According to a post hoc analysis, a greater percentage of patients receiving NP001 experienced a halt in disease progression which reached statistical significance when compared to the combination of concurrent and matched historical (placebo) controls. The high dose of NP001 (2mg/kg) halted progression in 27% of patients compared to 11% of patients on placebo. Further, NP001 was found to be safe and well-tolerated in the study.
Amyotrophic lateral sclerosis or ALS; Alzheimer’s disease, Parkinson’s disease, lytico-bodig and other similar neurodegenerative diseases have a common denominator. “Slowly, people in different places are coming on a realization that, probably, all these diseases have a common cause,” Steele said.
After decades of research, Guam and stateside neurologists have reason to believe the causes and possible cures for Alzheimer’s and other neurodegenerative diseases could be discovered on Guam. The most important question to answer, Steele said, is why lytico-bodig has slowly gone away during the last 50 years on Guam, while in all other parts of the world, related diseases such as ALS, Parkinson’s and Alzheimer’s are common.
Recognition that the mutations adversely impact regulation of RNA could lead to targeted therapy to correct the problem. The mutation’s location in the prion-like domain might also prove significant. Although the mutations in hnRNPA2B1 or hnRNPA1 appear to be rare, hundreds of other RNA-binding proteins have prion-like domains. Taylor said patients with unexplained neurodegenerative diseases may have mutations in these proteins.
Stevens thinks that their findings might shed light on diseases that involve synapse loss or dysfunction. In neurodegenerative diseases such as glaucoma, Alzheimer’s disease, Lou Gehrig’s disease (ALS), Huntington’s disease and Parkinson’s disease, scientists have noted subtle changes in synapses that might cause them to be targeted for elimination.