The FDA has just recently approved the first new drug for treating Alzheimer’s in nearly two decades, named aducanumab. It’s accelerated approval path is so remarkably parallel to that of another drug approved for ALS a handful of years ago that some wisdom might be gained from the comparison.
In late 2016 I was diagnosed with ALS, or Lou Gehrig’s disease. At the time, I was a 50 year old dedicated and successful athlete who was just beginning to face a terrible new problem. Emotions were high. The motivation to do something, to figure out a way to beat my impending demise, was off the charts.
The diseases of ALS and Alzheimer’s share some striking similarities. They are both degenerative neurological diseases, both are devastating and fatal, both have been historically resistant to understanding and perhaps most importantly, both have little in the way of effective treatments.
In early 2017, the Food and Drug Administration approved the first new medication for ALS in over two decades using a similar accelerated approval process that was recently used for the new Alzheimer’s drug, aducanumab. In an amazingly similar trajectory, Mitsubishi Tanabe Pharma of Japan took a leading theory of what causes neurons to die in ALS patients, devised a chemical that might prohibit that and continued with the appropriate double blind studies of their proposed drug, Radicava. The first study using a broader sample of ALS patients showed no benefit. The drug company repeated the study, this time using only newly diagnosed patients matching certain criteria and found a modest benefit, and thus won FDA approval for all patients, although no benefit was found in the earlier study with the broader sample. The brand new Alzheimer’s drug had a smiliar two study scenario, and will have a price tag of $56,000 per year, which doesn’t include MRI’s to watch for the 40% chance of brain swelling and brain bleeds noted as side effects on this newly “approved” drug.
With Radicava, the ALS community was excited. The advocacy groups were excited. The doctors were really excited. I was contacted to be on the front page of the newspaper and on TV to show the patient perspective. Finally, we have something, people exclaimed.
Before beginning the medication myself, I started to question the conflicting studies. When questioning my doctor about this, the FDA approval was used as evidence in itself.
I had a port surgically implanted and started getting daily intravenous infusions, with on/off cycles during the month. Most anything short term can be tolerated and justified but this was to continue for the rest of my life. The impact of an ongoing treatment process such as this on quality of life should not be underestimated. I remember mornings spent waiting to start with my day while tinkering with a slow IV drip. This was especially hard when attempting to travel. I have no clue as to whether the drug was or wasn’t slowing progression but I wasn’t going to cling to the strong desire that we all have to do something, especially in desperate situations. After about a year, I decided to quit with more or less no noticeable change in my progression rate.
Other small scale studies around the world have since painted a murky picture. I don’t think that any have reproduced the promising result as the one repeat study and thus, excitement has waned. Costing $137,000 a year and already approved, the manufacturer of the ALS drug Radicava has little incentive to rock the boat.
When a terrible disease is encountered and one has no cure, it is only natural to pin your hopes on any new thing. Some might say ‘what’s wrong with that?’ I say, from experience, lots. I am not alone. Of the FDA advisory board that unanimously advised the FDA against aducanumab approval, three have resigned over the FDA’s decision.
Excellent David. You have a perspective as a patient that is invaluable . You point out the true costs to patients of FDA approval with very murky evidence. Love to you, Lebe
I think it’s a real tribute to your critical thinking skills that you can research, think about and then write a critical review of something that impacts your physical and emotional health. It’s a very impressive bit of writing
Awe, shucks (wondering around and kicking a rock)