Posted by on Jul 24, 2015 in Blog | 14 comments

Patients Included In ResearchWe’ve gotten used to thinking about having cancer in your colon or lung or breast. We’ve systematized treatments and research of cancer by the organ that’s affected. And we’ve been fundraising that way too–pitting lung cancer against breast cancer against childhood cancers.  But what if this view of cancer is totally wrong?

That’s the question that is driving a new direction for NCI and researchers around the US. The new focus is on genes and how those genes influence the out-of-control, immortality of cancer cells.

A New Look At Outliers

The way research usually works is to concentrate on statistical significance: the search for confidence that something that you did made a difference and that it probably wasn’t just blind luck that caused it to happen. The number that is used to test this is the average or mean.

Let’s look at an example. Say a new medication is tested and it doesn’t work on most of the people who take it. Often that medication gets put on the shelf. But, what if there are some people who took the medication and had a miraculous recovery?  In statistics, those people are outliers…and though everyone is happy for their success, researchers have usually ignored them.

Patients Included In Research

Now, what if there was a way to find out why those miracles happened?  In an initiative described in NCI’s press release last fall,

“investigators will study the molecular characteristics of tumors of patients who had an exceptional response to a cancer therapy. In doing this, they hope to discover molecular features in the tumors that may predict benefit to a particular drug or type of drug.”

Washington Post article  describes how this type of research can have an enormous effect on someone’s life.  Review of data on a “failed” medication led to a woman with thyroid cancer who had a miraculous recovery.   Genetic sequencing of her tumor led to the discovery that she had a mutation in the gene that regulated a protein pathway that allowed her cancer to grow.   The “failed” medication inhibited that protein pathway.

Physicians at Dana Farber Cancer Institute looked in their database for other patients with that mutation.  There were several.  One,  a patient with advanced ovarian cancer,  received treatment with the “failed” drug and her cancer is in remission.

Patients as Partners

Corrie Painter, PhD, (who we introduced here) is a mother, scientist and patient advocate. In aPatients Included In Research previous life, she was headed for a tenure track position with a lab in Biochemistry. After walking away  from a million dollar grant,  she has taken a position at the Broad Institute of MIT and Harvard as their Associate Director of Operations and Scientific Outreach.  There she is using her experiences as a patient to initiate direct partnerships with patients.  The goal of the work she is doing is to accelerate discoveries and treatments for cancers through collaboration.

“Imagine what would happen if there was ample preliminary data for all scientists to share. Those scientists could write grants based on their ability to synthesize the most amount of information based on a robust and shared knowledge base,” Corrie wrote.

The first project, #MBCProject, was introduced on the #BCSM website.  The #MBC stands for metastatic breast cancer and the project is a new Broad Institute and Dana Farber Cancer Institute research initiative designed to accelerate discoveries and treatments for breast cancer metastases. The project is being done in partnership with advocacy groups like the Metastatic Breast Cancer Alliance, the Metastatic Breast Cancer Network, the Avon Foundation, Living Beyond Breast Cancer, the Young Survival Coalition, and the Inflammatory Breast Cancer Research Foundation.

Wait! Isn’t This Organ Based Research?

Why start by studying metastatic breast cancer?  Corrie answered this way.  First, “the study of extraordinary responders in and of itself will be a game changer for treating cancers by mutation.” Second, research specific to metastatic breast cancer has been limited. Third, since most patients in the US receive care at non-research facilities, their tumors are never studied. “By reaching out to patients online we will be able to work with people all over the country regardless of where they live.” Fourth, “we need to understand the mutations in the context of their biology and clinical behavior, and then we’ll have the infrastructure
to understand how to treat cancer by mutational profiles rather than simply by site of origin.” Finally Corrie explains, “we hope to move the needle on both fronts by performing a number of studies in mbc, leading with extraordinary responders.** Our goal is to partner with patients, conduct research and listen to their…voices, [to] pave a path forward by learning [from] both directions.”

A website is under construction that will allow any patient with metastatic breast cancer to sign up.  After signing up, the plan is to obtain medical records, tumor samples and saliva.  Genetic analysis will be conducted on the tumors and saliva.  But this work will not occur in a vacuum:  patients are collaborators in this research.

Changing The Culture of Academic Research

Having been an academic researcher, Corrie knows the culture.  “Every lab needs to establish an independent area of expertise. To publish, you need to discover or describe something that no one else has figured out. Think that happens by scientists talking openly and collaboratively about their ideas? Nope.”  Being “scooped” is the fear of academic medicine.  ” You can get scooped by scientists who hear about your unpublished work at conferences, by word of mouth, by “collaborators”, by reviewers who are reading your manuscript (by far the most nefarious). Best way to not get scooped? Work in a little silo and keep your data SECRET until publication.”

Patients Included In Research

This culture brings publications and facilitates the climb from non tenured assistant professor to full tenured professor with a well-funded lab and many graduate student workers, but does it save lives?  Corrie answers with a resounding NO.  “We can redefine the way that science is “tackling” cancer. When scientists care more about high impact papers, and getting tenure than they do about curing disease, it’s UNACCEPTABLE.”

Patients Included In Research

As a patient and a scientist, Corrie’s perspective was sought out by the Broad Institute.  The innovation is “patients included.”  This is not just at conferences, but also on the research bench. The goal is shared data, collaboration and breakthroughs.   Targeted therapies that are not organ-specific but are genetic-mutation specific are pushing this movement.

Corrie is hopeful, “Technology has reached a point where we can ask questions like, “what drives my cancer, why did I develop resistance to a targeted therapy, what combination of drugs might work best for me?” And when I say it in the first person, I mean it…from the patient, for the patient. And this brings me to my favorite point with respect to progress in cancer research. THE PATIENT IS BEING LISTENED TO.”

Metastatic breast cancer is just the beginning.  Corrie and the Broad Institute are interested in making this kind of collaboration a reality with other cancers as well.  Corrie states, “Our goal is to generate enough of an understanding of cancer at the crossroads between traditional site- specific studies and genomics that cancers will one day be treated by their mutational profiles rather than their site of origin.”

For more information email at

**Correspondence with Corrie