I talked to yet ANOTHER person from Karmanos today (so far, I’ve collected no less than six phone numbers from people involved in my case) and he told me that I’ll know next week which cohort of the trial I will be enrolled in. It’s taking longer than normal to get concrete answers about a trial spot because the sponsor (drug company) and investigators (lead oncologists at the cancer centers that are running the trial) need to have a conference call to determine whether the next cohort will be a dose escalation or dose expansion phase. Here’s a quick lesson on Phase 1 trials (to the best of my understanding).
Phase 1 trials are designed to do two things in particular: First, to find the maximum tolerated dose level; second, to identify potential side effects and adverse reactions (called ‘events’). There are usually two parts to a Phase 1 trial—dose escalation and dose expansion. With dose escalation, they start at a low dose of the drug, and with each cohort (new group of people enrolled in the trial), increase the dose until it is at the highest level tolerate by the patients. Once the maximum tolerated dose is established, the trial moves on to dose expansion, which means the size of the cohort increases. (Note: this is an explanation for a Phase 1 trial using a single agent—with multi-agent trials that use two or more drugs, dose escalation is a bit more complicated, and it doesn’t apply right now, so I won’t get into it.)
If the sponsor and investigators decide that the trial should continue in dose escalation, I won’t have a spot in the trial until the next cohort is enrolled in early April (which would likely mean starting treatment in mid-April). If they decide to move into dose expansion, the cohort will increase and I will get a spot in mid- to late-March. Currently, three patients are enrolled for each cohort; once dose expansion is established, this will likely increase to six patients in each cohort. A new cohort is generally enrolled every three to four weeks. If the sponsor decides to continue with dose escalation, I could still enter the trial sooner if one of the three people entering the next cohort fails the qualifying physical examinations.
However it plays out, it’s good news (for more reasons than simply being accepted to a trial). It’s preferable to be enrolled in a Phase 1 dose expansion rather than a Phase 1 dose escalation, as there is generally indication of therapeutic value of the drug by this point, and you have a much better idea potential side effects. If you are enrolled in dose escalation and are in an early cohort, chances are that the dose will be too low to be therapeutic. Basically, you can have the right drug but at the wrong dose level (which is what ultimately ended up happening to me on the MEK inhibitor trial once my dose was reduced—it was the right drug, but the wrong dose). If the sponsor is considering moving to dose expansion, I know that they must be close to finding the ideal dose, which means I shouldn’t run the risk of the trial failing because the dose level isn’t high enough. And if they decide this next cohort will be dose escalation, it’s likely that the cohort after that will be the expansion cohort. Either way, I should be starting the trial at the ideal (as far as anyone knows at this point) dose level.
The sponsor and investigators are having their conference call on March 7th or 8th, so I should know more about which cohort I’ll be enrolled in next week. Of course, there is always the chance that something will come up in my screening exam that could preclude me from the study, but it’s unlikely that will happen. In the meantime, I have a CT scan this week and an appointment with Dr. Siu to go over the results next Monday, so I’ll find out how my cancer is doing on its own, if it’s relatively stable or if it’s progressed since I stopped weekly chemo. I’m actually pretty interested to find out. I was on hormone-blockers for almost a month, and it’ll be interesting to see if they did anything. Tumor regression is unlikely with the hormone-blockers, but not an impossibility.
In fundraising news, I’m at 67 percent of my goal (which puts me at just over $33,515). We’ve got some fundraising activities in the works, which I’ll write more about as the details are defined. In the meantime, you can donate here if you are so inclined. Several people have asked if offline donations are an option, and they are. Contact me if you would like to arrange something. Donations that aren’t made through the site are still added to the total earned, and are reflected in the amount raised on the GoFundMe site.
It’s actually happening, guys. It’s still a ways away yet, but it’s happening.