2017CLINICAL CANCER ADVANCESA S CO ’ s 1 2 t h A n n u a l R e p o r t o n P r o g r e s s Ag a i n s t C a n ce rI N S I D E A S C O N A M E S A DVA N C E O F T H E Y E A R
EDITORSEXECUTIVE EDITORSHarold Burstein, MD, PhD, FASCO, Dana-Farber Cancer Institute, Boston, MADon S. Dizon, MD, FACP, Massachusetts General Hospital, Boston, MASPECIALTY EDITORSJeanny B. Aragon-Ching, MD, FACP, INOVA Schar Cancer Institute, Fairfax, VANancy N. Baxter, MD, St. Michael’s Hospital, University of Toronto, Toronto, CanadaE. Gabriela Chiorean, MD, University of Washington, Seattle, WAWarren Allen Chow, MD, FACP, City of Hope, Duarte, CAJohn Frederick De Groot, MD, University of Texas MD Anderson Cancer Center, Houston, TXSteven Michael Devine, MD, Ohio State University, Columbus, OHSteven G. DuBois, MD, Dana-Farber Cancer Institute, Boston, MAWafik S. El-Deiry, MD, PhD, FACP, Fox Chase Cancer Center, Philadelphia, PAAndrew S. Epstein, MD, Memorial Sloan Kettering Cancer Center, New York, NYJohn Heymach, MD, PhD, University of Texas MD Anderson Cancer Center, Houston, TXJoshua Adam Jones, MD, MA, University of Pennsylvania Health Systems, Philadelphia, PADeborah Mayer, PhD, AOCN, ANP-BC, University of North Carolina/Lineberger Comprehensive Cancer Center, Chapel Hill, NCRebecca A. Miksad, MD, Beth Israel Deaconess Medical Center, Boston, MANathan A. Pennell, MD, PhD, Cleveland Clinic, Cleveland, OHMichael S. Sabel, MD, FACS, University of Michigan, Ann Arbor, MILynn Mara Schuchter, MD, FASCO, University of Pennsylvania, Philadelphia, PANadine Tung, MD, Beth Israel Deaconess Medical Center, Boston, MAKaren Marie Winkfield, MD, PhD, Wake Forest Baptist Medical Center, Winston-Salem, NCLori J. Wirth, MD, Massachusetts General Hospital, Boston, MAASCO PRESIDENTDaniel F. Hayes, MD, FASCO, FACP, University of Michigan Comprehensive Cancer Center, Ann Arbor, MIASCO PRESIDENT-ELECTBruce E. Johnson, MD, FASCO, Dana-Farber Cancer Institute, Boston, MACHIEF EXECUTIVE OFFICERClifford A. Hudis, MD, FASCO, FACPCHIEF MEDICAL OFFICERRichard L. Schilsky, MD, FASCO, FACPACKNOWLEDGEMENTThe editors would like to thank Lada Krilov, PhD, Senior Specialist, Science Writing,for her work drafting this report.Copyright 2017 American Society of Clinical Oncology. All rights reserved.
TOCTABLE OF CONTENTSA MESSAGE FROM ASCO’S PRESIDENT2EXECUTIVE SUMMARY3ADVANCE OF THE YEAR: IMMUNOTHERAPY 2.0u Progress With Immune Checkpoint Inhibitorsu Emerging Clues on Patient Selectionu Checkpoint Inhibitors Work Well Against Hypermutated Cancers9101516CANCER RISK, PREVENTION AND SCREENINGu Additional Genes Linked to Ovarian Cancer Risk u PancreaticCancer Susceptibility Genes identified,and New Opportunities for Screening and Prevention u BroaderTesting Uncovers Unexpected Cancer PredispositionGene Mutationsu Many Children With Cancer May Carry Hereditary Gene Mutationsu Daily Vitamin B Reduces Skin Cancer Risku New Understanding of Barriers to Cancer Screening Among Latinas1920ADVANCES IN CANCER TREATMENT BEYOND IMMUNOTHERAPYu Targeted Therapyu Combined Modality Therapyu Chemotherapyu New Concerns About Laparoscopic Rectal Cancer Surgeryu Longer Hormone Therapy Further Reduces Breast Cancer Recurrence252731323333ADVANCES IN PATIENT CAREu ASCO Advances Patient Care Through Precision Medicine and Big Datau Better Way to Prevent Chemotherapy-Triggered Nauseau Patient Self-Reporting of Symptoms Improves Careu Patient Navigation Program Improves Compliance With Cancer Therapyu Addressing Health Literacy in the Era of Precision Medicine u Excellent10-year Survival for Patients With Early Prostate Cancer,Regardless of Treatment35363737383940 ADVANCES IN TUMOR BIOLOGYu Melanoma Develops Through Successive Genetic Changesu Therapeutic Options Expand With Precision Medicine Approaches414242LOOKING TO THE FUTUREu Liquid Biopsies Help Personalize Cancer Therapyu Expanding Targeted Therapy Options for Ovarian Cancer u CancerMoonshot: Galvanizing a Renewed Commitment toConquering Cancer43434546REFERENCES47APPENDIXu Additional Notable Advances u ASCOClinical Practice Guidelines5054INDEX OF ADVANCES BY CANCER TYPE552122222324To view this report online and learn more about progress against cancer, visit ASCO.org/CCA.This report was also published in the Journal of Clinical Oncology at ascopubs.org/journal/jco on February 1, 2017.
A MESSAGE FROM ASCO’S PRESIDENTI am pleased to present Clinical Cancer Advances 2017, which highlightsthe most promising advances in patient-oriented cancer research overthe past year. The report gives us an opportunity to reflect on what anexciting time it is for cancer research and how swiftly our understandingof cancer has improved.One year ago, the White House announced the national CancerMoonshot program to accelerate progress against cancer. This sharedvision of progress has reinvigorated the research community, identifiednew areas of scientific collaboration, and raised our ambitions regardingwhat may be possible beyond the progress we have already made.When I entered the field 35 years ago, I could not have imagined where we would be today. We can now detectcancer earlier, target treatments more effectively, and manage adverse effects more effectively to enable patientsto live better, more fulfilling lives. Today, two of three people with cancer live at least 5 years after diagnosis, upfrom roughly one of two in the 1970s.This progress has resulted from decades of incremental advances that have collectively expanded ourunderstanding of the molecular underpinnings of cancer. There is no better current example of this thanASCO’s 2017 Advance of the Year: Immunotherapy 2.0.Over the last year, there has been a wave of new successes with immunotherapy. Research has proven thisapproach can be effective against a wide range of hard-to-treat advanced cancers previously consideredintractable. Researchers are now working to identify biologic markers that can help increase the effectivenessof treatment and determine who is most likely to benefit from immunotherapy. This knowledge will enableoncologists to make evidence-based decisions so as many patients as possible might benefit from this new typeof treatment.Each successive advance builds on the previous hard work of generations of basic, translational, and clinicalcancer researchers. Importantly, the advances described in this report would not have been possible without theindividuals who volunteered to participate in clinical trials as part of their treatment.To turn the promising vision of a cancer moonshot into meaningful advances, we need sustained, robustfederal funding for continued research and innovation. Approximately 30% of the research highlighted in thisreport was funded, at least in part, through federal dollars appropriated to the National Institutes of Health or theNational Cancer Institute. Without this federal investment—unique internationally in scale, duration, and impact fordecades—I fear we may lose the forward momentum needed to further the progress we see highlighted inthis report.Federal lawmakers can further fuel progress by advancing initiatives that facilitate the use of big data to achievethe common good of high-quality care for all patients. Such programs, like ASCO’s CancerLinQ, will rapidlyincrease the pace of progress and dramatically expand the reach of treatment advances to the millions of patientswho are living with cancer today or who will do so in the future. This investment will yield medical, scientific,economic, and societal benefits for years to come.Much work still lies ahead. Many questions remain about how cancer develops and spreads and how best to treatit. As you read through Clinical Cancer Advances 2017, I hope you are as inspired as I am by the gains the clinicalcancer research community has made over the past year and by the promise of a new era of advances just overthe horizon.Daniel F. Hayes, MD, FASCO, FACPASCO President, 2016 to 20172CLINICAL CANCER ADVANCES 2017
u EXECUTIVE SUMMARYCancer is one of the world’s most pressing health care challenges.On the whole, research progress from one year to the next is incremental,and true breakthroughs are exceptional. Nevertheless, every year brings newknowledge and insights that help direct further research and ultimately improvethe outlook for patients with cancer. This report highlights the most importantclinical advances of 2016 and previews where cancer science is headed.Accelerated by the National Cancer Act of 1971 and then by a responsiveresearch infrastructure and increasingly innovative regulatory environment,cancer research today delivers new treatments to patients faster thanever. In just 1 year’s time (from November 2015 through October 2016),the US Food and Drug Administration (FDA) has approved 20 therapiesfor more than a dozen different types of cancer (Table 1). An example ofthis accelerating progress is cancer immunotherapy.CLINICAL CANCER ADVANCES 20173
A hundred years in the making, cancerimmunotherapy is now a standard treatmentoption for people with a growing number ofdifferent cancers. In 2016 alone, the FDA approvedimmunotherapies for advanced forms of lung,kidney, bladder, and head and neck cancers, aswell as Hodgkin lymphoma.For some people with these advanced-stagecancers, the advent of cancer immunotherapyis truly life changing. It often offers the onlychance to live longer and better. And manybelieve that this first wave of success with cancerimmunotherapy is just the beginning.Building on our initial success, a key next stepis to understand why fewer than half of patientscurrently selected for treatment actually benefitfrom immunotherapy and why the benefit, if itoccurs, may be short lived. In 2016, several reportsrevealed early insights into patient and cancercharacteristics (ie, biomarkers) that might predictwhether immunotherapy could work well in anindividual patient. For example, it seems that forsome cancers, the subset of tumors with manygenetic mutations can be more responsive tocurrent immunotherapy options.While we are trying to identify who willbenefit, we are also exploring whether combiningimmunotherapy treatments with one another orwith other cancer treatments, such as radiationtherapy and chemotherapy, might extendCancer by the Numbersthe impact of this new group of therapies.Together, these efforts mark the next phase ofimmunotherapy: Immunotherapy 2.0: ExpandingUse and Refining Patient Selection, the AmericanSociety of Clinical Oncology’s (ASCO) Advance ofthe Year.PRECISION MEDICINEThe advances highlighted in this report attest toa key trend that is driving progress against cancertoday: cancer therapies are becoming increasinglyprecise, thereby enabling a more personalizedapproach to treatment selection. The research intocancer biology is propelling rapid development ofnovel treatments targeting the key molecules thatallow cancers to grow and spread. In 2016 alone,this strategy resulted in new targeted therapiesfor people living with advanced cancers of thelung, breast, and kidney, as well as several hardto-treat forms of blood cancer.Today, new molecular technologies can quicklypinpoint molecular changes in the tumor orfree-floating cancer DNA in the blood. For agrowing number of patients, such changes can bematched to either existing targeted treatmentsor experimental treatments that are being testedin clinical trials. Although this approach is notyet routine, early research suggests it could openviable new treatment options for patients with awide range of hard-to-treat cancers (as describedin Looking to the Future).22M70%uWHAT’S NEXT FOR CANCERIMMUNOTHERAPY?The World Health Organization projects that the number of new cancer diagnoses will reach 22million per year in the next two decades, up from 14 million in 2012. In the same timeframe, cancerrelated deaths may increase by as much as 70%. Seven of 10 deaths resulting from cancer occurin Africa, Asia, and Central and South America, regions of the world with limited access to cancerscreening and treatment.1In the United States, an estimated 1.7 million people were diagnosed with cancer in 2016.2 Because ofthe growing population of older adults and changing demographics, US annual cancer incidence isexpected to reach 2.2 million per year by 2030.3The good news is that, for most people, a diagnosis of cancer is not as grim as it used to be. Today,68% of adults and 81% of children with cancer will be alive at least 5 years after a diagnosis. This isa big improvement from the 1970s, when only 50% of adults and 62% of children were surviving 5years.4 Although annual US cancer death rates have been declining in the last 20 years, the numberof deaths resulting from cancer remains high because of overall population growth.Stewart BW, Wild CP (eds): World Cancer Report 2014. Lyon, France, IARC Press, 20141American Cancer Society: Cancer Facts and Figures 2016. /documents/document/acspc-047079.pdf2Rahib L, Smith BD, Aizenberg R, et al: Projecting cancer incidence and deaths to 2030: The unexpected burden of thyroid, liver, andpancreas cancers in the United States. Cancer Res 74:2913-21, 20143National Institutes of Health: Fact Sheet: Cancer. .aspx?csid57544CLINICAL CANCER ADVANCES 2017
CARING FOR THE WHOLE PATIENTFEDERAL FUNDING SUPPORTSPIONEERING RESEARCHIn the 21st century, people with cancer are notonly living longer than ever, but also enjoyingClinical cancer research in the United States isa better quality of life than before. Indeed, anmade possible through funding from both publicentire field of cancer survivorship has emerged—and private sectors. When it comes to high-risk,studying ways to improve life as well as extendpioneering research, federal funding is oftenit. Although treating the physical illness remainsindispensable and has been a unique asset overa priority, more and more attention is being paidth