Cancer Treatment Research

An IV drip — Credit: National Cancer Institute

The Importance of Cancer Treatment Research

Research on the treatment of cancer is fundamental to improving outcomes for all patients affected by the disease. Treatment advances, in combination with innovative diagnostic tools, are leading to therapies that are increasingly tailored to the cancer’s unique traits.

But despite the tremendous progress made in recent decades in treating many types of cancer, effective therapies are still lacking for some cancers, including liver cancer, pancreatic cancer, and certain types of adult and pediatric brain cancer. And even when effective therapies are available, these often stop working as the cancer develops resistance to the treatment.

Also, many people with cancer experience severe side effects of the disease and its treatment. Some of these toxic effects have long-term consequences, including an increased risk of a second cancer. For childhood cancer survivors in particular, the long-term effects of cancer treatment can have a lifelong impact on their quality of life.

NCI-supported researchers are developing more effective and potentially less toxic treatments, such as targeted therapies, immunotherapies, and cancer vaccines, that are designed to spare healthy tissues more so than systemic treatments. In parallel, researchers continue to improve therapies that have existed for decades, such as chemotherapy, radiation therapy, and surgery. And some studies test whether less intensive therapy, with fewer side effects during and after treatment, can still effectively treat cancer.

Thanks to NCI-funded research, patients with cancer have a greater number of therapeutic options than ever before, many of which are more effective and less toxic than earlier options. But more research is needed to ensure the most effective treatment possible, including better ways to use existing therapies in combination, while maintaining the highest possible quality of life for every individual with cancer.

Selected NCI Activities in Cancer Treatment Research

For more than 50 years, NCI has played an active role in cancer drug development—from conducting preclinical studies in the laboratory to testing potential therapies in humans.

NCI researchers conduct clinical trials to test cancer treatments at the National Institutes of Health in Bethesda, MD, and the institute sponsors trials at cancer centers, hospitals, and community practices around the country. The Cancer Therapy Evaluation Program (CTEP) functions as the institute’s primary clinical evaluator of new anticancer agents, radiation treatments, and surgical methods.

While many companies and institutions around the world conduct research on cancer treatments, NCI meets needs that industry does not. These include developing and testing treatments for rare cancers and conducting trials to test the safety and effectiveness of using less treatment or no treatment at all.

Examples of NCI-supported activities in treatment research include:

Discovering New Cancer Drugs

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NCI Treatment Research and the National Cancer Plan

NCI supports a broad variety of research that aligns with the National Cancer Plan’s goal to develop effective treatments. Read about the plan and this goal.

NCI’s Developmental Therapeutics Program (DTP) provides free services and resources to the academic and private-sector research communities worldwide to facilitate the discovery and development of new anticancer agents, including targeted therapies that work through cancer’s unique genetic alterations. DTP supports all stages along the critical path of preclinical drug discovery and development, from high-throughput tumor cell–based screening to first-in-human clinical trials.
The NCI-60 Human Tumor Cell Lines Screen, which includes 60 human tumor cell lines representing nine different types of cancer, is a free resource available to the research community worldwide to evaluate compounds for anticancer activity. The NCI-60 screen tests up to 7,000 compounds yearly and prioritizes compounds with promising anticancer potential for further evaluation.
The Comparative Oncology Program is a unique program that helps researchers improve the assessment of novel therapies for humans by treating pet animals with naturally occurring cancers. The program gives these animals the benefit of cutting-edge research and therapy, and it provides researchers a better understanding of cancer biology and cancer's response to treatment.

Understanding Treatment Response

The Acquired Resistance to Therapy Network (ARTNet) uses team science to study the mechanisms of acquired resistance to cancer therapies and disease recurrence. This program replaces and builds on progress made by the Drug Resistance and Sensitivity Network (DRSN), an NCI and Cancer Moonshot–created program to explore why some cancer cells are innately sensitive to treatment and to identify strategies for circumventing drug resistance in tumors.
NCI contributes to the international Human Cancer Models Initiative, which generates novel human tumor-derived culture models with the goal of creating cancer models that replicate patients’ tumors as faithfully as possible. The models are annotated with genomic and clinical data and are available to the wider research community to define cancer pathways, determine mechanisms of drug resistance, and assess responses to small molecules.

Improving Current Cancer Treatments

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NCI FY26 Annual Plan & Professional Judgment Budget Proposal

Each year, NCI prepares a plan for advancing cancer research and proposes the budget required to fund a broad research portfolio.

NCI’s first-of-its kind precision medicine trial called Molecular Analysis for Therapy Choice (NCI-MATCH) tested the effectiveness of treating tumors in adults and children based on matching targeted therapies to specific genetic alterations in the tumors, regardless of tumor type. Using information learned from NCI-MATCH, three more precision medicine trials—called ComboMATCH, MyeloMATCH, and iMATCH—will test whether drug combinations can overcome resistance to treatment, evaluate new treatments for myeloid leukemia and myelodysplastic syndrome, and examine immune profiles and tumor markers for responsiveness to immunotherapy, respectively.
NCI’s Radiation Research Program (RRP) provides expertise to investigators who perform radiotherapy studies and helps direct radiation research. Methods that more precisely target radiation therapy to tumors while sparing as much normal tissue as possible are critical for maintaining patients’ quality of life and improving cure rates.
Immunotherapy is a type of cancer treatment that helps cells in a patient’s own immune system detect and eliminate cancer, including some difficult-to-treat tumors. Researchers are working collaboratively through the Immuno-Oncology Translational Network (IOTN) and the Pediatric Immunotherapy Network (PIN) to speed up the development of new immunotherapies to treat and prevent adult and pediatric cancers.
NCI brings together researchers from across the institute and the National Institutes of Health to work on cancer immunology and immunotherapy research to discover, develop, and deliver immunotherapy approaches that can prevent and treat cancer and cancer-associated viral diseases. This work has pioneered important research on the basic mechanisms of immune response, including how immune system cells and cancer cells interact, and on the development of better vaccines and immunotherapies.

Moving Discoveries into the Clinic

The Translational Research Program (TRP) supports efforts through the Specialized Programs of Excellence (SPOREs) to translate novel scientific discoveries from the laboratory to the clinic for testing in humans. This work includes determining the biological basis for observations made in cancer patients or in populations at risk for cancer.
The NCI Experimental Therapeutics Program (NExT) focuses on advancing discoveries in basic and clinical research into new therapies to treat cancer patients. This translational research effort unites the drug development expertise of multiple NCI research programs and the facilities at the NIH Clinical Center to advance new therapeutic interventions from both the private and public sectors.

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Supportive Care, Symptom Management, and the National Cancer Plan

NCI supports research on supportive care and symptom management to improve the lives of people living with cancer. This research aligns with the National Cancer Plan’s goal to deliver optimal care. Read about the plan and this goal.

Supportive Care and Symptom Management

The Supportive Care and Symptom Management program focuses on the prevention and treatment of acute and chronic symptoms and side effects related to cancer and its treatment, such as fatigue, musculoskeletal pain, nerve damage, and fertility issues. Researchers also study the effect of treatment on cancer patients' quality of life and the psychosocial issues and strategies for care at the end of life.
NCI has established a collaborative research consortium, called Improving the Management of Symptoms during and following Cancer Treatment (IMPACT), to determine the best approaches for symptom management in cancer care delivery. Research centers within this consortium are testing integrated symptom monitoring and management systems in the clinic and analyzing the effects of those systems on patient outcomes, cancer treatment delivery, and health care utilization using randomized designs.