New Directions

Dear Readers,

Your author is en route to Chicago to attend the annual meeting of the American Society of Clinical Oncology. It’s the first time in years I’ll be there, and I’m looking forward to it – the next phase of my career, a blend of academics and new writing.

So it seems a fitting time to put this blog on hiatus. Not to worry, as always, I’ll pay attention to the conference proceedings. I’ll take extensive notes on cancer science and drugs. And I’ll see some old friends in the Windy City.

You will hear from me again soon!

To all of my readers, and especially those who have commented here or otherwise connected through Medical Lessons over the years, I say thank you. I’ve learned a lot, and not just about medicine.

Moving forward,


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Palbociclib Appears to Prolong Progression Free Survival in Women with Metastatic Breast Cancer

Yesterday researchers at the annual AACR meeting announced the results of a clinical trial of a new drug with activity in some forms of breast cancer. Palbociclib (PD-0332991), a pill developed by Pfizer, was tested in women with metastatic breast cancer cells with estrogen receptors and lacking Her2. These ER+/Her2- tumors represent the most common breast cancer subtype, which is one reason so many people are eying the results of this relatively small, randomized study.

The phase 2 trial, called PALOMA-1 included 165 post-menopausal women with advanced ER+/Her2 negative disease. The research subjects were assigned to take either Letrazole (Femara, an aromatase inhibitor, a drug that inhibits estrogen synthesis) alone, or Letrazole and also the experimental drug, Palbociclib. The study found a highly significant difference in progression free survival (PFS), the intended endpoint: the mean time until disease progressed was 20.2 months among women who took Palbociclib, as opposed to 10.2 months for those assigned to Letrazole alone. The p-value for the difference between the arms (1-sided) was 0.0004. That’s a powerful  result.

But there was no statistically significant difference in overall survival between the two groups, a fact that was irksome to some observers, particularly in the biotech investment world, and to some who were reminded of the Avastin story and its fall-out. Most of the women lived for approximately 3 years after enrolling, with a trend of a few months favoring the Palbociclib arm. Another problem is that over half the patients were recruited to the study based on biomarker results, having to do with cyclin D1 amplification and/or loss of p16. So it could be the results are more relevant to breast cancer patients who have those particular changes. How those molecular features, enriched in the final study population, relate to Palbociclib’s usefulness in breast cancer and other tumor types warrants more evaluation, for sure.

My feeling is that this may prove to be a useful drug, not just in breast cancer. Any medication which interferes with cell growth by blocking cyclin-dependent kinases (enzymes) called CDK-4 and -6 could be useful in quite a few malignancies. The main side effect was suppression of the bone marrow (low blood cells). Some questions I’d like to ask the researchers, and which I hope they’ll address in the Phase III study, is if certain types of mets (e.g. lung vs. bone) or certain molecular subtypes are more tempered by this drug.

As for 10 months of PFS – if it pans out in a formal, published work, that’s valuable. Imagine that you’re 55 years old and living with metastatic breast cancer. A drug that is likely to delay, by most of 2 years, your tumor’s expansion into the lungs (causing shortness of breath), or bones (causing fractures and pain) or liver, and elsewhere can be worth a lot. It’s about the quality of life, whether or not it’s extended.

One final concern is that this study wasn’t blinded, so the doctors’ assessment of how the patients were doing, and the patients’ assessment of how they were feeling,  may have been influenced by their knowing which arm they were on. Also, because this new drug is a pill, some insurance may not cover it – a policy issue that applies to many new cancer drugs.

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A Film and Story-Telling Festival Focuses on Disability

Recently I had the opportunity to attend part of the New York ReelAbilities Film Festival. The 6th annual event in New York involved all five boroughs, but was based primarily at Manhattan’s Jewish Community Center. The program featured a dizzying spectrum of disability perspectives and concerns on film. It also included talks, photographs, parties and story-telling in presented by The Moth.

RealAbilities NY Disabilities Film Festival, 2014

RealAbilities NY Disabilities Film Festival, 2014

I liked everything about this festival. Perhaps the best aspect is that individuals with all kinds of issues can come, in real life, and meet other people with similar kinds of concerns. And so might their parents, or spouses and others who want to know, to gain a better sense of the experiences of people with varied physical forms. I don’t know that I could have imagined this kind of event happening, when I was a child or a young doctor.

For this post, I’ll stick to “the Moth” presentations, which numbered five. My instincts tell me not to declare favorites, so I’ll just provide a tidbit about each of the stories:

The first speaker walked onto the stage with just a bit of guidance. He was young, blind, handsome and funny. He spoke of growing up in a suburb. He was assigned chores and minded those. When in his early 20s, he signed up to participate in  a program that involved cleaning on Coney Island, the people in charge tried to keep him standing at the edge of the project, to not let him help out in a meaningful  way. He felt marginalized. By speaking with the other participants, gradually he entered the workspace. He got to get his hands dirty, doing grunt work with the rest of the crew. Happiness ensued.

Next, a dark-haired, smiling woman who has aphasia – difficulty speaking, casually stood as she told her story. Her name is Yvonne Honigburg, and she advocates for the National Aphasia Association. She described growing up with a sometimes secretive mother, of learning she was adopted, and of searching for her biological mother. Eventually the three met in a restaurant in New Haven, CT. Upon meeting Yvonne’s natural mother, the adoptive mother said something surprising. It ended well.

A woman in a wheelchair delivered the third, marvelous story. Millie Gonzalez has long curly reddish hair. She wore a sequined, shiny top and spoke of how she has always loved to dance. Evidently she has spina bifida, and after years of dancing with crutches, as a child and in high school, she’s learned to dance in a wheelchair. A while back she attended a previous ReelAbilities festival and saw the film Musical Chairs. After the event, upon trusting a man, perhaps the film director, he “twirled” her in the air, or something like that. Her heart stopped, momentarily, for the thrill of it all. It was very romantic. After that, she’s gone belly-dancing and advocating for people with disabilities.

The fourth speaker told of a moving story of her life with severe kidney disease and impaired vision. When she was a child, and the doctors finally explained to her what was wrong, she felt a sense of relief, knowing at least that there was an explanation for what she was experiencing. After some dark times, and dialysis, she received a kidney from her mother. Still, she lacked self-esteem, and hibernated. She spoke openly and vulnerably, about what led her to see the value of living. #uplifting

The final speaker walked on stage and, after a few minutes, mentioned that she had a prosthetic arm. She’d spent most of her childhood, adolescence and college years trying to hide her deformity. She didn’t want to be perceived as defective. In becoming a mother, she realized that her child had certain expectations….I cried, just a bit.

Seriously, you should listen to the Moth to find out what happened. And next year, or in your city, check out the ReelAbilities Film Festival.

No favorites. Each story is distinctly beautiful, and instructive (like people…)

All for a while,


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A Conference on Bioethics and Humanities, and Future Planning

Last week I traveled to Atlanta, Georgia, where the American Society for Bioethics and Humanities (ASBH)* held its annual meeting. Most of a thousand people participated in the four-day conference. The sessions drew a mix of nerdy physicians like me, nurses, professional bioethicists, philosophy professors, a few lawyers, historians and artists.

It was really a lot of fun. Fun, that is, if you’re into subjects like philosophy in medicine, literature in medicine, medicine in literature, ethics in medicine, technology and privacy, justice and parsimony in health care, etc. I hadn’t heard the word “epistemic” so many times since I was in college. I felt young and idealistic, talking seriously about philosophy, as though it matters. (For the record: it does.)  This was, clearly, a medical society meeting unlike others. For instance, an academic named Woods Nash, of the University of Tennessee, gave a talk on David Foster Wallace’s story, “Luckily the Account Representative Knew CPR.”

original cover image (Wikipedia) - link to Random House (publisher)

original cover image (Wikipedia), publisher: Random House 

On the first day, I walked into a provocative plenary talk by Julian Savulescu, an ethicist and Oxford professor. He presented an argument that that using medical tools for the purpose of moral bioenhancement might be a good thing. (If this topic brings to mind A Clockwork Orange, you’re on track. Think also of Huxley’s soma, as a questioner raised.) All very serious. The next day, a packed ballroom of people heard from Amy Gutmann, President of the University of Pennsylvania and Chair of the Presidential Commission for the Study of Bioethical Issues. She spoke about the concept of deliberative democracy, and the value of teaching ethics. Toward the end, she entered into a humorous and seemingly candid discussion of men and women in the workplace, “having it all,” and common sense. “Time is finite,” she mentioned.

I could go on, and list all the lectures and smaller sessions, but this post would get dry. Besides, I couldn’t possibly attend each one, nor can I give all the speakers’ due credit. Some talks were better than others, as meetings necessarily go. But I can’t resist a plug for the presentation by Rosemarie Garland-Thomson, a professor of women’s studies and English at Emory, on perspective and disability. Another favorite had to do with technology and science. David Magnus, of Stanford University, considered whether research accomplished through gamification – a means of crowd-sourcing science – on platforms like FoldIt, EteRNA and EyeWire should be covered by the usual rules for biomedical research. “Are the players scientists?” he asked.

The tone, overall, was intense. Intellectual, brain-stimulating… By contrast to other medical meetings I’ve attended, there was little glitz, scant makeup and limited Wireless. Perhaps the most surprising aspect of the ASBH conference was the distribution of freebies at booths in a display area, where attendees gathered for an opening evening reception and, on other days, breakfasts. Of course it was all minor stuff handed out, like pens and candy, mainly from university departments seeking applicants for fellowships, and academic presses selling books. The most substantive, and useful, gift I received (or “accepted” – a term with greater moral accuracy, from my perspective) was a green umbrella from the Hastings Center – a bioethics stronghold where I’d love to spend some time learning and doing research, in the future.

On Sunday morning, I attended one of the last sessions, on decision aids in bioethics. We lingerers were treated to three terrific talks. I can’t cover them all. So to close this post, I’ll refer to the promising work of Michael Green, a physician and bioethicist at the Penn State College of Medicine. He and colleagues have been developing an on-line decision tool for advanced care planning with grant support from the NIH, the American Cancer Society and elsewhere. The website,, enables individuals to detail their wishes through an interactive questionnaire. Green and his colleagues collect and publish data on users’ feelings upon using the decision aid. They can measure, for instance, if it gives people a sense of control, or reduces fear, and if patients’ families and doctors find the “outputs” useful. I, for one, intend to try out the MYWK website.

And I do hope to attend another ASBH meeting. Next year’s is planned for October, in San Diego.

All for a while,


*disclosure: I joined the society.

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A Theoretical Note to My Students, On a Breast Cancer Case and Future Learning

Last week my students – who are, necessarily, abstracted here – studied breast cancer. How the course goes is that we meet in a small group and, each week, work through a case by Problem Based Learning. The recent case concerned a woman who, at age 35, noted a small breast lump. Each day we acquired more information about the patient, such as the size and molecular features of her tumor and prognosis. We sorted through her treatment options.

a traditional lecture hall (Wikimedia)

a traditional lecture hall (Wikimedia)

It was a dense subject. Over 4.5 hours we discussed what kind of biopsy she needed – aspirate or core needle? We considered if excision in an operating room is required to establish a breast cancer diagnosis. (rarely) We reviewed breast imaging methods (mammograms, sonograms and MRI) and tumor staging. We covered some pathology techniques including OncotypeDx and Her 2 testing by IHC or FISH. We spoke about risk factors and BRCA testing – how that’s done, what it costs and when it might be indicated. We looked at the molecular biology of Her2 signaling, and how that might be pharmacologically blocked. We considered the nomenclature of LCIS and DCIS, and the concept of overdiagnosis. We talked about the woman’s decisions for surgery (lumpectomy or mastectomy) and sentinel node evaluation. We considered kinds of adjuvant therapy including hormone blockers, chemotherapy combinations, radiation, antibodies including Herceptin, and other treatments she might receive. We spoke about her prognosis and odds of recurrence.

We spent time on the statistical concept of lead-time bias. And more. Medical school isn’t easy.

What I hope for my students, real and in cyberspace, is that they’ll always try to do what’s best for their patients. Sometimes in PBL we use PowerPoint. So here’s a list of three things to keep in mind, on learning – not just about breast cancer, but about all aspects of medicine:

1. Keep studying. Patients want and rely on their doctors to stay up-to-date about medical and scientific knowledge in their field of practice.

2. Keep paying attention, so you’ll hear and recall your patients’ concerns and preferences, and offer care that’s mindful of their goals and values.

3. Keep thinking, constantly – how the data applies to the person, an individual, the real patient you’re trying to help.

Of course you should keep asking good questions, solicit advice from colleagues, be respectful of the people who entrust you with their lives…

The best presentations don’t cover too much ground, so I’ll stop here.

See you in the morning, or next week,


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Visiting an Exhibit on Early AIDS at the New York Historical Society

School’s back in session. With fall approaching, your author has resumed teaching and attending lectures. Today I had the chance to visit the New-York Historical Society where an exhibit, AIDS in New York: The First Five Years is winding down. The display closes in two days.

A group advocating AIDS research marches down Fifth Avenue in June, 1983. (Mario Suriani/AP) - NYHS image

A group advocating AIDS research marches down Fifth Avenue in June, 1983. (M. Suriani/AP image) NYHS 

The opening scene, by the first room’s entrance, is breathtaking in a way. There’s a huge picture of men, countless, basking in the sun on a Hudson pier. The men looked relaxed, comfortable and healthy – blissfully unaware of what lies ahead. The exhibit takes you through the late 70’s club scene, with just a few pictures of that, and then moves to confusing and odd reports of unusual infections in homosexual men, intravenous drug addicts, hemophiliacs and Haitians. The show moves on into the early 80’s, when science steps in slowly, and most politicians keep away.

What’s clear is that most doctors didn’t know what was going on. The young men weren’t sure either. There were rumors but also credible denials about a disease affecting the community. Gradually, the city’s Department of Health and CDC started tracking the problem. There were protests, and activists, and friends helping friends to die. There was no therapy back then, except to temper some of the infections and treat the once-rare cancers we were seeing with strange frequency.

I had the fortune of walking through the exhibit today among a group of suburban high school students – kids who were born after the invention of anti-retroviral therapy. Their questions – some simple and others intense, and the relatively young guide’s recounting of her experiences during the early AIDS years, made me realize how crucial is this history. It was a terrifying health problem, then.

Yes, the historical society’s exhibit is neat and tidy. I remember, well, caring for young people who died, hopelessly. The gravity of the epidemic isn’t captured. But it’s a worthwhile review, nonetheless – especially for its bits on low-end media, like typed bulletins from the early Gay Men’s Health Crisis and early posters on safe sex. Those frank messages provided the only information some people at risk received about the emerging disease. The display includes a few passages and images having to do with patients helping patients. That was the best part.

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Good News from SCOTUS on Gene Patents, But Questions Remain

Today the U.S. Supreme Court issued a unanimous decision on the Myriad Patent case, having to do with the company’s ownership of BRCA-1 and BRCA-2 gene sequences. The main opinion, authored by Justice Thomas, says this:

“A naturally occurring DNA segment is a product of nature and not patent eligible merely because it has been isolated, but cDNA is patent eligible because it is not naturally occurring.

At first glance, this is terrific news for patients world-wide. It means is that no company, university, other entity or individual can patent human genes.

Keep in mind – the case doesn’t just apply to BRCA and evaluating a person’s risk for breast and ovarian cancers. Rather, there are hundreds of human genes implicated in cancer that are potential targets for treatment, that might be evaluated, and thousands linked to other diseases. The decision continues:

“Myriad did not create or alter either the genetic information encoded in the BCRA1 and BCRA2 genes or the genetic structure of the DNA. It found an important and useful gene, but groundbreaking, innovative, or even brilliant discovery does not by itself satisfy the… <patent law>

West façade of U.S. Supreme Court Building. (Franz Jantzen)

West façade, U.S. Supreme Court (Franz Jantzen), gov’t image

What’s clear is that gene sequences, as they occur in human cells, can’t be owned just because they’re found, no matter how important they are. This circumstance should allow other researchers and firms to create cDNA from the natural sequences to develop new (competing and potentially less costly) assays and, even better – do their own work – tantamount to providing “second” and “third” opinions (etc. & n.b. IMO more is better!) research to understand how the genes lead cause disease in some people and might targeted for therapy. Great –

But the decision suggests that many lab-generated complementary DNA (cDNA) strands remain patentable, or up for grabs once created – which may be the reason some biotech stocks have rising values today. I’m neither a lawyer nor an analyst, but I do know from my experience as a researcher that it’s essentially trivial to generate cDNA from a short DNA segment, potentially with a mutation of interest. So how might the cDNA be patented, if anyone who has access to the original genetic sequence might form the cDNA by routine lab methods?

Near the end of the opinion, the justice writes:

“…but the lab technician unquestionably creates something new when cDNA is made. cDNA retains the naturally occurring exons of DNA, but it is distinct from the DNA from which it was derived. As a result, cDNA is not a ‘product of nature’ and is patent eligible under <patent law §101>, except insofar as very short series of DNA may have no intervening introns to remove when creating cDNA. In that situation, a short strand of cDNA may be indistinguishable from natural DNA.

The document clarifies the cDNA issue just slightly:

“It is important to note what is not implicated by this decision. First, there are no method claims before this Court… the processes used by Myriad to isolate DNA were well understood by geneticists at the time of Myriad’s patents ‘were well understood, widely used, and fairly uniform insofar as any scientist engaged in the search for a gene would likely have utilized a similar approach’…

Nor do we consider the patentability of DNA in which the order of the naturally occurring nucleotides has been altered. Scientific alteration of the genetic code presents a different inquiry, and we express no opinion about the application of <patent law §101> to such endeavors.

How I interpret this is that if a researcher generates a short cDNA segment based on a gene, that’s not patentable, but if it’s a long strand involving lots of clipped introns, that might be patentable.

Taking in all this, which is far from simple, I have a question and a wider point:

What goes unaddressed by the justices is the patentability of cDNA based on common genetic variants in cancer. Those are “naturally occurring” mutations, inasmuch as they arise in humans. But the cDNA generated from those sequences might remain patentable. There are loads of examples in this regard: Consider, for example, the genetic mutations in EGFR, and ALK, that are used in lung cancer diagnosis, treatment decisions and development of new targeted drugs. In the current issue of the New England Journal of Medicine, doctors report on SALL4, a gene that occurs in some liver cancers and might be a good, useful target for therapy in that disease.

The point is that the Supremes – and those would be lawyers – need to know about biology. Justice Scalia, sadly in my view, wrote his own opinion not because he disagreed with the others, but because he felt there was too much science in the decision. From the Scotus Blog today:

“Many readers no doubt will share the view of Justice Antonin Scalia, in a short, separate opinion refusing to join in a section “going into the fine details of molecular biology,” of which he said he had neither knowledge nor belief.  Scalia said he did understand enough …

This scares me, that one of the Justices, our most accomplished lawyers who might make decisions on cloning, and stem cells and who knows what in the future, copped out because he lacks science education – what should be required high school biology in  U.S. schools, public and private – to form an opinion that matters so much.

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Dr. Edward Shortliffe, on the History and Future of Biomedical Informatics

Last week I had the opportunity to hear and meet Dr. Edward Shortliffe at the New York Academy of Medicine. He’s a maven in the field of biomedical informatics (that would be the “other” BMI), and a pioneer at that. He mentioned that he began working on an electronic health record (EHR) when he was an undergraduate at Harvard in 1968.

Shortliffe emphasized the multidisciplinary nature of the field – that clinicians and computer science-oriented types need be involved for health information technology (HIT) to be effective. “Human health is at the core of it,” he said. The goal of biomedical informatics isn’t for computers to replace humans, he said, but for doctors to learn how to use it – as a tool – so that we (human doctors) can practice better medicine.

He reviewed the 50-year history of the field. The super-simple summary goes something like this: in the 1960s hospitals developed early information systems; in the 1970s, early decision support and electronic health records (EHRs) emerged at hospitals and large institutions; in the 1980s clinical research trials led to databases involving patients across medical centers; in the 1990s, progress in science (especially genetics) led to modern biomedical informatics. Now, the vast work includes clinical, imaging, biology (molecular, genomic, proteomic data) and public health.

Clinical informatics is the newest field supported by the American Board of Medical Specialties.  The first boards will be offered in October of this year, he mentioned.

If you’re interested in the future of health IT, as I am, you might want to take a glance at a perspective published recently by Dr. Shortcliffe and two coauthors, Putting Health IT on the Path to Success, in JAMA. The authors consider the slow pace of implementing HIT, and suggest that the solution rests with patient-centric Health Record Banks (HRBs):

“…Health record banks are community organizations that put patients in charge of a comprehensive copy of all their personal, private health information, including both medical records and additional data that optionally may be added by the patient. The patient explicitly controls who may access which parts of the information in his or her individual account.

I’d like to see these emerge.

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News on Occupational Exposure to N-PropylBromide, a Neuro-toxin

Yesterday’s NY Times drew my attention with a front-page article on the Occupational Safety and Health Administration (OSHA) and its inability to prevent harm among furniture and cushion factory workers in the U.S.  Even though hundreds of workers in North Carolina handling nerve-damaging glues have developed neurological toxicity, OSHA failed to suppress use of likely chemical culprits.

structure of n-propyl bromide (Wiki-image)

structure of n-propyl bromide (Wiki-image)

Regulating industry is complicated. The Times reporter, Ian Urbina, focuses on a compound, n-propyl bromide, aka nPB or 1-bromopropane, that’s used by “tens of thousands of workers in auto body shops, dry cleaners and high-tech electronics manufacturing plants across the nation.”

Problem is – it’s hard and possibly impossible, based on studies of factory workers, to prove cause and effect. He writes:

Pinpointing the cause of a worker’s ailment is an inexact science because it is so difficult to rule out the role played by personal habits, toxins in the environment or other factors. But for nearly two decades, most chemical safety scientists have concluded that nPB can cause severe nerve damage when inhaled even at low levels…

The lack of absolute proof – that a particular chemical substance has cause disease in an individual –is exacerbated by the fact that many cushion and glue workers’ symptoms, like numbness and tingling, are subjective: At one company, Royale, a ledger of employees’ illness is said to list “Alleged Neurologic Injury.” This phrase reflects the evaluators’ doubt of the handlers’ complaints and, by insinuation, adds insult to injury – some so severe the workers couldn’t button a shirt, feel a cut, bleeding foot, or stand for more than a few minutes.

The government agency that might respond, OSHA, is woefully understaffed. According to the Times:

“OSHA still has just 2,400 responsible for overseeing roughly eight million work sites — roughly one inspector per 60,000 workers, a ratio that has not changed since 1970. The federal budget for protecting workers is less than half of that set aside for protecting fish and wildlife…

Regulation of industry kills jobs, some say – it’s for this reason that some individuals most likely to suffer harm from manufacturing align with corporations. What’s more, if people lack education about chemistry and need employment, they may not choose or know what’s in their long-term best interests. This piece, like the story of Toms River, points to the unfortunate reality that many citizens tolerate and even take pride in a damaging local business, especially if the health problems it causes are insidious, affect some but not all exposed, and the facts aren’t in full view.


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Contemplating Breast Cancer, Beyond October 2012

It’s foggy today, October 3, ten years since the last mammogram I had and will ever need. I’ve been remiss in updating the blog. The reasons include family concerns and other projects. Meanwhile, I’ve been thinking about the big picture – what’s most important for progress against breast cancer in the decade ahead.

So here’s what I see, now – in terms of three priority areas: improving treatment, prevention, and education to inform treatment decisions.

Pumpkins, organized by subtype (WikiCommons image)

As an oncologist, I perceive huge strides in understanding BC since the time of my diagnosis. But these advances are largely invisible to patients because they’re in the realm of pathology and classification of different subtypes. What was essentially a 3-type malignancy with a handful of treatment options has expanded under the molecular microscope to a spectrum of 4, 10 or – what’s probably most accurate – hundreds or thousands of patient-particular conditions, depending on the level of precision by which you define a disease. I’m optimistic, because it looks as though, in my lifetime, BC treatment will be tailored to each patient. There’ll be less surgery and better drugs.

The hitch, now, is not so much with science as with funding– funding to analyze each patient’s tumor at the genetic and protein levels, funding to pay for treatments selected by patients (which might include less treatment and/or palliative care in advanced cases), and funding to educate doctors about BC subtypes and medical progress, so they might offer “modern” advice to each patient in ordinary clinics, apart from clinical trials and academic centers. Newer is not always better in medical care. Same goes for more treatment (especially when it comes to higher doses). Still, the lag between advances in BC science and application of distinct, targeted and better treatments is frustrating at best.

Some of my colleagues call for patience – emphasizing that studies need be confirmed, drugs tested in mice, etc. Their point is that we can’t jump from pathology research and new BC classifications to new therapy. But one lesson I take from progress against AIDS is that maybe we shouldn’t be so patient. At least not for young people with poor-prognosis BC subtypes or stage. We could do studies and studies of particular BC treatments, and studies of studies (those would be meta-analyses) and debate 8 or 10 years from now whether a particular drug or combination of drugs worked in clinical trials that selected for patients with an antiquated subtype of the disease. Or we could move toward “n=1” trials, with smart, well-trained physicians assessing each patient by a combination of old-fashioned physical exams and the most modern of molecular studies of the tumors, considering the options, and moving forward with individual, mini-experimental treatment plans.

I vote for the latter. If the drug works in a patient with advanced BC and the patient feels better, why not?

For people with early-stage BC, prescribing or taking new and essentially untested drugs makes less sense at first glance. That’s because standard treatments are “successful” – leading to long-term remissions and possible cures in over 80 percent of those affected. But these relatively good results may have, paradoxically, hampered development of better drugs that could obviate the need for breast-deforming surgeries and radiation in many women. The possible application of BC drug cocktails, in lieu of surgery for early-stage patients, is a huge question for the future, and one for which trials would be necessary. Just getting those projects going – applying BC science to treatment of early-stage cases – would be a step in the right direction.

As for BC prevention, of course that would be infinitely better than detecting or treating the disease. Unfortunately, I think we’re farther away from preventing the disease than we are from having effective and less brutal treatments for most patients. The problem with lifestyle modification – like staying active and not obese – is that it’s far from full-proof: You can be seemingly fit as a fiddle and get a lethal case of BC. Still, there are plenty of other health-related reasons for women to exercise and eat sensibly. As for avoiding carcinogens or, first, just knowing what chemicals contribute to BC formation and growth, the science isn’t there yet.  It’ll be a long haul before anyone can prove that a particular chemical causes this disease. That said, I advocate research in the slow-growing field of environmental oncology and wish there’d be more enthusiasm for regulating our exposure to likely-toxic chemicals.

The third priority is for improving education in math and science, starting at the elementary school level. Doctors need to understand statistics, but many don’t. They need to know about genomics and basic science in medicine. Patients need this kind of knowledge if they want to have a clue, if they want to engage meaningfully in decisions about which antibody to take, or pill, or whether they want to participate in a clinical trial of pills instead of surgery for a Stage II tumor with high levels of Her2, for example. That’d be a tough decision for an oncologist. I only wish that we could reach the point where we could have those kinds of truly informed conversations about clinical treatment of breast cancer, which happen every day.

We’ve got a lot of information in hand, but we need to learn how to apply that to more patients, faster and more openly.

All for a while. I’m open to ideas on this. Happy October!


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