Tag Archives: florey

Motor neurone disease breakthrough: Patient trial shows impressive clinical results

A new drug delays motor neurone disease progression and improves cognitive and clinical symptoms according to recently announced trial results.

Research at a glance: 

  • The copper-delivery drug CuATSM improved symptoms in MND patients over six months
  • Improvements were seen in lung function and cognition
  • Decline in motor disability was reduced in treated patients compared to standard-of-care patients
  • The researchers will begin a larger Phase 2 trial to confirm CuATSM’s effectiveness in motor neurone disease

A new drug developed by scientists at the Florey Institute of Neuroscience, and the School of Chemistry and Bio21 Institute at the University of Melbourne has dramatically improved clinical and cognitive symptoms of motor neurone disease, also called amyotrophic lateral sclerosis.

Motor neurone disease is a progressive, fatal neurodegenerative disease. Its key hallmark is the death of the brain cells that control muscle movements. This results in muscle weakness and eventually paralysis.

Patients usually die of respiratory failure within three years of diagnosis, and there are no treatments or disease-modifying therapies available.

In this dose-finding trial involving 32 patients, the group given the highest amount of the CuATSM compound showed improved lung function and cognitive ability, compared to the predicted declines observed in standard-of-care patients.

Further, treated patients showed a much slower overall disease progression as measured by a global disability score. The clinical trial was led by Professor Dominic Rowe at Macquarie University, and Associate Professor Susan Mathers at Calvary Health Care Bethlehem, and sponsored by Collaborative Medicinal Development Pty Ltd with support from FightMND.

Professor Ashley Bush, Chief Scientific Officer of Collaborative Medicinal Development and director of the Melbourne Dementia Research Centre, said “This is the first human evidence for a disease-modifying drug for motor neurone disease. It is a huge breakthrough, and we look forward to confirming the positive results in a larger study soon.”

Associate Professor Kevin Barnham of the Florey, Associate Professor Anthony White at the Queensland Institute of Medical Research, and Professor Paul Donnelly and Associate Professor Peter Crouch from the University of Melbourne, developed and tested CuATSM over a 15-year period.

After showing its therapeutic potential for motor neurone disease in pre-clinical models, the researchers founded a company and licensed the compound to Collaborative Medicinal Developments to take the drug into human studies.

Professor Donnelly said, “It is gratifying to see such promising results made possible by collaborative fundamental research at the interface between chemistry and biology.”

The results were reported at the 29th International Symposium on ALS/MND in Glasgow by Dr Craig Rosenfeld, CEO of Collaborative Medicinal Development.

The researchers plan to begin enrollment for a larger, randomised, placebo-controlled double-blind Phase 2 trial in mid- to late 2019. This trial will test CuATSM’s effectiveness in motor neurone disease / amyotrophic lateral sclerosis in a larger patient sample.

About the Florey:

The Florey Institute of Neuroscience and Mental Health is one of the largest and highly respected brain research centres in the world. Its teams work on a range of serious diseases including stroke, epilepsy, Alzheimer’s, Parkinson’s and motor neurone diseases, depression and addiction. The Florey is a world leader in imaging technology, stroke rehabilitation and large population studies to improve patient care around the world.

About the Bio21 Institute:

The University of Melbourne’s Bio21 Molecular Science and Biotechnology Institute seeks to improve human health and disease and the environment through innovation in molecular life science and biotechnology, driven by collaborative research and dynamic interactions with industry. It is home to more than 700 research scientists, making it one of the largest biotechnology research centres in Australia. The Institute houses major analytical and imaging technology platforms providing world-leading capability to researchers in the Melbourne Biomedical Precinct.

About Collaborative Medicinal Development
CMD is a privately-held biopharmaceutical company developing innovative therapies for neurodegenerative diseases.  The Company’s lead drug, CuATSM, was licensed from the University of Melbourne and entered clinical trials in ALS and Parkinson’s disease in 2017 at leading clinical centers in Australia. The CMD team includes Craig Rosenfeld, MD (CEO), Kay Noel, PhD (COO), Ashley Bush, MD, PhD (CSO), and Jim Babcock (Chairman, founder of Cthulhu Ventures LLC, CMD’s founding investor). 

This article is courtesy from proud precinct partner, Bio21 Institute

Professor Bernhard Baune appointed as Co-Head of the Florey’s Mental Health Theme

Professor wearing clear rim glasses and a blue suit smiles for the photo
Professor Baune has accepted an honorary appointment at the Florey Institute of Neuroscience and Mental Health and will co-head our mental health theme with Professor Andrew Lawrence

Prof. Bernhard Baune (PhD, MD, MPH, FRANZCP) is Cato Chair and Head of the Department of Psychiatry at The University of Melbourne, Australia.

Prof. Baune is a Fellow of the Royal Australian and New Zealand College of Psychiatrist (FRANZCP), Australia.

Prof. Baune’s strong background using preclinical rodent models and in translational research places his integrative research approach at the interface between clinical research, psychiatric neuroscience and molecular psychiatry, stimulating forward and backward translational research in the etiology and pathophysiology of major psychiatric disorders.

He leads an extensive research program into personalised psychiatry, prediction and biomarker research in psychiatry and treatment response prediction and in neuroimmunology. Key research achievements include an in-depth understanding of the interaction between the immune and nervous system, the development of a systems biology approach for response prediction and the establishment of innovative personalised clinical trials in major psychiatric disorders. He has a particular interest in cognitive function and psychosocial function in psychiatric disorders and in the severe course of mental illness, treatment response and recovery. Prof. Baune designs and conducts personalized randomized clinical trials that take clinical and biological information into account to inform treatments. He is developing novel pharmacological and psychological treatments for improving emotion processing, cognitive function and functional outcomes in mood disorders.

Bernhard’s translational work aims to make real-world differences to the lives of people with mental illness by integrating neurobiological and clinical information, by personalising treatments and by targeting the mechanisms of functional recovery.

Bernhard currently leads an international study on the genomics of cognitive function in depression; in addition, he founded and directs an international consortium on the genomics of severe depression and response to ECT in affective disorders (GenECT-ic) and leads an international research network on transnosological pharmacogenomics and transcriptomics of treatment response. His research is nationally and internationally funded and he has published more than 470 peer-reviewed articles, reviews and book chapters, edited several text books in Psychiatry, and most recently the books “Personalised Psychiatry” and “Inflammation and Immunity of Depression”. He is member of numerous editorial boards of international Journals in Psychiatry and related fields.

Please join us in welcoming Bernhard to the Florey. We look forward to his contributions to our research and the new opportunities he will bring to the Florey and the broader precinct.


This article is sourced from our Proud Precinct Partner, The Florey Institute

Could lab-grown human minibrains help treat Alzheimer’s and epilepsy?

A colourful network of brain pathways dual smad

This year, as the worlds of science, technology and literature marked the bicentenary of Mary Shelley’s Frankenstein, in some laboratories human cells were being coaxed into a tiny version of the organ that most defines us.

Depending on where you sit, these “minibrains” – formally known as cerebral organoids – could rival Shelley’s creation on the monstrosity scale, raising deep moral questions about consciousness and the nature of humanity.

But they also promise hope of a cure for illnesses ranging from childhood epilepsy to Alzheimer’s disease and brain cancer.

I’ve come to Melbourne’s Florey Institute of Neuroscience and Mental Health to hunt a minibrain down. I’m expecting to find it in a dish, on a bench or perhaps in a fridge.

As it happens, the first one we stumble upon is in the bin.

A special bin for biowaste, of course. The neural tissue has died after serving its purpose and is now merely a smudge of pink on a plastic slide.

Still, it seems inconceivable that this miracle of science could qualify as rubbish.

Steve Petrou

Prof Steve Petrou in the lab. Credit – Pat Scala

The contrast becomes more extreme when I sit down to chat with Florey Institute director Professor Steven Petrou. He is leading research that creates organoids to mimic the behaviour of the brains of children with rare, debilitating forms of epilepsy.

“Some of these kids can’t speak, are not mobile, they sit in a cot, they have 20 seizures a day and they die when they’re 12. So, absolutely devastating neuro-developmental disorders,”The researchers take skin cells from the children, turn them into pluripotent stem cells that can form almost any tissue in the body, then direct them to become neurons.

Through a microscope you can clearly see the slender bodies of those brain cells afloat in a watery matrix. Hook them up to electrodes and you get something mind-bending. These guys are talking to each other – the computer shows spikes of electrical activity as the neurons fire.

A multi-electrode array used to measure electrical activity in minibrains

But for the kids Petrou is trying to help, the chatter is out of whack. Some have a mutation in a gene called SCN2A that controls the passage of sodium in and out of the neuron.

“This is a gain of function of excitation, so this channel works too hard and produces epilepsy,” says Petrou.

Replicating that glitch in a dish has allowed the researchers to tailor a treatment right there on the bench; Petrou is on the verge of announcing a clinical trial of a gene therapy to treat one variant of the disorder.

And it won’t just aim to stop the seizures.

The idea with precision medicine in this application is if you can fix the fundamental disorder far enough back in the pathological chain, you should fix all the problems,” says Petrou.
If the treatment works, these kids could be spared the intellectual disability and movement disorders that go hand-in-glove with constant seizures.

Complexity is something of a buzzword in organoid research. Recent Australian research upped the stakes by 3D-printing brain tissue, bringing a replica brain on a bench into tantalising – if very distant – focus.

But Petrou stresses just how tricky that task is going to be.

“We know how fragile a real brain is. One genetic mutation, some trauma, and that brain doesn’t work anymore,” he says. “It is so easy to break and therefore that means it is probably going to be so difficult to reproduce.”

Nonetheless, as the technology advances you wonder if those organoids could grow to the point where one day they start to gain bona fide moral status.

If that happens, the first sign might just be a very slight pang as scientists toss their dreaming minibrains into the garbage.

This is an edited extract from a larger story by Paul Biegler which appeared in the Sydney Morning Herald

Article sourced from The Florey Institute

Appointment of new Clinical Director of the Florey

The Florey Institute Logo

The Florey Institute of Neuroscience and Mental Health has announced the appointment of Professor Trevor Kilpatrick to the role of Clinical Director,  effective 1 October 2018.

This is a joint position with the University of Melbourne which will provide high level clinical leadership in the discipline of Neuroscience. The role will also make major contributions in the area of academic leadership and research, including external engagement to build on successful partnerships with hospitals to enhance global translational outcomes in neurology and psychiatry.

Trevor Kilpatrick is an academic neurologist and PhD trained neuroscientist. His research interests include the neurobiology of multiple sclerosis, neural precursor cell biology and the study of genetic and environmental factors that contribute to MS as well as the translation of basic research discoveries to the clinic.

Professor Kilpatrick undertook his undergraduate medical degree at the University of Melbourne and clinical training at the Royal Melbourne Hospital. He then undertook graduate studies at The University of Melbourne in the Department of Medical Biology at the Walter and Eliza Hall Institute and gained a Doctor of Philosophy in 1993. He was appointed as a Howard Hughes Medical Postdoctoral Fellow at the Salk Institute (1993-5) and returned to Australia as laboratory head at the Walter and Eliza Hall Institute (Viertel Fellow [1995-2000], NHMRC SRF, [2000-03]). He then took up an appointment as Chair of Neurology in the Centre for Neuroscience at the University of Melbourne and was Director of the Centre from 2004-2013. In 2009 he was appointed the Director of the Melbourne Neuroscience Institute (MNI) at the University of Melbourne where he has continued to foster interdisciplinary research relevant to the Neurosciences over the last decade. He has also held concurrent appointments at the Florey Institute of Neuroscience and Mental Health as Head of the MS Division (since 2003-) and at the Royal Melbourne Hospital as Head of the MS Unit since 2000.

Professor Kilpatrick has been the recipient of the Sunderland Award (1994), AMRAD Postdoctoral Award (1995), inaugural Leonard Cox Award (2000), Bethlehem Griffiths Research Foundation Award for Medical Research (2004), the Australian Museum’s Jamie Callachor Eureka Prize for Medical Research (2008), the Stephen C. Reingold Research Award by the US MS National Multiple Sclerosis Society (2010) and the Bethlehem Griffiths Research Foundation Medal for outstanding leadership in medical research (2013). He was admitted to the Australian Academy of Health and Medical Sciences in 2016. His total career publications number 207, with 15,765 citations (Google Scholar). Since 2013, he has authored 64 publications with 8,701 citations (Google Scholar). He has published in Nature, Nature Medicine, Nature Genetics, Nature Communications, JAMA, PNAS, BMJ, Neuron and JCI.

The Melbourne Biomedical Precinct Office looks forward to the tremendous contributions that Professor Kilpatrick will make to the Florey and across the broader precinct.