The Critical Role of Biospecimens in Cancer Research

The Critical Role of Biospecimens in Cancer Research

NCI OBBR Director Dr. Carolyn Compton explains how biospecimens begin and end with the patient.

Biospecimens

Balintfy: In our last episode, we talked to the Deputy Director of the National Cancer Institute about studying the cancer genome. This next interview is a great follow-up, because researchers can't study things like cancer tumors - or healthy cells and tissues for that matter—without samples, or bio-specimens. So this month, we're talking with Dr. Carolyn Compton. She's the director of the Biorepositories and Biospecimen Research Office at the National Cancer Institute. We started by asking her, what exactly is a bio-specimen?

Dr. Compton: A bio-specimen is anything that comes out of or off of the human body and reflects the biology of the person from whom it was derived. So it can be a tissue, an organ, a fluid, like urine or blood that's derived from a person and reflects their unique biology.

Balintfy: What are the key issues facing bio-specimens, in particular regarding collection, processing and storage?

Dr. Compton: I think the most important thing to remember about bio-specimens is that they are alive. That they are living entities; they are living cells and living tissues that we kill by fixing or freezing in order to store them. But up until the point when we stabilize them by fixing them or freezing them for use in the future, research use in the future, they are still living and still capable of reacting to biologic stress. And so the way that we derive them, the way we collect them, the way we process them and even the way we store them over time can affect the integrity of their biology, can in fact produce stresses that change their biology and can affect the quality of the bio-molecules that are in the cells. So what we're looking at is a challenge to make sure that we are using processes of collection processing and storage that are of the highest possible quality, consistency, and are standardized across the country.

Balintfy: Consistency and standardization are important in any kind of research, aren't they?

Dr. Compton: It's true. It's important for any research in any disease process using human specimens, but it's especially important in cancer research because that's where most of the specimen collections in this country are targeted, is towards cancer research.

Balintfy: Dr. Compton, can you explain the NCI's role with bio-specimens and issues surrounding them?

Dr. Compton: So we create and distribute standards and guidance, and we educate people about the implementation of those standards and guidance. We are also in the process of exploring the possibility of creating a national resource for this country, a national bio-bank which we do not have in the United States, which other countries ironically do have in countries that are much smaller than the United States but are ahead of us because they have national healthcare and are able to coordinate the collection of human specimens through their health care system. We don't have that advantage, so we have to create an ingenious way to collect standardized specimens in our rather heterogeneous health care system. And we're exploring a way to do that to create a center that would be accessible to all researchers to be able to find bio-specimens that would match their needs and that would be of known standards with known amounts and types of clinical annotation, that is data about the patient from whom the specimens were derived.

Balintfy: Who are some of the different players involved with bio-specimens?

Dr. Compton: There are many people involved in what I like to think of as the life cycle of a bio-specimen. It certainly does start with the patient and the patient consenting to allow the banking of his or her bio-specimen. And most of the bio-specimens in cancer research are derived from therapeutic procedures. In other words, the tissue is removed for a medical reason; you undergo a cancer operation for example.

But the patient, him or herself, must consent to allow that tissue to be used for research. So it certainly does start with the patients consent. And then the people who explain the use of the specimen in research are also key to this process. Those would be mostly nurses or clinical research assistants who educate the patient about the use of the specimen in research so they know what they're consenting to. And then there's the surgeons who actually remove the tissue and the way that they handle the tissue and their communication with the pathologist to whom they pass the tissue after it's removed from the body. They're also absolutely key in the process.

So there are a number of individuals who are involved in this entire process, and the entire process must be optimally coordinated, which means that all of these people must do their jobs very well, and there must be communication among them.

Balintfy: Dr. Compton, would you say that the patient has an especially important roll with regards to bio-specimens?

Dr. Compton: The patient is key. Without the patient's consent and understanding and full participation in the process of research, we will never change medicine for the better. And so despite all of the bio-bankers efforts, we could create the perfect standard operating procedures and everyone in the operating room and the pathology suite and the bio-bank could be doing a perfect job, but in fact if the patient hasn't consented to use the specimen in the project for which its intended, we wouldn't be able—all would be lost. We wouldn't be able to use the specimen at all for those all-important projects that are going to change medicine.

Balintfy: How is medicine being changed and what is the role of bio-specimens in that change?

Dr. Compton: Well, I think we are standing at a watershed between what we now know as standard of care medicine that is rather generic in terms of cancer treatment, where we have one drug that we generically use to treat all cancers of a certain type. Say colon cancer, and we are looking forward on the other side of this watershed to an era of what we're thinking of as molecular medicine where the treatment will be tailored to the specific colon cancer from the specific patient. And there will be targeted therapies that will target the specific molecular defects in that patient's cancer.

Well, the only way we're going to get to this new era of molecular medicine is through the bio-specimen, which is both the fuel that will drive the research that will get us to this new era of medicine but will also be the currency of the practice of molecular medicine in the future. Because, in fact, that's where the molecules are so we must optimally preserve the molecular integrity of the specimens because that is the reflection of the biology of the patient and the biology of the patient's disease. And it will be the basis on which we will treat patients in the future, and certainly it is exactly the anaglyph of importance, it is the centerpiece for our research, our ongoing research that is going to take us to this new era of medicine.

Balintfy: Can the public get involved with this new era of medicine, perhaps by donating bio-specimens?

Dr. Compton: Absolutely. And it's timely that you ask this question because some of the biggest scientific initiatives that are now being launched surround normal human biology. In fact, we have powerful technologies now that we can direct at this study of disease, but it's ironically easier to get diseased tissue because it's removed for therapy for the patient than it is to get normal tissue. Obviously if you're a human being with normal tissues you like to hang on to them for your own purposes, and so they're very difficult to come by, and we are, here at the NIH, are just starting a brand new project. It's an NIH roadmap project, with the National Human Genome Research Institute taking the lead, to study the transcriptome [spelled phonetically], in other words, how genes are transcribed in different cells and tissues in the body. Obviously, every cell in your body has exactly the same genome, but those genes are transcribed differently in different cells and tissues because they do different jobs biologically.

But we have never had the opportunity to use our most powerful technologies to understand how these molecular differences occur. And in order to do that, we need large numbers of normal tissues from normal people, and those are challenging to acquire. And some of the ways that we are thinking about addressing this is through, again, something that the patients can participate in, in terms of education and direct participation is rapid autopsy programs, where you actually leave a living will to consent to having your tissues used in research. And although everyone is very familiar with the consent that we give, say at the Bureau of Motor Vehicles when we go to get our driver's license to consent to have our normal tissues used for transplantation, for therapeutic reasons, we don't think to consent to have our normal tissues used for research where they are just as valuable.

Balintfy: Thank you Dr. Compton. Is there anything else you'd like to add?

Dr. Compton: Just remember that the bio-specimen is the fuel for translational research that is taking us towards a new era of molecular medicine. And in the era of molecular medicine will be the center of the universe. It's where the molecules are.

Balintfy: Thanks again to Dr. Carolyn Compton, Director of the Biorepositories and Biospecimen Research Office at the National Cancer Institute. For more information about her office and bio-specimens, visit the website biospecimens.cancer.gov.