Targeted Cancer Therapies - A Peek Behind the Curtain

Lions, and Tigers and Bears! Oh my! It took Dorothy courage to look behind the curtain in the city of Oz. She found the Wizard.

I recently took a peek behind the curtain. For me, more so than courage, it was curiosity and a desire to make a personal contribution which prompted me to attend the June 2011 American Society of Clinical Oncology (ASCO) conference, Patients Pathways Progress. This is the premier annual oncology conference in the world. It attracted 30,000 oncology professionals. There were hundreds of sessions ranging from the kickoff of a large keynote address to meetings with individual researchers who were peer-selected to display their ground breaking research.

I expected to find a few presentations on the biology of cancer and how our growing knowledge of genetic science would lead to targeted cancer therapies. As with Dorothy in The Wizard of Oz, what I found behind the curtain surprised me. Unlike Dorothy, I found a set of true wizards comprised of oncologists, molecular biologists, epidemiologists, and statisticians who were willing to share their latest breakthroughs in the hopes of collectively and collaboratively finding cures to cancers.

Out of the dozens of presentations in which I participated, my personal favorite was the one delivered by Apostolia M. Tsimberidou, MD, PhD, titled Personalized Medicine in a Phase I Clinical Trials Program: The M. D. Anderson Cancer Center Initiative. The hypothesis of her study is, “Genetics and molecular analyses of patients’ cancers will identify biomarkers for targeted therapies that will improve clinical outcomes.” A phase I non-randomized trial within M. D. Anderson’s Investigational Cancer Therapeutics department was begun in 2007. The trial used tissue from patients referred for treatment to this trial to identify genetic mutations. If a genetic mutation was found and a drug regimen was available that targeted that specific mutation, the patient was enrolled in this phase I trial. Of the 1,144 patients whose tissue sample was analyzed for molecular aberrations, 40.2% were identified with one or more cancer-suspected genetic defects. This patient set was then placed on either a “Matched Therapy” or a “Therapy without Matching” regimen. Here’s the bottom line. The “matched therapy” group showed a 27% positive response rate whereas the “therapy without matching” group showed a 5% positive response rate. The conclusion, based on rigorous statistical methods, was that the matched therapy group faired far better than the non-matched group. By my way of thinking, more than 5 times better. The studies long term goals were twofold. First, over the next five year they hope to develop the capability to test all of M. D. Anderson’s 30,000 new patients a year for any genetic/molecular aberration in order to assign targeted therapy. Second, to perform research that proves efficacy so that the tests are reimbursed and become the standard of care.

This one presentation was one of hundreds during this five day conference focused on genetics and targeted therapies. This is what elevates my hope for a cure. Although targeted therapies are a relatively nascent science, the acceleration of discovery is shifting into high gear. As Dorothy said to Toto upon her arrival in Oz, “We’re not in Kansas anymore.”