The Heat Is On

Aducanumab, they wayward child of Alzheimer's disease treatments, will get her day in court on November 6.  Here is the entry in the Federal Register announcing the meeting when the FDA will consider Biogen's request for approval.  And the pressure on the FDA to approve is considerable.  

What must the FDA consider in their decision?  First is safety, and the second is efficacy.  Is it safe, and will it work?

First -- safety:  The monoclonal antibody is known to cause microhemorrhages in the brains of some test subjects.  None of these had serious consequences, but the test subjects were taken off the treatment immediately when the hemorrhages were detected.  All test subjects required frequent MRIs to detect the hemorrhages, and it's anticipated that anyone treated with an approved drug will require the same MRI regimen.

Second -- efficacy:  During Biogen's trial, fear of microhemorrhages caused the researchers to significantly cut the dose for many of the test subjects.  When the data was analyzed at the end, the researchers had a mixture of data from both doses.  From this, they initially concluded that the treatment was ineffective and declared it a bust.  But the researchers worked late into the night to discover that, if you broke out the test subjects who were exposed to the full dose, maybe it worked after all.  It wasn't a cure, but it appeared to be the first pharmacological treatment that would actually attack the disease mechanism and slow its progress.

There are some who believe that the trial was botched, and they should have a do-over.  But that would take four more years.  Others say that we understand both the risks and the potential well enough that we should just go ahead and make it generally available.  We'll see what they decide.

I actually first began hearing back in 2016 that we should just make it available before even performing the drug trial.  I don't know if I could find the article now, but I believe I saw a doctor making the case for it in Parade Magazine.  The thought was that Alzheimer's disease is so onerous and so many victims would not outlive the trial that it should be available to anyone willing to take the risk.  That didn't happen, but I'm guessing that the FDA will approve it without the customary level of evidence for its efficacy.  It's likely they will agree with Biogen that the side effects and risks are sufficiently well understood to let it go forward.