Editor's note: May is National Cancer Research Month, and May 20 is Clinical Trials Day.

A cancer diagnosis is an emotional experience. Learning that you have cancer can create feelings of hopelessness, fear and sadness. This is especially true if your cancer is advanced or available treatments are unable to stop or slow its growth.

"Often, when patients are diagnosed with cancer, they feel hopeless and scared. Clinical trials are one way patients can be proactive. They can make a choice in how their care is going to be," says Matthew Block, M.D., Ph.D., a Mayo Clinic medical oncologist.

Cancer clinical trials help physician-scientists test new and better ways to control and treat cancer. During a clinical trial, participants receive specific interventions, and researchers determine if those interventions are safe and effective. Interventions studied in clinical trials might be new cancer drugs or new combinations of drugs, new medical procedures, new surgical techniques or devices, new ways to use existing treatments, and lifestyle or behavior changes.

Clinical trials provide access to potential treatments under investigation, giving options to people who otherwise may face limited choices. "Clinical trials open the door to a new hope that maybe we can fight their cancer back and give them a better quality of life," says Geoffrey Johnson, M.D., Ph.D., a Mayo Clinic radiologist, nuclear medicine specialist and co-chair of the Mayo Clinic Comprehensive Cancer Center Experimental and Novel Therapeutics Disease Group.

You will receive cancer treatment if you participate in a clinical trial. "I think one common misperception about clinical trials is that if you enter a clinical trial, you may not get treatment (receive a placebo). And that's actually very much not true. Most clinical trials are looking at one treatment compared to another treatment," says Judy C. Boughey, M.D., a Mayo Clinic surgical oncologist, chair of Breast and Melanoma Surgical Oncology at Mayo Clinic in Rochester, Minnesota, and chair of the Mayo Clinic Comprehensive Cancer Center Breast Cancer Disease Group.

"I think one common misperception about clinical trials is that if you enter a clinical trial, you may not get treatment (receive a placebo). And that's actually very much not true. Most clinical trials are looking at one treatment compared to another treatment."Judy C. Boughey, M.D.

Watch this video to hear the experiences of people who have participated in cancer clinical trials and to hear Drs. Block, Johnson and Boughey discuss the importance of clinical trials in cancer care:

Clinical trials are a significant part of cancer care at Mayo Clinic Comprehensive Cancer Center. Cancer care teams work together across specialties to make sure the right clinical trials are available to serve the needs of people with cancer who come to Mayo Clinic.

"We are very particular in how we select the clinical trials that we have available for patients," says Dr. Boughey. "We want to have the best trials available for our patients. Some of the clinical trials are evaluating drugs — we are so excited about those drugs, but we can't prescribe those drugs for patients without having that trial. And so we will actually fight to try to get that trial open here to have it available as an opportunity for our patients."

If you choose to participate in a clinical trial, you will continue to receive cancer care. "For most patients that we evaluate, there's always the standard of care treatment option for those patients. And then, in many situations, there's also a clinical trial that the patient can participate in," says Dr. Boughey.

People who participate in clinical trials help make new and better cancer care available for future patients. The treatments available for cancer patients today exist because of the clinical trial participants of yesterday. "We couldn't advance medicine if it wasn't for people volunteering for trials. And the promise from our side is to say we're not going to put patients on trials or offer trials for them to consider unless we think there's a good chance that they'll get a benefit or that society at large will get a benefit," says Dr. Johnson.

"We couldn't advance medicine if it wasn't for people volunteering for trials. And the promise from our side is to say we're not going to put patients on trials or offer trials for them to consider unless we think there's a good chance that they'll get a benefit or that society at large will get a benefit."Geoffrey Johnson, M.D., Ph.D.

Participating in a clinical trial may give you access to cutting-edge treatment, improve your quality of life and extend your time with loved ones.

"It's definitely worth reaching out to your healthcare provider and asking, 'What clinical trials could I be a potential candidate for?'" says Dr. Boughey. "And remember, you can ask this of your surgical oncologist, your medical oncologist, your radiation oncologist, or any of the physicians you're seeing because there are trials in all disciplines. There are also ongoing trials that require the collection of tissue or the donation of blood. They can also be important in trying to help future generations as we continue to work to end cancer."

Participating in a clinical trial is an important decision with potential risks and benefits. Explore these FAQ about cancer clinical trials, and ask your care team if a clinical trial might be right for you.

This article first published on the Mayo Clinic Comprehensive Cancer Center blog.

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Scott Spaulding's relationship with Mayo Clinic in Florida started before he learned he had throat cancer. He'd been coming to the campus for liver issues and was impressed with the care he received.

"They were responsive, they were on time, they had so many resources available, including the latest technologies and equipment," Scott says. "It made an impression on me."

Still, when a spot on his neck — what he described as "a little BB under my skin" — led to a diagnosis of throat cancer, he initially went somewhere closer to his home in St. Marys, Georgia, for cancer treatment. About a year later, though, Scott's cancer came back, and his thoughts immediately turned to Mayo Clinic.

"I was already going to the Mayo Clinic, and I really liked it," he says."So I decided to talk to them about treating my cancer."

Because it was his "second time through with cancer," Scott wanted to make sure he did his homework. He met with the hospital where he was originally treated, another cancer center, and Mayo Clinic Comprehensive Cancer Center to hear their plans for how they would attack his disease.

"I really wanted to be at the Mayo Clinic because I thought there would be a lot of value in having all of my issues treated in one place," he says. "But I also wanted to be really careful. Because I’m not a doctor, I needed someone to help me make these serious treatment decisions."

In Scott's opinion, Dr. Yujie Zhao, an oncologist at Mayo Clinic Comprehensive Cancer Center who specializes in head and neck cancer, presented the best case.

"She told me that because the cancer had spread from lymph nodes in my neck to lymph nodes in my chest, we should start with chemotherapy combined with immunotherapy first to treat my whole body and then do radiation therapy," he says, adding that Dr. Zhao explained it would be a multipronged attack on the cancer to kill cancer cells that had spread to other areas.

"Others proposed radiation therapy first," Scott says. "I wasn't sure which way to go, but Dr. Zhao did the best and most thorough job of explaining why we should approach it the way she proposed. So I told her, 'Let's get going.'"

"The outside recommendation for upfront radiation was based on the impression that the disease was a primary lung cancer," notes Dr. Zhao. "However, after a thorough workup, we confirmed that it was metastatic disease (or cancer that had spread) from the previous head and neck cancer, leading to a different treatment approach."

Comprehensive care for complex cases

Scott's throat cancer was challenging because it wasn't his only health condition, Dr. Zhao says. However, Mayo Clinic has the experience and expertise to navigate complex diagnoses.

"Head and neck cancer is a multidisciplinary disease," she says. "It requires working across specialties as a team to provide the care patients like Scott need."

After completing his treatments, Scott has been in remission from cancer for about a year. 

"So far, my cancer's all gone," he says. "You keep your fingers crossed, but you have to have a positive attitude about it."

Recently, Scott resumed some activities he enjoys, like golfing and fishing. 

"Going through cancer and treatment for cancer takes it out of you for quite a while," he says. "But it's better than the alternative, I'll tell you that."

The high point throughout the journey, Scott says, is how much help and support he has received from Mayo Clinic. 

"The resources you have available to you, the fact that they're all in one place is so helpful," he says. "I still have an issue with a chronic cough. A lot of times, you get scarring in your throat from radiation therapy. So they're trying to figure out whether it's nasal drainage getting trapped in my throat because of the scar tissue or allergies or sleep apnea. They're very thorough. They're evaluating everything. 

"They're also very honest. They'll say to me, 'Hey, you've got scarring in your throat. We may not be able to fix this 100%, but let's figure out what we can do to help it as much as possible.' I like that attitude."

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Multiple myeloma is a type of blood cancer caused by a buildup of abnormal plasma cells in the bone marrow. As the abnormal plasma cells accumulate, they crowd out healthy cells and cause symptoms such as bone pain and tiredness.

In 1980, Drs. Robert Kyle and Philip Greipp, Mayo Clinic hematologists, coined the term "smoldering multiple myeloma" to describe an accumulation of abnormal plasma cells in the bone marrow without any symptoms. Hematologists now know this condition usually has all the biological features of cancer and is associated with a high risk of progression to multiple myeloma.

Cancer researchers have since been investigating how, why and when smoldering multiple myeloma (SMM) progresses to active multiple myeloma and what can be done to delay progression. 

Dr. S. Vincent Rajkumar and Dr. Rafael Fonseca, Mayo Clinic Comprehensive Cancer Center hematologists, explain what SMM is and how treating it could delay or prevent multiple myeloma.

What is smoldering multiple myeloma?

The first phase of abnormal plasma cells building up in the bone marrow without causing symptoms or complications is a benign (noncancerous) condition called monoclonal gammopathy of undetermined significance (MGUS). Approximately 10% of people diagnosed with MGUS will develop multiple myeloma in their lifetime.

If abnormal plasma cells continue to accumulate and acquire additional biological abnormalities, MGUS evolves into SMM.

"Multiple myeloma is a malignancy (cancer) often associated with symptoms such as anemia, hypercalcemia and renal (kidney) failure. Smoldering multiple myeloma has many features of malignancy, but it is asymptomatic, so people don't know they have it," says Dr. Rajkumar, the Edward W. and Betty Knight Scripps-Edward C. Rosenow III, M.D., Professor of Medicine.

These conditions are more alike than not. "Through careful genetic analysis, we have learned that we cannot differentiate SMM from the active form of the disease," says Dr. Fonseca, the Getz Family Professor of Cancer.

When does smoldering multiple myeloma progress to multiple myeloma?

Dr. Rajkumar says researchers are trying to understand better when SMM progresses to multiple myeloma. "Some patients have low-risk smoldering multiple myeloma, which can stay dormant for a period of time but has a higher risk of progression than the precancerous MGUS stage, while other patients have high-risk smoldering multiple myeloma, which is more like full-blown multiple myeloma that is asymptomatic," he says.

High-risk SMM is more likely to progress to multiple myeloma than low-risk SMM.

"We expect disease progression in the first two years in about 50% of patients with high-risk SMM. After that, the rate of progression goes down, but we have followed patients from older clinical trials and found that the vast majority of people with high-risk SMM will eventually develop multiple myeloma," he says.

Dr. Rajkumar recommends that people diagnosed with MGUS or SMM seek care at a healthcare institution that specializes in myeloma and other blood disorders. "Patients need to be carefully evaluated to find out whether they have SMM, MGUS or active multiple myeloma. If they do have SMM, they need to know if it is low or high risk."

These questions sometimes require a second opinion. "Many of the referrals I get are from physicians and patients struggling with the question: 'Should we treat or not treat?' Even in high-risk SMM, the numbers and the condition's features matter. So, we are careful when counseling patients," says Dr. Rajkumar.

Is there screening for smoldering multiple myeloma?

There are no screening recommendations for SMM, so it is usually diagnosed by accident. As abnormal plasma cells accumulate, they release monoclonal proteins, or M proteins, into the blood. MGUS and SMM are sometimes discovered because a blood test conducted for another reason indicates the presence of these M proteins.

Dr. Rajkumar and other researchers are still weighing the risks and benefits of screening more people for SMM. Until they learn more, he does not recommend screening people with average risk.

"There might be some populations with high risk who should be considered for screening," says Dr. Rajkumar. "That includes people with two or more relatives with a history of multiple myeloma or people with African ancestry who have a relative with myeloma."

Hope for slowing multiple myeloma development.

Active surveillance is currently the standard of care for SMM. However, Dr. Rajkumar recently led a clinical trial to investigate whether treating patients with high-risk SMM could slow its progression.

"In this trial, we found that by the six-year mark after SMM diagnosis, 80% to 90% of patients in the observation arm of the trial had enough progression that many physicians would begin myeloma treatment," says Dr. Rajkumar.

Study results published in December 2024 showed promise toward a path to treat SMM. "The trial involved 390 patients with high-risk SMM. Half of the patients had careful active monitoring. The other half received daratumumab, a drug used to treat active multiple myeloma. The trial found that early intervention using daratumumab, which was given for three years, delayed progression to active myeloma and prolonged overall survival," says Dr. Rajkumar.

Dr. Rajkumar says multiple myeloma treatment often involves three to four drugs, bone marrow transplant and continuous therapy, which affect patients' quality of life. Slowing SMM's progression to active multiple myeloma also delayed the need for treatment. "The trial accomplished this with reasonable safety and without adversely affecting quality of life," he says. "This is a major finding."

"While there is still some debate about how to select these patients, I have no doubt this will be the future of timely intervention. Ideally, we'll be able to treat patients before they have to suffer from complications of the disease," says Dr. Fonseca. "It is because of rigorous clinical trials like Dr. Rajkumar's that we can start envisioning a brighter future for people with myeloma."

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When an aggressive lymphoma threatened to derail a couple's happily ever after, the Mayo Clinic Comprehensive Cancer Center helped them find a future.

Joanne McDonnell likens her longtime romance with her husband, Paul, to that of June Carter and Johnny Cash. The two have known each other since they were kids — specifically, third grade for Joanne and fifth for Paul. "She was my best friend's little sister," Paul explains. 

So, two years ago, when doctors told Joanne there wasn't anything else they could do for Paul's diffuse large B-cell lymphoma — an aggressive form of blood cancer — Joanne refused to believe their story was over.

"I know death comes for everyone, but I also knew it wasn't his time," she says.

Paul McDonnell learns his cancer is in remission.

Instead, Joanne, a retired human resources specialist for the Department of Defense, spent the next several months relying on two sources she'd come to trust: research and Dr. Madiha Iqbal, Paul's hematologist and oncologist at the Mayo Clinic Comprehensive Cancer Center in Jacksonville, Florida.

Facing hurdles

Paul learned he had cancer four years ago. He'd scheduled an appointment with an ear, nose and throat (ENT) specialist because he had swelling along the jawline on one side of his face. A biopsy a day or two later revealed it was lymphoma.

Initially, Paul underwent six months of chemotherapy-based treatments, but they weren't working well, so Paul's doctor, who knew Dr. Iqbal, referred him to her. Paul's doctor hoped that Dr. Iqbal, who specializes in bone marrow transplant and a form of immunotherapy called chimeric antigen receptor (CAR)-T cell therapy, might be able to help.

Dr. Iqbal first considered a bone marrow transplant for Paul. Bone marrow transplant, also called stem cell transplant, involves replacing a patient's damaged bone marrow with healthy cells. However, Dr. Iqbal said she and her team became a "little hesitant about the transplant" when they started to encounter obstacles with the process. Paul's body wasn’t producing enough stem cells to use for a transplant and his bone marrow was showing signs of damage from the chemotherapy treatments he'd received. 

"These hurdles derailed the plans we had for him," Dr. Iqbal says. 

Then, the team found signs his lymphoma had spread.

A novel treatment for aggressive lymphoma

In the past, oncologists didn't have much to offer patients with lymphoma, specifically diffuse B-cell lymphomas, who'd had two chemotherapy-based treatments and still had disease, Dr. Iqbal says. Today, however, they have a new tool in CAR-T cell therapy. CAR-T cell therapy works by altering a patient's T cells, or white blood cells, so they can recognize and destroy cancer cells.

"CAR-T cell therapy has significantly changed outcomes for patients with aggressive lymphomas, especially for those whose disease isn't very responsive to chemotherapy," Dr. Iqbal says. "Close to half of patients who, in the past, would have succumbed to their disease, are now able to go on with their lives thanks to this innovative treatment."

Dr. Iqbal started the process for Paul to receive CAR-T cell therapy. Then she faced a major hurdle: Paul's cancer had metastasized, or spread, and a tumor had grown so big it had penetrated his bowel and ruptured it. Paul was admitted to the hospital in critically ill condition — too sick for CAR-T cell therapy, and too sick to even undergo surgery to repair his bowel. 

Instead, doctors placed a tube into his bowel for drainage and placed him on total parenteral nutrition, meaning he had to have all his nutrition delivered directly into his bloodstream.  

"It was a serious situation at that time," Dr. Iqbal says. "While hospice was discussed as an option, I knew Joanne didn't want to give up on him."

After talking with Joanne and Paul, Dr. Iqbal decided to pursue a treatment course that would help get Paul's disease under control so they could try CAR-T.  

Navigating a road forward

Although nervous, Joanne was grateful to have a potential path forward for Paul. "Dr. Iqbal educated us, gave us books, answered our questions," she says. "She never discredited anything we said or asked. She was a big help for me as Paul's advocate."

Dr. Iqbal prescribed two rounds of chemotherapy to get Paul's disease under control. Soon afterward, he began to feel better. His tumor had also shrunk enough that Dr. Iqbal's team was able to collect his cells, and, about a month later, infuse them back into his body to target and kill cancer cells. 

The therapy was successful.

"Paul was far from an ideal patient to go into CAR-T cell therapy," Dr. Iqbal says. "We never want to put a patient through an intensive treatment if they're not healthy enough to withstand it. But because we were able to give him that stabilizing chemotherapy treatment that got him into a bit of a better place, it provided us with a window of opportunity to go ahead with the CAR-T cell therapy. And he was lucky, obviously, that it's resulted in him having a long-term remission."

A year and a half after Paul’s CAR-T cell therapy, he still has no evidence of disease. "He's a changed man ever since," Dr. Iqbal says, noting Paul's quality of life has improved drastically.

Joanne agrees: "She saved Paul's life."

Now, the married couple of nearly 43 years is "trying to figure out how to live again," Joanne says. "I tell Paul every day that God spared him for a reason. Thank God Dr. Iqbal didn't give up on him."

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In the U.S., it’s estimated that 4.5 million adults are diagnosed with chronic liver disease. It develops over time and may be caused by a number of conditions including, hepatitis, genetics, alcohol overuse or cancer. Chronic liver disease is different than acute liver disease, which can come on quickly and may be the result of an injury or a virus.

Regardless of the cause, Dr. Bashar Aqel, a Mayo Clinic transplant hepatologist, says when the liver can no longer function, a life-saving transplant may be needed.

Watch: The Mayo Clinic Minute

Journalists: Broadcast-quality video (1:17) is in the downloads at the end of this post. Please courtesy: "Mayo Clinic News Network." Read the script.

The liver is the largest internal organ of the body. It is essential for metabolism, digesting food and ridding the body of toxic substances. Liver failure can happen quickly, or over time.

"Once liver disease advances, the only way we can reverse the process and give the patient their life back is by replacing the liver, " says Dr. Aqel.

When patients become a candidate for a liver transplant, they are added to a waitlist. Unfortunately, there are more people waiting for a liver than there are available organs.

"As the patient is waiting on the list, they usually are anxiously waiting for that call when a donor becomes available. And that's what we call the deceased donor liver transplantation."

Another option is a living liver transplant. It's a major surgery where a person donates two-thirds of his or her liver. Donors needs to go through an extensive evaluation to make sure they can donate safely.

"A healthy liver is able to regenerate, and you will be surprised to know that with living donor liver transplantation, both segments of the liver will grow back to almost the normal size within 90 days after transplantation."

Being a living organ donor may not be an option for everyone, but there are ways to people can sign up to be an organ donor. It can be as easy as checking a box on your driver's license.

"Donating your organs is really donating the gift of life to people who are in urgent need for organ transplantation," Dr. Aqel says.

For the safety of its patients, staff and visitors, Mayo Clinic has strict masking policies in place. Anyone shown without a mask was either recorded prior to COVID-19 or recorded in a nonpatient care area where social distancing and other safety protocols were followed.

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ROCHESTER, Minnesota — It is a large part of the body that lies deep inside, out of sight and perhaps out of mind: your abdominal core. A new and rapidly developing area of medicine focuses on abdominal core health, including how people can incorporate it into a healthy lifestyle and how to address complex medical problems that arise when it is compromised. Dr. Charlotte Horne, a metabolic and abdominal wall reconstructive surgeon at Mayo Clinic, explains what abdominal core health is, how to protect it and risk factors for problems that may require surgery.

"The abdominal core is the outside muscular container of your abdominal wall," Dr. Horne says. "This starts at the diaphragm and goes all the way down to the pelvic support muscles. Most of the core is muscle and connective tissue. It's a muscular container that holds your internal organs in. Every time you breathe, bend, bear down to have a bowel movement, you're using these muscles."

The abdominal core includes abdominal muscles that people may think of as the "six-pack," and oblique muscles and tissue that wrap all the way around the upper part of the abdomen and connect to the midline of the body, Dr. Horne adds. Many nerves lie between the layers of muscle and tissue, including those that extend to the groin, thighs, back and hips.

Those muscles function as a unit and that unit needs to operate well for you and your body to perform daily activities, Dr. Horne says. One way to strengthen the structural integrity of your abdominal core is to engage it during your normal activities, she explains: You do not have to do thousands of sit-ups or become a bodybuilder.

"One of the things we're realizing is that we need to educate people how to appropriately engage those muscles when they do everything from going from lying to sitting and sitting to standing, lifting objects and other basic movements in their daily lives," she says. "When people do yoga or Pilates, they think about pulling their belly button into their spine. That helps stabilize the deeper muscles of the abdominal wall."

Improving abdominal core health involves conscious engagement of the abdominal muscles, "bringing everything in and holding it in," Dr. Horne says. 

Risk factors for abdominal core problems are wide-ranging. They include cancer treatment; inflammatory bowel disease; chronic or severe coughing; and complications from pregnancy (diastasis recti) and surgery. The most common problems are hernias, when part of an organ or tissue bulges through a weak spot in muscle.

"Coughing can cause large hernias. When you're coughing, you're bearing down and there is a sudden, acute change in intra-abdominal pressure, almost like punching from the outside in or from the inside out," Dr. Horne explains. "When you're doing that all of the time, it causes significant stress to your abdominal wall."

Avoiding smoking and excessive alcohol consumption can help protect abdominal core health. Smoking increases the risk of chronic coughing, while heavy alcohol consumption to the point of cirrhosis can cause hormonal changes that in turn weaken the abdominal wall, Dr. Horne says. 

As knowledge about abdominal core health grows, approaches to protecting it and healing it are advancing, Dr. Horne says. For example:

• Pregnancy causes muscles to expand to accommodate a baby, and sometimes those muscles do not go back to normal. Exercise regimens during and after pregnancy can help to stabilize them.
• Healthcare experts are realizing that restrictions on movement after surgery may not help and sometimes may be harmful. Rather than telling people not to lift anything, it may be more appropriate to explain how to safely reengage those muscles and tendons, Dr. Horne suggests.
• People with inflammatory bowel disease are likelier to have surgery and therefore are likelier to develop hernias. Surgeons now know that in those patients, mesh should be placed in different anatomic planes to prevent the mesh from touching the bowel and potentially causing problems later, Dr. Horne says.
• Pelvic floor physical therapy can help women experiencing urinary or fecal incontinence after pregnancy or menopause, she says.
• There is growing recognition that mesh used to repair hernias isn't one-size-fits-all. Dr. Horne's research focuses on hernia repair in women, including mesh and mesh techniques.

In her practice, Dr. Horne specializes in complex hernia surgeries.

"Most of the patients that I care for have more of their abdominal contents outside of their abdominal cavity than inside," she explains.

Even with hernias as large as 10 to 15 centimeters, surgeries can be performed with a robot to minimize the incision, or can be done with a mix of robotic surgery and minimal open surgery, Dr. Horne says. She uses 3D-printed models to help plan surgeries. Sometimes Botox is used to lengthen the abdominal wall muscles, she adds.

"The best part is that patients go from a dysfunctional abdominal wall to one that is functional within about a week in the hospital," Dr. Horne says. "Seeing them at their checkup a year later is the best thing because they go from saying `I couldn't do anything' to `I've gone on all these trips, I've done all these things that I've wanted to do for years but haven't been able to.' They are so happy that they can put that whole thing behind them, which is great."

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Media contact:

• Sharon Theimer, Mayo Clinic Communications, newsbureau@mayo.edu

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Dr. Denise Harnois, a Mayo Clinic transplant hepatologist, explains how this innovative strategy is expanding treatment options and improving outcomes.

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Learning you have advanced colorectal cancer that has spread to the liver can be scary. In some cases, a liver transplant may offer hope, providing better outcomes when other treatments aren't an option. 

"In those circumstances where patients have colon cancer that's gone to the liver that doesn't seem to be anyplace else within the body, but they're not eligible for consideration of doing a surgical resection — in those circumstances, we can consider replacing the entire liver, and that involves a liver transplant," explains Dr. Harnois.

It's a complex surgery requiring experts from multiple teams working together.

"We're working together as a team to make sure we are offering the best options for the potential for cure for these patients," she says.

Liver transplants have greatly improved survival rates. One-year survival is 80% to 100%, three-year survival is around 80%, and five-year survival is between 60% and 80%.

"Without consideration of liver transplant, their five-year survival rate would have been 15%. So we can offer a dramatic improvement in survival rates in patients that undergo liver transplant," says Dr. Harnois.

Related posts:

• Liver transplants offer new hope for colorectal cancer patients with liver metastasis
• Mayo Clinic Q and A:  Understanding colorectal cancer
• Mayo Clinic Minute: Warning signs of colorectal cancer in younger adults
• Innovative treatments for rectal cancer

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DEAR MAYO CLINIC: A friend was just diagnosed with colorectal cancer. She's only 30. I didn't know young people could get this cancer. Can you tell me more about it?

ANSWER: Unfortunately, your friend is among a growing number of adults under 50 who are being diagnosed with colorectal cancer. Many younger adults don't consider themselves at risk, since screening for colorectal cancer typically doesn't start until age 45.

That's why it's especially important to know the warning signs of colorectal cancer and not put off getting medical attention. Early diagnosis is essential to surviving colorectal cancer. If this cancer is found when it's only in the colon or rectum, the five-year survival rate is over 90%. That survival rate drops to roughly 15% if the cancer spreads beyond the colon and rectum.

 What is colorectal cancer?

It's not one type of cancer but two: colon and rectal cancer. The large intestine has several regions. The last part of the large intestine is known as the rectum. The other areas of the large intestine are called the colon. The treatments for cancers of the colon and rectum are different.

Who is at risk for this cancer?

Risk factors for colorectal cancer include:

• Older age
• Black race 
• Low-fiber, high-fat diet 
• Not exercising regularly
• Diabetes 
• Obesity 
• Smoking
• Drinking alcohol 

Those who are at higher risk for colorectal cancer tend to have at least one of these factors:

• Medical condition that places them at higher risk.
• Genetic condition that increases their risk. 
• Family history that places them at higher risk. For example, this is could be a first-degree family member such as parents or siblings who have had a diagnosis of colorectal cancer or advanced polyps.

Are there warning signs?

Four warning signs of colorectal cancer are:

• Abdominal pain
• Rectal bleeding
• Diarrhea
• Iron deficiency anemia

Although these symptoms can be embarrassing, you need to share them with a healthcare professional — don't put it off.

You may not easily notice blood loss after bowel movements, but that bleeding can lead to low iron levels. If a routine blood test reveals iron deficiency in a young adult, it can prompt the primary care clinician to consider colorectal cancer.

What is the screening process for colorectal cancer?

Your primary healthcare professional may order a screening that can help detect colon polyps or cancer. These tests include:

Stool DNA test. This test uses a stool sample to look for changes in cells' genetic material. Certain DNA changes are a sign that cancer is present or that it might happen in the future. The stool DNA test also looks for hidden blood in the stool.

If your stool DNA test returns positive (abnormal), you'll need a follow-up colonoscopy.

Colonoscopy. This test looks inside the colon. For it to be most effective, you need to thoroughly cleanse your colon, which involves reducing the fiber in your diet. The last step is consuming a special solution to ensure your colon is clean so the gastroenterologist is able to get a clear view. Although preparing for the procedure can be unpleasant, your care team can provide tips to make it more doable.

If the gastroenterologist finds any precancerous growths, called polyps, these will be removed during the colonoscopy and sent for testing. The number, size and type of polyps removed help determine when the next colonoscopy should be scheduled. If cancer is found, your care team will discuss treatment options with you.

How can I reduce my risk for colorectal cancer?  

No matter your age, there are five steps you can take to avoid developing colon cancer:

1. Eat your vegetables and healthy fats. Research has shown that the Western diet correlates to higher colorectal cancer rates. People who eat high-fiber diets are less likely to develop the disease.
2. Get moving. Exercise benefits your heart and helps you maintain your weight. It also can lower your colon cancer risk. Aim for 30 minutes of moderate exercise on most days of the week.
3. Watch your weight. Talk to your health care team if you need help losing weight.
4. Limit alcohol and don't smoke. If you choose to drink alcohol, do so moderately. That means no more than one drink a day for women or two drinks a day for men. And if you smoke, quit.
5. Follow colorectal cancer screening guidelines.

Derek Ebner, M.D., Gastroenterology, Mayo Clinic in Rochester

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The body has a built-in defense: the immune system, which consists of white blood cells, lymph system tissues and organs that work together to identify and destroy infections and abnormal cells. However, cancer cells use genetic changes to hide their abnormality or interfere with the immune system's ability to detect and destroy them. Immunotherapy helps the immune system overcome these defenses.

"We're working with what the body naturally does but has not done perfectly, as it has allowed cancer to develop. We're trying to reinforce the body's natural defenses," says Svetomir Markovic, M.D., Ph.D., a Mayo Clinic Comprehensive Cancer Center medical oncologist who researches immunotherapy for melanoma and non-Hodgkin lymphoma.

"Immunotherapy leverages the patient's immune system to fight cancer. It activates and pushes it to find the tumors and kill them," says Haidong Dong, M.D., Ph.D., a Mayo Clinic Comprehensive Cancer Center cancer immunologist. "You can use different tools — T cells, vaccines, targeted drugs — but they all use the patient's immune cells to fight their tumors."

Dr. Markovic and Dr. Dong discuss the history of cancer immunotherapy at Mayo Clinic, how it works, its benefits and risks, and its future:

The roots of cancer immunotherapy at Mayo Clinic

In the late '90s, Dr. Dong and his colleagues at Mayo Clinic discovered that a protein called PD-L1 regulates the immune system's response to cancer cells and other threats. PD-L1 acts as a check or brake by binding to another protein in T cells called PD-1. This discovery led them to develop an antibody that blocks PD-L1's function.

"Using the antibody to block PD-L1 restores the immune system's ability to kill the tumor," says Dr. Dong, the Iris and Winston Clement Professor of Research. "This breakthrough laid the foundation for immunotherapy drugs that target the PD-L1 pathway."

In 2002, Dr. Dong and his team reported their findings, paving the way for the development of new immunotherapy drugs called immune checkpoint inhibitors. In 2014, the Food and Drug Administration approved pembrolizumab as the first immune checkpoint inhibitor to treat melanoma. Today, pembrolizumab is approved to treat 18 types of cancer.

How does cancer immunotherapy work?

Cancer immunotherapy drugs don't directly target cancer cells. Instead, they enhance the body's immune cells, improving their ability to recognize and destroy cancer cells.

"Immunotherapy helps the immune system see where the tumor is in the body. The immune system can see a single cancer cell. That's powerful," says Dr. Dong.

"Immunotherapy helps the immune system see where the tumor is in the body. The immune system can see a single cancer cell. That's powerful."Dr. Haidong Dong

"It's almost like cancer therapy by proxy," says Dr. Markovic, the Charles F. Mathy Professor of Melanoma Research. "We're engaging the body to do its job in ways that make it uniquely effective."

Some of the most common immunotherapies used in cancer treatment include:

Immune checkpoint inhibitors

Immune checkpoint inhibitors are immunotherapy drugs that block checkpoint proteins, such as PD-L1, on cancer cells from binding to their partner proteins on immune cells (T cells). This allows the immune system to destroy cancer cells. Pembrolizumab is one example of an immune checkpoint inhibitor. Eleven immune checkpoint inhibitors have now been approved to treat cancer.

Most of these drugs are used for advanced cancer or cancer that has spread (metastasized). However, some immune checkpoint inhibitors are now being used in earlier stages of cancer to prevent its spread and recurrence. Clinical trials are also investigating combinations of these drugs with other treatments and new immune checkpoint inhibitors.

Learn about immune checkpoint inhibitors and related research at Mayo Clinic:

• Immune checkpoint inhibitors: Immunotherapy for people with triple-negative breast cancer
• From molecule to melanoma: A foundation of research brings new cancer immunotherapies to patients

Oncolytic viruses

Oncolytic viruses use naturally occurring or lab-made viruses to infect cancer cells, causing them to rupture. This stimulates the immune system to attack the remaining cancer cells.

Most oncolytic virus treatments for cancer are still in clinical trials. A weakened form of herpes simplex virus type 1 given by injection is approved to treat melanoma on the skin or in lymph glands.

Learn about oncolytic virus research at Mayo Clinic:

• Novel oncolytic immunotherapy shows promise for patients with bladder cancer
• Unleashing viruses aimed at killing cancer
• Mayo Clinic researchers load CAR-T cells with oncolytic virus to treat solid cancer tumors

Personalized cancer vaccines

Personalized cancer vaccines train the immune system to recognize a specific cancer-related protein from a person's tumor. As the immune system learns to recognize the protein, it can fight it. This treatment is still being studied in clinical trials.

Learn about personalized cancer vaccine research at Mayo Clinic:

• Mayo Clinic Minute: How personalized vaccines target cancer tumors
• Vaccines: The next frontier of lung cancer treatment and prevention
• Tomorrow's Cure: How personalized vaccines combat cancer
• Mayo Clinic researchers design personalized vaccines to fight cancer
• Harnessing the immune system to fight ovarian cancer

Cellular therapies

Cellular therapies involve collecting cells from blood and modifying them to attack cancer cells. These include chimeric antigen receptor (CAR)-T cell therapy and tumor-infiltrating lymphocyte (TIL) therapy.

• CAR-T cell therapy: For this therapy, white blood cells called T cells are removed from a person's blood and genetically modified to produce chimeric antigen receptors. CARs allow T cells to recognize markers on the surface of cancer cells, activating the T cells to kill them. The modified CAR-T cells are then infused into the patient's body to identify and destroy their cancer. The FDA has approved CAR-T cell therapy to treat several types of blood cancer, and clinical trials are exploring its use for solid tumor cancers.
• TIL therapy: This therapy involves surgically removing a person's cancerous tumor, extracting immune cells (lymphocytes) from it, and enriching them. During this process, the patient receives chemotherapy to prepare to receive the enriched immune cells, which are later infused into their body to target any remaining cancer. "The first treatment the patient receives warms up the immune system — improves it to a degree ­— but not enough to kill the tumor. TIL therapy gives the immune cells more power to finish the job," says Dr. Dong. In 2024, the FDA approved the first TIL therapy, lifileucel, for people with melanoma that has spread or cannot be removed by surgery.

Learn about cellular therapy research at Mayo Clinic:

• What is CAR-T cell therapy?
• Understanding CAR-T cell therapy for cancer: Mayo Clinic expert explains how it works
• Unleashing CAR-T cell therapy to destroy solid tumors in thyroid cancer
• Using molecular scissors to improve CAR-T cell therapy
• Preparing to biomanufacture a new CAR-T cell therapy for B-cell cancers

Cytokines

Cytokines are proteins made by white blood cells that signal the immune system to either activate or slow down. Cytokines used as immunotherapy drugs include interleukins and interferons:

• Interleukins: These act as chemical signals between white blood cells. Aldesleukin is a lab-made interleukin (IL-2) that increases the growth and activity of certain white blood cells. It is used to treat melanoma and kidney cancer that has spread throughout the body.
• Interferons: These help the body resist viruses and cancer cells. Interferon alfa-2b is a type of interferon (IFN-alpha) used to treat hairy cell leukemia, melanoma, follicular lymphoma and AIDS-related Kaposi sarcoma.

Bispecific antibodies

When the immune system detects cancer cells or other abnormal cells, it produces immune cells called antibodies to attack them. Bispecific antibodies are lab-made antibodies that can bind to both cancer and immune cells simultaneously, bringing them close together to enhance the immune system’s ability to destroy cancer cells.

The FDA has approved seven immunotherapy drugs that use bispecific antibodies to treat cancer.

Learn about bispecific antibody research at Mayo Clinic: "New research discovers a new combination of therapy for people with a type of leukemia, leading them to live longer."

What are the benefits and risks of immunotherapy?

Immunotherapy is effective against many types of cancer because it uses the immune system to recognize and attack cancer cells. "We don't yet know which immunotherapy best suits all malignancies. As we learn the biology of all this, as our intervention tools become more sophisticated, the number of cancers we can treat increases," says Dr. Markovic.

"We don't yet know which immunotherapy best suits all malignancies. As we learn the biology of all this, as our intervention tools become more sophisticated, the number of cancers we can treat increases."Dr. svetomir Markovic

Immunotherapy can also produce long-lasting responses to treatment by training the immune system to recognize cancer and respond quickly if it returns.

However, immunotherapy can cause side effects. "That’s the price we pay," says Dr. Markovic. "When turned on, the immune system attacks the cancer but may also attack noncancerous tissue, producing side effects similar to autoimmune disease symptoms. They mimic allergic reactions."

Side effects vary based on the type of immunotherapy. Some can be serious, but most can be managed, treated and resolved. Common side effects include redness, itching or blistering of the skin, flu-like symptoms, diarrhea, swelling and weight gain.

Mayo Clinic scientists and colleagues worldwide are researching how to reduce immunotherapy's side effects and increase its benefits. "As we get better with cancer immunotherapy, our treatments become more effective, less toxic and help more people," says Dr. Markovic.

The future of cancer immunotherapy

Scientists are researching ways to improve immunotherapy outcomes and reduce its limitations. Dr. Dong says research is exploring these key areas:

• Understanding why the responses vary from person to person.
• Combining immunotherapy with other treatments, such as chemotherapy, radiation therapy, or targeted drug therapy, to produce the best outcomes.
• Reducing side effects.
• Identifying biomarkers to help predict which people will respond to immunotherapy.

Dr. Dong says this research could improve the effectiveness of immunotherapy and patients’ quality of life. It will also help care teams make more informed treatment decisions and may decrease the financial burden of cancer treatment.

"Immunotherapy has fundamentally changed cancer medicine with the possibility of a cure for cancers that have been considered completely incurable," says Dr. Markovic. "It was once unheard of for patients with metastatic, advanced melanoma to be cured of their disease. Today, a person with this diagnosis has a one in three chance of being cured. That’s a dramatic improvement in outcomes."

Learn more

Drs. Dong and Markovic encourage you to ask your care team specific questions about your treatment. Other trusted sources of information about immunotherapy include:

• American Cancer Society
• Cancer Research Institute
• National Cancer Institute
• National Comprehensive Cancer Network

Find a clinical trial at Mayo Clinic.

Join a support group on Mayo Clinic Connect, an online community moderated by Mayo Clinic for patients and caregivers.

This article first published on the Mayo Clinic Comprehensive Cancer Center blog.

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ROCHESTER, Minnesota — Dr. Eric Moore has been appointed medical director, International at Mayo Clinic. Dr. Moore will begin his new position in April.

Dr. Moore exemplifies dedication to patient care and is globally recognized as a leader in his field. He brings more than three decades of experience in clinical care, education and research to the role. He is a professor and chair of the Department of Otolaryngology — Head and Neck Surgery at Mayo Clinic in Rochester.

Dr. Moore is known for advancing care for complex ear, nose and throat conditions and leveraging research expertise to innovate therapies. He is passionate about sharing knowledge and expertise to improve global healthcare. Dr. Moore is a pioneer in minimally invasive transoral robotic surgery and has improved outcomes for patients with HPV-related cancers and oropharyngeal cancers.

As a dedicated mentor, Dr. Moore has contributed to otolaryngology education and received multiple awards for his work. He is a five-time recipient of the Mayo Fellows Teacher of the Year Award.

Before joining Mayo Clinic, Dr. Moore was a major in the U.S. Air Force. He attended medical school at Jefferson Medical College in Philadelphia and completed residency training at Mayo and a fellowship in Austria.

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About Mayo Clinic
Mayo Clinic is a nonprofit organization committed to innovation in clinical practice, education and research, and providing compassion, expertise and answers to everyone who needs healing. Visit the Mayo Clinic News Network for additional Mayo Clinic news.

Media contact:

• Elizabeth Baxter, Mayo Clinic Communications, newsbureau@mayo.edu

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