A Transformative Moment for Cancer Research

A Transformative Moment for Cancer Research Photo by Ernie Branson

Annual Progress Report from the National Cancer Institute

by Douglas R. Lowy, MD, acting director of the National Cancer Institute

“An investment in knowledge,” Benjamin Franklin once wrote, “pays the best interest.”

Although he was writing specifically about financial advice, Franklin’s words could no doubt apply to decades of scientific investment that is opening up numerous, exciting opportunities for progress against cancer. From the rapid emergence of immune-based therapies to the continued advances in cancer genomics that are forever altering how we think about and approach prevention, diagnosis, and treatment – this is truly a transformative moment for cancer research.

As the leader of the U.S. National Cancer Program, the National Cancer Institute is focused on seizing this moment. Over the last year, since the readers of Coping last received an annual report from NCI, I can confidently say that we have made important progress. And during that time, much has changed.

In March 2015, after five years as NCI Director, Dr. Harold Varmus stepped down from the position. Among his many achievements while at NCI, Dr. Varmus launched important initiatives that will have a lasting impact on our ability to reduce the burden of cancer not only in the United States but also in other countries. 

I was honored to be asked by President Obama to serve as acting NCI director, and it was an offer that I enthusiastically accepted. Because after more than three decades of conducting research at NCI, I believe we have reached a crucial moment in cancer research. 

Perhaps at no other moment in history, in fact, have we had such a singular opportunity to make monumental leaps in our understanding of the collection of diseases that we call cancer, and in our ability to prevent and treat it.

Although NCI already supports a large portfolio of cancer health disparities research, it’s one of several areas where I believe the Institute can more sharply focus its efforts.

Part of this belief is rooted in President Obama’s January 2015 announcement that he was launching a Precision Medicine Initiative. The President called precision medicine “one of the biggest opportunities for breakthroughs in medicine that we have ever seen,” and I wholeheartedly agree. NCI will play a critical and leading role in the President’s Precision Medicine Initiative as we seek to make precision medicine a regular part of cancer care in the very near future. 

In general, precision medicine involves routinely using detailed genetic and other molecular information about a person’s cancer to select effective, patient-specific remedies to treat it. For nearly ten years, NCI has been at the forefront of precision medicine – launching important programs like The Cancer Genome Atlas and, more recently, the Exceptional Responders Initiative. A large component of the Precision Medicine Initiative is devoted to cancer, and NCI will continue to initiate new research to explore the benefits of precision medicine in cancer prevention and treatment.

Our focus will be on conducting precision medicine clinical trials, better understanding tumor resistance to treatment, and developing robust information platforms that facilitate the study of the molecular drivers of cancer and the incorporation of precision medicine into everyday patient care.

Perhaps at no other moment in history have we had such a singular opportunity to make monumental leaps in our understanding of cancer.

In June, we announced a rigorous, large-scale precision medicine trial called NCI-MATCH (Molecular Analysis for Therapy Choice). In this clinical trial, which will enroll as many as 1,000 people with different cancer types, participants will be assigned to smaller component trials in which their treatment will be based not on their type of cancer but on certain genetic abnormalities thought to be driving their cancer (based on genomic testing of their tumor samples). Treatments will be selected from more than twenty FDA-approved and investigational agents to “match” a specific genetic abnormality in each person’s tumor.

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Importantly, a pediatric counterpart to NCI-MATCH is being planned that will enroll children with advanced cancer that has progressed despite standard-of-care treatments. This trial will offer a unique opportunity to study whether many of the targeted therapies used in or being studied for use in adults can benefit children with cancer. 

The adult and pediatric NCI-MATCH trials are just two of a series of new precision medicine trials being conducted under the auspices of NCI’s National Clinical Trials Network (NCTN). Last year at this time, the NCTN – the product of a complete revamping of NCI’s prior clinical trials program, known to many as the Cooperative Groups – was just launching. I’m happy to report that the new program is now in full swing and is offering people across the country easy access to all NCI-supported trials, including precision medicine trials such as NCI-MATCH. 

We must remain committed to funding excellent science. Because excellent science, by its very nature, will always promote progress.

Last year, NCI also launched the NCI Community Oncology Research Program (NCORP). This program, which replaced NCI’s previous community-based clinical trials program, will bring cutting-edge trials to more communities across the country – to people where they live. 

NCORP will also conduct studies to assess and improve how everyday cancer care is delivered, and to help address what in my view is an extremely serious public health issue: disparities in cancer incidence and death among racial, ethnic, and socioeconomic groups. Examples of these disparities are discouragingly abundant, such as the higher rate of prostate cancer death among African American men and the high incidence of cervical cancer among Hispanic and Latino women.

Although NCI already supports a large portfolio of cancer health disparities research, it’s one of several areas where I believe the Institute can more sharply focus its efforts. Two other areas in which I think NCI can bring more clarity to our efforts are prevention and early diagnosis and basic research.

With health disparities, I believe it’s critical that we focus on what we consider to be the most serious and important concerns and take the necessary steps to better address this issue. The same holds true for prevention and early diagnosis. We’ve had some important and high-impact successes in prevention – including dramatic reductions in smoking rates and the development of HPV vaccines – but with 1.6 million new U.S. cancer cases and 600,000 cancer deaths forecast for 2015, clearly there are opportunities for greater progress.

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Basic research accounts for approximately half of NCI’s budget. And although that research is often viewed through the prism of its likelihood of leading to a clinical application, such as a new treatment or screening test, many of our most important advances have come from basic research that had no such ambitions. In short, we must remain committed to funding excellent science. Because excellent science, by its very nature, will always promote progress.

Beyond changes at NCI, the past year has seen a number of noteworthy, practice-changing advances – progress that is due in no small part to NCI-conducted and supported research. 

For example, immunotherapies continue to generate much-deserved enthusiasm. The U.S. Food and Drug Administration has already approved immunotherapy drugs known as checkpoint inhibitors for the treatment of melanoma and, in March 2015, for treating people with advanced non-small cell lung cancer. Based on a growing body of data from ongoing clinical trials, many in the research community expect that immunotherapies will become part of our standard treatment arsenal for many cancers. 

The FDA also recently approved a third HPV vaccine that protects against nine different types of the human papillomavirus, seven of which can cause cancer. While we already know that the vaccines protect against cervical cancer, there is emerging evidence that they also protect against other cancers, including head and neck cancer, the rates of which have increased dramatically over the past decade, particularly in men.

And we now have the first approved treatment for children with a high-risk form of neuroblastoma, the most common solid tumor in children – the immunotherapy drug Unituxin. For the better part of two decades, NCI supported the development of this agent, which is now the standard of care for children diagnosed with high-risk neuroblastoma.

In short, I’m happy to report that progress is happening on many fronts. And I look forward to penning this report a year from now, confident that I will have other important successes to share and that our investment in cancer research will continue to lessen the burden of cancer and improve the health and well-being of our nation.

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

If you would like to learn more about precision medicine, the NCI-MATCH trial, or any of the other information presented in this article, call NCI’s Cancer Information Service at 1-800-4-CANCER or visit the NCI website, cancer.gov.

This article was published in Coping® with Cancer magazine, July/August 2015.