June 21 marks the annual Amyotrophic Lateral Sclerosis (ALS) Awareness Day, a global initiative dedicated to raising awareness of this progressive neurodegenerative disease. The day serves as a platform to inspire crucial research funding and empower those living with ALS.
First chosen by the International Alliance of ALS/MND Associations in 1997, and officially designated as World ALS Day in 2000, June 21 symbolizes a turning point in the fight against ALS. Marked by the summer solstice—a time of change—this day aims to bring us closer to effective treatments and, ultimately, a future free from ALS.
On this day, organizations, medical professionals, caregivers, and advocates come together to educate the public about ALS, its symptoms, and the challenges faced by patients and their families. Events include informational campaigns, fundraisers for research, public talks by medical experts and patients, social media campaigns sharing personal stories, and community gatherings to provide support and solidarity.
This year, significant strides have been made in Asia, where cutting-edge technology and collaborative research are offering new hope in the battle against ALS.
An international research team, including scientists from the Chinese mainland’s Tsinghua University, Harvard Medical School, and Johns Hopkins School of Medicine, has utilized artificial intelligence to identify potential new treatments for ALS. Using Insilico Medicine’s AI-driven target discovery engine, PandaOmics, the team identified 28 gene targets with therapeutic potential.
In animal models, the researchers validated that 18 of the identified gene targets were functionally correlated with ALS, and suppressing eight of them significantly reduced neurodegeneration.
“This study demonstrates how AI can accelerate the drug discovery process and opens up new opportunities for therapeutic interventions,” said Lu Bai, professor of pharmaceutical sciences at Tsinghua University. “AI can reduce the cost and time but raise the success rate of drug development, especially for neurodegenerative diseases.”
The findings were published in the journal Frontiers in Aging Neuroscience, highlighting the potential of AI in combating complex diseases like ALS.
Meanwhile, a research team from the Chinese University of Hong Kong (CUHK), in collaboration with the University of Oxford, uncovered a novel molecular mechanism leading to the pathogenesis of ALS. Led by Professor Edwin Chan Ho-yin from CUHK’s School of Life Sciences, the team studied the underlying mechanisms of gene transcriptional control in ALS.
The team demonstrated that the function of a transcriptional factor, YY1, is disrupted in the spinal motor neurons of ALS patients, inducing a pathway that leads to neuronal cell death. These insights offer additional mechanistic understanding of ALS and open avenues for developing new drug candidates. The research findings were published in the journal Nature Communications.
“Elucidating the underlying disease mechanisms not only allows us to better understand ALS pathogenesis but, more importantly, directs the development of new therapeutic strategies against this devastating condition,” said Professor Chan.
Furthermore, a group of researchers in the Chinese mainland has created a gene-targeted therapy drug for ALS. SineuGene Therapeutics, a biopharmaceutical company, successfully completed a clinical drug delivery attempt using an adeno-associated virus vector gene therapy for the disease.
“Treating ALS is a worldwide challenge,” said Peng Lin, co-founder and CEO of SineuGene Therapeutics. “Based on the basic research achievements in neuroscience from the Tsinghua University School of Medicine, we developed the drug and completed the transformation of the research results within two years.”
These advancements demonstrate the significant impact of technological innovation and international collaboration in the fight against ALS. On this ALS Awareness Day, the progress brings renewed hope to patients and underscores the importance of continued research and support worldwide.
