Dr. E on the patient voice and thinking outside the pill

Dr. Eleni Efstathiou has a hypothetical question about your high-grade prostate cancer diagnosis that typifies her approach:  You alone can answer it, and you wonder why nobody asked you before.

The straight-talking Dr. E, an AnCan advisory board member and genitourinary oncology section chief at Houston Methodist, shared her views Monday night in a conversation with our High-Risk/Recurrent/Advanced prostate cancer group, which includes several of her patients.

Medicine needs to start thinking outside the pill, she said, tackling not only development of drugs but development of therapies. How should we be deploying drugs and other interventions? What timing, combinations, and sequences will achieve highest benefit and lowest toxicity?

One target for this rethinking is localized or locally advanced prostate cancer.  She’s gotten exceptional long-term results from more than two-thirds of patients by administering 3 to 6 months of ADT plus abiraterone, followed by prostatectomy.

Much of her philosophy is reflected in this work. First, therapeutic thinking led to strong results by putting drugs in a novel setting. Second, because 30% of patients don’t benefit from the treatment, biomarkers are needed to identify them and to develop treatments for them as well. Third, emphasis is needed on early-stage high-grade disease. Fourth, the patient gets a voice in whether to go forward.

She came to Monday’s meeting hoping to hear more of these voices. She asked what our own choices would be: Diagnosed with localized or locally advanced high-grade cancer, would we accept a prostatectomy in hopes of an excellent prognosis without further ADT?

Results were surprising. Half the men in the meeting still would decline the prostatectomy in favor of radiation and long-term hormones.  This opened a path for future discussion — what would make prostatectomy a stronger alternative?

In the 90-minute session, Dr. E also stressed the importance of using real-world evidence in addition to randomized clinical trials where results come slowly and have narrow applicability. She described the work leading to the development of second-generation antiandrogens.

And she hinted that groundbreaking news would be coming from the GU ASCO conference in February.

An edited transcript of the talk, with slides, is available.