Annual Progress Report from the National Cancer Institute
W. Kimryn Rathmell, MD, PhD
Being impatient for patients and survivors with cancer
by W. Kimryn Rathmell, MD, PhD, director of the National Cancer Institute
I’m thrilled to write this year’s annual progress report for Coping Magazine readers as the new, 17th director of the National Cancer Institute.
Throughout my career, I have been both a physician and a researcher. I’ve had days in the lab studying how cancer behaves at the microscopic level and days at the bedside with patients and their loved ones where their cancer is not visible, but their symptoms are. This dual physician-scientist role has taught me to think about cancer from multiple angles and, importantly, to focus on people at the center of our work.
Now as the director of the National Cancer Institute, I have an even broader perspective. We have a growing number of cancer survivors in the country, projected to increase to 22.5 million survivors by 2032, that we must consider at the center of all that we do. NCI supports the highest quality cancer research—this is essential to developing more effective approaches for cancer treatment, diagnosis, prevention, and survivorship. Our ultimate impact is helping drive faster progress for patients and survivors with cancer, and those at risk for cancer, everywhere. I am happy to report on meaningful advances NCI has made since last year, as well as new opportunities to be optimistic about.
Transforming the way we include participants in clinical research
In the past three decades—during which NCI has continuously shared annual progress with Coping readers—we as a society have witnessed significant progress in cancer research and care. Examples include immunotherapy, scientific discoveries about key drivers of cancer, cutting-edge technologies that improve cancer detection or predict treatment response, and methods that improve quality of life and wellbeing for cancer survivors.
An important part of the research process is translating what we discover in the lab into new options for cancer treatment, detection, prevention, and survivorship approaches. The outcomes of research must reach everyone who can benefit from them, even and especially those with financial, language, or other barriers to optimal care. The researcher in me understands that science takes time. The physician in me is impatient for patients and survivors who have run out of options.
NCI is doubling down to speed progress in clinical research, the essential step required to find better ways to beat cancer. Here are a few examples of exciting progress we’ve led in the past year.
Pragmatic studies
Ensuring that scientific insights lead to real advances for people requires testing new leads and proving they work through clinical research. But many studies are not as efficient as they need to be. So NCI is making important changes in how clinical research is done, through our Clinical Trials Innovation Unit, which focuses on “pragmatic studies,” or those that address the most pressing questions with direct relevance to patients, such as whether a combination therapy for advanced lung cancer, using drugs that are already approved, improves survival (the Pragmatica-Lung study). Pragmatic studies enroll quickly due to broader enrollment criteria and simpler data-gathering requirements.
Bringing research to communities
Too many people who are eligible to participate in clinical research are unable to, due to the costs and complicated logistics of being far from a study center. NCI recently created an expert group charged with investigating ways to bring research into remote communities. People with lived experience of cancer must be welcomed as true partners in progress to make sure the onus is on the researchers, not the patients, to make clinical research happen. This is an area of high potential that has not been addressed enough. We also launched a pilot Virtual Clinical Trials Office in response to the health care staffing shortages during the COVID-19 pandemic. This centralized, online model boosts clinical research staff capacity for screening patients for eligibility, enrolling patients to studies, educating patients about research, and performing data entry.
Addressing access is also about leadership that champions these initiatives. NCI recently launched the Cancer Equity Leaders, a diverse group of leading experts across the country that will reimagine the future of cancer health equity in terms of the workforce and community outreach and engagement.
Making data more robust and accessible
The Childhood Cancer Data Initiative (CCDI) continues to add new datasets from children with cancer around the country and the world; expand enrollment for molecular testing, including for children with rare cancers; and generate resources and infrastructure for the childhood cancer community. The initiative recently launched the Childhood Cancer Clinical Data Commons (C3DC), an open-access database that provides pediatric cancer clinical data that is standardized to common data elements, empowering researchers to conduct important studies and analyses with more precision and efficiency. CCDI is a promising testing ground for data approaches that will be applicable in other populations and for other cancer types as well.
Research participants should be owners of the data they contribute to research—of their data. Toward this end, NCI has joined the Advanced Research Projects Agency for Health (ARPA-H) to develop an advanced toolbox, called the Biomedical Data Fabric, that will make data sharing and use easier for patients, clinicians, and researchers. This is a big step forward in putting data into the hands of patients.
Innovations in screening and early detection
I always say the best cancer is the one that is prevented. The next best is the one that is detected early enough so that it can be swiftly treated and eliminated. Screening and early detection can be lifesaving. Earlier this year, NCI launched a clinical trials network to evaluate emerging technologies for cancer screening. The Cancer Screening Research Network plans to launch a pilot in 2025—the Vanguard Study on Multi-Cancer Detection—to evaluate the feasibility, benefits, and harms of using blood tests to detect multiple types of cancer.
NCI is also putting into the hands of people knowledge about their personal risk for cancer. Earlier this year, I was honored to announce, at the White House Cervical Cancer Forum, a multisite study to evaluate self-testing for HPV to address those considered to be in the “last mile,” or those at risk for cervical cancer but who lack access to screening. This recently launched multisite study is the Self-collection for HPV testing to Improve Cervical Cancer Prevention (SHIP) Trial, and I couldn’t be more excited about the potential of modern cancer screening technology to accelerate progress toward eliminating cervical cancer.
Engaging people with cancer as true partners
Putting people at the center of our research includes personalizing approaches to treatment. NCI has launched new editions of the MATCH (Molecular Analysis for Therapy Choice) studies testing therapeutic options based on specific genetic drivers of cancer. One of these studies, MyeloMATCH, just opened and, for the first time, offers people with acute myeloid leukemia and myelodysplastic syndromes an opportunity to enroll in a precision medicine trial at any point during their disease. The ComboMATCH study is also open for enrollment and is the largest study of its kind to test combinations of precision cancer drugs targeting specific genetic changes.
A highly personalized form of treatment is using a patient’s own immune system as cancer therapy. In the last several months, new breakthrough research, pioneered at NCI over decades, has led to evidence that a cellular immunotherapy approach may be effective in treating solid tumors. Even more recently, this approach has shown potential for advanced cancer that has spread.
We’re not slowing down
We know we’ll have beaten cancer when it becomes a manageable disease with effective treatment options, and, in most cases, is prevented from ever occurring. I’m a big believer in consistent progress leading to transformative breakthroughs, like the ones I shared in this message. We won’t slow down until there is no more fear associated with the word “cancer.”
The National Cancer Plan, launched last year, describes the progress needed to create that future that we want, in two broad areas: health-centric goals (mirroring the patient experience) and empowering goals (societal forces that enhance efforts of the health-centric goals). The health-centric goals of the plan are prevention, early detection, treatment, and care delivery. The empowering areas are data utility, equity, workforce, and engaging every person.
While NCI is committed to driving progress toward all eight goals, engaging every person is now recognized as more important than ever before. We are in a new, modern era where the perspectives of patients, survivors, and advocates are equally valued alongside those in the traditional science and medical professions. I hope that many of you reading will consider how you can get involved, whether by participating in clinical research, scheduling recommended cancer screenings, or learning more about advocacy. Your participation will supercharge our national effort to end cancer as we know it.
Since 1994, the National Cancer Institute has written an exclusive annual report for Coping.
