New ALS drug Radicava comes with new questions as well
It is the first new drug for amyotrophic lateral sclerosis in 22 years.
In this study, we have demonstrated that the Aβ-binding region is exposed on the SOD1 surface through the conformational changes due to misfolding of SOD1. Interestingly, we found that the intracellular aggregation of Aβ is enhanced through the direct interaction of Aβ with the Aβ-binding region exposed to misfolded SOD1. Ultimately, increased Aβ aggregation by this interaction promotes neuronal cell death. Consistent with this result, Aβ aggregates was three-fold higher in the brains of G93A transgenic mice than those of non Tg. Our study provides the first direct evidence that Aβ, an AD...
Nature. doi:10.1038/550S105a Author: Brian Owens
New England Journal of Medicine,Volume 377, Issue 16, Page 1602-1602, October 2017.
CONCLUSIONS: We show that in fibroblasts of FUS P525L mutation carriers, FUS mislocalized to the cytoplasm where it redistributed into stress granules with likely a dose effect, i.e. a higher number of cells with granules, which persist longer, than in controls and ALS cases. These data represent an early molecular change occurring before ALS onset, suggesting a transient preaggregative state. PMID: 29035885 [PubMed - as supplied by publisher]
Conclusions: We show that in fibroblasts ofFUS P525L mutation carriers, FUS mislocalized to the cytoplasm where it redistributed into stress granules with likely a dose effect, i.e. a higher number of cells with granules, which persist longer, than in controls and ALS cases. These data represent an early molecular change occurring before ALS onset, suggesting a transient preaggregative state.Neurodegener Dis 2017;17:292-303
Brainstorm Cell Therapeutics (NSDQ:BCLI) said today that the first patients have been enrolled in its Phase III trial evaluating NurOwn as a treatment for amyotrophic lateral sclerosis, or ALS, at Massachusetts General Hospital and UC Irvine Medical Center. The trial, which is slated to enroll 200 patients, is designed to include the pre-specified patient subgroups who saw superior outcomes in the NurOwn Phase II ALS trial. BrainStorm said it expects top-line data from the trial in 2019. Get the full story at our sister site, Drug Delivery Business News. The post BrainStorm enrolls first ALS patient in late-stage NurO...
Trial supported by a $16M non-dilutive grant from CIRM Top-line data are expected in 2019 HACKENSACK, N.J. and PETACH TIKVA, Israel, Oct. 16, 2017 -- (Healthcare Sales &Marketing Network) -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) today an... Regenerative Medicine, Neurology BrainStorm Cell Therapeutics, NurOwn , stem cell, amyotrophic lateral sclerosis
Developments in eye-gaze technology – which converts minute movements of the eye into spoken words – are opening up undreamed of opportunities for people with motor neurone syndromeSteve Thomas and I are talking about brain implants. Bonnie Tyler ’s Holding Out For a Hero is playing in the background and for a moment I almost forget that a disease has robbed Steve of his speech. The conversation breaks briefly; now I see his wheelchair, his ventilator, his hospital bed.Steve, a software engineer, was diagnosed with ALS (amyotrophic lateral sclerosis, a type ofmotor neurone disease) aged 50. He knew i...