Abstract
Developing an integrative approach to early treatment response classification using survival modeling and bioinformatics with various biomarkers for early assessment of filgrastim treatment effects in amyotrophic lateral sclerosis patients. Filgrastim, a hematopoietic growth factor with excellent safety, routinely applied in oncology and stem cell mobilization, had shown preliminary efficacy in ALS.
Highlights
Amyotrophic lateral sclerosis (ALS) is a severe neurodegenerative disorder with a median survival time of 15.8 months from diagnosis [1]
Dose and application modes were individualized, and detailed description may be seen in Supplementary Figure 1
Filgrastim was well tolerated, and safe, minor adverse events were mild to moderate bone pain after injections, and—as expected— leukocytosis
Summary
Amyotrophic lateral sclerosis (ALS) is a severe neurodegenerative disorder with a median survival time of 15.8 months from diagnosis [1]. Filgrastim is a neuronal growth factor with neuroprotective and regenerative properties [3, 4], enhancing immunocompetence [5], reducing neuroinflammation [4], increasing motoneuron functional activity [4, 6], and improving motor function and survival in ALS mouse models [4, 7]. Since its approval in the early 1990s, filgrastim has been applied in millions of patients and healthy donors. It is an established and well-tolerated clinical compound, so far in some preliminary studies with ALS patients [4, 8,9,10,11,12]
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