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Cancer Treatment Research

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. Despite the tremendous progress made in recent decades in treating many types of cancer, effective therapies are still lacking for some forms of the disease, including liver cancer, pancreatic cancer, and certain types of adult and pediatric brain cancer. More than 600,000 people are projected to die from cancer in the United States in 2020.

Also, too many patients whose cancer has been successfully treated experience long-term adverse effects of the disease and its treatment, including increased risk of a second cancer. Therefore, cancer treatment research includes developing ways to prevent or lessen the side effects of treatment. More research is needed to ensure that all patients with cancer have safe and effective therapies and the highest possible quality of life.

Thanks to NCI-funded research, patients with cancer have a greater number of more-effective and less-toxic therapeutic options than ever before. NCI has played a vital role in cancer drug discovery and development for more than 50 years. NCI’s support for cancer treatment research extends from studies of the fundamental biology of cancer, the development of treatments that target cancer cell abnormalities, and the testing of new cancer therapies in clinical trials.

NCI’s contributions are reflected in the fact that:

  • Approximately half of the drugs currently used to treat patients with cancer were discovered and/or developed by NCI-supported researchers. These include imatinib (Gleevec), the first small-molecule molecularly targeted therapy; ipilimumab (Yervoy), the first immune checkpoint inhibitor; and tisagenlecleucel (Kymriah), the first genetically engineered cell-based immunotherapy.
  • In 2019 alone, 11 new cancer treatments were approved by the Food and Drug Administration, and NCI-funding contributed to the development and/or testing of most of them.
  • A study published in 2019 showed that nearly half of the phase 3 clinical trials conducted by the SWOG Cancer Research Network, one of five National Clinical Trials Network clinical research groups, were associated with changes in cancer clinical practice guidelines or new drug approvals.

The Future of Cancer Treatment Research

Breakthroughs in molecularly targeted therapies and immunotherapy have revolutionized the cancer treatment landscape for patients. Additional cancer treatment innovations are on the horizon. For example, recent research is creating optimism that, one day, there may be targeted treatments for so-called “undruggable” cancer targets, including the oncoproteins RAS and MYC, and for restoring the tumor-suppressor function of proteins such as p53 and PTEN. Although more development and clinical testing are needed, the availability of these targeted treatments will be a hallmark of unprecedented progress for patients who have few therapeutic options.

Many other important research opportunities exist to improve the care and treatment of the individual patient. One day, not only will it be possible to molecularly characterize a patient’s cancer cells, but the cellular components of their tumor—and even the composition of their intestinal or tumor microbiome—will inform treatment decisions. With this information, doctors will select therapies, or combinations of therapies, for each patient and avoid ones that will have unacceptable side effects. This future will only be possible through additional research investment.

NCI’s Plan for Cancer Treatment Research

NCI prepares an Annual Plan & Budget Proposal for the President and Congress that describes the Institute’s scientific priorities and promising opportunities from across the research continuum. Included in the plan is the following strategic vision and approach for improving cancer treatment.

Vision

All patients with cancer will have safe and effective treatments.

Approach

Additional investments in cancer treatment research will further improve the outlook for both adults and children with cancer. Fully realizing the potential to identify, study, and test new cancer therapies requires additional research to achieve the following goals:

1) Discover and develop new cancer therapies, including those that involve molecularly targeted therapies and immunotherapies, as well as treatment combinations

Treatments that target the molecular changes in a person’s cancer and immunotherapies that unleash the power of the immune system against the disease are revolutionizing the potential of cancer care. Because these newer therapies provide durable clinical benefits to only a small proportion of patients, new or revised therapeutic approaches must be developed. Among our major objectives of this research are:

  • Identifying and characterizing new targets for cancer treatment, such as abnormal proteins that are responsible for cancer cell survival, growth, and spread
  • Developing new ways to leverage the rapid progress in cancer immunotherapy to benefit more patients, including identifying predictive biomarkers of efficacy or toxicity, developing novel immune targets, and combining therapies
  • Understanding the mechanisms of drug resistance, a major cause of treatment failure in patients, and developing strategies that target these mechanisms, including the use of combination therapies
  • Identifying and developing additional biomarkers to monitor treatment benefits and harms and to aid clinicians in selecting the most appropriate treatments for patients

2) Improve traditional cancer treatment approaches, including surgery, radiation therapy, and chemotherapy

Surgery, radiation therapy, and chemotherapy remain important options for cancer treatment. NCI funds research to improve the effectiveness and use of these treatments. We must learn to use them more precisely and minimize their side effects. NCI’s major objectives include:

  • Understanding how to combine therapies, including different types of treatment (for example, radiotherapy with immunotherapy)
  • Tailoring treatments to avoid overtreatment and avoidable toxic side effects (for example, by conducting de-escalation studies)
  • Advancing the development of precision radiotherapy to target tumors more precisely and spare the surrounding normal tissue from radiation damage
  • Supporting innovations in cancer surgery, including approaches to minimize the impact on normal tissues