Today's FDA Avastin Decision

There's a lot of news out there about the FDA's 6 to 0 vote today recommending that Avastin not gain full approval for treating women with metastatic breast cancer. This case has been watched closely by both drug companies and cancer patient advocate groups. The final decision for withdrawing full approval rests with the FDA Commissioner, Margaret Hamberg. According to one news source, that decision will be rendered later this summer.

I've followed this issue closely. I'm not going to comment on the accelerated approval process granted by the FDA which allowed on label use of this drug sooner than later. With some fear of upsetting people who sided against the FDA, based on what I've read and after careful consideration, the FDA panel is making a sound recommendation.

I base this on the assumption, which I believe to be true, that the NCCN (National Comprehensive Cancer Network) Compendium will continue to list Avastin as a treatment option for breast cancer. This compendium is used by many insurance companies and Medicare to determine which treatments will be reimbursed. The panel's recommendation and ultimate decision to remove the breast cancer approval will not prevent doctors from prescribing Avastin to their breast cancer patients. In addition, Avastin is a serious drug with potentially deadly side-effects (internal bleeding). Unfortunately for patients, Genentech, and our hope for a cure, Avastin showed only marginal benefit in two recent studies. There are many, myself included, that for some people Avastin is the answer and our medical community is hard at work to find the bio-markers which show which patients have the greatest chance of success without endangering women who will not benefit.

Today's recommendation is a win for patients, doctors, and the FDA. Patients will continue to have access to this treatment regimen, insurance companies will continue to pay for the treatment, doctors will still be able to prescribe it to patients who they believe it will benefits, and the FDA is protecting the American people against unnecessary risks.

In closing, I credit my medical team who had the foresight to treat me with Avastin (in off-label use for head & neck cancer) with significantly prolonging my life although I can't prove that it was the Avastin that did the trick. I wish the two recent Avastin breast cancer studies on which the FDA panel based their recommendation had shown great benefit for breast cancer patients, but they didn't.

Lastly, I apologize to anyone I may offend for having sided with the FDA on this one. It is not about the money, it is not about death panels, it is not about Obama care; it is about the science.

Take care.

What a H&N Cancer Patient doesn't want to have...

A sore throat. For most people, me included, a sore throat was (past tense) just that and nothing more. We all get them. That was then; now it dredges up a lot of bad memories and negative possibilities. I've been feeling a bit under the weather for about two weeks with frequent migraines, more than the usual fatigue and loss of appetite, and a SORE THROAT. I went to the doctors today for my annual physical (it's actually been a few years). They swabbed my throat and it came back positive for type A strep. I began a 10 day regimen of an antibiotic. In researching this on the Internet, it appears that this is a pretty common illness and that whatever contagion I may have will be gone within 24 hours of beginning my medication. For now, the best cure is a little extra rest.

I can't not wonder how I got this. I realize this is a stretch with possibly scant medical backing. For me, I believe it to be a somewhat compromised immune system from my cancer and all my cancer treatments and a potential side effect of the stress and close people contact associated with traveling (that trip to ASCO 2011 in Chicago two weeks ago). I've mentioned in the past that travelling is a challenge for me, this is one of a number of those challenges... real or imagined.

Take care everyone.

Ed

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.”

"Major Shift in War on Cancer"

This is the headline of an article in today's (June 6 2011) Wall Street Journal regarding the 2011 ASCO (American Society of Clinical Oncologist) conference currently being held in Chicago at the McCormick Place conference center. There are about 25,000 attendees. Although traveling is a challenge for me, I am here as a patient research advocate volunteer and am honored and somewhat humbled to be one of those in attendance.

The WSJ article goes on to say, and I quote...

"New research is signaling a major shift in how cancer drugs are developed and patients are treated—offering the promise of personalized therapies that reach patients faster and are more effective than other medicines.

At the heart of the change: an emerging ability for researchers to use genetic information to match drugs to the biological drivers of tumors in individuals."

Here are some of the highlights from my perspective based on people I met, presentations attended, and the overall conference materials:

• About 30% of the several hundred large presentations are on a deeper understanding of the genetics within many different cancers... head & neck, lung, ovarian, breast, melanoma, etc. In sessions I attended there is a focus on finding combinations of mutant genes which lead to cancer (or a prediction of cancer) versus a prior focus of finding one single gene mutation. Finding these gene combinations is akin to finding a needle in a haystack, but large teams of medical doctors, molecular biologists, and other researchers across cancer institutions and international boundaries are attacking focused research and making progress.  
• I spent an afternoon meeting with drug company representatives. The larger companies like GSK, Abbot Laboratories, Genentech, Pfizer, etc have 1,000 square foot plus exhibition spaces. Most of them have a subset space for discussions on current research staffed by highly knowledgeable researchers and molecular biologists. This resource allocation confirmed for me the conference’s focus on finding cures through deeper genetic understanding.
• About 20% of the exhibition space was dedicated to companies specializing is what appeared to be the most current technologies and biology's available for genetic mutation detection. The cost of testing is falling rapidly. There are three primary testing techniques. Single gene testing costs begin at about one hundred dollars while testing an individual’s entire genome is about ten thousand dollars. Elapse time for gene testing from patient consent to results has been shortened, but is still a 3 to 4 week process.
• Researchers are beginning to test genetic defects across cancer types. This is the paradigm shift I wrote about in my prior blog entry. It is too early to know what fruit this will bear.
• Multi drug cocktails are resulting in evidence-based patient benefits.
• Genetic testing is still part art and part science. Testing methods and processes are being refined and as these refinements are put into practice better data will lead to quicker and more accurate results.
• Genetic mutations are complex. There are missing gene pieces and gene mismatches. Some gene mutations promote tumor grow while other gene mutations affect genes which, when working properly, inhibit tumor growth. The downstream effect of promotion and inhibiting have very different biological effects that can affect many (up to about 100) cell processes. This is one of the factors that make finding a cure, even after finding a mutation, so complex.

It is hard to summarize this annual gathering of the world’s leading oncologists. My takeaway is a true sense of breakthroughs, progress, and an excitement from the participants about winning the war on cancer. For some patients it will come too late; for others it will mean the difference between life and death.

Take care everyone.