在最近发表于《European Journal of Human Genetics》的研究中，科学家基于病理生理机制识别出3种不同的ALS生物型（子类型），同时开发出一种机器学习模型，能够使用人口统计学和疾病相关信息将ALS患者划分到3组之中。研究人员表示，这些结果和方法将为未来的临床试验提供启示，从而解决ALS患者的异质性问题，并开发特定靶向不同ALS生物型的治疗方法。https://www.nature.com/articles/s41431-025-01920-y
Published recently in the European Journal of Human Genetics In the study, the scientists identified three different ALS biotypes (subtypes) based on pathophysiological mechanisms and developed a machine learning model that can use demographic and disease-related information to divide ALS patients into three groups. "These results and approaches will provide implications for future clinical trials to address the heterogeneity of ALS and develop therapies that specifically target different ALS biotypes," the authors write. https://www.nature.com/articles/s41431-025-01920-y
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Cellenkos announced that the FDA has granted orphan drug designation for its regulatory T cell (Tregs) therapy CK0803 for the treatment of ALS. The CK0803 is currently undergoing phase 1 / 1b REGALS clinical trials and is expected to receive top-line data next year.
By analyzing millions of mRNA molecules in the progression of ALS, researchers discovered that certain neurons, such as those that control eye movements, which are resistant to ALS, may not respond significantly to the disease due to their high levels of several neuroprotective factors. Neurons that are sensitive to ALS, on the other hand, activate both harmful and protective responses. These findings on the different basal and induced gene activities in various types of neurons open up new possibilities for treatment. The study was recently published in Genome Research. https://genome.cshlp.org/content/early/2025/08/08/gr.279501.124
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Prilenia Therapeutics and Ferrer announced today that they will launch a phase 3 clinical trial of oral therapy Pridopidine for ALS in January, based on promising results from the Healey ALS trial. Participants will be randomized to receive either Pridopidine or a placebo for 48 weeks, followed by a 48-week extended period. Pridopidine is a small molecule drug designed to activate the sigma-1 receptor (S1R), reduce stress and improve nerve cell survival.