The new Cell study on the failed BIIB078 ASO (for C9orf72 gene) shows the drug hit the CNS, but didn't clear the toxic G4C2 RNA in brain tissue, thus, toxic pathologies (DPRs, pTDP-43) remained.
Significance: CSF biomarkers were misleading—they didn't match the brain!
The new Cell study on the failed BIIB078 ASO (for C9orf72 gene) shows the drug hit the CNS, but didn't clear the toxic G4C2 RNA in brain tissue, thus, toxic pathologies (DPRs, pTDP-43) remained.
Significance: CSF biomarkers were misleading—they didn't match the brain!
A Science paper finds when heart muscle is weak, other cells (fibroblasts) panic, multiply, & strangle it stiff.
Value: Block one switch (p38) in them & the heart is saved. A new target!
Flaw: It's a genetic edit in mice that prevents, not reverses.
A Science paper finds when heart muscle is weak, other cells (fibroblasts) panic, multiply, & strangle it stiff.
Value: Block one switch (p38) in them & the heart is saved. A new target!
Flaw: It's a genetic edit in mice that prevents, not reverses.
Upshot: An Alzheimer's gene (ABCA7) messes up fats in brain cells , breaking their batteries (mitochondria).
Value: They fixed it in a dish with a simple supplement (CDP-choline)! It even lowered amyloid gunk.
Flaw: This was in a lab dish, not a person. Big leap.
Upshot: An Alzheimer's gene (ABCA7) messes up fats in brain cells , breaking their batteries (mitochondria).
Value: They fixed it in a dish with a simple supplement (CDP-choline)! It even lowered amyloid gunk.
Flaw: This was in a lab dish, not a person. Big leap.