Understanding Cancer The Power and Promise of Cancer Research

Annual Progress Report from the National Cancer Institute

Dr. Norman Sharpless  (Photo by H. Darr Beiser)
Each summer, for nearly a quarter-century, Coping® has asked the NCI Director to write an article for the magazine. Having been sworn in as director last October, I am honored to share my thoughts with so many people across the country through Coping®.

Engaging with patients always has been, and always will be, one of the most important parts of my work. I started my career back in the early 1990s as a clinical oncologist caring for patients. I have to admit that, as a new oncologist, I sometimes did not feel up to the task. I was afraid of making a mistake, of missing something important, and of letting my patients down. I read every research article I possibly could and attended clinical oncology conferences in an effort to become the best oncologist I could be and to give my patients the very best possible care. 

Although I loved caring for my patients, I eventually decided that I could have a greater impact if I transitioned from the clinic to the lab. Experiences with numerous patients led me to this decision, but one person, in particular – a charming, upbeat woman in her forties with metastatic breast cancer – remains etched in my mind. 

After learning that her chemotherapy was no longer working, the woman confided in me that her wish was simply to live long enough to see her 11-year-old daughter graduate from high school. While six more years did not seem like much to ask, I knew it was an unlikely possibility given the woefully inadequate options for therapy that were available back then. 

A Time of Hope and Optimism

Fast forward 20 years, and oncologists have more and better treatment options to offer their patients. Many patients are living longer and fuller lives after treatment for their cancer. The most recent Annual Report to the Nation on the Status of Cancer reports a steady decline over the past 15 years in cancer deaths for men, women, and children in all major racial and ethnic groups. In addition, with survival rates continuing to trend upward, the number of U.S. cancer survivors has doubled since the 1990s. 
Discoveries in the cancer field are occurring on a daily basis, and it is truly a time of great hope and optimism.This progress has not occurred by accident. It’s been a hard-won effort, the result of decades of painstaking, intricate basic research to understand the biology of cancer; clinical researchers working around the clock to find new approaches to treating the disease; and passionate survivors, family members, and friends banding together to advocate for increased awareness and research funding. It’s the entire cancer community working in partnership, tirelessly, to accelerate the pace of progress. And we are finally reaping the benefits. Discoveries in the cancer field are occurring on a daily basis, and it is truly a time of great hope and optimism.

One of the major breakthroughs in our understanding of cancer is how we actually think about cancer. What we know now is that cancer is not one disease, but a collection of thousands of biologically distinct diseases. Lung cancer is different from liver cancer or melanoma, for example. And lung cancer, itself, is not even one disease; more than 100 different subtypes of lung cancer have been identified.

In addition, we’ve learned that where the tumor starts growing and how it continues to grow can also differ among patients. And, interestingly, tumors that arise in different organs may be more similar than tumors from the same organ. So, for example, a tumor in a person’s bladder may be more similar molecularly to colon cancer than to bladder cancer. 

Last year, the U.S. Food and Drug Administration approved the first cancer drug in a “site-agnostic” fashion, meaning it doesn’t matter where in the body the tumor originated. If it has a specific genetic marker, it can be treated with the drug pembrolizumab. This drug approval was a huge step forward for precision oncology, an approach to diagnosing and treating cancer based on the molecular features of the disease rather than the location of the tumor.

We have also deepened our understanding of the tumor microenvironment. A tumor isn’t simply a clump of cancer cells growing in isolation. A tumor lives within an environment comprising nearby blood vessels, immune cells, noncancerous cells, hormones, microbes, and lipids. These components and cells have diverse roles: some help the cancer cells survive and spread, while others prevent them from doing so. And they are in constant communication with one another. Researchers are currently investigating ways to block these communication signals to, hopefully, stop cancer cells from spreading.
We owe it to patients to make progress against all cancers, not just some of them.In addition, we have come to appreciate that every person diagnosed with cancer has unique characteristics, including their race, ethnicity, behaviors, beliefs, socioeconomic status, and long-term goals. The interplay between biology and lifestyle is uniquely different in each person and plays a role in how cancer develops and how it responds to treatment. It’s this staggering heterogeneity of cancer that translates into an astronomical level of complexity and, thus, no “magic bullet” for cancer. On the other hand, we have made many incremental and important advances in prevention, early detection, diagnosis, and therapy that, in sum, have moved the needle forward.

Indeed, a greater understanding of how cancer cells interact with other cells in the body have led to new treatments. For example, several new therapies have been developed (called immunotherapy) that target the patient’s immune system to help the body fight cancer cells, as opposed to targeting tumor cells themselves. This is a huge advance, and immunotherapy is now considered an additional pillar of cancer treatment, along with surgery, chemotherapy, 
radiation, and targeted therapy.

Key Focus Areas

As NCI director, I am determined to do everything I can to accelerate the pace of progress. To do that, I spent a good portion of my first six months in my new role on a “listening and learning tour,” engaging with and learning from people across the cancer community – patients, advocates, scientists, and clinicians.

At the end of this tour, I announced four key focus areas for the NCI: Basic Science, Workforce Development, Big Data, and Clinical Trials. These are not new areas for NCI. However, with our new understanding of the heterogeneity of cancer, our approach to studying cancer, providing treatment, and conducting clinical trials must also change. A more intensive focus on these areas (summarized below) will help ensure the research community maximizes our chances for achieving rapid progress in cancer research and care. 
• Basic Science: By investing in basic science, the cancer research community will continue to deepen its understanding of the molecular basis of cancer and make advances in precision medicine and immunotherapy. It will also, ultimately, help us tackle those cancers, such as brain, pancreatic, and liver cancer, that have been resistant to therapy. We owe it to patients to make progress against all cancers, not just some of them.
• Workforce Development: NCI will be dedicating funding of training grants and professional development opportunities to encourage development of the right skills – proficiency in immunobiology, geriatrics, and data science, for example – to be a successful scientist today and in the future. 
• Big Data: Innumerable studies have generated and continue to generate huge amounts of data. Data from every patient, including those we have successfully treated and those we have not, is key to furthering our understanding of cancer and developing new and better treatments. It is critically important that we collect and aggregate data in a way that makes it accessible to and usable by all researchers.
• Clinical Trials: Clinical trials are a primary way scientists discover more effective treatments. They represent hope for all of us in the research community, and most of all for patients. In fact, every one of today’s standard-of-care therapies is available because of past clinical trials. NCI is focused on revamping the way investigators conduct clinical trials. 

We must ensure that clinical trials are more accessible to patients and healthcare providers. Currently, only about 5% of adults with cancer enroll in cancer trials, and many trials never get completed due to insufficient enrollment. That, unfortunately, slows the pace of research and discovery of new treatments. 

NCI is committed to making trials available in community settings across the country where patients from diverse populations live. Trials must be convenient so that more people can enroll and take advantage of state-of-the-art therapies. This would result in a win for patients, a win for community oncologists, and a win for research. The NCI-MATCH precision medicine trial is an example of this innovative trial design, enrolling more than 6,000 patients in cutting-edge therapeutic trials at 1,100 sites across the country.

The Power of Research

What have the power and promise of research given us? Almost every day we learn of new discoveries, advances, and approaches that show promise. I’m happy to say we have options. We have treatments. And, sometimes, we now do have cures. It is my hope that we will soon reach a time when no person with cancer has to ask for just a few more years and when everyone can have not only the expectation, but also the reality, of a long and healthy life.


Since 1994, the National Cancer Institute has written an exclusive annual report for Coping’s July/August Celebration issue.

This article was published in Coping® with Cancer magazine, July/August 2018.