Aducanumab DOA -- Probably

It's all over the news.  The FDA's Peripheral and Central Nervous System (PCNS) Drugs Advisory Committee voted against recommending that the FDA approve Aducanumab.  The FDA is not bound by the committee's conclusion, but I would be surprised if the FDA disregarded their recommendation and went ahead with approval.

It's not surprising that Biogen's presentation painted the monoclonal antibody in a positive light.  But it surprised me that the PCNS criticized the FDA for speaking positively about aducanumab when they tasked the committee with their evaluation.

There were 10 voting members of the committee, but only one member supported pursuing approval.

The sponsors, led by Biogen, conducted multiple trials of the drug, but only one trial yielded positive results.  That was the one where the full dose was administered throughout the trial, and Biogen's case was efficacy was proven in that trial.  One of the committee members said that was like shooting an arrow at the side of a barn and then painting a target around it with the arrow in the bull's eye.

You will recall from Beating the Dementia Monster that I auditioned for the third phase trial of aducanumab but was rejected.  I wasn't disappointed in this because I doubted that the drug would be successful.  The treatment had shown promising results in the first phase (probably 12 statistically problematic test subjects), but had not shown promise in phase 2 with a larger cohort of test subjects.  I wondered how they justified proceeding with the third phase.  My conclusion was that there were so few other prospects for an effective pharmacological treatment that grasping at aducanumab seemed to be the only promising near-term option.  This explains the great wailing and gnashing of teeth that followed Biogen's initial conclusion that the treatment was ineffective. 

All of this underscores that the multi-domain lifestyle interventions we describe in Beating the Dementia Monster are, for the moment, what we have for holding back the advance of Alzheimer's disease.