In its national coverage determination, Medicare limits Aduhelm coverage to clinical trials, but OKs registry-based studies for traditionally approved anti-Aβ antibodies. This may change once new efficacy data comes out.
Most observational cohorts are on pause, and many clinical trials have stopped dosing. The long-term effects on the integrity of AD studies are unclear.
Months before mice develop plaques, faulty lysosomes swollen with Aβ commandeer plasma, ER, and Golgi membranes, creating toxic rosettes around the nucleus. The neurons eventually burst, leaving behind plaques.
While the FDA weighs aducanumab’s marketing license application, Alzheimer’s researchers agree that the agency’s own statistician correctly assessed the data as weak. Most prefer that one more trial be done.
After shutting down a Phase 3 program last March, Biogen now says the futility analysis it did was incorrect, and that a new analysis of a larger dataset in fact supports filing for FDA marketing approval next year.
The indefatigable Colombian neurologist leaves behind a legacy of Alzheimer’s prevention research, which, at the time of his passing, stands on the cusp of success.
Scientists at CTAD found the Phase 3 trial well-done, the data consistent, and expect the drug to be approved. Priorities: grow the effect, understand the bleeding risk.
Using a collection of isogenic iPSC-derived neurons harboring different familial AD mutations, researchers found that, across the board, the mutations meddled with endosomes via elevated β-CTF.