New ALS drug slows disease progression in groundbreaking clinical trial
Motor neurone disease (MND), also known as amyotrophic lateral sclerosis (ALS), is a devastating disease that quickly kills the brain cells in charge of muscle control, usually claiming the lives of patients within a few years. But now a new drug developed by a team of Australian scientists has shown promise in a Phase 1 clinical trial, improving symptoms and slowing progression of the disease.
Work on the drug, known as CuATSM, began over 15 years ago. At first it was thought that an excess of copper in the brain was the problem, but the researchers soon realised the opposite may be true.
"The initial focus of our work was on Alzheimer's disease where it was thought that copper drove the formation of amyloid plaques," Professor Kevin Barnham, co-developer of CuATSM said back in 2017. "We reasoned if you could just remove this copper you could eliminate plaques, and you'd have the solution. Then we thought, perhaps it's the other way around? Maybe cells are expelling the copper and so we've actually got a situation of copper deficiency. So, what happens if you deliver the copper back?"
And that's just what the new drug does. CuATSM is a compound that can penetrate the blood-brain barrier and deliver copper to the cells that need it. After tests in vitro and in animal models showed promise, a Phase 1 human clinical trial began in late 2016 to find the right dosage.
The trial consisted of 32 participants and it was found that those who received the highest amount of CuATSM showed the most marked improvements. Those participants regained some lung function and cognitive ability, and the overall disease progression was found to be much slower.
"This is the first human evidence for a disease-modifying drug for motor neurone disease," says Professor Ashley Bush, director of the Melbourne Dementia Research Center. "It is a huge breakthrough, and we look forward to confirming the positive results in a larger study soon."
The Phase 2 trial, which will be larger, randomized, placebo-controlled and double-blind, is due to begin later this year.