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Advances in Prostate Cancer Research

Nanoparticles are tested as a means to deliver drugs to prostate cancer cells.

Nanoparticles are tested as a means to deliver drugs to prostate cancer cells. Credit: National Cancer Institute

NCI-funded researchers are working to advance our understanding of how to prevent, detect, and treat prostate cancer.  Most men diagnosed with prostate cancer will live a long time, but challenges remain in choosing the best treatments for individuals at all stages of the disease.

This page highlights some of the latest research in prostate cancer, including clinical advances that may soon translate into improved care, NCI-supported programs that are fueling progress, and research findings from recent studies.

Studying Early Detection for Men at High Risk

Men with certain inherited genetic traits are at increased risk for developing prostate cancer. Examples of such traits include inherited BRCA gene mutations and Lynch syndrome. No clear guidelines exist for when or how—or if—to screen men at high genetic risk for prostate cancer.

NCI researchers are using magnetic resonance imaging (MRI) of the prostate in men at high risk to learn more about how often and how early these cancers occur. They’re also testing whether regular scans in such men can detect cancers early, before they spread elsewhere in the body (metastasize).

Diagnosing Prostate Cancer

Improving Biopsies for Prostate Cancer

Traditionally, prostate cancer has been diagnosed using needles inserted into the prostate gland in several places under the guidance of transrectal ultrasound (TRUS) imaging to collect samples of tissue. This approach is called systematic biopsy.

However, ultrasound does not generally show the location of cancer within the prostate. It is mainly used to make sure the biopsy needles go into the gland safely. Therefore, biopsy samples using ultrasound guidance can miss cancer altogether, or identify low-grade cancer while missing areas of high-grade, potentially more aggressive cancers.

Some doctors, concerned that a systematic biopsy showing only low-grade cancer could have missed a high-grade cancer, may suggest surgery or radiation. However these treatments are for a cancer that may have never caused a problem, which is considered overtreatment.

Using MRI and ultrasound. Scientists at NCI have developed a procedure that combines magnetic resonance imaging (MRI) with TRUS for more accurate prostate biopsies. MRI can locate potential areas of cancer within the gland but is not practical for real-time imaging to guide a prostate biopsy. The new procedure, known as MRI-targeted biopsy, uses computers to fuse an MRI image with an ultrasound image. This lets doctors use ultrasound guidance to biopsy areas of possible cancer seen on MRI.

NCI researchers have found that combining MRI-targeted biopsy with systematic biopsy can increase the detection of high-grade prostate cancers while decreasing detection of low-grade cancers that are unlikely to progress.

Testing machine learning. Researchers are testing the use of machine learning, also called artificial intelligence (AI), to better recognize suspicious areas in a prostate MRI that should be biopsied. AI is also being developed to help pathologists who aren’t prostate cancer experts accurately assess prostate cancer grade. Cancer grade is the most important factor in determining the need for treatment versus active surveillance.

Finding Small Amounts of Prostate Cancer Using Imaging and PSMA

NCI-supported researchers are developing new imaging techniques to improve the diagnosis of recurrent prostate cancer. A protein called prostate-specific membrane antigen (PSMA) is found in large amounts—and almost exclusively—on prostate cells. By fusing a molecule that binds to PSMA to a compound used in PET scan imaging, scientists have been able to see tiny deposits of prostate cancer that are too small to be detected by regular imaging. The Food and Drug Administration (FDA) has approved two such compounds for use in PET imaging of men with prostate cancer.

This type of test is still experimental. But the ability to detect very small amounts of metastatic prostate cancer could help doctors and patients make better-informed treatment decisions. For example, if metastatic cancer is found when a man is first diagnosed, he may choose an alternative to surgery because the cancer has already spread. Or doctors may be able to treat cancer recurrence—either in the prostate or metastatic disease—earlier, which may lead to better survival.

As part of the Cancer Moonshot℠, NCI researchers are testing whether PSMA-PET imaging can also identify men who are at high risk of their cancer recurring. Such imaging may eventually be able to help predict who needs more aggressive treatment—such as radiation therapy in addition to surgery—after diagnosis.

Prostate Cancer Treatment

Treatments for prostate cancer that has not spread elsewhere in the body are surgery or radiation therapy (RT), with or without hormone therapy. Active surveillance is also an option for men who have a low risk of their cancer spreading.

Hormone Therapy for Prostate Cancer

Over the last few years, several new approaches to hormone therapy for advanced or metastatic prostate cancer have been approved for clinical use.

Many prostate cancers that originally respond to treatment with standard hormone therapy become resistant over time, resulting in castrate-resistant prostate cancer (CRPC). Three new drugs have been shown to extend survival in men with CRPC. All three block the action of hormones that drive CRPC:

The survival benefit for these drugs has been seen regardless of whether men have previously received chemotherapy.

In addition, both enzalutamide and the drug apalutamide (Erleada) have all been shown to decrease the risk of metastases in men with CRPC that has not yet spread to other parts of the body. Darolutamide has been shown to increase the amount of time men live without their cancer metastasizing.

Abiraterone, apalutamide, and enzalutamide have been shown to improve the survival of men with metastatic castrate-sensitive prostate cancer when added to standard hormone therapy.

Scientists are continuing to study novel treatments and drugs, along with new combinations of existing treatments, in men with metastatic CRPC.

Immunotherapy: Vaccines for Prostate Cancer

Immunotherapies are treatments that harness the power of the immune system to fight cancer. These treatments can either help the immune system attack the cancer directly or stimulate the immune system in a more general way.

Vaccines and checkpoint inhibitors are two types of immunotherapy being tested in prostate cancer. Treatment vaccines are injections that stimulate the immune system to recognize and attack a tumor.

One type of treatment vaccine called sipuleucel-T (Provenge) is approved for men with few or no symptoms from metastatic CRPC.

Immunotherapy: Checkpoint Inhibitors for Prostate Cancer

An immune checkpoint inhibitor is a type of drug that blocks proteins on the immune cells, making the immune system more effective at killing cancer cells.

A checkpoint inhibitor called pembrolizumab (Keytruda) has been approved for the treatment of tumors, including prostate cancers, that have specific genetic features. Pembrolizumab has also been approved for any tumor that has metastasized and has a high number of genetic mutations.

But relatively few prostate cancers have these features, and prostate cancer in general has largely been resistant to treatment with checkpoint inhibitors and other immunotherapies, such as CAR T-cell therapy.

Research is ongoing to find ways to help the immune system recognize prostate tumors and help immune cells penetrate prostate tumor tissue. Studies are looking at whether combinations of immunotherapy drugs, or immunotherapy drugs given with other types of treatment, may be more effective in treating prostate cancer than single immunotherapies alone.

PARP Inhibitors for Prostate Cancer

Some prostate tumors have genetic defects that limit their ability to repair DNA damage. Such tumors may be sensitive to a class of drugs called PARP inhibitors, which also block DNA repair.

Two PARP inhibitors, olaparib (Lynparza) and rucaparib (Rubraca), have been approved for some men whose prostate cancer has metastasized, and whose disease has stopped responding to standard hormone treatments.

Targeted Radiation Therapy and PSMA

Scientists are also developing targeted therapies based on PSMA, the same protein that is being tested for imaging prostate cancer. For treatment, the molecule that targets PSMA is chemically linked to a radioactive compound. This new compound can potentially find, bind to, and kill prostate cancer cells throughout the body.

In a recent clinical trial, men with a type of advanced prostate cancer who received a PSMA-targeting drug lived longer than those who received standard therapies. Ongoing and planned clinical trials are testing PSMA-targeting drugs in patients with earlier stages of prostate cancer, and in combination with other treatments, including targeted therapies like PARP inhibitors and immunotherapy.

Personalized Clinical Trials for Prostate Cancer

Research is uncovering more information about the genetic changes that happen as prostate cancers develop and progress. Although early-stage prostate cancer has relatively few genetic changes compared with other types of cancer, researchers have learned that metastatic prostate cancers usually accumulate more mutations as they spread through the body.

These mutations may make men with metastatic prostate cancers candidates for what are called “basket” clinical trials of new drugs. Such trials enroll participants based on the mutations found in their cancer, not where in the body the cancer arose. In the NCI-MATCH trial, a high percentage of enrolled men with advanced prostate cancer had mutations that could potentially be targeted with investigational drugs.

NCI-Supported Research Programs

See a full list of prostate cancer research projects that NCI funded in FY 2018.

Many NCI-funded researchers working at the National Institutes of Health campus, as well as across the United States and world, are seeking ways to address prostate cancer more effectively. Some of this research is basic, exploring questions as diverse as the biological underpinnings of cancer and the social factors that affect cancer risk. And some is more clinical, seeking to translate basic information into improving patient outcomes. The programs listed below are a small sampling of NCI’s research efforts in prostate cancer.

Clinical Trials

NCI funds and oversees both early- and late-phase clinical trials to develop new treatments and improve patient care. Trials are available for prostate cancer preventionscreening, and treatment.

Prostate Cancer Research Results

The following are some of our latest news articles on prostate cancer research:

View the full list of Prostate Cancer Research Results and Study Updates.

  • Reviewed: 

“Advances in Prostate Cancer Research” was originally published by the National Cancer Institute.

Pursuing a Cancer Prevention Vaccine

For more than two decades, Olivera Finn has tirelessly pursued one goal in her research: to develop a vaccine to prevent cancer. She has had this goal since 1989, when her research team discovered the first tumor antigen recognized by a type of immune cell that can kill cancer cells. That antigen—an abnormal version of a protein called MUC1—is produced by the cells of more than 80% of cancer types, including cancers of the breast, pancreas, colon, lung, and prostate.

Although she started her research career as an organ transplant immunologist, the discovery of MUC1 was a pivotal point in Olivera’s career trajectory. “Once we discovered tumor antigens,” she said, “I never looked back.” Olivera received her first NCI grant in 1991 and has been funded ever since to study the biology of tumor antigens and develop them as targets for cancer prevention.

Cancer can take many years—even decades—to develop. Some cancers arise from precursor growths that can be detected by current screening methods. For example, colorectal polyps called advanced adenomas, which can be detected by colonoscopy, can progress to colorectal cancer. These adenomas can be removed surgically, but in many patients, new ones continue to develop and some will become malignant. Olivera’s lab found that the cells of advanced adenomas and the precursors of pancreatic, lung, and many other types of cancer all produce abnormal MUC1 protein.

The presence of abnormal MUC1 on premalignant growths may make it a good target for a vaccine that would prevent their progression to cancer or the development of new precursors. To test this idea, Olivera’s group conducted the first ever clinical trial of a cancer prevention vaccine based on a tumor antigen in healthy people without cancer who were at increased risk of developing the disease.

In the NCI-funded trial, reported in 2013, individuals with a history of advanced adenomas were given an MUC1 vaccine. The vaccine was shown to be safe and to elicit a strong immune response and a long-lasting immune memory. NCI is currently sponsoring a phase II trial testing whether the vaccine will prevent the regrowth of colorectal polyps.

Looking forward, Olivera envisions, “If you are in your 60s and your doctor discovers you are at high risk for cancer, the idea would be to vaccinate to boost the immune system’s ability to keep any abnormal cells in check instead of waiting to see if cancer develops.”

Olivera says that funding from NCI is critical for her research and for cancer prevention research in general. Cancer prevention research is complex, and translating laboratory discoveries into new ways to prevent cancer requires sustained investments over many years—investments that the private sector is often reluctant to make. But “building the evidence that vaccines are an effective way of controlling cancer will go a long way toward getting companies interested,” she said.

The field of cancer immunology has expanded dramatically and has led to immunotherapies for the treatment of advanced cancers as well as vaccines against some viruses that cause cancer. Boosting the immune system to prevent cancers that are not caused by viruses may now be within reach. “The opportunities are amazing,” she added.


Understanding Cancer Research Study Design and How to Evaluate Results

Approved by the Cancer.Net Editorial Board, 04/2018

Doctors and scientists conduct research studies to find better ways to prevent and treat cancer. Depending on the questions they want to answer, researchers can design these studies in different ways. No study design is perfect. Each has strengths and drawbacks. It is important to understand a study’s design. By doing this, you can understand the results to know if they apply to your situation.

In cancer research, there are 2 main types of research studies:

  • Experimental studies. This type of study provides an intervention, such as a new treatment. The intervention is given to a group of people. Then, researchers compare their results to those of another group that does not receive the intervention. This other group is known as the control group. The researchers choose who does and does not receive the intervention either randomly or through a selection process. Experimental studies help researchers learn more about how cancer starts or spreads. These studies can also test new imaging techniques and explore quality of life issues.
  • Observational studies. This type of study involves observing groups of people in a natural setting and looking at a specific result. A result may include whether 1 group of people has more cancer diagnoses than another group. In these studies, the researchers cannot control the intervention, such as a person’s weight or whether they took vitamin supplements. These studies are often described as epidemiologic. Epidemiology involves studying how different risks cause or spread a disease in a community.

Types of experimental studies

Experimental studies are more reliable than observational studies. This is because the volunteers are placed in the intervention or control group by chance. This reduces the likelihood that the assumptions or preferences of the researchers or volunteers will change the study results. Such assumptions or preferences are called bias.

This type of study also helps researchers to better find and control other factors, such as age, sex, and weight. These factors can affect the results of the study.

Researchers may also consider certain factors when choosing people to enroll in an experimental study. They could be based on type of cancer, stage of the disease, or whether the cancer has spread.

One of the most common types of experimental studies is the clinical trial. This is a research study that tests a medical intervention in people. Clinical trials test:

  • The effectiveness or safety of a new drug or combination of drugs
  • A new approach to radiation therapy or surgery
  • A new treatment or way to prevent cancer
  • Ways to lower the risk of cancer coming back

Doctors and researchers conduct clinical research in segments called phases. Each phase of a clinical trial provides different answers about the new treatment. For instance, it can show the dose, safety, and efficacy of the treatment. The efficacy is how well the treatment works. There are 4 phases of clinical trials.

In a clinical trial, volunteers are usually selected by chance to either be in the treatment or control group. Researchers can prevent bias in a clinical trial by keeping volunteers and/or themselves from knowing how the volunteers are grouped. This is a process known as “blinding.”

Types of experimental studies include:

  • Double-blind randomized trial. Most scientists believe this type of clinical trial will produce the best evidence in a study. Neither the volunteers nor the researchers know who belongs to a treatment or control group until the study ends.
  • Single-blind randomized trial. In this type of trial, the volunteers do not know whether they belong to a treatment or control group. But the researchers know.
  • Open/unblinded trial. Both volunteers and researchers know who belongs to each test group in this type of study. This occurs when it is not possible to use blinding. For instance, the study could compare a surgical treatment to a drug.

Types of observational studies

In observational studies, researchers have less control over the study volunteers. This means that certain factors could affect the results. These studies, however, are useful in providing initial evidence that can help guide future research.

Types of observational studies include:

  • Case-control studies. These types of studies compare 2 groups of people. For instance, they could compare those who have cancer (the case) and those who do not (the control). Researchers may look for lifestyle or genetic differences between the 2 groups. By doing this, they hope to find out why 1 group has a disease and the other group does not. These studies are conducted retrospectively. That is, they are researching what has already happened.
  • Cohort studies. These studies are prospective, which means that researchers study the event as it occurs. They monitor a group of people for a long time and track something. For example, they could track any new cancer diagnoses. This type of study can assess whether certain nutrients or actions can prevent cancer. This approach can also find cancer risk factors. For instance, cohort studies have looked at whether postmenopausal hormone replacement therapy increases the risk of breast cancer.
  • Case reports and case series. These studies are detailed descriptions of a patient’s medical history. The individual patient descriptions are called case reports. If many patients receive a similar treatment, the case reports may be combined into a case series. The results of case series studies are descriptions of patients’ histories within a specific group. As such, they should not be used to determine treatment options.
  • Cross-sectional studies. These studies examine how diseases interact with other factors within a specific group at a point in time. But because these studies only measure interactions at a single point in time, they cannot prove that something causes cancer.

Types of review articles

A large number of cancer research studies are published every year. Given this, it is challenging for doctors, as well as interested patients and caregivers, to keep up with the latest advances. Research studies published in journals are constantly shaping and reshaping the scientific understanding of that subject. No single study provides the final word on a topic, type of cancer, or treatment. As a result, review articles, which evaluate and summarize the findings of all published research on a certain topic, are extremely helpful.

Types of review articles include:

  • Systematic reviews. These articles summarize the best available research on a specific topic. Researchers use an organized method to locate, gather, and evaluate a number of research studies on a particular topic. By combining the findings of a number of studies, researchers are able to draw more reliable conclusions.
  • Meta-analyses. These studies combine data from several research studies on the same topic. By combining these data, a meta-analysis can find trends that are hard to see in smaller studies. But if the single studies were poorly designed, the results of the meta-analysis may not be useful.

Evaluating research studies

Here are some tips for finding out the quality of a research study:

  • Find out if the journal uses a peer-review process. Results from a study are more reliable if they are peer-reviewed. This means that researchers who are not a part of the study have looked over and approved the design and methods.
  • Look at the length of the study and the number of people involved. A study is more useful and credible if the same results occur in many people across a long time. Studies of rare types of cancer or cancers with a poor chance of getting better are an exception to this rule. This is because there are a small number of patients to study. Also, when looking at the length of the study, it may be suitable for some clinical trials to be shorter. For instance, cancer prevention trials are often much longer than treatment clinical trials. This is because it usually takes longer to figure out if a prevention strategy is working compared to a treatment.
  • Consider the phase of the study when learning about new treatments. Phase I and II clinical trials usually tell you more about the safety of a treatment and less about how well it works. These studies tend to have a smaller number of patients compared to phase III clinical trials.  Phase III clinical trials compare a new treatment with the standard of care. “Standard of care” means the best treatments known. Doctors consider phase III clinical trials to be the most reliable.
  • Find out if the study supports or contradicts current research. New results are exciting, but other researchers must validate the results before the medical field accepts them as fact. Review articles like systematic reviews are of special interest. They review and draw conclusions across all of the published research on a specific topic.
  • Watch out for conclusions that overstate or oversimplify the results. Each study is a small piece of the research puzzle. Medical practice rarely changes because of the results of a single study.

Questions to ask your health care team

Always talk to your health care team about what you find in an abstract or study. If you have reviewed a study that suggests a different approach to cancer treatment, do not stop or change your treatment. First talk with your health care team about how the study relates to your treatment plan.

Consider asking your health care team the following questions:

  • I recently heard about a study that used a new treatment. Is this treatment related to my type and stage of cancer?
  • What type of journals should I read to learn more about my type of cancer?
  • Should I consider being a part of a clinical trial?
  • What clinical trials are open to me?
  • Where can I learn more about clinical trials?

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.

Immunotherapy to Treat Cancer

Immunotherapy is a type of cancer treatment that helps your immune system fight cancer. The immune system helps your body fight infections and other diseases. It is made up of white blood cells and organs and tissues of the lymph system.

Immunotherapy is a type of biological therapy. Biological therapy is a type of treatment that uses substances made from living organisms to treat cancer.

As part of its normal function, the immune system detects and destroys abnormal cells and most likely prevents or curbs the growth of many cancers. For instance, immune cells are sometimes found in and around tumors. These cells, called tumor-infiltrating lymphocytes or TILs, are a sign that the immune system is responding to the tumor. People whose tumors contain TILs often do better than people whose tumors don’t contain them.

Even though the immune system can prevent or slow cancer growth, cancer cells have ways to avoid destruction by the immune system. For example, cancer cells may:

  • Have genetic changes that make them less visible to the immune system.
  • Have proteins on their surface that turn off immune cells.
  • Change the normal cells around the tumor so they interfere with how the immune system responds to the cancer cells.

Immunotherapy helps the immune system to better act against cancer.

What are the types of immunotherapy?

Several types of immunotherapy are used to treat cancer. These include:

  • Immune checkpoint inhibitors, which are drugs that block immune checkpoints. These checkpoints are a normal part of the immune system and keep immune responses from being too strong. By blocking them, these drugs allow immune cells to respond more strongly to cancer.Learn more about immune checkpoint inhibitors.
  • T-cell transfer therapy, which is a treatment that boosts the natural ability of your T cells to fight cancer. In this treatment, immune cells are taken from your tumor. Those that are most active against your cancer are selected or changed in the lab to better attack your cancer cells, grown in large batches, and put back into your body through a needle in a vein.T-cell transfer therapy may also be called adoptive cell therapy, adoptive immunotherapy, or immune cell therapy.Learn more about T-cell transfer therapy.
  • Monoclonal antibodies, which are immune system proteins created in the lab that are designed to bind to specific targets on cancer cells. Some monoclonal antibodies mark cancer cells so that they will be better seen and destroyed by the immune system. Such monoclonal antibodies are a type of immunotherapy.Monoclonal antibodies may also be called therapeutic antibodies.Learn more about monoclonal antibodies.
  • Treatment vaccines, which work against cancer by boosting your immune system’s response to cancer cells. Treatment vaccines are different from the ones that help prevent disease.Learn more about cancer treatment vaccines.
  • Immune system modulators, which enhance the body’s immune response against cancer. Some of these agents affect specific parts of the immune system, whereas others affect the immune system in a more general way.Learn more about immune system modulators.

Which cancers are treated with immunotherapy?

Immunotherapy drugs have been approved to treat many types of cancer. However, immunotherapy is not yet as widely used as surgerychemotherapy, or radiation therapy. To learn about whether immunotherapy may be used to treat your cancer, see the PDQ® adult cancer treatment summaries and childhood cancer treatment summaries.

What are the side effects of immunotherapy?

Immunotherapy can cause side effects, many of which happen when the immune system that has been revved-up to act against the cancer also acts against healthy cells and tissues in your body.

Learn more about immunotherapy side effects.

How is immunotherapy given?

Different forms of immunotherapy may be given in different ways. These include:

  • Intravenous (IV)
    The immunotherapy goes directly into a vein.
  • Oral
    The immunotherapy comes in pills or capsules that you swallow.
  • Topical
    The immunotherapy comes in a cream that you rub onto your skin. This type of immunotherapy can be used for very early skin cancer.
  • Intravesical
    The immunotherapy goes directly into the bladder.

Where do you go for immunotherapy?

You may receive immunotherapy in a doctor’s office, clinic, or outpatient unit in a hospital. Outpatient means you do not spend the night in the hospital.

How often do you receive immunotherapy?

How often and how long you receive immunotherapy depends on:

  • Your type of cancer and how advanced it is
  • The type of immunotherapy you get
  • How your body reacts to treatment

You may have treatment every day, week, or month. Some types of immunotherapy given in cycles. A cycle is a period of treatment followed by a period of rest. The rest period gives your body a chance to recover, respond to the immunotherapy, and build new healthy cells.

How can you tell if immunotherapy is working?

You will see your doctor often. He or she will give you physical exams and ask you how you feel. You will have medical tests, such as blood tests and different types of scans. These tests will measure the size of your tumor and look for changes in your blood work.

What is the current research in immunotherapy?

Researchers are focusing on several major areas to improve immunotherapy, including:

  • Finding solutions for resistance.
    Researchers are testing combinations of immune checkpoint inhibitors and other types of immunotherapy, targeted therapy, and radiation therapy to overcome resistance to immunotherapy.
  • Finding ways to predict responses to immunotherapy.
    Only a small portion of people who receive immunotherapy will respond to the treatment. Finding ways to predict which people will respond to treatment is a major area of research.
  • Learning more about how cancer cells evade or suppress immune responses against them.
    A better understanding of how cancer cells get around the immune system could lead to the development of new drugs that block those processes.
  • How to reduce the side effects of treatment with immunotherapy.
Find more information about immunotherapy clinical trials near you HERE.

Immunotherapy to Treat Cancer was originally published by the National Cancer Institute.