An N of 1


The most recent roller coaster ride seems to be coming to an end, though that sounds remarkably like “famous last words” – never good!

For those keeping score, here’s the update:

About four months ago I started on my third-line treatment – everolimus and exemestane. I started the treatment with trepidation. After nearly three months it seemed, to me, that I wasn’t seeing the results I wanted. Tumor markers were rising (that’s not good) and the side effects weren’t great. So I started looking for “what’s next.” Time to look at my options – clinical trials.

Quick overview of trials for the uninitiated:

Phase I trials are generally about establishing a safe dose of an experimental medication. Side effects are also identified in Phase I, and they are usually conducted on a small group of patients, increasing the dose with every few patients until unacceptable side effect or the maximum expected dosage is reached.

Phase II trials look at effectiveness and safety issues. These trials are conducted on a larger group of patients.

Phase III trials are conducted with still larger patient pools to confirm effectiveness, monitor side effect, and collect the information necessary to seek FDA approval. Generally there are “gold standard” randomized control trials – half the patients receive the trial drug or procedure, the other half receives the “standard of care.”

And new to me, Phase I Expansion trials take drugs that have completed escalation and reached an established dose will often add additional patients, perhaps with other cancers than the one underlying the trial.

Why does this matter? In this world of targeted therapies and personalized medicine it seems like studies are becoming increasingly specific, with ever-narrower foci. Finding trials we fit into can be a challenge when you consider breast cancer subtypes, exclusion based on the drugs we’ve taken, and the one’s we haven’t, and then there are specific targets. And for me, perhaps even more importantly, if I’m going into a trial – I want the goods! While Phase III trials have fewer safety concerns, there’s only a 50/50 chance I’d get the trial drugs.

If the current meds are working – and we’re not yet sure – they will, at some point, stop. Of course, there are a number of already-approved therapies, but before I exclude myself from more trials, I wanted to know what else is out there. Of the trials I heard about, these are the two most promising and for which I’m eligible:

Cedars has an anti-PDL1 Phase I expansion. This class of drugs, all still in development, works by turning off a cancer cell receptor that blocks the immune system’s T-cells. The anti-PDL1 renders PDL1 ineffective, allowing the T-cell to return to doing its job in prompting cell death. The “vaccine” trial is not only showing success, but much of it’s breast cancer success seems to be in TNBC (triple negative) patients.

Another option for me is a trial that targets the WNT pathway, where vantictumab is given along with a traditional chemotherapy. While the chemo targets cancer cells, the trial drug targets cancer stem cells. Cancer stem cells are found within the heterogeneous tumor, and since they aren’t killed by chemo they remain to evolve into cancer again, and they are thought to be at the heart of metastasis.


Clinical trials are the foundation of new therapies, a critical step in the approval of any medication. There are risks, but then again there are risks for what I’m taking now. It may not work, but there are no guarantees anything else will either.

That’s my update. I remember when I was a new, uninitiated cancer patient. I was told again and again that each patient is unique – how we respond to treatment, which side effects we will have, how bad those side effects get, and whether our cancer will recur – that couldn’t be predicted. It was hard to imagine, but it is true. Treatment, trials, responses, relapses – we are each an N of 1.

6 Comments (+add yours?)

  1. marcy westerling
    May 27, 2014 @ 09:21:13

    Great, clear explanations. This is the first I have heard the phrase Phase I Expansion trials but it explains my own confusion. I am a participant in cohort four of an ongoing phase one trial. No end in site of the Phase One phase. I know someone who did the exact same protocol in this Phase One seven years ago! They keep tinkering with the “icing” if you will, for example my cohort takes an aspirin each night. I guess I am in a Phase I Expansion trial!

    I am a fan of the PDL1 stuff. Good luck!

    Marcy Westerling


    • Lori
      May 30, 2014 @ 10:34:34

      I, too, had not heard of Phase I Expansion trials – and they may be replacing Phase 2 trials, though it’s too soon to tell. And I agree, the anti-PDL1s are very promising. Everyone is trying to fast-track them. I’ve also heard that they are especially promising in TNBC patients, and they CERTAINLY need something!


  2. Allison Wood
    May 27, 2014 @ 12:01:07

    Good luck, Lori! I’m glad to be aware of what’s going on with you and will keep you in my thoughts for health and wellness. xoxo


  3. Jackie Frager
    May 27, 2014 @ 14:00:20

    I’m on the roller coaster with you my dear friend. Thanks for this very informative overview of how clinical trials work. So interesting, and so glad that you were able to find and qualify for a new trial. I’ll be riding along with you!


    • Lori
      May 30, 2014 @ 10:36:24

      Thank you, Jackie – I do know you’re along for the ride! I just want to be clear that I am NOT currently opting for a trial. I’m sticking with this new but tested and approved combination for now. Unfortunately these trials may be closed by the time it’s my turn, but I’m certain other will be open! XOX


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