Exploring how America's premier cancer research agency is revolutionizing treatment through groundbreaking science, innovative therapies, and collaborative research models.
When President Franklin D. Roosevelt signed the National Cancer Act of 1937, he established the United States' first dedicated weapon against a disease that would become one of humanity's most persistent foes. Today, the National Cancer Institute (NCI) stands as the federal government's principal agency for cancer research and training, coordinating a massive scientific enterprise that spans the nation 1 6 . With a team of approximately 3,500 professionals and a budget provided by Congress, NCI serves as the backbone of America's cancer research infrastructure, supporting everything from basic laboratory science to cutting-edge clinical trials 1 .
Professionals working at NCI
Cancer survivors in the U.S. (2018)
Clinical trial sites nationwide
The impact of this coordinated effort is measurable and profound. Over the past several decades, cancer survival rates have improved dramatically, with the number of cancer survivors in the United States more than doubling from 7 million in 1992 to over 18 million in 2018 1 . This progress stems from advances in detection, diagnosis, and treatment that have translated scientific discoveries into real-world therapies. As the largest funder of cancer research in the world, NCI manages a broad range of research, training, and information dissemination activities that reach across demographic and geographic divides, bringing the promise of scientific discovery to all people 1 .
The National Cancer Institute's mission encompasses a comprehensive approach to cancer research, from fundamental biological studies to the implementation of evidence-based interventions in clinical and community settings. The institute coordinates and supports research across the entire cancer continuum—prevention, detection, diagnosis, treatment, and survivorship.
For decades, the conventional approach to cancer research followed a linear path—from laboratory discoveries to clinical applications. However, a significant paradigm shift has emerged, championed by thought leaders at institutions like MD Anderson Cancer Center. Rather than moving exclusively "from bench to bedside," transformative cancer research increasingly begins with critical observations from clinical practice that then inform laboratory investigations 4 .
This "bedside to bench" approach ensures that research addresses the most pressing challenges faced by patients. For instance, when oncologists noticed that certain cancers responded differently to treatments than predicted by laboratory models, this clinical observation sparked investigations that revealed previously unknown resistance mechanisms and cancer subtypes 4 . By grounding research in real human cancer experiences, scientists can ask more relevant questions and develop more meaningful models, ultimately leading to treatments that are more likely to benefit patients.
Modern cancer research requires expertise across so many disciplines that the traditional lone-scientist model has become increasingly inadequate. Recognizing this, NCI has championed team science—the collaborative effort of researchers from diverse fields working toward common goals 5 . The complexity of cancer demands integration of knowledge from biology, chemistry, physics, computational science, engineering, clinical medicine, and behavioral science.
NCI's Team Science Toolkit provides resources to support these complex collaborative efforts, integrating knowledge from disciplines as varied as public health, communications, management sciences, and psychology 5 . This approach prevents unnecessary duplication of efforts and maximizes the efficiency and effectiveness of cancer research initiatives. The growing body of knowledge about how to optimize team science is emerging as a critical component in accelerating progress against cancer.
One of the most promising recent advances comes from a Phase II clinical trial presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting. Researchers tackled anaplastic thyroid cancer with BRAF V600E mutation, an aggressive form of the disease that often proves fatal because it's typically diagnosed at an advanced, inoperable stage 2 .
The study, led by Dr. Mark Zafereo, tested a novel approach for these high-risk patients. Rather than proceeding directly to surgery, patients first received neoadjuvant therapy (treatment before primary surgery) with a three-drug combination: pembrolizumab (an immunotherapy drug) plus dabrafenib and trametinib (targeted therapies) 2 . This DTP combination specifically targets the molecular vulnerabilities of the cancer while engaging the immune system.
After this preoperative treatment, surgeons removed any remaining cancer. The results were striking: two-thirds of patients had no residual anaplastic thyroid cancer after the combination therapy and surgery 2 . Historically, patients with this diagnosis have had extremely poor outcomes, but this approach resulted in a two-year survival rate of 69%—a remarkable improvement for a cancer that once offered little hope 2 .
Researchers enrolled patients with Stage IV BRAF V600E-mutated anaplastic thyroid cancer, a population with limited treatment options and poor prognosis 2 .
Patients received the three-drug DTP combination (pembrolizumab, dabrafenib, and trametinib) before surgery. This preoperative treatment aimed to shrink tumors and eliminate microscopic cancer cells 2 .
After the neoadjuvant therapy, surgeons operated to remove any remaining cancer. The preoperative treatment made these surgical procedures more successful than in untreated patients 2 .
Pathologists examined the removed tissue to determine whether any viable cancer cells remained, finding that 67% of patients had no residual anaplastic thyroid cancer 2 .
Researchers followed patients for two years, documenting survival rates and monitoring for potential recurrence 2 .
The dramatic success of this approach—67% of patients achieving no residual cancer and a 69% two-year survival rate—represents a potential new standard of care for this aggressive cancer type 2 . The findings demonstrate the power of molecularly targeted treatment strategies combined with immunotherapy, showing that strategically sequencing treatments (neoadjuvant therapy followed by surgery) can yield dramatically improved outcomes even for advanced cancers.
| Outcome Measure | Result | Significance |
|---|---|---|
| Rate of no residual cancer | 67% | Indicates high degree of tumor destruction before surgery |
| Two-year survival rate | 69% | Dramatic improvement over historical averages |
| Surgical success | Enhanced | Preoperative therapy made subsequent surgery more effective |
This research exemplifies the modern approach to cancer treatment: identifying specific molecular features of a cancer, selecting drugs that target those features, and creatively combining modalities to maximize effectiveness. The trial also highlights the importance of clinical trials in advancing cancer care, as these structured studies allow researchers to rigorously test new approaches and generate evidence needed to change medical practice 2 3 .
Modern cancer research relies on sophisticated tools and reagents that enable scientists to probe the mysteries of cancer biology and develop new interventions. These fundamental resources, many supported by NCI, form the foundation of discovery across the cancer research enterprise.
Function in Research: Target-specific proteins on cancer cells
Application Examples: Used in immune checkpoint inhibitors (e.g., anti-PD-1) 2
Function in Research: Block specific cancer-driving molecules
Application Examples: Targeted therapies like dabrafenib that inhibit BRAF V600E 2
| Tool/Reagent | Function in Research | Application Examples |
|---|---|---|
| Lipid nanoparticles (LNPs) | Delivery vehicle for therapeutic molecules | Used in mRNA vaccines to protect and transport genetic material 2 7 |
| mRNA constructs | Blueprint for protein production | Encodes therapeutic proteins like bispecific antibodies 2 7 |
| Monoclonal antibodies | Target-specific proteins on cancer cells | Used in immune checkpoint inhibitors (e.g., anti-PD-1) 2 |
| Small molecule inhibitors | Block specific cancer-driving molecules | Targeted therapies like dabrafenib that inhibit BRAF V600E 2 |
| Animal models | Test therapeutic approaches in living systems | Study treatment effects in immune-compromised and immune-competent mice 4 7 |
| Cell lines | Provide reproducible systems for laboratory studies | Model cancer behavior and test drug responses 4 |
Building on the success of COVID-19 vaccines, cancer researchers are harnessing mRNA technology to develop innovative cancer treatments. One exciting advance comes from University of Florida Health, where researchers have developed an experimental mRNA vaccine that boosted the effects of immunotherapy in mouse models 7 .
"This paper describes a very unexpected and exciting observation: that even a vaccine not specific to any particular tumor or virus—so long as it is an mRNA vaccine—could lead to tumor-specific effects."
Unlike earlier approaches that target specific cancer proteins, this vaccine takes a different tack—it simply revs up the immune system generally, as if fighting a virus. The surprising result was that this general immune activation still generated a strong anti-cancer response, particularly when combined with immune checkpoint inhibitors 7 .
This research suggests a potential third paradigm in cancer vaccine development: instead of targeting specific antigens expressed in many people with cancer or creating fully personalized vaccines, scientists might develop "off-the-shelf" universal cancer vaccines that work by generally sensitizing the immune system to cancer 7 .
The translation of scientific discoveries into patient benefits depends critically on clinical trials—the structured research studies that evaluate new treatments in human volunteers. NCI supports and coordinates clinical trials across 3,100 sites nationwide, enabling patients to access cutting-edge therapies while contributing to medical knowledge 1 3 .
Primarily assess safety and determine appropriate dosing 3 .
Evaluate effectiveness and further monitor side effects 2 .
| Therapy | Cancer Type | Key Finding | Stage |
|---|---|---|---|
| BNT142 | CLDN6-positive tumors (testicular, ovarian, NSCLC) | First clinical proof-of-concept for mRNA-encoded bispecific antibody 2 | Phase I/II |
| VLS-1488 | Cancers with chromosomal instability | First-in-class oral KIF18A inhibitor showing anti-tumor activity 2 | Phase I/II |
| Pivekimab sunirine (PVEK) | Blastic plasmacytoid dendritic cell neoplasm (BPDCN) | High, durable complete remission responses in rare leukemia 2 | Seeking FDA approval |
The landscape of cancer research and treatment has been transformed since NCI's founding in 1937. From the early days of chemotherapy development to today's sophisticated immunotherapies and targeted treatments, the steady progress fueled by scientific investigation has yielded tangible benefits for patients. The number of cancer survivors in the United States is expected to rise to 26 million by 2040—a testament to these advances 1 .
As research continues, the future promises even more sophisticated approaches—from universal cancer vaccines that can "wake up" the immune system 7 to increasingly personalized treatments matched to the unique molecular profile of each patient's cancer 2 . The National Cancer Institute remains at the center of this effort, supporting the scientists, institutions, and collaborations that drive progress against this disease.
While challenges remain, the trajectory is clear: through continued investment in basic science, innovative clinical trials, and collaborative research models, we are moving closer to a world where cancer is no longer a feared diagnosis but a manageable condition. The scientific journey continues, with each discovery building on the last to transform hope into healing.