia to develop a competitive grant and a few months later, the grant was awarded to Irish-born Neurologist Dr Colin Mahoney.

Described by the University of Sydney as 鈥溙�, Dr Mahoney has also been a recipient of one of the university鈥檚 10 prestigious international postdoctoral fellowships following a global search for outstanding applicants focused on tackling 鈥渢he greatest challenges of our time.鈥�

Gold: the promise of neuroprotective therapies

It was in March 2020, as a confused world came to grips with the unfolding COVID-19 crisis, that Dr Mahoney quietly began enrolling patients in a new human trial for MND called听Rescue ALS,听at the听University of Sydney's Brain and Mind Centre.

The trial involved the use of a novel gold nanoparticle protocol for MND (which is also known as听Amyotrophic Lateral Sclerosis or ALS in some countries).

Early animal studies in听2019听found gold nanocrystals to be neuroprotective, reducing the loss of nerve cells and improving the function of remaining cells.

It鈥檚 thought this helps increase energy production in cells and reduce oxidative stress which in turn may slow down the clinical impact of MND, (which causes a progressive inability to talk, walk and breathe).

Gold also has important anti-inflammatory properties, frequently used in conditions such as rheumatoid arthritis and听. And unlike many medications, this new formulation of gold can successfully cross the blood-brain barrier.

鈥淚 must admit that at the start of lockdown it was very hard to get a clinical trial up and going,鈥� says Dr Mahoney.

鈥淏ut we enrolled听45 patients form across NSW and South Australia 听and the one good thing about COVID-19, is that it has spurned听听So instead of trialling one drug we can do many more at the same time and ultimately this will speed up the time it takes to find medicines to slow, stop or reverse the progression of MND. A cure for the genetic forms of MND is close, probably 3 years away, and we are hoping for a cure for the sporadic form (90% of patients) within the decade.鈥�

Judy, who has been part of the trial for 12 months, does not know if she was on the active arm of the trial or the placebo arm for the first nine months.

However, in recent months all participants in the blinded phase of the trials were offered the active drug, after combined results on the first 42 patients showed a听听Some patients even had an听improvement听in function from baseline.

鈥淭hese studies are early but very encouraging because it suggests that improving cellular bioenergetics may have neuro protective affects,鈥� says Dr Mahoney.

The next big question

鈥淥f course, we 肠补苍鈥檛 hold our breath until top line results are out at the end of this year. If the study shows no clear difference between the placebo arm and the active arms, the treatment won鈥檛 be what we are all hoping for. However, if the improvements are shown to be in the active treatment arms this could potentially represent a new paradigm in the treatment of MND.鈥�

The world-first human trial听 - a joint project with chief investigators Dr William Huynh from the BMC and Associate Professor Parvathi Menon at Westmead Hospital; FightMND and Clene Nanomedicine -听 was extended in late 2020 to include other global MND centres and institutions such as Massachusetts General Hospital and Harvard University.

With the additional support from MNDRIA's Angela Worthington Award, Dr Mahoney and his team will be able to cast their net wider, with funding for an additional 70 to 80 people to participate in a further study.

Living with MND

For Judy and her husband Don, living with MND involves at least six to eight medical appointments a month, and multidisciplinary care with her neurologist, occupational therapist, exercise physiologist, speech pathologist, neurology specialist nurse, respiratory physician and dietitian to increase kJ intake.

But despite Judy鈥檚 devastating diagnosis, neither has lost their sense of humour.

鈥淵ou always need to be one step ahead of the disease,鈥� says Don.

鈥淎s MND causes a lot of word slurring that can be tricky to understand, we decided early on to do 鈥渧oice banking鈥�. This is听 where we 鈥渂ank鈥� about 250 words and 20 key phrases. Then Judy can just play the relevant phase on her iPad when she needs them and can create her own sentences. So, Judy recorded all the important messages in life in her lovely Sussex accent.

鈥淗er favourite of course is 鈥榞et me a gin and tonic please Don!鈥欌��

Judy also jokes that MND is 鈥榯he only disease鈥� where doctors encourage you to eat as many chips and chocolate as your like, with a high protein, high KJ diet linked to improved survival outcomes and better quality of life. She exercises听several days a week, which includes Feldenkrais, gym and walking. She also spends many active hours with her听6听grandchildren, and reads, listens to music plays piano and does jigsaws.

鈥淚 feel so privileged to have such a wonderful husband and family, and such world-class multidisciplinary care in Colin and his team. This a very big part of living well with MND.

鈥淕etting a diagnosis like this is dreadful for anyone. But a lot of it comes down to your attitude. You can sit in a chair and get depressed or treat every day as a gift. I鈥檝e accepted that what will happen will happen, but I鈥檓 not ready to give up. Being on a clinical trial may or may not help me but I hope it may help others in the future.

鈥淛ust like Dr Worthington, we cannot let the things we听肠补苍鈥檛听do, prevent us from doing the things we听can.鈥澨�