An open letter to Larry ‘ Let’s Muzzle the Cancer Patients’ Husten

Larry Husten writes CardioBrief for Commenting on the recent FDA Avastin hearings,  he declared that he is “sick and tired of the excessive role given to patients in news stories about health and medical issues… Nearly every story included respectful, uncritical and often fawning coverage of the patients who spoke at the hearing … They all said the same things: Avastin saved my life. I wouldn’t be here without Avastin. I demand the right to choose Avastin.”

Husten said good journalists would ask hard questions such as: What is the evidence for your claim? How do you know the drug saved your life?

He praised a MedPage journalist for ” bravely tell[ing] the harrowing tale about how during the FDA hearing the fanatical self-appointed patient advocates bullied and harassed FDA officials and anyone else with a different viewpoint.”

Husten concludes: “When reporters cater to these type of people they not only foster fuzzy thinking, they encourage a mob mentality that tears down any semblance of rationality or any possibility of intelligent discourse. Medicine, of course, is all about the patient. But that doesn’t mean that every patient is right, or deserves a public voice, or that uncritical journalists should assist them in metastasizing their views.”

Dear Mr. Husten,

On behalf of the estimated 155,000 U.S. women currently living with metastatic breast cancer, I would like to apologize. How  dare these women disturb the cathedral-like confines of an FDA hearing? The nerve of these hussies! With so many of them being young mothers, I am appalled that this is the example they are setting for their children.

I am so sorry.

Ladies: Please remember, when you go to an FDA hearing, you should treat it  like like the “March of the Siamese Children” in “The King and I.”  Your are not to turn your back on Richard  Pazdur, head of FDA’s cancer drug division . Since he is The Man, however, it is his right to turn his back on you, as he did for virtually the entire hearing.

Some of you have never been to an FDA hearing. Let me remind you that reverent silence should prevail. It’s no different than being in a library, church or a cemetery. And, since we all know that in 2011 that metastatic breast cancer is incurable, of course we all want to ensure our families won’t disgrace us at our funeral services or grave side ceremonies with unseemly emotional outbursts. Practice makes perfect! Yes, my sisters, this FDA hearing presents a teachable moment. Carpe diem!

You are so right about that mob mentality, Larry. You know, a lot of these cancer ladies drive mini vans. And those vans are a rolling armory  of potential weapons: Wet Wipes, Harry Potter books and smelly soccer shin guards are all within arm’s reach. There’s no telling what these “fanatics” as you so aptly put it, might do. (Provided they are having a good day.)

I am so ashamed about bullying Pazdur experienced. The poor man, it seems to happen to him a lot. Remember in the fall of 2007? “Dozens of protestors gathered outside the FDA’s headquarters to complain about the agency’s handling of cancer medicines,” according to Ed Silverman.  “Two prostate-cancer patients blamed the agency for rejecting the Dendreon vacccine known as Provenge, which they said helped keep them alive. Later, the group broke into a chant: ‘Pazdur Must Go! Pazdur Must Go!'”

The meanies. And then the cancer girls had to go pick on him, too. I hope Mrs. Pazdur fixed Dickie a nice hot mug of Ovaltine after his harrowing day.

You are so right to suggest muzzling patients like those who  testified at the Avastin hearing. We, the people with metastatic breast cancer should stay strong, be positive and defer to medical and government officials who know what is best for us and can be relied upon to act in our best interests. Remember  Rose Kushner?

Per Wikipedia, in 1974,  Kushner objected to the  then standard-treatment, in which a tumor biopsy and radical mastectomy were performed in a single surgical operation while the patient was under anesthesia. She resisted the then-standard radical mastectomy procedure in favor of a less invasive procedure.

She traveled to Europe to learn about breast cancer treatment there, finding that the radical mastectomy was not used as widely as in the United States. In 1975 she wrote a book: Breast Cancer: A Personal History and Investigative Report. It was widely criticized by other doctors and the American Cancer Society.

Kusher attended numerous meetings of medical professionals, interrupting presentations, questioning conclusions, and speaking against the prevalent practices of one-step breast cancer surgery and radical mastectomy. In 1975 she was “booed off the stage” at a meeting of the Society of Surgical Oncology, whose members objected to her challenges to traditional treatments.[3]

In spite of her unpopularity with the mainstream medical profession, Kushner’s work was well-received in the public and won increasing respect in official circles. In June 1977, she was the only lay member appointed to a ten-member National Institutes of Health (NIH) panel that evaluated treatment options for primary breast cancer. In 1979, the panel issued its findings, concluding that the Halsted radical mastectomy should no longer be the standard treatment for suspected cases of breast cancer, instead recommending total simple mastectomy as the primary surgical treatment.[1][4][5] Additionally, Kushner convinced her fellow panel members to include a statement calling for an end to the one-step surgical procedure.[4] At the time of her death, Dr. Bruce A. Chabner of the National Cancer Institute said she was “probably the single most important person” in ending the practice of one-step surgery for breast cancer, because of her persistence and because she brought medical information to a wide public audience that otherwise might have remained unaware of the options.[3]

I was learning my multiplication tables during the peak of these advocacy efforts. But clearly, this Kushner woman was some kind of lunatic. The “fanatical” patients at the FDA hearing must be just as nefarious as anyone who would dare to suggest mastectomies didn’t have to be painful, disfiguring and potentially disabling.

Larry, I am going to go out on a limb (so to speak) and assume your boobs remain intact. Well, I am missing “Lefty,” and man, I am so pissed off  that thanks to busybodies like Rose Kushner that I couldn’t have a good old-fashioned hack job. I mean if it was good enough for Betty Ford, it’s good enough for me, dammit!

You are also so very right about the fawning treatment accorded metastatic breast cancer patients and related developments. In 1999, for example, Business Week gushed about  Herceptin:

“The results mark the first time in 40 years that any treatment has improved the survival rate of women with breast cancer that has metastasized...the risk of death for women who took Herceptin in the late stages of breast cancer was 22.5% lower than for women who were treated with standard chemotherapy.

“In real terms, this difference may not seem like much — women on Herceptin had a median survival rate of 24.8 months after treatment, vs. 20.8 months on standard treatment. However, metastatic breast cancer is almost always fatal, so any improvement in survival rates is a cause for rejoicing.

Some of these nutty cancer broads actually got their doctors to testify, too. What’s up with that? According to TIME:

Beth DuPree, a Pennsylvania breast cancer doctor who as a surgeon does not prescribe Avastin but does have patients who are on it, mocked the supposed scariness of the 1% chance of death from the drug — for use by women with a 100% chance of dying of Stage 4 breast cancer. “It sounds bad to say,” she told [the TIME reporter] outside the hearing, “but if you take the drug and it doesn’t work for you, you die right away,” not because of the drug, but because of the cancer.

DuPree said she has had patients get a couple of extra years of life as a result of Avastin since it was provisionally approved — pending further clinical-trial data — for patients with late-stage breast cancer in 2008. Since then, Roche has failed to prove that it sufficiently extends or improves the quality of life for women with terminal breast cancer. Yet, for some reason, it does seem to help a minority of patients.”I had one woman who got to watch her child go from 12 to 14″ because of Avastin, DuPree said. Is that a big deal? If you’re that woman or her child, of course, that’s not even a serious question… And if you happen to be one of the roughly 17,500 patients in the United States for whom the drug is working, you don’t really want to hear that your experience is statistically insignificant..Can 17,500 women really be anecdotal?

Again Larry, I am so sorry you were subjected to these patients’ presence and their trivial concerns.

We, the people with metastatic breast cancer, account for 90 percent of the deaths from breast cancer, yet metastatic breast cancer receives less than 3 percent of all research funding. Every year, 45,000 of us people with metastatic breast cancer die.  Obviously our silence is imperative to preserving this awesome track record.


Katherine O’Brien

P.S. I see that you drove a taxicab in New York City before embarking on a career in medical journalism. I can only surmise once a hack, always a hack!

25 thoughts on “An open letter to Larry ‘ Let’s Muzzle the Cancer Patients’ Husten

  1. Kathi says:

    Oh, Katherine, I would laugh except that this makes me want to cry. When I recover a little from my extreme frustration with this entire subject of the pitiful amount of research directed at metastatic breast cancer, perhaps I may be able to say something more cogent. But then again, perhaps that’s utterly beside the point.

    Mostly, I just wish we were in the same room so I could give you a hug.

  2. Raymond Prince says:

    Your letter to Larry is well done. Health care is an evolving science and our minds need to be kept open.
    PS: I never drove a cab

  3. Debbie says:

    Katherine, your words are priceless. So many people do not understand what we go through, living with metastatic breast cancer. Every treatment is a crap shoot and you are grateful for any time you get without tumor progression; even if it is just 2 months. To some women, those months may be the difference of seeing their child graduate from high school or college, or seeing their first grandchild. How many women out there that might respond to Avastin, will never get the chance?

  4. Leslie says:

    I would love to read this self appointed judge and jury’s response to your letter. It was full of wit and information. I love the way you addressed each of his point with previously published articles, Names of well known and respected Oncologists, as well as tongue in cheek “support and sympathy” for the poor big baby FDA pane,l that the woman who have their life clock ticking in their ears, harassed. In my opinion, you deserve more respect for the one letter you wrote than Larry or Mr. Pazdur deserve for their yellow journalism or being a cog in the FDA’s machinery. You rock!

  5. katherinembc says:

    Advocate Musa Mayer clears states the MBC facts. Expertly annotated. Thank you, Musa!

    Click to access Mayer_NBCC_2011_0.pdf

  6. Musa Mayer says:

    You’re welcome, Katherinembc! You may be surprised to know, however, that advocate Musa Mayer actually supports Christine Brunswick of NBCC, and the few others with the courage to speak out on behalf of evidence-based decision-making at the Avastin hearing. I support FDA’s withdrawal of the Avastin indication for metastatic breast cancer, and I’d like to tell you why, at the risk of being mocked and vilified here.

    While I certainly don’t agree with Larry Husten about muzzling cancer patients, I think it’s crucially important for those who are attempting to influence the process to understand the dynamics of drug development and regulation. This is not just about access. It’s about requiring–no, demanding–good evidence of clinical benefit before letting drug companies market their products.

    Since 1992, Accelerated Approval has brought 49 indications for cancer drugs to patients years soon than full approval would have made possible–only 10% have failed to confirm benefit in subsequent confirmatory trials. All the others except Avastin have been voluntarily withdrawn.

    Ask yourself why Avastin has such marginal results in breast cancer in the clinical trials overall–no increase in overall survival benefit, and a few weeks to a month or two before the disease progresses. Is it that we don’t know how to select those patients who do respond dramatically, the “super-responders” who testified at the Avastin hearing last week? Their meaningful results get diluted to almost nothing in the larger patient population–yet all are exposed to the toxicities and pay the costs. And the company, of course, reaps the benefit.

    What I’m asking you to think about is WHY. Why, with the everexpanding potential of genomic science, has so little progress and research investment in finding predictive biomarkers for Avastin (and other drugs) been made?

    Advocates have been raising the issue of identifying Avastin responders with Genentech ever since 2005 when the first Avastin trail in metastatic breast cancer failed dismally. Their position has always been that there is absolutely no subset of patients who does not respond to Avastin. And now, suddenly threatened with losing the breast cancer indication, in a move clearly driven by lawyers, not scientists, Genentech proposes mounting a new trial to confirm Taxol as the preferred chemotherapy partner, with correlative science to determine responders? And leave Avastin approved while doing so? I don’t think so.

    Where are the incentives for industry to co-develop and validate predictive biomarkers with targeted therapies like Avastin? Think about it. At its height, Avastin was selling over 11 billion dollars a year worldwide, more than any other cancer drug. Why on earth would Roche (owner of Genentech) be interested in developing an assay to determine the fraction of metastatic breast cancer patients who benefit? Why would they want to cut their profits? If they did develop such an assay, wouldn’t they then be pressed to do the same in colorectal, lung, renal and the other cancers for which Avastin is approved?

    The thing is, I don’t expect otherwise from industry. To do so is like expecting lions not to hunt and eat their prey. It’s the nature of the beast. They can always be expected to put shareholders before patients, however skillfully they attempt to convince us otherwise. That means that we, as advocates and educated patients, need to be particularly mindful of the incentives driving drug development and regulation.

    If a drug’s effect is so diluted among a general patient population that we see no overall survival benefit, and only weeks of progression-free survival, in the face of significant toxicity, should that drug really be FDA approved for that entire population, in the name of treatment choice? Is that a wise public health decision? I know that doctors and patients want to have Avastin in the toolbox just in case. I have no doubt that this “I should be able to have/prescribe whatever treatment I want” perspective drives the guidelines, which are after all only partially evidence-based. Much has been made of the rights for doctors and patients to make treatment choices unencumbered by government interference via the FDA. But how useful are these rights, when not informed by evidence?

    Surely the happy marketing message that retention of the Avastin indication in metastatic breast cancer would send to the rest of pharma is that it’s not necessary to do the expensive and demanding scientific work to identify responders. The bar would be lowered. We’ve sent the message to pharma that one size fits all is fine. A host of mediocre but costly treatments with minimal benefit would march through the open door of accelerated approval, which will have lost its integrity as a mechanism for getting really promising drugs to market sooner.

    Hence, we can look forward to another depressing decade of subjecting patients with metastatic breast cancer to toxic, expensive drugs for marginal benefits, at best. I’m all for choice, folks, but let’s be really clear about what we are choosing. I’m convinced that we are NEVER going to make real progress in fighting metastatic breast cancer this way. Keep your eyes on the prize!

    For more on how I learned this perspective the hard way, read this:

    And for those interested, our free web course: “Understanding Evidence-Based Healthcare: A Foundation for Action” can be found here:

  7. katherinembc says:

    Hi Musa!

    No risk of vilification here. I read your MBC book and it was huge help to me, as are so many of your articles. I have referred many people to the excellent brain mets site you help develop: www,

    Thank you, for all you do.

    Larry Husten’s blog was downright nasty.

    Your points are excellent.

    Accelerated approval, as I understand it, was driven by AIDS patients. They were desperate. Are we any less so?

    Why doesn’t the official NBCC statement make any mention of “metastatic”? Why does it only refer to women? Why is NBCC only convening a metastatic breast cancer committee next month? If you fault the drug companies, can you fault them any less ?

    At the Project LEAD meeting I attended in FL, the featured oncologist, presenting the SABC highlights, literally said NOTHING about mets. He just said he wasn’t going to get into it. He didn’t explain (possibly because there wasn’t a lot of mets news at SABC). He just skipped right over it.

    There was a brief explanation of the metastatic process.But I honesty think many of the attendees didn’t grasp that most people who die from breast cancer die from metastatic breast cancer.

    NBCC says it wants to end breast cancer by 2020. Musa, nobody know the statistics better than you. What are we supposed to do until then?

    Realistically, very few of the women with metastatic breast cancer in 2011 will still be here.

    Imagine you are walking over a bridge. A woman has fallen into the water and is yelling for help.

    Would you tell her to tread water for nine years?

    Or would you throw her a rope, even if the rope was frayed and could break and with the sure knowledge that the woman would soon fall back into the water and drown and that at best you were only delaying the inevitable drowning?

    We are drowning, Musa.

    Who will throw us a life preserver? Can we wait that long?

    Are you with us?

  8. Musa Mayer says:

    Katherine, of course I am with you. If I had it in my power to keep all metastatic women alive, would I not do it? Believe me, I understand the urgency…what I don’t understand is what action you are proposing that is going to make a difference.

    Avastin will not be taken from you, whatever the FDA Commissioner decides. Medicare has clearly said they will cover Avastin, and virtually all MBC patients are eligible for SSD and Medicare. Private insurers will come into line, as pressure is brought to bear. But I still strongly believe FDA should not approve it until and unless it can be shown in randomized controlled trials to have actual clinical benefit.

    If you see really Avastin as a “life preserver,” you haven’t talked to the surviving familiy members of women who’ve died after fatal bleeds and perforations. Rare, yes, but so is extended survival on Avastin. Frankly speaking, the vast majority of women taking Avastin as first-line treatment have no way of knowing if it’s the Avastin helping them, or the companion chemotherapy drug, as it is not uncommon for women on Taxol alone to have complete responses and extended periods of NED.

    You may scoff at clinical trials and statistics, in favor of patient testimony, but medicine would be in the dark ages, and we would have no modern treatments at all without trials.

    Women with mets made the same desperate pleas about getting bone marrow transplants back in the 1990’s, and as a result tens of thousands were subjected to a very toxic, costly treatment that was no better than standard chemotherapy, but killed so many. AIDS patients found out within very few years that they paid a huge price in toxicity for early access to the first drugs developed for HIV/AIDS. What made a huge difference in the end for HIV/AIDS research was the discovery of viral load as a short term endpoint for real clinical benefit, meaning that clinical trials of new drugs could be completed within a few months. In breast cancer, we have no such measure.

    As for your attacks on NBCC, may I just point out that the presentation you so admired was at the NBCC Annual Advocacy Training Conference. There is no other organization that would have had such a presentation, nor that would have a Metastasis Prevention Summit. Attack them if you like, but they are not the enemy.

  9. katherinembc says:

    Avastin is not an option for me nor is it ever likely to be. I am stable on tamoxifen. I can’t say what I would do if were the shoes of the Avastin patients. I don’t know.

    I don’t scoff at clinical trials or statistics. I do believe clinical trials are the gold standard.

    And yet what do we tell someone who doesn’t have any alternatives or very few? (Like someone who is triple negative.) I struggle with that.

    I try to keep an open mind.

    Musa, I am asking because I want to understand: Why is Avastin not facing the same problems with the FDA’s European counterpart?

    I am speaking broadly of “life preservers.” I have many options. But what about those who don’t?

    I went to a Y-Me meeting. Do you remember “The Beautiful 8” Roz Kleban’s taped session with eight young women with metastatic breast cancer? Young Survivors Coalition distributes it. Anyhow, I think that DVD was new two years ago when I was diagnosed. At my meeting the other week, they showed “The Beautiful 8.” Afterwards they explained that only one woman is still alive. That has stayed with me.

    I am not attacking NBCC. I just don’t see how they can promote a deadline of 2020 that seemingly makes no allowance for those who don’t have that luxury of time. It makes me feel invisible.

    I did ask those questions of NBCC last Thursday. I e-mailed the contact on the statement. No response. and do have such presentations, altho they are generally more from the vantage point of the metastatic vs. preventing the mets. Also, as you know this fall there will be the First International Advanced Breast Cancer Conference (ABC1):

    Thank you for the articles. They do provide prospective. I will keep reading and learning.

  10. Katherine – I’ve been watching intently this latest round in the Avastin controversy. Whilst I tend to side with what Musa is saying with respect to the science, there’s a couple of points that I’d like to add.

    Firstly, the language in the Forbes article is just disgraceful and disturbing. No matter one’s views on Avastin, the use of such aggressive language is just simply uncalled for.

    Secondly, your points about the “life preservers” are well taken. Honestly, I’m not sure how I would react if one of my current medications (which appears to be doing the job) were potentially no longer available to me, or only with a mortgage of my house or other severe financial penalty.

    In none of the commentary, nor from any of the bc advocacy organizations, have I seen any suggestions as to what the women who are CURRENTLY on Avastin (the so-called “super-responders) are supposed to do going forward. It’s seems to be fine if they’re on Medicare as they’ll be covered, but what about those who are at the mercy of fickle insurance companies? If it’s their INFORMED decision to carry on with the medication on an off-label basis, what is being done to advocate for an arrangement between Genetech and insurance companies to ensure that these women aren’t penalized financially? If Genetech wants to keep studying these women, perhaps with an eye to identifying a particular biomarker as Musa mentions, then are they prepared to also foot the bill?

    Third, you are right. There is most certainly a need to help the women with MBC who are here today. For me, the fact that metastatic breast cancer is even “on the table” now is a great start. But to your point we always need more and I’d also like to be here to ring in the bell at 2020. I wish I could go to the Lisbon conference but I don’t think it’s on the cards right now, but I’ll be watching with avid interest as to what comes out of the NBCC summit, the Lisbon conference, and indeed MBCN’s conference (which I hope to be able to get to). I’m keeping my eyes peeled for action items that we can take up in social media to get the word out and hopefully help get things moving for BC mets. It’s high time.

  11. Hi All,
    Musa, I have enormous respect for your work. But I have less confidence in the accuracy of medical “science” and evidence as presented at meetings and published in oncology journals.

    There is no biomarker for Avastin responsiveness, because it’s based on the blood supply to tumors as opposed to a genetic mutation. Some women’s tumors or their mets may be more vascular than others.

    Thanks, Katherine, for adding to this discussion.

  12. Musa Mayer says:

    Elaine, there are many scientists/researchers would would disagree–iincluding past ASCO president George Sledge. Are you familiar with Bryan Schneider’s work? As I understand it, he is currently validating the findings in this study.

    Schneider BP et al; ECOG 2100. Association of vascular endothelial growth factor and
    vascular endothelial growth factor receptor-2 genetic polymorphisms with outcome
    in a trial of paclitaxel compared with paclitaxel plus bevacizumab in advanced breast
    cancer: ECOG 2100. J Clin Oncol 2008;26(28):4672-8.


    Purpose No biomarkers have been identified to predict outcome with the use of an antiangiogenesis agent for cancer. Vascular endothelial growth factor (VEGF) genetic variability has been associated with altered risk of breast cancer and variable promoter activity. Therefore, we evaluated the association of VEGF genotype with efficacy and toxicity in E2100, a phase III study comparing paclitaxel versus paclitaxel plus bevacizumab as initial chemotherapy for metastatic breast cancer.

    Patients and Methods DNA was extracted from tumor blocks of patients from E2100. Three hundred sixty-three samples were available to evaluate associations between genotype and outcome. Genotyping was performed for selected polymorphisms in VEGF and VEGF receptor 2. Testing for associations between each polymorphism and efficacy and toxicity was performed.

    Results The VEGF-2578 AA genotype was associated with a superior median overall survival (OS) in the combination arm when compared with the alternate genotypes combined (hazard ratio = 0.58; 95% CI, 0.36 to 0.93; P = .023). The VEGF-1154 A allele also demonstrated a superior median OS with an additive effect of each active allele in the combination arm but not the control arm (hazard ratio = 0.62; 95% CI, 0.46 to 0.83; P = .001). Two additional genotypes, VEGF-634 CC and VEGF-1498 TT, were associated with significantly less grade 3 or 4 hypertension in the combination arm when compared with the alternate genotypes combined (P = .005 and P = .022, respectively).

    Conclusion Our data support an association between VEGF genotype and median OS as well as grade 3 or 4 hypertension when using bevacizumab in metastatic breast cancer.

    Full text of this study is available here:

  13. katherinembc says:

    State-of-the-Art Treatment for Triple Negative Breast Cancer:Talking With the Experts

    Click to access State-of-the-Art%20Treatment%20for%20TNBC.pdf

    Dr. Sledge:
    Other agents, including the receptor tyrosine kinase inhibitors such as sorafenib and sunitinib, have been tried. Sunitinib has failed in multiple phase 3 trials so it is not going to be developed any further in metastatic breast cancer. The jury is still out on sorafenib because the trials are not as far along but certainly, the vascular endothelial growth factor (ie, VEGF) story has not been a very promising one.

  14. Kathi says:

    This is precisely the sort of back-and-forth that we need about this subject. Too bad Larry Husten didn’t invite such a discussion, instead of demonstrating the limits of his empathy and intelligence, as he did. It’s a relief to see some intelligent discourse among real stake-holders. We don’t all have to agree, but we can disagree with informed respect.

  15. MJ says:

    Katherine and Musa, both of you make excellent points. I completely fault Genentech for not developing tests to predict who will respond and who will not respond to Avastin. Boo his on them, and boo his on people who nastily mock patient’s participation in the drug development and approval process.

  16. Liz Schulte says:

    Thank you, Musa. I agree wholeheartedly. We need better treatments quickly. It makes much more sense to take a more systematic approach to develop treatments that have greater benefit for larger numbers of patients and that we take the time to learn who benefits before prescribing it to those who won’t.

  17. Musa Mayer says:

    Katherine, I want to try to respond to some of the questions you raised in earlier posts, that I hadn’t had a chance to get to.

    I don’t know why the European Medicines Agency (EMA) made the decision to approve Avastin in MBC for the EU–their proceedings are closed, so it’s anyone’s guess. But I’m not necessarily assuming it’s based solely on evidence. Remember that each of these countries, via their national health programs, have other ways of restricting access. Given the drug’s cost, I’d be surprised to see many national health programs actually paying for Avastin in breast cancer. Avastin is not approved or available through the NHS for use in any cancer for the UK.

    More about NBCC, since you asked. NBCC has always taken policy positions on behalf of ALL women with breast cancer, which does include metastatic patients, after all, as it does all the other subsets: young women, women with IBC, men with breast cancer , HER2+ patients, triple-negative patients, BRCA-positive patients, women with DCIS, and so on. Each of these at one time or another has offered up compelling reasons for NBCC to advocate specifically for them, and some have felt rebuffed as you do. After all, no argument is more compelling than those brought forward by women with mets, who are, as you and many others have pointed out, are the ones who die of the disease.

    Over 20 years, many women who’ve been important to NBCC’s growth have had metastatic disease, and have died of breast cancer. In the early years, the exhibit “Faces of Breast Cancer” displayed photos and stories of 2 women who’d died from each of the 50 states. Nothing was more powerful, or more devastating, than seeing that exhibit in the rotunda of the Senate Office Building. Every NBCC session begins with a moment of silence for a lost advocate. So I think NBCC has always embraced metastatic disease as a central concern, even as it advocates for all women touched by breast cancer. Unlike many organizations who have avoided or pinkwashed the issue, from the beginning, NBCC has told the real truth about breast cancer and welcomed advocates with metastatic disease wholeheartedly into the work that needs to be done.

    Having said that, in my experience, and I speak for myself alone–not representing NBCC–they have steadfastly resisted a single focus on any one group of patients–with the possible exception of getting diagnostic testing and treatment for poor women without insurance. There may be other exceptions, where a wrong can be righted via legislation. NBCC is an umbrella organization, representing all, including many, many organizations that do represent particular groups of patients.

    Advocates are told, as part of their advocacy training: “It’s not about YOUR breast cancer.” Sometimes that is hard to hear. We are asked to take a larger, public health and evidence-based perspective, in the conviction that looking beyond immediate needs to the larger picture is the only way to create meaningful change for all women with breast cancer and at risk of getting breast cancer.

    The 2020 Deadline is a dedicated, intense focus on making the kind of difference in breast cancer we haven’t seen in the three decades of research, to move beyond incrementalism through collaborative effort on key issues. It’s a bold declaration that the status quo is simply not getting us where we need to go. Most say that 2020 is too little time. You say it’s too long a time, that we need to end breast cancer sooner because women are dying now. Would that we could! Would that we already had the drugs to prolong lives that you seem to believe are being withheld.

    The voices of metastatic women are crucial to this effort, in my opinion. Are you with us?

  18. njbookwoman says:

    Wow-wonderful discussion here and a lot to digest and think about. I’m late in responding, since I just got back from a digital free vacation, but i do have a few comments:

    seems like you’re assuming that there will in fact be a cure by 2020 and the only issue is those of us with mets who are left out. I think it’s a big assumption that there will be a cure by 2020–curing the multiple types of breast cancer disease is infinitely more complicated than the engineering feat of going to the moon, to which this campaign is often compared. Even if you argue that the focus should be on a vaccine that prevents mets that is a huge step. Consider AIDS which was relatively quickly identified as being caused by a virus—and still no vaccine. How much more complicated for breast cancer which is really multiple diseases.

    As for Avastin and TN: Genentech trials I believe did track by cancer type and overall TN did not have any better results than ER+ or HER2+. What people fail to realize is that TN is really the “other” category, which just lumps together everyone without the other known receptors. There is so much unknown and many oncologists believe that eventually TN will be identified as several different types of bc.

    As for myself, (sorry, Musa, but in the end it comes down to the personal), I am TN and on Avastin and xeloda and have been stable for 2 years. But, is it because of the xeloda alone or is the avastin a crucial contributor? I still have mets, so I am not one of the miracle outliers whose disease disappeared. I’m not sure if I would be really upset if I lost the avastin. My onc believes in the maxim that you don’t change anything when it appears to be working and that does seem reasonable. But I agree with Musa that we have to hold a high standard. It’s just too common for the newest drug to be hailed as “the cure” and then have it fail. Accelerated approval should work as intended. You have to prove it within the extended time period. And yes, folks, cost does enter into it. There are only so many dollars in the health care pie and if we waste so much money on a drug that doesn’t really work, it’s our loss. You can say how can you put a dollar value on 2 months of someone’s life, but money is not infinite and what you spend one place, you can’t spend elsewhere. So the medical economy imposes choices and always will.

    I can’t thank Katherine enough for her brilliant blog. She’s funny, gifted, and right on target for raising the issues. I love to read every blog!! Musa- I know your name as an advocate and appreciate your bringing light and not just heat to the subject. Thank you both and all who are working for progress with mbc.

  19. Hi Katherine, Musa and everyone else who has commented,
    What a fantastic discussion this is. Still, I hold that, until better drugs come along, which may or may not happen before 2020, Avastin is worthwhile for a small number of patients who tolerate it without serious side effects.

    The lack of biomarkers is unfortunate, but the reality for this drug now and probably indefinitely, because when the drug works it does so, at least in principle, by cutting off the blood supply rather than directly damaging the tumor cells themselves. So if the cells have a mutation, it wouldn’t make a difference, except if that genetic change or marker renders them susceptible to another drug available now or in the pipeline. We’re talking about women who don’t have a response to other drugs, in 2011.

    Musa, as for Bryan Schneider’s work, and predictors of hypertension, I don’t see that as influencing the discussion either way. High blood pressure is usually manageable.

    As far as what “we” learned from the bone marrow transplantation (BMT) experience, and what was found to be not a good treatment in the period around 1990-1993, I agree with you. But that high-dose intense treatment is far more toxic than Avastin, and is an all-or-none kind of treatment. Once you give the treatment for BMT, you can’t reverse it. With Avastin, you can give a dose or two, with a taxane as is the usual, and see how the patient does.

    Several people have brought up costs. My personal opinion is that we should consider costs, and need to limit medical care in the U.S., but that those restrictions shouldn’t be for middle-aged women in their 40s and 50s who can walk, talk and give informed consent.

    Thanks again, Katherine, for initiating and hosting this civil (exemplary!) conversation among women who care about this issue.

  20. Lisa says:

    Ladies-keep up the good work. It is all valuable.

  21. Musa Mayer says:

    Elaine, my comparison with BMT was meant to be indirect, not literal. I meant to refer to what can happen when medical care isn’t based on high-quality evidence, when on a very large scale, patients and doctors use treatments that are either untested in randomized trials, or as in the case of Avastin, where they are tested, but are shown to have only marginal benefit in unselected populations. To lake a less harmful example, this is not really so very different than 20 years ago when most gynecologists were persuaded by observational evidence that their older postmenopausal patients needed to take postmenopausal hormone therapy, i.e. Premarin, to avoid heart disease. These physicians were so sure it helped these women, just as so many oncologists were so sure the BMTs worked for their patients. Now they are sure that Avastin is helping too, clinical trials be damned. They know what they see in the clinic. My point being that clinical judgment can be and has been wrong, as it was then, in these examples.

    But Avastin is clearly different in one respect: we have level one evidence from several well-controlled randomized blinded trials of Avastin’s statisitically significant impact on PFS in first through third line HER2-negative metastatic breast cancer. Taken together, that evidence clearly shows this drug does not prolong life (OS) and that it extends the time to disease progression (PFS) only minimally, by only a month or two. By the same token, Avastin does cause some serious, even fatal, side-effects, and it requires careful monitoring for hypertension and proteinuria, which are common. These side effects are sufficient for the drug to have a “black box” warning on its label. We do not know from any of the trials if quality of life is affected or not by the added toxicity, because there is no data.

    You seem to be agreeing with FDA on one thing–that is, that there are no particular subsets of patients who respond, no “super-responders,” and further you state that there is no possible way of determining who will respond and who will not. So, if the responses are distributed pretty equally across the population of patients who take the drug, could you explain to me what Avastin’s clinical benefit actually is? Why is a brief delay until the cancer progresses worth the added toxicity?

    If a woman has a great response to first-line Avastin + Taxol, how do you as her doctor determine if that response is due to the addition of Avastin? Could it not be due to the Taxol alone? The research on single agent first-line Taxol shows plenty of extended responses and stable disease, doesn’t it? If you then withdraw the Taxol after six or eight cycles, and leave her just on Avastin, how to do you know continued time to progression is not merely a remission following chemotherapy that is not uncommon? In other words, how can you ascribe what you see to Avastin?

    I feel as if there is something missing in my understanding here.

  22. To be clear, I do think there’s a distinct, albeit small, subset of advanced BC patients who respond well to Avastin. The problem is that we can’t predict who those women are or use a biomarker to define them.

    Most cancer drugs are given in combination; teasing out which component of an Avastin + taxane regimen is no more difficult than it is for interpreting data for other drugs. Since you mention it – and this is a divergence of topic- I was one of the minority among doctors who didn’t recommend HRT for most women, because I was skeptical of those data.

    This issue is nothing like that: I’m not advocating Avastin be prescribed for most women, or for most women with advanced BC, all I’m saying is that the drug should be available.

  23. Musa Mayer says:

    I understand that antibodies, like Herceptin, usually need to be given in combination with chemotherapy, but I thought that when it came to more toxic agents, like chemotherapy drugs, sequential monotherapy was in general preferred over combination chemotherapy in the treatment of hormone-receptor negative or refractory HER2-negative MBC–except when there is “visceral crisis.” I know that carboplatin isn’t active when given alone, but most of the women I talk to, unless there’s heavy liver and/or lung involvement, are not on combination chemotherapy.

    Forgive me, Elaine, I’m not trying to be difficult–but I think it’s critically important to think through the implications of what you’re advocating. I really want to understand what standards, if any, do you think are reasonable for FDA approval? To me that’s really the question here.

    Let’s say you were sitting on the Oncologic Drugs Advisory Committee. What would be your position on the approval of a drug that appears to help a few women, but in several large randomized trials in an unselected patient population, the goal of statistical significance is just barely met, on an endpoint whose value to patients is unproven? Should such a drug get an accelerated approval? What kind of evidence, if any, would you require to convert that drug to full approval? What if the drug had rare, but life-threatening toxicities?

    You say you want the option open to prescribe Avastin, because it works in some women. There are dozens of drugs under development that show some activity (i.e., work in a few patients) otherwise they wouldn’t be advancing through the pipeline. Would you want them all in your toolbox, whether or not they meet current FDA standards? In other words, should FDA be looking just for documentation of individual patient benefit when it reviews data from reglistration trials, and not require overall clinical benefit in large randomized studies? FDA is supposed to weigh harm vs. benefit–how would that be possible? Without evidence from randomized trials, how would doctors and patients know how to select unproven treatments in the clinic?

    I think implications and precedent in regulatory action are vitally important.

  24. Hi Musa and everyone else following,
    I’m not sure how it’s best to take this conversation further here. I agree that this is a hard, significant issue for patients and for the FDA.

  25. katherinembc says:

    Phil McCartin & wife Lorraine were featured in this story:

    Today, Phil writes on his FB page:

    So, who should you be more angry at ? A ” scientific ” bureaucracy that is sincerely deluded, buried in piles of stats , and cant look at the ” Living Proof ” on the tv screen ? that thinks it is Saving Stage IV cancer pts from ” bad drugs ” ?… Theyre Stage IV ! Or an advocacy group that has no scientific credibility, is supposed to be helping pts, but undermines their fundamental right to an informed choice, and denies Stage IV TNBC Survivors the benefit of any doubt that some stats dont tell the whole story, when its said that only 20 % of TNBC Survivors live 5 yrs ?? Hmmm , tonight I vote the Stage IV Raspberry, Ethical Meltdown Award to the National Breast Cancer Coalition ! For their taking the time to testify against TNBC Survivors at the Avastin Hearings. Then continue to try and defend the decision w/ their ” scientific ” rationales. None of which carry any real weight in the Stage IV world. Lorraine said it best about this FDA ” I guess they have never had a close personal family mbr or friend w/ Stage IV BC ” Whats NBCC’s excuse ? sure they ll have one , or two, three, Did you know that only about 6 individuals or groups testified against Avastin during the FDA Public Hearings ? I know, I was there. NBCC and BC Action were 2 of the 6 negatives. Over 30 individuals / groups testified in favor of TNBC ‘ers. Breast and stepped up and testified in favor. Komen at least put out a press release. But the minority NBCC got equal press. And BC Action, which went way extreme yrs ago, last yr asked FDA to decline approval of t dm-1. Not based on any scientific facts, just an ” all chemos are poison ” bizarro position. Sad that NBCC would go w/ them.Organizations that did the right thing in the 90’s w/ herceptin , would do this. Must be new “leadership “. Well, thats my venting for tonight. I do try to stay focused on t dm-1 approval .But the avstin issue is linked to ours. I got tired of the NBCC rationalizations Ive been seeing. They should soul-search, and do the right thing . Support Stage IV pts, fight this new FDA ” Overall Survival ” hoop that will kill Stage IV pts !
    Philip McCartin 9:05pm Aug 3

Leave a Reply

Fill in your details below or click an icon to log in: Logo

You are commenting using your account. Log Out /  Change )

Google photo

You are commenting using your Google account. Log Out /  Change )

Twitter picture

You are commenting using your Twitter account. Log Out /  Change )

Facebook photo

You are commenting using your Facebook account. Log Out /  Change )

Connecting to %s

%d bloggers like this: