As the organization that represents and connects the global community of clinicians who discover new treatments for cancer and deliver the latest advances to patients, ASCO has developed, for the first time, a set of Research Priorities to Accelerate Progress Against Cancer. These priorities represent promising areas of research that urgently need greater attention and have the potential to significantly improve the knowledge base for clinical decision-making and address vital unmet needs in cancer care.
The current list reflects ASCO’s mission—conquering cancer through research, education, and promotion of the highest quality patient care—as well as the diversity of needs and opportunities in oncology. It focuses on cancer prevention strategies, increasing equity in access to and participation in research, optimizing treatment, and improving long-term health for the growing number of cancer survivors around the world.
Over time, ASCO’s Research Priorities will evolve with the cancer research landscape and will be periodically updated to reflect advancing science and unmet clinical needs. Current priorities are listed in no particular order:
Identify Strategies That Better Predict Response to Immunotherapies
Cancer immunotherapy encompasses a broad range of medicines and treatment approaches, including vaccines, immune checkpoint inhibitors, and most recently, cellular therapies. These interventions have improved the outlook for multiple cancers, producing long-lasting remissions that can last for years in some patients. At present, however, long-term disease control occurs in just a minority of patients. In addition, immunotherapies can cause substantial adverse effects that can be life-threatening and, in some cases, permanent. Methods to identify patients most likely to benefit from immunotherapy and those at high risk for severe adverse events are urgently needed. The ability to adequately assess benefits and risks of immunotherapy for each individual will lead to better outcomes for patients.
Priority focus areas:
- Identify factors that predict response, long-term disease control, prolonged survival, treatment resistance, and adverse events for all types of immunotherapies
- Develop blood- and tissue-based biomarkers and novel immune-response signatures that predict treatment benefit
- Develop predictive models and algorithms that assign risk of severe immune-related toxicities based on readily available patient data
Better Define the Patient Populations That Benefit From Postoperative (Adjuvant) Therapy
A wide range of therapies are recommended to patients after surgery. These therapies, referred to as adjuvant treatments, aim to reduce the risk of recurrence and cancer-related death. Although such therapy has been associated with dramatic improvements in survival for some patients, studies have shown that the risks can outweigh the benefits for others. It is important to ensure that patients who receive adjuvant therapy are the ones most likely to benefit. Eliminating its use in those who do not benefit will be an important step in optimizing care and eliminating unnecessary adverse effects and costs for patients in whom the benefits are unlikely to outweigh the risks.
Priority focus areas:
- Determine factors that identify patients most likely to benefit, or those unlikely to benefit, from adjuvant therapy, including, but not limited to, clinical, pathologic, genomic, biochemical, immunologic, and environmental or social factors
- Develop analytically and clinically valid biomarker tests with proven clinical utility to identify recurrence risk after treatment of the primary tumor and determine the best options for patients with different degrees of risk
A Path Forward to Increase Access to Clinical Trials
ASCO is working with key stakeholders to address under-representation of certain populations in clinical trials. Recently, the society issued a policy statement on the financial barriers that prevent patients from participating in clinical trials. ASCO’s recommendations include the following: improve insurance coverage of routine care costs for clinical trial participants, increase transparency about out-of-pocket costs, remove impediments to ethically appropriate direct payments to patients, and incentivize research on the financial costs of trial participation.
This statement builds on ASCO’s work with Friends of Cancer Research to address under-representation in cancer clinical trials through more inclusive eligibility criteria. The two organizations developed comprehensive recommendations to address eligibility criteria in the following five areas: minimum age requirements for trial enrollment, HIV/AIDS status, brain metastases, organ dysfunction, and prior and concurrent malignancies.
Translate Innovations in Cellular Therapies to Solid Tumors
Recent FDA approvals of chimeric antigen receptor (CAR) T-cell therapies in leukemia and lymphoma are true milestones in cancer therapy, as signified by ASCO’s 2018 Advance of the Year designation. Although cellular therapies that use a patient’s modified cells to harness the immune system are transforming care for some patients with blood-based cancers, there are limited data to show whether this strategy can be expanded to patients with solid tumors.
Priority focus areas:
- Identify and validate novel antigenic targets uniquely present in solid tumors
- Explore the safety and activity of promising cellular therapies in solid tumors
- Develop strategies that mitigate current challenges in delivering cellular therapies to patients, including exploring the use of cellular products that do not have to be individually manufactured for each patient
- Examine and optimize the cancer care delivery systems needed to safely administer cellular therapies to all who might benefit
Increase Precision Medicine Research and Treatment Approaches in Pediatric Cancers
Genomic tools have been widely deployed for adult patients to characterize common mutations across different types of cancer. In certain cancers, the use of these tools has accelerated development of new targeted therapies that have improved and extended patients’ lives. Despite this success in adult patients, precision medicine treatment approaches have yet to be widely integrated into the treatment of pediatric cancers.
Priority focus areas:
- Identify genomic or other molecular alterations in pediatric cancers that can serve as potentially actionable treatment targets
- Develop effective therapeutic agents that can target genomic or other molecular alterations in childhood cancers
- Explore the efficacy of existing targeted therapies in pediatric patients with tumors that have mutations shown to be responsive to medicines that work in adult populations
Optimize Care for Older Adults With Cancer
Although adults age 65 years and older represent the majority of people with cancer, few cancer clinical trials focus specifically on this population. Older patients who do participate in clinical trials are generally not representative of the older patients who oncologists typically see in daily practice. As a result, clinicians face challenges applying clinical trial data to older patients who may have additional health conditions, varying levels of functional ability, and different goals from clinical trial participants. The lack of evidence in this area combined with the inherent diversity of aging populations impedes the delivery of high-quality care for the largest and most rapidly growing segment of patients with cancer.
Priority focus areas:
- Develop standardized methods to characterize physiologic aging, such as geriatric assessment and biomarkers of aging, to more reliably predict risk of treatment-related adverse effects in older patients with cancer
- Investigate the impact of cancer treatment on physical function, cognition, and quality-of-life to inform tolerability of cancer therapies in older people
- Investigate the efficacy and toxicity of therapies among older adults most under-represented in clinical trials, such as those with impaired functional status, comorbidities, or frailty
- Conduct clinical trials testing the role of geriatric assessment-guided management to improve outcomes using personalized care; important focus areas include strategies that minimize undertreatment of fit patients and overtreatment of vulnerable or frail patients, supportive care interventions, and care delivery interventions
Increase Equitable Access to Cancer Clinical Trials
Certain patient populations are consistently under-represented in cancer clinical trials. These include patients from racial and ethnic minorities, rural areas, and lower socioeconomic groups and patients older than age 65 years as well as adolescents and young adults age 15 to 39 years. Decreased representation among these groups can limit access to the promising treatments offered through these trials and means that research findings may not fully account for the diversity of biologic, social, and cultural factors that influence outcomes. Additional research is needed to ensure that every patient with cancer, regardless of race, ethnicity, age, geographic location, or socioeconomic status, benefits from research advances.
Priority focus areas:
- Improve understanding of the barriers to trial enrollment among various under-represented groups, taking into consideration patient, practice, community, and trial-specific factors
- Develop and test interventions that enhance clinical trial enrollment among under-represented populations (examples may include use of educational tools, telehealth, and community-based involvement and participatory research)
- Evaluate novel strategies to improve access to clinical research resources in areas with large proportions of under-represented minorities
- Develop mechanisms that improve awareness and education about clinical trials among under-represented groups and the physicians treating them
- Investigate differences in cancer incidence, prevalence, natural history of disease, and treatment experience, including efficacy and toxicity, among under-represented populations
Reduce the Long-Term Consequences of Cancer Treatment
Advances in cancer treatment have resulted in a record number of cancer survivors—more than 15.5 million.63 Although this is a tremendous accomplishment, survivors still face long-term consequences of cancer, including adverse effects of cancer therapies that affect quality of life. These adverse effects, which commonly include peripheral neuropathy, cognitive impairment, and cardiotoxicity, pose a substantial burden not only to patients but also to the health care system.
Priority focus areas:
- Identify genetic variants associated with increased risk of treatment-related toxicities
- Deepen understanding of the underlying mechanisms of toxicities from targeted treatments, determine their contribution to long-term effects, and develop novel strategies to mitigate or eliminate such toxicities
- Develop new tools to facilitate long-term tracking of patient outcomes that include patient-reported outcome measures
Reduce Obesity’s Impact on Cancer Incidence and Outcomes
The incidence of obesity has dramatically increased over the past several decades.64 Despite being the second leading preventable cause of cancer, a recent ASCO survey14 found that only 35% of Americans recognize excess body weight as a cancer risk factor. Obesity is associated with poorer cancer survival and can contribute to increased risk of treatment-related adverse effects. If current trends continue over the next 20 years, it is estimated that obesity will lead to more than 500,000 additional cases of cancer each year in the United States65 and will surpass smoking as the leading preventable cause of cancer.
Priority focus areas:
- Improve the understanding of the mechanisms through which weight and energy balance, including physical activity and dietary factors, contribute to cancer development and progression
- Investigate how obesity affects response to therapy, risk of cancer recurrence, and long-term cancer outcomes
- Assess the impact of energy balance interventions, such as weight loss, increased physical activity, and improved dietary quality, on cancer risk, recurrence, and mortality
- Identify effective interventions that optimize energy balance in people at risk and who are living with cancer
Identify Strategies to Detect and Treat Premalignant Lesions
Many cancers begin as high-risk lesions that invariably progress to invasive cancer. Currently, little is known about the genetic makeup, heterogeneity, microenvironment, and what causes some of these lesions to progress to invasive cancer. Increased knowledge will help guide new approaches to intercept and eradicate high-risk lesions before their transformation to malignancy.
Priority focus areas:
- Identify specific molecular pathways that drive progression of preinvasive lesions to invasive cancer and develop interventions that can delay or prevent progression to malignancy
- Identify features of the microenvironment of premalignant lesions that are associated with progression to invasive disease
- Investigate novel methods for evaluation of premalignant lesions to better inform the risk or likelihood of progression to invasive disease
Voices for Cancer Research: Renee Bakos-Pournaras and Yano Pournaras
“We wouldn’t be here today if it weren’t for what research did for us in 2009. I never even had to think about my son participating in a clinical trial–some other mother gave permission for her child so that my child could be in the position he is in today. It’s a domino effect.”
In 2008, 2-year-old Yano’s health began to deteriorate. He developed fevers that would not subside, lost his appetite, and had trouble walking. His mother, Renee, knew something was wrong. “Things were just off, things were not right,” she said.
Yano’s condition did not improve after multiple visits to the doctor, many rounds of bloodwork, and treatment with intravenous antibiotics. Then one morning he woke up with what appeared to be bruises around his eye sockets. Renee rushed him to the hospital where he underwent scans that revealed a large tumor on his spine. On his third birthday, Yano was diagnosed with stage IV terminal cancer.
Less than 24 hours after his diagnosis, Renee returned to her hometown of Chicago with Yano to begin his treatment. Yano received surgery, chemotherapy, radiation, and stem cell transplants as part of his care. His mother was determined to save his life and served as his biggest advocate, ensuring he received the latest treatment options available.
“He went on almost every clinical trial the doctors presented us with. We tried everything, literally everything,” she said. One of the treatments he received as part of a clinical trial is now a standard of care for patients with high-risk neuroblastoma.
Today Yano is a thriving 12-year-old, thanks to the incredible care of his doctors and the clinical trials he was offered. Based on his experience with cancer, Yano is now working on an anti-bullying campaign aimed at helping children with cancer. He knows first-hand that side effects from treatment, such as hair loss, can make children with cancer feel different from their peers and wants to help all children understand and overcome these perceived differences.
Renee has great admiration for her son, “He’s truly my miracle.” Once Yano finished treatment and went into remission, he officially graduated into long-term care and was introduced to his current oncologist, Dr. Henderson. “Yano’s doctors are the most compassionate people I have ever met. They know all the side effects of his treatments and they are constantly searching for ways to make things better because they truly care,” said Renee.
Voices for Cancer Research: Tara Henderson, MD, MPH
“Pediatric oncology is one of the great success stories of medicine because of our nation’s commitment to curing this disease.”
Fascinated by science at a young age, Dr. Henderson participated in a cancer research laboratory program during her junior year of high school. The experience led her to combine her knack for science and desire to positively impact the world with a career in cancer research.
Today, Dr. Henderson directs a cancer survivor clinic in one of the biggest cities in the United States. The clinic’s program is aimed at preventing and treating the long-term health issues that pediatric and adult survivors of cancer may face after treatment. Her ongoing research efforts, many of which are federally funded, are dedicated to keeping patients healthy for the rest of their lives.
“I wake up every day excited to be part of the amazing success story of pediatric oncology,” said Dr. Henderson. “I’m working hard to make sure that all kids can live healthy lives like my own children.”
Dr. Henderson’s current research focuses on better understanding the lingering effects of treatments for children with high-risk neuroblastoma. She wants to ensure that the recent advances in the treatment of pediatric cancers don’t lose their momentum. She notes that the overall survival rate for children with cancer is now more than 80%,63* compared with 58%in the mid-1970s. 63** Many of these pioneering research advances can be attributed to clinical trials and studies funded by the federal government.
While Dr. Henderson is proud of the successes in her field, she acknowledges that we still have a long way to go. She believes that federal investment in cancer research must continue now to “make sure that the progress continues in 10, 20, or 30 years for children all over the world.” She envisions a future where no child dies of cancer and aspires to ensure a future where no child dies of the consequences of cancer treatment, either.
Dr. Henderson is a pediatric oncologist and director of the Childhood Cancer Survivors Center at University of Chicago Medicine. Disclosures for Dr. Henderson include research funding from Seattle Genetics and other relationships with the company.
To learn more about why Dr. Henderson lives to conquer cancer, visit asco.org/live-to-conquer-cancer.