Ashish Kamat: Hello and welcome to UroToday's Bladder Cancer Center of Excellence. I'm Ashish Kamat from MD Anderson Cancer Center in Houston, Texas and it's my pleasure to welcome today Karen Sachse who's a patient advocate for the Bladder Cancer Advocacy Network and who is here today to share with us her perspective from a patient perspective on nonmuscle-invasive bladder cancer, the various trials that are ongoing and the future of the different studies. So, Karen, the stage is yours.

Karen Sachse: Thank you. Thank you for the opportunity to be here. I was diagnosed in 2010 at age 54 with high-grade nonmuscle-invasive bladder cancer. Well, like many women, my symptoms had persisted for two years and the thinking was that they were gynecologic in their nature. I had frequent urinary tract infections and discomfort upon urination. My bladder tumor was discovered on an intervaginal sonogram and I was referred to a community urologist who removed my papillary tumor and treated me with 12 doses of BCG. When I still had persistent disease six months later, I transferred my care to an NCI designated bladder cancer center, which was about an hour and a half away. The cancer had spread to the lower third of one of my ureters, and so they inserted a stent and I received eight doses of intravesicular mitomycin-C. Since then I've had three small recurrences, all treated with one dose of chemotherapy, and I've been disease-free since 2014.

I have been a patient advocate for BCAN for the last four years, I'm involved in several pharmaceutical projects and on two study advisory panels. Last year, I spoke at BCAN's Think Tank sharing my story about being a patient and then also a caregiver. This year, I participated in the Virtual Think Tank, in the panel following presentations by clinicians and researchers. So what I wanted to bring to peoples' awareness is the opportunities of how patients can get involved, and these are efforts specifically related to nonmuscle-invasive bladder cancer.

So the first opportunity is something called the CISTO study. It's comparing intravesical therapy and surgery as treatment options. It's an 8.5 million project funded by the Patient-Centered Outcomes Research Institute and the principal investigators are Dr. John Gore and Dr. Angela Smith. There is an eight-member patient advisory board, of which I'm a member. There are 28 sites, nine community-based and 19 university-based, 12 have been enrolled so far. This was as of May, I believe there have been a few more enrolled as of today.

It's an observational trial so that the treatment option is selected by the patient in conjunction with their physician. The goal of this study was that there were patients with high-grade nonmuscle-invasive bladder cancer whose disease recurred or did not respond to BCG to compare what are the outcomes if you receive more medication versus a cystectomy. Patient enrollment, there have been 18 out of 900, so it's going to be a very large study. So far, there are eight patients who have chosen surgery, and 10 patients who have chosen more medication. There is going to be a caregiver component and that will be done as a survey throughout the project. Patients complete a questionnaire upon enrollment in the study and then at designated time points for four years. Patient-reported outcomes will, of course, help further patients make decisions about treatment options.

The next opportunity for patients specifically with nonmuscle-invasive bladder cancer came out of a working group that started last year at the 2019 Think Tank. And clinicians, Dr. Gary Steinberg and Noah Hahn met with patients and caregivers to determine how it was really best to provide input to the FDA to improve clinical trials for this specific type of bladder cancer. Questions involving how patients view trade-offs between risks and side effects and benefits of new therapy. In August of this year, the survey was sent out by email from BCAN getting the perspectives of the clinicians, caregivers, and the patients, and currently, they're in process of tabulating all that data. The patient and caregiver voice is so important. It really helps the clinicians to formulate their study and go in a direction that really makes sense. And that's all I have. Thank you.

Ashish Kamat: Great. Thank you so much, Karen. I mean, that was really good to hear about your perspectives and what's going on from a patient advocate and obviously from a survivor's standpoint when it comes to bladder cancer and BCG and mitomycin and so have you. If I could ask you a couple of questions, because oftentimes when we as clinicians that are designing clinical trials or even pharmaceutical companies that are looking to sponsor these clinical trials in the past have designed such studies, there hasn't been as much input from our patients as I would personally like and I'm really pleased to see that all this is changing. I remember being at the very first Think Tank even before it was the Think Tank, and it was just five of us sitting around at a round table trying to come up with ideas in getting patients involved, and now it's a huge effort, a huge community.

But if I had to ask you from your perspective and feel free to talk about your own personal perspective or what you've gleaned from being part of these different working groups, what are some of the top or I guess the top three things that you would recommend to anyone that's thinking about designing a clinical study in this space?

Karen Sachse: Well, honestly, partnering with BCAN is a great way to go in that they will work with the clinicians and the researchers too. You would then have actually access to a very large patient and caregiver population. So depending on what group or what your clinical trial is targeting, they can certainly help with that.

Ashish Kamat: Great. And when it comes to the CISTO study, for example, the one that Angie Smith and John Gore are leading, the way it's designed is clearly to get a patient perspective as to which are the choices and which pathway someone would like to go down. And again, from your comments, I got the sense that you have been involved with some of the questionnaires. What is your sense as to the validity of those questionnaires when it comes to bladder cancer patients, per se? Because those questionnaires haven't really been developed specifically for bladder cancer patients, but they've been adopted. What is your sense when looking at those questionnaires as to how useful you think they will/will not be and where do you think we should go next?

Karen Sachse: Well, the tools that the questionnaires were adopted from were certainly questionnaires that were validated. I feel like between the eight of us, we have come from different perspectives and in terms of different socioeconomic backgrounds, some of us are women, some of us are men, some of us are caregivers. And I think that the questions are excellent and really get at what is the root of the issues.

Ashish Kamat: Great, great. Okay, good. Yes, because from a provider viewpoint the questionnaires seem to make sense to us but from a patient perspective, I'm glad to hear that you have the same sense as to the questions in the questionnaire. Now, I'm going to ask you a question to not really put you on the spot, but kind of put you on the spot a little bit because during the panel discussion, and I'm sure you are aware that pembrolizumab was obviously approved for the treatment of BCG unresponsive disease and there are two other agents, nadofaragene and pertuzumab that might be approved. So if we assume that all three are approved and available for the patient, there's a lot of discussion amongst investigators about, well, if one's even a therapy, it's systemic, patients don't want an IV.  On the other hand, other people say, "Well, patients don't want intravesical therapy because it's a catheter that has to be placed in the bladder." When I talk to my patients, the responses I get are all over the place.  What is your personal, either bias, and or your sense, being part of the advocacy group, as to, if at all, if patients do have a preference of one mode of administration over the other, or is it purely based on the efficacy of the agent?

Karen Sachse: Well, I think that the efficacy really drives the number one priority and so looking at the outcomes of the clinical trials, I think is going to be important. Having said that, there are also some patients that have had a horrible experience with intravesical therapy and so they would do anything to get away from that and do something else. And I think then it's really kind of individual and maybe what sort of support people have, how frequently the medication needs to be delivered, how does that work with someone's lifestyle, getting to the appointments, et cetera.

Ashish Kamat: And I agree with you there, it clearly has to be individualized for each patient, not everybody's the same. But I am glad you said what you did because at least it provides a counterargument to the groups that say, "Well, patients are never going to do A, B or C." There's never a never, each person is an individual and the socioeconomic status and access to appointments and all of that makes a huge, huge difference. In closing, Karen, if I could ask you to share with our listening audience, which is obviously made up of physicians, nurses, mid-level providers, but also patients listening to this video and podcast that we do, what would be some of the closing thoughts you want to leave our audience with?

Karen Sachse: Well, as far as patients, I say, you really need to get involved in order to make a difference. And certainly, in my experience, I have gained a tremendous, not only just knowledge but camaraderie and through BCAN, I met women that had bladder cancer that I had never met before and I have developed some really amazing friendships and support because of it. Thank you.

Ashish Kamat: Thank you very much. And these are trying times for everybody, and most of all our patients with bladder cancer who tend to be older and sometimes can't access health care systems, and having someone like yourself share your experience and also sharing your thoughts is really beneficial to the community at large. So I do want to thank you once again. Stay safe and stay well.

Karen Sachse: You too. Thanks for the opportunity.