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.
All patients with cancer will have safe and effective treatments.
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
Highlighted Areas in Cancer Treatment Research
Cancer Treatment Research was originally published by the National Cancer Institute. Updated August 30, 2020.