Researchers in Australia have identified a three-drug combination that greatly extends the survival of motor neuron cells in lab tests, providing new hope for motor neuron disease (MND) patients.
Scientists used an innovative stem cell platform to test more than 100 drugs, converting skin cells donated by people with MND into motor neurons, the nerve cells affected by the disease, said a Tuesday statement by The Florey in Australia, the largest brain research centre in the Southern Hemisphere.
MND, or amyotrophic lateral sclerosis, causes progressive paralysis affecting movement, speech, swallowing, and breathing. Patients typically live two to three years with current treatments, with riluzole, the only approved drug, prolonging survival by about three months, it said.
The researchers discovered that a triple combination of riluzole, an anti-inflammatory drug and a medication developed to treat dementia, was 6.5 times more effective than riluzole alone at prolonging the survival of nerve cells in the laboratory.
The model paves the way to better understanding MND and the development of new treatments, according to the study, published in Nature Neuroscience.
Associate Professor Chris Bye, who co-led the study with Professor Bradley Turner at The Florey, said MND is difficult to study because its causes are complex and incompletely understood.
“About 10 per cent of cases have a known genetic cause. Our focus is on finding treatments for the 90 per cent of ‘sporadic’ or unexplained cases,” Bye said.
“Drugs are traditionally developed in the lab, tested in mice, and if they show promise, they might make it to human trials. Since the discovery of riluzole over 30 years ago, more than 170 drugs for sporadic MND discovered with this approach have failed in clinical trials,” Turner said.
The innovative platform at The Florey enables testing drugs directly on motor neurons from MND patients for the first time, he added.
