Cancer Research Starts Here

Much of the recent improvement in 5-year survival rates for all cancers combined is the result of discoveries across the past five decades that have shaped our understanding of what cancer is, its biological and social risk factors, and how it grows and spreads. Thanks to the individuals who perform this research, more lives have been saved and great strides were made in preventing, diagnosing, and treating this collection of diseases.

Earlier Breakthroughs That Paved the Way for Progress

From left, Drs. Joseph Fraumeni, Jr., Harold Varmus, Joan Steitz, Jim Allison, and Steven Rosenberg.

Credit: National Cancer Institute, Copyright held by, and used with permission of, The Board of Regents of the University of Texas System through The University of Texas MD Anderson Cancer Center

Dr. Joseph Fraumeni, Jr.

Developed the first computer-generated maps linking cancer and the environment

Back in the 1970s when computers still weren’t in most homes, Dr. Joseph Fraumeni, Jr., created the first computer-generated maps that showed groupings of cancer deaths in US counties. These maps opened our eyes to the link between cancer and the environment and helped solidify Dr. Fraumeni’s position as a pivotal figure in public health. Even before that, together with NCI colleague Dr. Frederick Li, he discovered Li-Fraumeni syndrome—a rare, inherited disorder that greatly increases risk for several types of cancer, particularly in children and young adults. And as founding director of NCI’s Division of Cancer Epidemiology and Genetics, he was an early advocate for disparities research at the institute. Across a trailblazing career, Dr. Fraumeni has attributed his success to a series of mentors and colleagues, and to his wife, whom he credits with encouraging him and keeping him focused over more than 40 years of marriage.

Dr. Harold Varmus

Discovered that cancer comes from mutations in normal genes

More interested in talking about plays and poetry in college, Dr. Harold Varmus graduated with two literature degrees before pursuing medicine. Feeling drawn to the study of the scientific basis of disease, he came to NIH in 1968 and set his sights on a career in basic research. On a backpacking trip to California the next year, Dr. Varmus met Dr. J. Michael Bishop, the man he’d go on to share a Nobel Prize with in 1989 for their novel theory on the origin of cancer. They found that cancer comes from mutations in certain normal genes in a range of species and that these mutations are triggered by random errors in normal cell division or by other external causes. Along with this major discovery, Dr. Varmus has spent decades advancing scientific knowledge and has held several key positions, including as director of NIH, and later, NCI.

Dr. Joan Steitz

Pioneered the study of RNA biology and RNA’s role in cancer development

With no female professors or women running labs around her in the 1960s, Dr. Joan Steitz initially never thought to aspire to such a role herself. Even her first choice for doctoral advisor turned her away, stating that, as a woman, she would just get married and have a family. Despite this, she went on to head her own lab at Yale University in 1970, just one year after the school first accepted female undergraduates, and became a pioneer in the field of RNA biology. Her lab has made fundamental discoveries about the roles of non-coding RNAs in many areas of biology, including cancer. She’s obtained many prestigious awards, including the National Medal of Science as well as a 2018 Lasker Award for her research and mentorship and support of women in science. As a mentor, Dr. Steitz has tirelessly campaigned for inclusive practices within the scientific community and research workforce, speaking out about biases that harmfully affect women and minorities in science.

Dr. Jim Allison

Created a therapy using one’s own T cells to fight cancer

Among the many talented scientists that make up The CheckPoints band, harmonica player Dr. Jim Allison stands out—and it’s not just because he has played with Willie Nelson or because he is the subject of a 2019 award-winning documentary, Jim Allison: Breakthrough. Dr. Allison, of the University of Texas MD Anderson Cancer Center, developed a way to unleash T cells (a type of white blood cell) to attack cancerous tumors, allowing one’s immune system to fight cancer. The invention of the first “immune checkpoint blockade” therapy earned him the 2015 Lasker Award and the 2018 Nobel Prize in Physiology or Medicine, among many other awards. For someone who originally did not set out to study cancer, his contributions to the field of immunotherapy have saved countless lives and pushed the frontiers of our knowledge of both cancer and the immune system.

Dr. Steven Rosenberg

Developed the first human cancer immunotherapy

Raised by Jewish immigrants from Poland, then 6-year-old Dr. Steven Rosenberg watched as his parents learned that many of their relatives had been killed in the Holocaust. Having witnessed “so much evil in the world,” he decided at an early age that he wanted to do something to help people. That desire later culminated in an idea: what if there was a way to activate a person’s immune system to attack and treat cancer without surgery or radiation? It’s a question that led Dr. Rosenberg to begin developing the first immunotherapy in 1976, though it took until 1984 and his 67th patient to confirm his hypothesis, resulting in the first FDA-approved human cancer immunotherapy. More than 30 years later, that 67th patient is still in great health, having effectively been cured of her widespread melanoma.

Accelerating Our Understanding of Cancer into the Future

From left, Drs. Mary-Claire King, Francis Collins, John Carpten, Ashani Weeraratna, Candelaria Gomez-Manzano, and Juan Fueyo.

Credit: National Cancer Institute, National Institutes of Health, Copyright held by, and used with permission of, The Board of Regents of the University of Texas System through The University of Texas MD Anderson Cancer Center

Dr. Mary-Claire King

Proved the existence of BRCA1, a gene that can cause breast and ovarian cancer

As a young assistant professor, Dr. Mary-Claire King began studying families devastated by breast cancer. The genetics of these families led her to BRCA1—a gene that can have inherited mutations that cause breast and ovarian cancer. It took 17 years for her to prove the existence of BRCA1, and now, 47 years after enrolling her first study participants, Dr. King continues to shed light on inherited cancers. As an accomplished geneticist and human rights activist, she also developed—with the Grandmothers of the Plaza de Mayo—genealogical matching to reunite kidnapped children with their families in Argentina, as well as the first use of DNA sequencing to identify victims of the Argentinian “Dirty War.” Her current research interests include inherited breast and ovarian cancer, and genetics of severe mental illness and immunological disorders in children.

Dr. Francis Collins

Led the sequencing of the human genome as well as the mapping of 33 cancer types at a molecular level

The word “genomics” was only recently coined in 1980, and Dr. Francis Collins has been involved in its study since the beginning. Dr. Collins grew up on a farm, the son of a drama professor father and playwright mother—the latter of whom taught him at home before he started going to school in 6th grade. Driven by the thrill of discovery and motivated to help people, he became a physician and geneticist. Known for his leadership of the international Human Genome Project, which sequenced the entire human genome in 13 years, and The Cancer Genome Atlas, which mapped 33 cancer types at a molecular level, Dr. Collins is also highly regarded for his discoveries of various disease genes. Serving as NIH director since 2009, he still manages to run a prolific lab, studying diseases such as progeria and diabetes, play guitar and piano, and ride his motorcycle.

Dr. John Carpten

Led first genome-wide scan for prostate cancer susceptibility genes in African-American people

Dr. John Carpten has spent a lot of his career understanding which cancers disproportionately affect underserved minorities and other communities, and why. He has spearheaded several high-impact studies in prostate cancer, myeloma, and breast cancer. He also conceived the African American Hereditary Prostate Cancer Study Network, which led to the first genome-wide scan for prostate cancer susceptibility genes in African-American people. Not only is he determined to reduce cancer disparities and increase minority representation in clinical trials and precision medicine studies, he’s also a dedicated mentor for the next generation of cancer researchers. Another doctor, Melissa Davis, described him as a “big brother” to all of the minorities in molecular sciences while he was at The Ohio State University. There, he organized support groups and facilitated networking for minority students, encouraging them to excel.

Dr. Ashani Weeraratna

Reshaped clinical practice with research findings on age-related differences in cancer treatment response

Bloomberg Distinguished Professor Dr. Ashani Weeraratna found that there are age-related differences in how people respond to certain cancer treatments—a groundbreaking finding now reshaping clinical practice. As a skin cancer researcher, she has also led public health initiatives to install sunblock dispensers throughout Philadelphia and to teach children about the dangers of sun exposure. A Sri Lankan who grew up in Lesotho in southern Africa before emigrating to the United States, Dr. Weeraratna is a fierce advocate for the contributions of immigrant scientists and has spoken passionately about her experiences with racism and harassment in this country, and about her belief in the American Dream. She is also a champion of and mentor for junior faculty, women, and people of color in science.

Dr. Candelaria Gomez-Manzano and Dr. Juan Fueyo

Created a new therapy that uses a common cold virus to attack brain tumors

Husband and wife duo Drs. Candelaria Gomez-Manzano and Juan Fueyo created an experimental therapy that harnesses a common cold virus and transforms it into something that can attack glioblastoma—the most common and deadly of brain tumors. This so-called “smart bomb virus” is an immunotherapy that has the potential to destroy these tumors without radiation or chemotherapy. Both Drs. Gomez-Manzano and Fueyo are neuro-oncology professors who emigrated to the United States from Spain and work side by side in their laboratories at the University of Texas MD Anderson Cancer Center in Houston.

Some Questions to Ask if You’re Offered a Clinical Trial

Dr and patient discuss cancer clinical trialIf you are offered a clinical trial, feel free to ask any questions or bring up any issues concerning the trial at any time. The following suggestions may give you some ideas as you think about your own questions.

The study

  • What is the purpose of the study?
  • Why do researchers think the approach may be effective?
  • Who will fund the study?
  • Who has reviewed and approved the study?
  • How are study results and safety of participants being checked?
  • How long will the study last?
  • What will my responsibilities be if I participate?

Possible risks and benefits

  • What are my possible short-term benefits?
  • What are my possible long-term benefits?
  • What are my short-term risks, such as side effects?
  • What are my possible long-term risks?
  • What other options do people with my disease have?
  • How do the possible risks and benefits of this trial compare with those options?

Participation and care

  • What kinds of therapies, procedures and /or tests will I have during the trial?
  • Will they hurt, and if so, for how long?
  • How do the tests in the study compare with those I would have outside of the trial?
  • Will I be able to take my regular medications while in the clinical trial?
  • Where will I have my medical care?
  • Who will be in charge of my care?

Personal issues

  • How could being in this study affect my daily life?
  • Can I talk to other people in the study?

Cost issues

  • Will I have to pay for any part of the trial such as tests or the study drug?
  • If so, what will the charges likely be?
  • What is my health insurance likely to cover?
  • Who can help answer any questions from my insurance company or health plan?
  • Will there be any travel or child care costs that I need to consider while I am in the trial?

Tips for asking your doctor about trials

  • Consider taking a family member or friend along, for support and for help in asking questions or recording answers.
  • Plan ahead what to ask — but don’t hesitate to ask any new questions you think of while you’re there.
  • Write down your questions in advance, to make sure you remember to ask them all.
  • Write down the answers, so that you can review them whenever you want.
  • Ask about bringing a tape recorder to make a taped record of what’s said (even if you write down answers).


Getting Cancer Clinical Trials in the Community

The NCI Community Oncology Research Program (NCORP) is a national network that brings cancer clinical trials and care delivery studies to people in their own communities.  NCORP is comprised of 7 research bases and 46 community sites, of which SCOR is one. SCOR has affiliated hospitals and clinics located in 6 states across the Southeast – Florida, Georgia, North Carolina, South Carolina, Tennessee and Virginia.  In this short video, researchers in the NCI Community Oncology Research Program (NCORP) describe this national network and how it brings clinical trials to diverse populations in their own communities.


TMIST Ramps Up to Illuminate Breast Cancer Screening

TMIST (Tomosynthesis Mammographic Imaging Screening Trial), the large NCI screening study to learn about the best ways to find breast cancer in women who have no symptoms, is ramping up with 16,505 participants enrolled to date at 84 certified mammography clinics in the United States, Canada and Argentina.

Photo of Etta Pisano

Etta Pisano, MD, chief research officer at the American College of Radiology, and the principal investigator of TMIST

The number of women and sites participating is likely to increase rapidly in the months ahead. Etta Pisano, MD, chief research officer at the American College of Radiology, and the principal investigator of TMIST, heads the efforts to get clinics involved.  “We have 15 approved clinics working on the logistics to begin enrollment, and another 34 have committed to joining. We are pleased that there has been a great deal of interest from international sites and expect to bring on additional sites in Europe and Asia in the next 6 months,” Dr. Pisano said.

TMIST is a randomized phase 3 trial comparing standard digital mammography (2-D) with the newer tomosynthesis mammography (3-D). The goal is to follow all participants to see if they are diagnosed with breast cancer or a precancerous condition, and to record their treatment and outcomes from the time of randomization until the end of the study.

TMIST was designed to provide evidence about the role of mammography screening in reducing the incidence of life threatening, advanced breast cancers. This is a stand-in for breast cancer mortality, the usual endpoint for screening trials, because a trial using that endpoint would take many more years to complete.

All told, 164,946 healthy women ages 45 to 74 who are already planning to get routine mammograms will be enrolled. Women will be randomized to either 3-D or 2-D screening mammograms for 5 years. Each woman is assigned to a screening frequency of every 1 or every 2 years, based on her individual risks for developing breast cancer.

While Medicare and many private insurance programs provide full cost coverage for screening mammograms, with no co-pay, copays are still allowed for some mammograms in some states.  Based on requests from women taking part in TMIST, some states are now funding co-payments or completely covering the participants’ cost for mammograms, according to the American College of Radiology (ACR).  The study also has some funding for uninsured patients subject to the charity care policies of the participating institutions.

States with Laws that Mandate
NO Co-pay for Screening Mammograms
States with Laws that Allow
Co-pays for Screening Mammograms
Arizona, Colorado, Connecticut, Illinois, Kansas, New Hampshire, New Jersey, New York, Oklahoma, Pennsylvania, Texas, Vermont, Washington Kentucky, Louisiana, Maryland, Montana

TMIST is supported by the NCI Community Oncology Research Program (NCORP) and co-led by the ECOG-ACRIN Cancer Research Group. “About 50 million screening mammograms occur every year in the U.S. and it’s important to note that despite the technology disbursement, there is a research study going on to inform the science and the clinical practice,” said NCORP Director Worta McCaskill-Stevens, MD, MS.

Toward Personalized Screening Recommendations

TMIST researchers also are collecting evidence of significant secondary endpoints including false positives, recall rates, biopsy rates, and overdiagnosis. Data is collected on the results of every mammogram, whether the imaging shows no signs of cancer, findings suspicious of cancer, or a breast cancer. Medical follow-ups, such as more imaging or biopsies, also are reported.

Portrait of Worta McCaskill-Stevens

NCORP Director Worta McCaskill-Stevens, MD, MS

“Any increase in sensitivity with a new technology could be of benefit, harm, or a mixture, but only a randomized trial can sort it out,” Dr. McCaskill-Stevens added. “The average-risk woman will need personalized guidance about screening tests to make informed decisions around potential risks and benefits in the future.”

A key component of the trial is a biorepository with detailed clinical annotations. The subsequent molecular characterization of specimens and accompanying demographic information could help advance a personalized approach to screening recommendations that determines mammography use and intervals for each person based on their genetics and individual risk factors for developing breast cancer.

“The molecular evidence is the first step to personalizing breast cancer screening recommendations,” Dr. McCaskill-Stevens said. “The resulting dataset of clinical images and biospecimens will help us tailor future screening to each woman’s individual risk.”

More information and a list of participating sites is available from NCI and ECOG-ACRIN. For a discussion about the science involved in the study, see the article “Why I choose to play a dual role in TMIST” by Dr. McCaskill-Stevens in The Cancer Letter. “TMIST Trial Aims to Provide Clarity on Breast Cancer Screening Approaches” in the NCI Cancer Currents Blog offers more background on 2-D vs 3-D technology.

For more information about cancer clinical trials near you, click here.

 TMIST Ramps Up to Illuminate Breast Cancer Screening was originally published by the National Cancer Institute.

Facebook Live Dialogue on Cancer Disparities, Prevention, and Research

Posted: April 30, 2019

During Minority Health Month on April 18, from 12 pm -1 pm EDT, the National Cancer Institute (NCI) hosted a Facebook Live event called “A Dialogue on Cancer Disparities, Prevention, and Research.”

Many factors contribute to cancer disparities or differences in cancer outcomes. Cancer can affect all populations in the United States, but some groups may bear a disproportionate burden.

The Facebook Live event featured Worta McCaskill-Stevens, MD, MS, director of the NCI Community Oncology Research Network (NCORP), Brid M. Ryan, PhD, MPH, of the NCI Laboratory of Human Carcinogenesis, and Christina Dieli-Conwright, PhD, MPHExternal Web Site Policy, of the University of Southern California. They discussed cancer disparities and the work being done to help reduce them.

NCORPs have an important role in providing the opportunity for minorities to take part in clinical studies, with Minority/Underserved NCORP sites having an additional focus on underserved populations.