June 21 marks Amyotrophic Lateral Sclerosis (ALS), also known as Motor Neurone Disease (MND), Awareness Day. ALS Awareness Day serves as a global platform to raise awareness for the progressive neurodegenerative disease, inspire crucial research funding, and empower those living with ALS.

The date of June 21 was first chosen by the International Alliance of ALS/MND Associations in 1997, and got its official designation of World ALS Day in 2000 at the International ALS Conference held in Denmark. Marked by the symbolism of the summer solstice, a turning point in the year, the ALS community hopes June 21 will similarly mark a turning point in the fight against the disease, bringing people closer to effective treatments and a future free from ALS.

On this day, organizations, medical professionals, caregivers and advocates come together to educate the public about ALS, its symptoms, challenges faced by patients and their families, and ongoing research efforts. It serves as an opportunity to honor those affected by ALS and to support initiatives aimed at finding effective treatments and, ultimately, a cure.

Awareness events may include informational campaigns, fundraisers for research, public talks by medical experts and patients, social media campaigns to share personal stories, and community gatherings to provide support and solidarity.

By highlighting ALS Awareness Day, we not only acknowledge the courage of individuals battling this disease but also emphasize the importance of continued research and support for improving the quality of life for ALS patients and their families worldwide.

Tech vs. ALS

ALS is a neurodegenerative disease. Patients progressively lose muscle strength, eventually becoming paralyzed and unable to speak, move, swallow or breathe. Despite being known to medicine for years, current ALS treatments are unable to halt or reverse this loss of function. Scientists have struggled to identify the molecular causes of ALS that can lead to therapies.

Using an artificial intelligence (AI) platform, an international research team has identified more than two dozen new targets with therapeutic potential for ALS, according to a study published in the journal Frontiers in Aging Neuroscience.

Researchers from China's Tsinghua University, Harvard Medical School, Johns Hopkins School of Medicine, alongside drug discovery company Insilico Medicine, used Insilico's AI-driven target discovery engine, called PandaOmics, to identify 17 high-confidence and 11 novel therapeutic targets for the disease.

In animal models, the team validated that 18 of the 28 identified gene targets were functionally correlated to the ALS and found that in eight of them, suppression could strongly reduce neurodegeneration. 

Co-author Lu Bai, professor of pharmaceutical sciences with Tsinghua, said the study demonstrates how AI speeds up the target discovery process, and opens up new opportunities for therapeutic interventions. "The study represents a new trend, in which AI can reduce the cost and time but raise the success rate of drug development, especially for neurodegenerative diseases," said Lu.

In addition, the Chinese University of Hong Kong (CUHK) also announced that its research team, collaborating with the University of Oxford, uncovered a novel molecular mechanism that leads to the pathogenesis of ALS.

To identify ALS therapeutic targets, the research team led by Edwin Chan Ho-yin, a professor from the School of Life Sciences at CUHK, studied the underlying mechanisms of gene transcriptional control in ALS.

The team demonstrated that the function of a transcriptional factor YY1 is perturbed in ALS patient spinal motor neurons, which subsequently induces a pathway and consequently leads to neuronal cell death. This offers additional mechanistic insights into ALS pathogenesis.

Chan said that elucidating the underlying disease mechanisms will not only allow researchers to better understand ALS pathogenesis but, more importantly, will also direct the development of new drug candidates against this devastating neurodegenerative condition. The research findings have been published in the scientific journal Nature Communications.

Moreover, a group of Chinese researchers have created a gene target therapy drug for ALS. SineuGene Therapeutics, a biopharmaceutical company, successfully completed a clinical drug delivery attempt of adeno-associated virus vector gene therapy for ALS.

The company's co-founder and CEO Peng Lin acknowledged that treating ALS is a worldwide challenge. "Based on the basic research achievements in neuroscience of the Tsinghua University School of Medicine, we developed the drug and completed the transformation of the research result within two years," Peng said.

(With input from Xinhua)