6/27/2016 - Answers to FAQ about the Trial

Hello there!  Here's some answers to some FAQ I've been asked related to the trial, along with an update on side effects.

What is a Clinical Trial?: I've been asked this question a few times so here's a quick analogy.  Let's say you're Microsoft, and want to release a new product.  Before you do so, you spend lots of time doing research and development, and get to a reasonably complete project and need user input to move forward.  

At this point, you want to test it on as many users as you can to get their feedback, to see what works, and what crashes.  To make sure you get enough samples of various users and their machines, you tweak the drug, learn more, and adjust accordingly.  After enough tests and thousands of people's input, and associated tweaks, you get approval from your boss to release the product.

In my situation, relating to the analogy above, the drug company is Microsoft, and the new product is the drug I am taking.  They have spent tons of money on R&D, and now need to test it on a bunch of people to see how it affects us, both positively and negatively.  They require several thousand people to test it and years of information before seeking FDA approval, which is the final gate to revenue.  There are several phases of the trial, and I happen to be in the very first phase, and one of the first humans the drug has been tested on (first 40).

Drug Information: The trial drug I am on is called INCB-054329, and is an oral pill I take twice a day (20mg), for 21 days.  For the first cycle, there are two long days where they take as many as 10 blood draws at many points during the day, along with 3 EKGs and urine tests to see how the drug is affecting me.  I also have many appointments with my doctor and nurse practitioner so they can document the side effects and how this drug has made me feel so far.  After the first 21 days, if the drug is not toxic to me, we'll increase the dose for another 21 days, and repeat until 9 weeks has passed.

As mentioned previously, the drug alters my genes, targeting a set of amino acids to prevent a specific protein with a variance of unknown significance (BRD2 or BRD3 in my case) from replicating. I think that's what she said, but don't quote me on that.

What is also interesting is that the bottle that contains the drug warning has the usual "Caution: anti-neoplastic material-handle properly", but also has another warning that says "CAUTION: New Drug Limited by Federal (or United States) law to investigational use".  I also have to turn in the bottles when they are empty because I assume they want to see if the bottle breaks down, or if the reside does anything interesting.

Side Effects So Far: I've been taking the drug for about 1.5 weeks, and am happy to say that the side effects are minimal.  I have a bit of fatigue, and nausea when taking the pills, but so far that's it.  Hopefully it stays that way throughout the trial.

Is it working?: At that point I'm not sure, and we won't know until the end of the trial.  We did a baseline CT scan and blood draw prior to starting the drug, and the final report card will be the CT scan at the conclusion of the 9 weeks to see how the tumors have reacted.  Until then, we'll monitor my blood, EKG, and urine to make sure everything's good so we don't need to end it early, but the drug needs time to work, so we'll give it the proper 9 weeks.  At that time, if we determine the trial is working, I'll continue the drug, and if not, I'll seek another clinical trial and try that out.

Thanks for reading, and I hope you have a great July and a great summer wherever you are in the world!

- Jerome