Hong Kong researchers discover mechanism that may provide ‘new angle’ in developing treatments for ALS
The Chinese University of Hong Kong and Oxford University collaborated for the study on the incurable rare neurodegenerative disorder, with further tests still to come
‘If we can develop methods or treatments targeting this newly discovered signalling pathway, we may be able to suppress the death of motor neurons,’ expert says
Researchers at the Chinese University of Hong Kong (CUHK) have identified a mechanism for amyotrophic lateral sclerosis (ALS), a finding that may shed light on developing treatments for the currently incurable rare neurodegenerative disorder.
Further experiments on animals were still needed before moving onto clinical trials for an ALS treatment, which was still at a preliminary stage of development, researchers said on Monday.
ALS is a rare neurodegenerative disease that affects nerve cells in the brain and spinal cord, which can cause muscle weakness, loss of coordination and slurred speech, among other things.
Patients can also suffer from respiratory failure when breathing muscles are affected.
Experts say the mechanism can shed light on developing ALS treatments. Photo: Yik Yeung-man
The study, conducted in collaboration with Oxford University, found that ALS patients had an uneven distribution of a protein named YY1 in their cells.
Such a distribution pattern results in a dysfunction when regulating the level of another protein named Fuzzy, which is essential for neuron functions.
ALS patients’ level of Fuzzy protein was found to be lower than in normal people, a disorder that could activate a pathway and lead to the death of motor neurons and contribute to the disease’s onset.
“If we can develop methods or treatments targeting this newly discovered signalling pathway, we may be able to suppress the death of motor neurons,” said Stephen Chen Zhefan, a postdoctoral fellow at CUHK’s school of life sciences who co-authored the study.
The study findings were published last month in the leading scientific journal Nature Communications.
Edwin Chan Ho-yin, a professor at the same school and leader of the study, said they had already identified two molecules related to the YY1 protein that could suppress the death of motor neurons based on preliminary studies.
Experiments on the efficacy of drug candidates were being done on cells and flies, he said, stressing that his team was hoping to conduct tests on mice next.
Chan added they were looking for funding to conduct experiments on bigger animals, such as monkeys, to test the molecule toxicity.
He also said that if funding had been secured, experiments could be completed in nine months before moving on to clinical trials.
The study gives hope to ALS patients, including Sze. Photo: Yik Yeung-man
Since 2017, Chan’s team has also been developing another medication targeting ALS based on a different mechanism. This medication development has already entered the preclinical stage and is expected to be tested on larger animals next.
Sze Chi-wai, an ALS patient for more than seven years, said he was looking forward to the research outcome.
“I’m very happy as there could be treatment for the disease,” he said. “At least there is hope here.”
Sze was 49 years old when he was first diagnosed with ALS. It started with the loss of mobility in a single finger.
He started using a ventilator to help him breathe three years ago. He has also lost mobility in his limbs and is forced to rely on a wheelchair to move around.
According to the city’s first ALS patient registry, established by the Hong Kong Neuro-Muscular Disease Association, about 200 patients suffer from the condition locally.
The average age of developing symptoms is 55, while the survival time ranges from a few months to 10 years, with a median of three years.
Renowned physicist Stephen Hawking battled ALS for 55 years before he died in 2018.
