The Value of Cancer Treatment Today
The Fight Against Cancer
Innovative biopharmaceutical research companies are committed to fighting cancer. Biopharmaceutical companies have made tremendous progress in the fight against the more than 200 diseases we call cancer. Continued research has expanded our knowledge of how cancer develops and how to target medicines for specific cancer types — resulting in more effective therapies for patients. Thanks to innovation from America’s biopharmaceutical research companies, the cancer death rate has fallen 34% from its peak in 1991, in large part due to innovations from targeted therapies along with immunotherapy.
Small and Large Molecules Medicines: Why We Need Both to Fight Cancer
Small molecule medicines, which represent the majority of cancer medicines, typically come in the form of a tablet or capsule, are taken by mouth and contain a single chemically synthesized active ingredient. Due to their size, small molecules can more easily reach therapeutic targets inside of cells, cross the blood-brain barrier, and are often available in oral dosage forms which offer greater flexibility and convenience in their administration and ultimately reduce barriers to treatment adherence and factors that can drive health disparities. For cancer specifically, targeted small molecule therapies can act upon specific proteins or genetic material inside cancer cells, causing cancer cells to die. Given cancer begins with genetic changes occurring inside cells, targeted small molecule medicines provide an essential tool in combating the cause of cancer.
Biologics, also referred to as large molecule medicines, in contrast are made by or from living cells, are structurally complex and are generally administered in a doctor’s office or hospital setting via injection or infusion. Due to their larger size, biologics are generally unable to enter cells, but rather are designed to reach therapeutic targets on the surface of cells.
Together, these two types of medicines offer patients and health care providers a wide choice of treatment options that are needed and are indispensable in the treatment arsenal against cancer.
Fact Sheet
40 Years of Cancer Immunotherapy Research
Immunotherapy
Revolutionizing Cancer Care with Immunotherapy
CAR-T
Immunotherapy Through the Years
The 1980s
1980s – Foundational Research Begins
Early 1980s
- Scientists begin to research new ways to use T-cells and monoclonal antibodies to treat cancer. During the same period, the role of the T-cell receptor (TCR), a type of immune cell that recognizes and binds to foreign substances, is determined.
Mid 1980s
- The first immune checkpoint molecule, cytotoxic T-lymphocyte antigen number 4 (CTLA-4), is discovered.
Late 1980s
- First human testing and use of genetically engineered T-cells that can recognize and kill cancer cells.
Scientist injecting liquid into vile
The 1990s
1990s – Foundational Research Continues, Early Therapeutic Successes and Setbacks
Early 1990s
- First tumor-specific antigen, the melanoma antigen gene (MAGE), discovered by melanoma researchers in Belgium in 1991, opens up new ways to use tumor antigens to stimulate the immune system to better fight cancer cells. A second immune checkpoint protein, programmed cell death-1 (PD-1), is discovered by researchers at Kyoto University in Japan (1992).
Mid 1990s
- The concept of modifying Chimeric antigen T-cells (CAR T-cells) is introduced but fails in initial 1990s clinical studies due to technical intricacies and knowledge gaps.
Late 1990s
- The first mAbs for cancer—rituximab for non-Hodgkin’s lymphoma (1997) and trastuzumab for HER2 positive breast cancer (1998)—are approved by the FDA, and the first evidence that gene-expression profiling can distinguish between cancer types is published (1999).
Nurse showing something to patient on tablet
The 2000s
2000s – New Targets Identified and Medicines Developed
2000
- Clinical trials launched to test the first immune checkpoint inhibitor drug containing a mAb targeted against CTLA-4 (ipilimumab for melanoma).
2001
- Two separate in-vivo studies show that certain tumor cells are destroyed by natural killer (NK) cells—a type of white blood cell that has small particles with enzymes that can kill tumor cells or cells infected with a virus—establishing a new mechanism for how NK cells recognize tumor cells and laying the groundwork for them to become key components of multipronged therapeutic strategies for cancer.
2004
- More mAb treatments (including cetuximab and avastin for metastatic colorectal cancer) are approved by the FDA.
2008
- First PD-1 targeted immune checkpoint inhibitor enters Phase I trials.
Woman looking at prescription pill bottle
The 2010s
2010s – Novel Immunotherapies Reach Patients
2010
- The FDA approves first therapeutic cancer vaccine, sipuleucel-T, for prostate cancer.
2011
- The FDA approves first checkpoint inhibitor targeting the CTLA-4 protein, ipilimumab, for metastatic melanoma. It is the first drug of any kind ever shown to extend survival in metastatic melanoma.
2014
- The FDA approves two more immune checkpoint inhibitors, pembrolizumab and nivolumab, both of which target the PD-1 pathway.
2015
- The FDA approves first oncolytic virus therapy, a new class of immunotherapies. Talimogene laherparepvec is approved for metastatic melanoma. It is a genetically engineered virus that has been tweaked to preferentially kill cancer cells.
2016
- The FDA approves fourth checkpoint inhibitor, atezolizumab, for bladder cancer. It targets the PD-1 pathway and is later approved for use in a total of six different cancers.
2017
- The FDA approves first CAR T-cell therapy, tisagenlecleucel, to treat adults with certain types of large B-cell lymphoma. FDA approves the fifth and sixth checkpoint inhibitors, which target the PD-1 pathway: avelumab, for Merkel cell carcinoma, and durvalumab, for bladder cancer, both of which are later approved for use in several additional cancers.
2018
- The FDA approves second CAR T-cell therapy, axicabtagene ciloleucel, for the treatment of adult patients with several types of large B-cell lymphoma, and a seventh checkpoint inhibitor, which targets the PD-1 pathway, cemiplimab, for cutaneous squamous cell carcinoma.
Group of 3 people working together in meeting room
The 2020s
2020s
The industry’s innovation, along with the FDA’s regulatory guidance and review, continues to expand the arsenal against cancer. Cutting-edge research and development adds new treatment options for patients year after year.
Liquid sample on tray
The Biopharmaceutical Pipeline
After decades of research to advance cancer progress, an average of 68% of medicines in the oncology pipeline today are likely to be first-in-class, meaning they use a new and unique mechanism for treating a disease. The pipeline is also ripe with innovative therapeutic approaches, like mRNA, with the potential to transform a wide range of cancers — many which have already seen approvals in recent years. For example:
- Immunotherapies, which include monoclonal antibodies and CAR-T, is an approach that works by unleashing the immune system to target and kill cancer cells.
- Gene editing involves manipulation of DNA at particular locations in order to treat a specific cancer.
- Oncolytic viral therapies work by zeroing in on cancer cells, to replicate and cause them to rupture.
- Antibody drug conjugates target specific cancer cells with cytotoxic agents without harming normal cells.
Looking Ahead
America’s innovative biopharmaceutical companies remain deeply committed to fighting cancer, driven by decades of progress that have transformed once‑fatal diagnoses into treatable conditions. Continued research is expanding our understanding of how cancer develops and enabling more precise, targeted therapies—contributing to significant declines in the cancer death rate since its 1991 peak. With more than 1,600 cancer medicines and vaccines in development, the industry is steadfast in its mission to bring forward the next generation of treatments and improve survival and quality of life for every patient facing the disease.
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