Good opinion piece on targeted therapy

Fallleaves

From "No Bull's Eye Without a Target" by Dr. Mark Kris, http://www.medscape.com/viewarticle/821575

Before we could find a target, or a way of selecting patients who would benefit from a treatment on the basis of a specific molecular aberration in their tumor, every patient received the same therapy. A few patients derived benefit, but we saw even more patients who either failed to benefit or simply suffered, and blessedly for many of the targets that we have, that day is now over. It is very important that we focus on changing our practice to make sure that we take the greatest advantages of targeted therapy to make those targeted therapy drugs available to the patients who can benefit as quickly as possible, as close to diagnosis as possible, and conversely spare patients who don't have the target from those drugs.

An example is the recent American Society of Clinical Oncology's second "top 5 list"^[1] They were asked to make a top 5 list previously^[2] when the Institute of Medicine came out with ways to cut down on waste and inefficiencies in the medical system, but they have done it again, and I want to highlight number 5 on the list:

"Do not use a targeted therapy intended for use against a specific genetic aberration unless a patient's tumor cells have a specific biomarker that predicts an effective response to the targeted therapy."

That reflects an understanding over the past decade that patients with an EGFR-mutant lung cancer will receive significant benefit, but those without a mutation will not. In the IPASS data,^[3] there was a 70% response rate in patients who "fit the profile" with an EGFR mutation, but a 1% chance of benefit in patients who did not have a mutation but had that same clinical profile....

It has been a great 10 years, but we have learned an important lesson: Targeted therapies only work if you have the target, and despite the fact that these drugs are available and approved, we must pay attention to the molecular profile of each patient's tumor. We must treat as precisely as possible to get the best results for our patients.