Notes from NCI GUSC Meetings at 2020 GU ASCO

Yesterday I attended several meetings sponsored by NIH's National Cancer Institute (NCI) in conjunction with the GU ASCO in San Francisco. Here are my notes:

Genitourinary Steering Committee (GUSC) Session

Discussion of “platform trials” in GU cancers by screening by biomarker and stratifying therapy based upon classifications. A platform trial is single overarching protocol developed to evaluate multiple hypotheses. FDA defines a master trial as“[A trial designed to] study multiple targeted therapies in the context of a single disease in a perpetual manner, with therapies allowed to enter or leave the platform on the basis of a decision algorithm”. Siden, et al., Reportingof master protocols towards a standardized approach: A systematic review, Contemp Clin Trials Commun. 2019 Sep; 15: 100406, quoting FDA Draft Guidance on Efficient Clinical Trial Designs (2017)). It’s similar to the UK’s STAMPEDE trial. Gilson, et.al, Incorporating Biomarker Stratification intoSTAMPEDE: an Adaptive Multi-arm, Multi-stage Trial Platform, Clin Oncol (R Coll Radiol). 2017 Dec; 29(12): 778–786. At the meeting, some researchers think platform trials are an exciting opportunity, but other researchers say those type of trials are very complex to implement and run. The discussion was inconclusive.

We also reviewed the recent and forthcoming clinical trials for the GU cancers: bladder, prostate, and kidney.

Bladder Task Force Session

Jason Efstathiou: We want to increase the visibility of patient advocacy. At a future meeting, maybe they’ll have a presentation from the patient advocates, and a checklist of things to consider from a patient advocate standpoint when designing clinical trials.

Jason also reviewed of chart of bladder bancer clinical trial concepts evaluated by GUSC. Generally, we’ve been successful in bringing trials forward. We’ve had some more recent trials that have had problems with accrual, and we’re trying to understand why. In looking at the chart of trials for all BC cancer types, virtually all types have pending or planned trials. There is some overlap, especially on the trimodal side.

Matt Milowsky: Current clinical trials planning meeting (CTPM) priorities: NCI wants to focus on biomarkers in NMIBC (where most patients are at). The majority of our trials are NMIBC. Do we stick with that? (Note: COXEN and CALGB 90601 are mets trials). I observed that BC has a high mutation burden with lots of biomarkers, and how in 2012 Dr. Apolo told me that they would identify the mutations, but didn’t know what to do about them. I said that my impression was that not a lot had changed in the past 8 years. I asked whether it’s even reasonable to tease out and test single biomarkers with BC, which typically has dozens of mutations (unlike most prostate cancers, for example). This triggered a good discussion on unmet needs, especially on HG NMIBC that is unresponsive to BCG. Bill Shipley pointed out how the median age of BC is (about age 73) among the oldest of common cancers. It would be great to develop therapies that don’t necessarily require RC, given the harsh recovery time. We wrapped up with a goal of sharpening the CTPM goals and providing greater communication and coordination between research groups.

Before and after the meetings, I spoke with several committee members, including Jason and Matt, co-chairs, and other committee members. I will be following up with to see how I can sharpen the role of patient advocates on the committee. I also spoke with Andrea Apolo, the NIH doctor who has been following my case since April 2012. It's been a couple of years since we had seen each other in person, and was delighted to see that I was not dead yet. I thanked her for her continuing role in keeping me alive, and looked forward to many more years of pleasant surprises. 

My next infusion in next week. I'll update my status then.