Australian researchers have developed a groundbreaking drug with promise of treating the debilitating and fatal motor neurone disease, not just the symptoms.
Participants had improved lung function and cognition and a reduction in the decline of their motor disability.
The results of a 32 patient trial in Sydney and Melbourne was the first human evidence for a disease-modifying drug for motor neurone disease.
Motor neurone disease is a progressive, fatal neurode generative disease with the death of brain cells which control muscle movements, eventually causing paralysis.
Patients usually die of respiratory failure within three years of diagnosis and there are no treatments or therapies available.
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.
“It’s a huge breakthrough,” said Professor Ashley Bush, chief scientific officer of collaborative medicinal development and director of the Melbourne Dementia Research Centre.
The researchers plan to begin enrollment for a larger, randomised, placebo-controlled double-blind Phase 2 trial in mid- to late 2019.