Centaur

September, 2020

The New York Times recently published an article (https://www.nytimes.com/2020/09/02/health/als-icebucket-treatment.html) on AMX0035 and the Centaur study in which I participated. Because you have nothing better to do, I decided to bore you with my involvement in the trial.

When I was first diagnosed, I dove into everything I could find about potential therapies. Notwithstanding ALS is particularly frustrating when it comes to developing new treatments, there is a lot of research happening. I searched papers in the National Institute of Health website even though I could only understand every fifth word. For some reason I enjoyed nerding out on protein misfolding and mitochondrial malfunction, both of which I dare you to google.

I soon got in touch with my inner lab rat and wandered over to clinicaltrials.gov. This nifty site allows you to search for drug and other therapies under investigatio. Not only can you find trials in your area, but you can review exclusion criterIa for each trial, which is a nice way of saying bugger off if you don’t satisfy the criteria. One near universal exclusion criteria for ALS is that you must have been diagnosed within the past 18-24 months. Yikes! No time to lose.

After a bit of searching I came across Centaur, a Phase 2 trial having a site at the Forbes Norris ALS Institute about an hour away. The medication is a combination of two compounds, the names of both of which are completely unpronouncable by mere mortals. One compound has been shown to impact neuron oxidation and the other impacts mitochondrial malfunction. Don’t worry. I don’t understand what that means either. What I did understand is that following the six month trial I would be guaranteed the real thing for at least the following two years.

I contacted the research assistant on the project, an engaging lad who, true to his Irish citizenship, turned me on to a great writer (Roddy Doyle if you’re interested). A few weeks later I spent a day being assessed and found out I had passed and could participate in the trial. Good thing because as soon as the paperwork was submitted I saw a press release saying the trial was fully enrolled. In under the wire.

One of the innovations in this trial was the greater use of objective data to determine disease progression through changes in muscle function. This was accomplished using a contraption that looked like a disembowled weight machine, but colored blue. You are transferred into this beast and various limbs are contorted into a variety of positions. Once settled in the appendage being tested is pressed against a pressure sensor bluetoothed to a research laptop. Suitably configured the research assistant berates you to push push push then rest. You do this over 8 configurations 2 – 3 reps a piece for a total of, well you do the math. Let’s just say it’s a good thing the breathing tests precede the blue beast.

Next stop is medication, or a placebo if I am one of the one out of three that drew the short straw. The preparation must be drunk twice a day and my tasting notes are contained in my blog entitled My ALS (search: Drano). Drinking this stuff before breakfast and dinner was like having your premeal libation replaced with battery acid. The things I do for science.

A few weeks after I completed my trial and went on, for certain, the real thing, preliminary results were announced showing clinical efficacy in slowing disease progression. Whoop whoop!!

So what are the takeaways?

First, I think it helps me by giving me more energy throughout the day. Not surprising considering the medication impacts the mitochondria, aka the cellular power plant. Whether it slowed disease progression in my case is hard to say because progression varies over time and from person to person.

Second, this stuff needs to be approved by the FDA as soon as possible. It took my sister the nurse about ten minutes to find the two compounds contained in this medication on Amazon and determine the appropriate dospage. I am hearing anecdotally of pALS availing themselves of this biohacking approach to therapy and expect it to increase as word gets out. pALS deserve a pharmaceutical grade doctor supervised administration of this therapy rather than an online sourced concoction mixed on the kitchen counter.

Drink up!