The appropriate use of focal therapy in the treatment of low-risk and perhaps intermediate-risk prostate cancer is a very controversial topic (despite the heavy promotion of this type of treatment by a relatively small number of clinicians).
In a new article in the Journal of Clinical Oncology, Giannarini et al. have laid out the arguments for why the value of focal therapy is unproven to date. The full text of this article is available on line and is important reading for any prostate cancer advocate or support group leader (whether you agree with the authors or not).
The authors’ basic premise is that we have very limited data to support the premise that focal therapy is clinically effective by comparison with simpler (and less invasive) expectant management for low-risk disease. We certainly have insufficient data to support the idea that focal therapy is effective in the management of intermediate-risk disease.
It is absolutely the case that there is a huge range of unanswered questions about the efficacy, safety, and long-term benefit (or lack of benefit) of focal therapy for localized prostate cancer — regardless of the type of focal therapy used (e.g., cryotherapy, laser ablation, high-intensity focused ultasound [HIFU], and others). Never one to hide his opinions under a bushel, Dr. Gerald Chodak has already commented on this issue on his blog site on Medscape, where he states that:
The only way we will ever know whether this therapy has merit is to conduct a randomized trial. Case series simply do not allow us to evaluate the safety and efficacy of focal treatment because many patients may have a cancer that simply is not life-threatening. To use focal therapy and report that patients did well in the long term is really not evidence that this is a justifiable treatment.
In my opinion, only IRB-approved protocols should be used to treat patients. Men certainly should not be charged for this experimental therapy, and really, without a randomized trial, we will never know whether focal therapy works in the long term.
We have considerable sympathy with the perspective of Giannarini et al. and Chodak: without well structured clinical trials, we are never going to know whether focal therapy is any better than expectant management for men with low-risk disease — even though the idea of less invasive treatment may sound good to the layman. It is time for the leadership in the urology community (and the radiation therapy community) to make a stand and start to insist on proper clinical trials of new types of surgical, radiotherapeutic, and other treatments for localized prostate cancer. Just because this didn’t happen in the past is no excuse for it not happening now.
At the other end of the scale, there are always going to be those patients who will willingly pay to have types of treatment that are unproven because they think that such treatments may be more beneficial than the standard of care. Such patients are most certainly entitled to make such decisions, but it is hard to see why insurance companies or Medicare should be covering the costs of focal therapy here in the USA without much better data to support the value. And if appropriate clinical trials were put in place (even if it was just a high quality registry trial with a centralized pathology system) then that would offer another mechanism to generate the data we need to be able to place a value on focal therapy compared to other forms of management in highly-defined sets of patients.