You Try Pronouncing Regorafenib and Nivolumab Really Fast

In June an abstract was presented at ASCO from a phase 1 study out of Japan. It combined 2 drugs currently being used in cancer treatment in the US, and the results of the study were promising - promising enough to get the serious attention of GI oncologists and patients. Serious enough that I took screenshots of the presentation slides I saw come across Twitter from those in the Twitterverse that were in attendance. 

The study treated gastric and colorectal cancer patients with microsatellite (MSS) tumors with a combination of Nivolumab and Regorafenib. Regorafenib, or Stivarga as it’s known for people who can’t spell or pronounce fancy drug names like myself, is already in line as standard therapy for colorectal cancer MSS patients. Though approved by the FDA, Nivolumab (Optivo) is not approved for my cancer type (MSS) because to date there has been no clinical data to support that this tumor type would respond to this immunotherapy.

In the study, the response rate in colorectal cancer patients with MSS tumors was 29%. As this was a phase 1 study, the number of patients enrolled was small (50 over all, 25 colorectal). These results were exciting, unexpected, and encouraging enough that there is talk of opening this as a phase III trial in the US. But I ain’t got time to wait for that!

The timing couldn’t have been more perfect, as I was a couple of weeks from starting my first clinical trial with its one-and-done short run of 42 days, and would need to line up my treatment plan after its completion. 

The week ASCO concluded (and the same week these results were presented in the abstract) I traveled down to MD Anderson in Houston to check out their trials. While meeting with the oncologist (and his fellow) about potential phase 1 trials available, he brought up this study. Of course I had the screenshots of the presentation slides on my phone and whipped them out! As he talked, the fellow pulled up the abstract to read, and within minutes we were all in agreement that mimicking this trial was the way to go. Fortunately I have an oncologist who is equally trusting of my ideas who immediately put this plan into action. 

Since these two drugs are in circulation in the US, I could duplicate the study. As Nivolumab was not approved for my tumor type, my insurance company would not cover the cost and I’d have to get it “off-label.” This means asking the drug manufacturer to give me the drug for free. The process wasn’t complicated, and in-house staff at my cancer center took care of it. Within days it was done.

Once I wrap my current trial with a scan on Monday, July 30, I will begin this treatment plan on Friday, August 2. The side effects from Regorafenib can be rough, but ironically the best results of the study came with the lowest dose of the drug. Knowing this, I'm hoping the suffering is kept to a minimum - as the drug is known for destroying hands and feet.

I wouldn’t exactly call this going rouge, as the results from the phase I study have clearly inspired US researchers to actively create their own study. I’m just getting a head start. I know that if I expect to survive this disease or (at the very least) continue to prolong my life - I have to take risks and venture off the paved path. 

Those of us with MSS tumors have been sitting on the sidelines for a while watching everyone else get to successfully play in the game, and a study like this can only be likened to the coach telling us to get off the bench and start warming up. I just hope I can do my fellow MSS patients proud as I get called in early to play.

How Now Mad Cow

I finally pulled the trigger on a trial, and I will admit it wasn't easy.

It was very easy for me to say yes (to that clinical trial dress), but what wasn't easy was finding a trial in a timely manner - or at least timely enough to keep my anxiety from creeping in to every corner of my life. To say it was a weird few months is an understatement, and my head space was clouded over in ways I've never experienced. I found myself withdrawing from the world - not because I was in a depressed state, but because I didn't have an ounce of anything to give as long as my mind was consumed with the reality that I had no treatment options on the table.

It has been nothing short of bizarre to have had a plan for 7+ years, and then all the sudden be left stranded on an island waiting for the phone to ring. All the while knowing cancer was continuing to grow, and the horrid cough I had reminding me daily that things were not improving.

I opted for a phase 1 trial primarily because that's what is available to me and my tumor type. As they say, beggars can't be choosers, and I was at the point of begging. I know most patients cringe at the idea of a phase 1 trial, as it's when the drugs are first tested in humans and the main purpose is to determine proper dosage (meaning how much of the trial drug can be given before side effects/toxicity because an issue).

The catch is the drugs they are studying have been shown to work in a lab, and by "lab" I mean in animals. Are you offering yourself up to be a human guinea pig? Absolutely. But are you also offering yourself up to be the first to get drugs that could work? Absolutely!!!

What I found most surprising once I jumped into the clinical trial pool was how hard it was to actually get into a phase 1 trial - primarily because of space available and the timing in which that space becomes available. Phase 1 trials have limited numbers of patients, and it's often doled out at a "one at a time" rate. Meaning in some cases I couldn't start a trial until the previous participant had completed their run.

Over 3 months after my last round of chemo the stars finally did align, and a trial I signed up for over 2 months prior locked in my spot. Ironically it was a trial I had considered last summer, and opted to pass on due to the option of revisiting a previous therapy. I'm playing the long game here, and I knew going in my goal would be to buy as much time as I could. By "buy time," I don't just mean life, I mean time for trials to progress, data to come in, trial arms to open, and new trials to begin.

In my case, the trial I was considering last summer changed it up a little from when I first considered it. They went from doing an intratumeral injection of the virus (injecting it right into the tumor) to doing an IV infusion of the virus (a drip from the bag) and found they were getting better results from the IV infusion. It's a clear example that "buying time" bought me a potentially better outcome.

The trial itself, one I've taken to calling Mad Cow Disease, is one and done. It is what's called oncolytic virus therapy, and if you click on that hyperlink you can better understand the use of viruses to fight cancer. I was infused with the virus, spent 24 hours in the hospital dealing with magnified flu-like symptoms, and spent the following week trying to get through the day without an all out crashing nap. We'll see if anyone I work with reads this, as I'll admit to putting my head on my desk for a power nap at work while listening to a webinar because I just couldn't go a minute more without face planting on the nearest flat surface. And they thought my door was shut just to filter out the ambient noise.

Roughly 6 weeks after my infusion I'll have a scan to see if it worked, and then I'm free to move about the clinical trial world again. I do have a few spoons in the pot, and something exciting that's brewing that I'm not prepared to talk about until it's actually underway. But the relief I have now is that I at least have options - something I spent 13+ weeks feeling like I didn't have, and that's a very frightening place to be.