Sunday, November 29, 2009

A Smarter War on Cancer

The headline in the Washington Post caught my eye: “Fighting a smarter war on cancer,” [sorry, you must register to read the article] an opinion piece by Dr. John Marshall, a faculty member at the Lombardi Cancer Center in Georgetown. It is about the intersection between health care reform and cancer care - it seemed like just the thing to read while drinking my morning coffee. But halfway through the article I found myself feeling marginalized, and that got me thinking. Although Dr. Marshall makes some good points, I think he missed a golden opportunity to propose changes that could make a real difference in our lifetimes.

How did Dr. Marshall make me feel marginalized? He wrote the following about chemotherapy:

“The most common approach to treatment involves exposing large populations of patients to highly toxic poisons in the hopes that the treatment will kill the cancer cells and not the patient. This strategy has succeeded with several types of less-common cancers, curing some patients with leukemia, lymphoma, testicular cancer and most childhood cancers [emphasis is mine]. But it has not worked for more common forms of the disease, including breast, prostate, lung, colon, pancreas, stomach and ovarian cancers. These cancers represent an enormous public health problem, consuming the majority of our cancer-specific health-care costs and research dollars.”

I realize that childhood cancer is rare, and that to make an impact on the total health care system in our country will require real progress against such public health menaces as breast and lung cancer. However, I believe that rather than brushing aside the approach that transformed childhood cancer from a death sentence to a treatable disease, the medical oncology world needs to embrace it.

Most oncologists know this, but in a single generation, collaborative clinical research in pediatric oncology, exemplified now by the Children’s Oncology Group, has revolutionized the way childhood cancer is treated in this country. Once uniformly fatal, there are now some types of childhood cancer that are cured 95% of the time. This transformation did not come about by chance. It came about because of a culture change that is now a huge gulf between pediatric oncology and medical oncology: the role of the clinical trial.

Dr. Marshall hints at this in his article: “In cancer medicine, fewer than 5 percent of all patients in the United States enter clinical trials. That means more than 95 percent are treated with the ‘standard of care’ -- a legal term denoting a minimum level of care for an ill or injured person.” In contrast, being entered on a clinical trial IS the standard of care in pediatric oncology. Partly this is born out of necessity – childhood cancer is rare enough that unless nearly every child with cancer is treated on a trial, not enough patients can be studied to yield reliable results, making progress impossible.

The cultural difference in the view of clinical trials between medical and pediatric oncology is readily apparent in my every day practice, particularly when I have to approach an insurance company about enrolling a patient of mine on a trial. In the “adult” world, a clinical trial is something offered when there is nothing “standard” to offer, just as Dr. Marshall implies. What this means is that insurance companies often feel justified in denying coverage for trials, since these are “experimental therapies” and “not standard.” In the “pediatric” world, everyone goes on a clinical trial. Coverage is routine, because the trial IS the standard.

Dr. Marshall makes an excellent point about where the future of cancer therapy lies. He believes, as do I, that the future is in “personalized medicine,” meaning treatments that are individualized for each patient, based on the molecular and cellular composition of their individual cancer. Unfortunately, therein lies the problem. In the world of “personalized medicine,” there will be no standard. What works for Patient A will not necessarily help Patient B. Destruction of the concept of “standard of care” in oncology will make coverage decisions by insurance companies far more complex. This will need to be accounted for as we move towards a reformed health insurance system.

More importantly, demonstrating the value of “personalized medicine” will require more clinical trials. Only by enrolling patients in such trials, painstakingly dissecting the molecular changes in each patient’s tumor, and carefully proving that tailoring therapy based on these changes dramatically improves outcomes can real progress be made. Because these molecular changes will, by definition, vary widely from patient to patient, treating only 5% of adults with cancer on clinical trials will never get us to where we need to be. The medical oncology world needs to learn from the successes of pediatric oncology. Being treated on a clinical trial needs to be the standard, not something that is reserved for use when “standard of care” fails.

Instead of marginalizing pediatric oncology, hold our system up as the model. Only then will real progress be made in the war on cancer.

Related Posts:
Is the Medical Community Complicit?


Megan said...

"Instead of marginalizing pediatric oncology, hold our system up as the model. Only then will real progress be made in the war on cancer."

What a compelling statement. I hope this blog post reaches a large audience--one filled with policy makers and adult oncologists and insurance companies!

Doctor David said...

Thanks, Megan! If you really feel that way, feel free to mention it on Twitter, Facebook, Reddit, whatever...

Megan said...

Okay, I posted to facebook. There's 300 or so that are now aware. It's a start!

Will FitzHugh said...

Great perspective, thanks a lot, David. Do you have any thoughts on what next steps could be taken to change the culture you talked about? Somehow you'd have to convince patients and payers that bypassing standard of care to try an experimental treatment in a clinical trial is a good idea. It's a good idea for medicine because it will help find better treatments, but is it better for the individual patient facing this decision?

Doctor David said...

Excellent questions. Unfortunately, the whole issue is far too complex for blog posts and comments, but... it's a good place to start (and maybe it just means I need to keep posting about this).

To some degree, the value to the patient (as opposed to the value to society) depends on how good "standard care" is, and on what benefit the patient might derive from participation.

A good place to start would be with creative Phase III studies, where patients would be randomized between arms that are based on standard care. That way, participation does not deny the patient what is standard, and progress still occurs. That is the basis for COG studies for the common childhood diagnoses, like Acute Lymphoblastic Leukemia.

Such studies, however, bring up another point Marshall made that I did not address (yet)... the incremental nature of most clinical trials. Most are designed to detect relatively small benefits, which means progress towards a "cure" is incremental. But that is a topic for another blog post.

Kyle Alfriend said...

I just found your blog, and find it very interesting. Thank you for the information.

I will say that my experience with many pediatric oncologists was very different. When my son was fighting cancer, I found a wall blocking us from information, trials, and personalized treatment. We felt like we were in some massive assembly line.

We traveled to several hospitals, and eventually found doctors willing to openly discuss options. But our initial months were filled with a lot of mis-information from the doctors. My son's cancer was agressive (stage IV Burkitt's), and we did not have the time to fight the hospitals.

Thank you for your work in saving our children.

Lorin Decker Buck said...

Thanks for blogging on this article, David! I read it in The Post -- saved it, actually, because I wanted to ask your opinion about it. I was confused by it. Through our experience in ped-onc, we've gotten used to thinking of clinical trials as offering the best opportunity for cure. I'd completely forgotten that a clinical trial has a different connotation in adult oncology -- that it's often offered as a last resort.

I'd encourage you to write up your blog as an editorial or letter to the editor and submit it to The Post. I have a contact there (David Brown, an MD who co-taught my medical-writing course at Hopkins last fall; he's The Post's medical writer), if that would be helpful.

Doctor David said...

Kyle, thanks for your kind words. I'm glad you found the blog and hope you enjoy reading it.

Lori, I'd be willing to submit it to The Post, but would love to chat with your contact first. Let's talk about it "offline." I'm thrilled you liked this post that much!

Shannon said...

Hey David.
So, I'm still in high school and thinking about becoming a pediatric oncologist. I'm doing a project on it now and wanted to know some of the skills necessary to do what you do. For example, being a people person.



david said...

Hi Dr.David,
Very informative article. I am always interested to know more about pediatric oncology and I feel glad that I found your blog here. This is a best method to find the better treatment offered. Blogs are best starters to convey the message.
Do keep posting more interesting articles like this.
Thanks for sharing healthy information here.

outre said...

I'm so glad I live in a state where it is a state law for insurance companies to cover clinical trials for cancer patients(except phase I, I think).

Part of the reason for low enrollment rate in the adult population I think is due to patient hesitance.

I encouraged a woman I met through the ACOR listserv dx'ed with MPNST to enroll in a chemotherapy trial but she was hesitant. She thought it'd be too miserable and in addition, she thought she'd be ok because they told her they had gotten clear margins (less than a centimeter, which, given the location and size, to me was not enough of a margin). I know that the cancer center she was treated at, while great, doesn't see that many sarcoma patients, and are in the 'no chemo for MPNST because it dosen't work' school of thought. True to a degree, except... part of the reason no one knows what drugs work is the lack of patients being treated with chemo.

So I got her the info for a trial she qualified for and would have been able to participate despite her lack of insurance but she was afraid and less then six months after her diagnosis there was an email in my inbox that broke my heart.

In children, the caretaker usually makes the decision and they I think are much more willing to fight for every last bit. It's 'they have so much to live for' rather then 'well, I had a good life, I don't want to be miserable with the whatever time I have'

Obviously it's not as simple as that... but I've seen this one too many times in the adult MPNST population. 'Well, they told me it only works in 12% of the patients, so I'm not going to try.' But, you, you can be in that 12%...

Doctor David said...

Outre... great comments, as always. You make some really good points, especially about how aggressive parents/caregivers are likely to be with treating their kid's cancer, compared with how conservative many adults will be (to avoid the misery). One school of thought holds that THAT is the one of the main reasons kids do better than adults (topic for another day).

Of course, you also reinforce a point I was trying to make... one reason chemo for MPNST isn't so great is because there aren't enough trials to let us figure it out.... our center is participating in one run through SARC, but initially by the NCI. It has been open for 6+ years, and is still trying to recruit patients. It's hard to get adults to participate... in large part, I believe, because of the thought that a clinical trial means nothing left to offer in the adult world. That's what needs to change.

Thanks for still reading and still commenting!

Anonymous said...

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Todd said...

One field thats always interested me as an undergraduate in Biology and MS1 is the field of cancer immunology, which to me epitomizes the idea of personalized medicine for fighting cancer.

Instead of using specific cancer molecular markers to target chemotherapy agents, one can use these markers to isolate and activate populations of cancer-recognizing cytotoxic T cells via adoptive cell transfer or cancer vaccine therapy.

I had a couple of questions though. First, as a practicing oncologist, how do you feel about the current research behind cancer immunotherapy? Secondly, as cancer immunotherapy is highly personalized, how do you feel personalized medicine will fare in an area of tightly regulated costs by health insurance organization, as personalization seems like it would be much more expensive?

Thanks a lot.

Doctor David said...

Great questions, Todd!

Regarding the current status of immunotherapy, I think it is in its infancy. This is a tremendously complicated field, far beyond what we can cover in this format... but suffice it to say the field has a long way to go.

But that doesn't mean it won't get there.

Costs.. that's a whole other set of issues. I think that insurance companies can be coerced to pay for expensive things if they work... so eventually it can be covered.

Keep in mind that "immunotherapy" is a very broad term... and drugs like Herceptin can fall into that category.

This sounds like something to cover in future blog posts...

Anonymous said...

As a member of the COG and involved in cancer research for both pediatric and adult cancers I agree! This an excellent post and an awesome site which I just found and I'm enjoying reading your perspectives very much!

Unknown said...

Hello Dr. David, first of all I want to congratulate you. You have created such a good blog giving knowledge and generating conscience about many things that are just in our thought or that simply we do not know. The problems you express in your blog stimulate the people to continue trying improving the medicine that exists nowadays, to be concerned and try to change this reality looking for better conditions for the people who really need help.
I believe that this publication makes us open the eyes on hidden issues that the medicine has. With your post we know that it can change and more than that we are assured of improving this situation. Now I have the hope of curing every disease even the most dangerous for people but if the specialists start to work together getting better results and giving us more hopes and more life.

Types of cancer said...

Good post . Good site. Keep it that way !

discountmedicalsupplies said...

The health care system should place the brakes on treatments with marginal (<2-3 month) survival benefit, exhorbitant cost, and poor side-effect profiles.