Patient trial heralds motor neurone breakthrough

A new drug delays motor neurone disease progression and improves cognitive and clinical symptoms, according to trial results announced by a spin-out company from the Florey Institute and the University of Melbourne.

Image-of-a-person-with-motor-neurone-disease-in-a-wheelchair

The 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 Cu(-ATSM) 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.

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 Cu(-ATSM) over a 15-year period.

After showing its therapeutic potential for motor neurone disease in pre-clinical models, the researchers founded a company, Collaborative Medicinal Development, 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 enrolment for a larger, randomised, placebo-controlled double-blind Phase 2 trial in mid to late 2019. This trial will test Cu(-ATSM)’s effectiveness in motor neurone disease/amyotrophic lateral sclerosis in a larger patient sample.