The need for study of rare cancers

Part of why I started this blog was to give myself practice in writing on science topics for a general audience. As I learned professional technical writing during my graduate studies, several mentors suggested that I examine writers whose papers I enjoyed reading. My own favorite is Bob Eisenman at the Univ of Washington - his early 90s Cell papers on Myc, Mad, and Max in oncogenesis still make for engaging reading of great clarity.

So, when I started experimenting with medical journalism, I chose Amy Dockser Marcus as my role model. Based in Boston for the Wall Street Journal, Amy was awarded the 2005 Pulitzer Prize for beat reporting, stemming from a series of articles on cancer survivors.

What I love most about Amy is that she writes in both understandable and scientifically-accurate technical detail, while also bringing an emotional component to the subject matter that makes me proud to be a scientist. In 2004, Amy brought to our attention Andy Martin, a Tulane medical student diagnosed with a very rare cancer, SNUC, or sinonasal undifferentiated carcinoma. I urge you to read her chronicle of Andy's struggle to grow his own tumor cells in the laboratory under the guidance of Dr Tyler Curiel. (Tyler and his wife, Ruth Berggren, MD, deserve their own separate post for their heroic treatment of patients in New Orleans in the aftermath of Hurricane Katrina.). Following Amy's story, NPR's Robert Siegel did a lovely interview with Andy - Andy is no longer with us, but Amy also reported on Tyler's successful post-Katrina rescue of Andy's SNUC cell lines.

As with many of us, cancer has come to roost in Amy's family. Amy's Mom, Golda Dockser, had her gall bladder removed about a year ago and learned it was cancerous. You probably haven't heard much about gall bladder cancer - no green ribbons, races for the cure, etc. - simply because it afflicts just under 7 500 Americans a year, as compared with over 200 000 each for breast and prostate cancer. Amy's account of her journey with her mother, A Cry in the Dark, appeared in this past Monday's WSJ (subscription req'd). However, I'd be happy to send you a 7-day free access invitation to read it if you send me an e-mail request. Her editor, Lawrence Rout, introduced it best:

"Amy Dockser Marcus writes about a different uncomfortable truth: If you have a rare cancer, you have few places to turn. When Amy's mother was diagnosed with gallbladder cancer, Amy, who won a Pulitzer Prize for her reporting on cancer, set out to help her mother learn about the best, and latest, treatments. She quickly saw there was little to learn."

"Amy's essay lays out the frustration of having few options. But it's also a daughter's plea for help, and a reminder that behind every faceless patient is a mother, a brother, a daughter, a friend. And you can't get a truth more simple than that."

Frankly, I can't tell you how many times I've heard on NIH grant review panels, "Why are they studying X cancer since it affects so few people?" But Amy's article got me thinking about why we should study rare cancers. Certainly, government research budgets are limited to the point that we must fund the best-designed studies that have the greatest potential to help the largest number of people.

However, I'm not terribly certain that the increased funding, say, of breast cancer research, has led to advances in treatment in proportional magnitude to the much higher investment. Once the US Dept of Defense started funding breast cancer research in the early 1990s, dozens of my colleagues suddenly started working with breast cancer cell lines - not because they were interested in breast cancer, but because it was a way to get funding to maintain their laboratories and support trainees.

In fact, most advances in cancer biology and treatment come from the least likely sources. The widely heralded anticancer drug, cisplatin, credited with saving Lance Armstrong and revolutionizing the treatment of testicular cancer, was not a result of directed study of testicular cancer. Instead, cisplatin was discovered serendpitously as a by-product of testing the effects of electric fields on bacterial growth. (Cisplatin was then identified from some careful control experiments as an electrochemical product of the platinum electrodes and the amine-containing buffer to which the bacteria were exposed; in fact, cisplatin had been synthesized as early as 1844 when it was named Peyrone's chloride).

In the couple of days since Amy's essay appeared, I've been looking for evidence to support why the study of rare cancers is not only good for those people afflicted with rare malignancies, but because such study might reveal truths and targets of relevance to other more common cancers. For example, exactly why is gall bladder cancer so rare relative to breast cancer? Might study of the gall bladder tell us something about why its cells so rarely undergo transformation? How could those pathways could be turned on in more common cancers?

I'm sure that this topic has been discussed and studied, and I'm aware that the US NCI has a special office on the study of rare cancers. However, I turn to you, o faithful readers, many of whom might have experience in studying rare cancers. For those of you that do, what are your reasons for studying rare cancers? How do you write the "significance" section of your grant applications to convince reviewers of the broad significance of your work?

I can close this post no better than did Amy and our mutual friend, Dr Curiel:

"After my mother's diagnosis, I called Tyler Curiel, the head of the hematology and medical oncology program at Tulane University. I knew that Dr. Curiel's lab had done work on several rare cancers. But that day, I was searching for comfort, not answers.

"A cancer is only rare until you know someone who has it," he told me.

"As my mother undergoes another round of chemotherapy, I cling to this idea. It offers the possibility of what I was searching for all along, an avenue of hope. So consider these words an introduction to my mother, Golda Dockser. Now you know someone with gallbladder cancer."