There is new hope in the fight against glioblastoma, the deadliest and most aggressive form of primary brain cancer.

Currently there is no cure, but results of a new study conducted at Mayo Clinic show patients experienced improved overall survival while maintaining quality of life after undergoing a novel approach to treatment.

Watch: Breakthrough in the fight against glioblastoma

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Richard Casper was one of the study participants. The Arizona man enrolled in the clinical trial at Mayo Clinic in Phoenix after his diagnosis of glioblastoma in 2019. Richard was given only months to live, but his family says thanks to his strength, perseverance, and innovative medical care, he survived nearly double the amount of time.

"To be almost two and a half, three years in after being told you only have a few months, it's quite remarkable," says Susan Casper, Richard's daughter.

Richard succumbed to the disease in 2023. During the treatment, his family says he had little to no side effects. In the months before his passing, Richard stated, "I feel great. If someone didn't tell me I have the glioblastoma, I wouldn't even know it."

The clinical trial was led by Dr. Sujay Vora, a radiation oncologist at Mayo Clinic in Arizona. The small, single-arm study incorporated the use of advanced imaging technology combined with cutting-edge radiation therapy in patients over the age of 65 with newly diagnosed World Health Organization (WHO) grade 4 malignant glioblastoma.

"The patients lived longer than we expected. This patient population is expected to live six to nine months. Our average survival was 13.1 months. There were some patients that were out closer to two years. The results exceeded our expectations. We are very pleased," says Dr. Vora.

The study is published in The Lancet Oncology.

Why is glioblastoma so deadly?

Glioblastoma is among the most challenging cancers to treat. The disease is aggressive and invades healthy brain tissue with hairlike tentacles. "That is why doing a complete surgery is very difficult, as compared to say breast cancer, where a lumpectomy can be performed to remove not only the tumor, but a healthy rim of tissue around it," explains Dr. Vora.

Surgery for glioblastoma presents a different set of obstacles. "When it comes to glioblastoma, it is challenging to do that level of surgery. You try to surgically remove whatever you can safely without leaving the patient worse off after surgery."

Another factor that makes glioblastoma so lethal is that it can be fast- growing and unresponsive to treatment.

"These cancer cells are quite challenging to overcome," says Dr. Vora. "There are some patients we see after their surgery, and by the time we are ready to start their treatment, they've already had a recurrence of the disease."

An estimated 14,500 people will be diagnosed with glioblastoma in the U.S this year. "In the best of circumstances the average survival rates are in the 14-15 month range. But for patients 65 and older, the group that was the focus of our study, patients do even worse. The prognosis for this population is between six and nine months," says Dr. Vora.

Symptoms of glioblastoma

• Headache
• Nausea and vomiting
• Confusion or decline in brain function
• Memory loss
• Personality changes
• Vision changes
• Speech difficulties
• Trouble with balance
• Muscle weakness
• Seizures

Attacking glioblastoma with a triad

For the clinical trial, Dr. Vora and his team mapped out a plan that would allow them to be more intentional and precise with treating the location of the glioblastoma.

The imaging incorporated the use of ¹⁸F-DOPA PET and contrast-enhanced MRI. "¹⁸F-DOPA PET is an amino acid tracer that can cross the blood brain barrier, and it can accumulate within the glioblastoma cells itself," says Dr. Vora.

Researchers combined these images to determine the location of the most metabolically active "hot spots" of the cancer in the brain.

Taking aim with proton beam therapy

Study investigators used one of the most advanced forms of radiation treatment, called proton beam therapy.

"With standard radiation, the beams go through the brain tissue, so there's an entrance dose and the exit dose. But with proton beam therapy, we dial up how deeply we want the radiation to go," explains Dr. Vora. "It drops off its energy in the tumor, and then there's basically no radiation after that. It allows us to be more preferential into the delivery of radiation and protect more of the healthy surrounding tissue."

During proton beam therapy, a patient lies on a table while the machine rotates around the patient's head targeting the tumor with an invisible beam. The patient is awake for the procedure. It is painless with many patients reporting fewer to no side effects.

Unlike traditional radiation for glioblastoma, which is typically delivered over the course of three to six weeks, treatment with proton beam therapy for the study was conducted in one to two weeks.

"I am hopeful that this is the first step of many where we can continue to move the needle and allow patients to live longer and live well," says Dr. Vora. "The goal is to improve the outcomes for our patients allowing them to spend more time with their families."

Nadya's story

2022 was a tough year for Nadya El-Afandi. She was on the verge of celebrating a long but successful battle with breast cancer.

"Out of the blue, I had a seizure. I went to the hospital, they did an MRI. After additional tests they told me the news: 'You have a glioblastoma,'" recalls Nadya. She asked her doctor if she should continue her breast cancer treatment. "He said, 'No'-meaning the glioblastoma would kill me before the breast cancer."

Nadya was not about to give up.

Nadya is a wife and mother of four children. She lives just outside of Rochester, Minnesota. "My children said to me, 'Mom, you're a unicorn. Of course you'll live.' My mother has had a number of medical conditions and we never expected her to live this long. And she's still alive and with us," says Nadya. "My children have also said, 'You've got grandma's blood in you. You will live.'"

Nadya is receiving care at Mayo Clinic in Rochester. That's where she learned about a new clinical trial called SAGA, or stereotactic ablative radiation treatment for glioblastoma. The phase 2 clinical trial is building upon Dr. Vora's research and studying a larger group of patients. The study is being led by Dr. William Breen, radiation oncologist at Mayo Clinic in Rochester.

It has been 15 months since Nadya began treatment for glioblastoma. So far, there is no sign the glioblastoma has returned. "Nadya has already exceeded the time that's expected time to have a tumor recurrence, and she continues to do well," says Dr. Breen. "Nadya is beyond some measures of what the average, overall survival time would be."

While Nadya's progress in encouraging, Dr. Breen says it is important to note that it is too early to draw any conclusions about safety or efficacy of this approach to treatment until the study is completed.

Meanwhile, Nadya is focused on living her life to the fullest. Fifteen months after undergoing treatment, Nadya embarked on an adventure to Hawaii, where she spent time taking helicopter tours, snorkeling and hiking.

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• https://cancerblog.mayoclinic.org/2021/12/21/glioblastoma-in-older-adults-improving-survival-and-quality-of-life/
• https://cancerblog.mayoclinic.org/2024/05/15/technology-clinical-trials-offer-hope-after-glioma-diagnosis/

The post (VIDEO) Breakthrough in the fight against glioblastoma appeared first on Mayo Clinic News Network.

If talking were an Olympic sport, Marty Kedian would be a gold medal winner.

After undergoing a history-making larynx transplant at Mayo Clinic in Arizona, the Boston-area native is back home drawing lots of attention with his gift for gab. Marty was greeted with a spirited welcome home celebration including family, friends and a pack of news reporters, all abuzz with excitement to hear his voice for hope.

Watch: Larynx transplant patient inspires hometown with his voice for hope

Journalists: Broadcast-quality video pkg (1:30) is in the downloads at the end of this post. Please courtesy: "Mayo Clinic News Network." Read the script.

Silenced by cancer

For the past decade, Marty battled a rare form of laryngeal cancer called chondrosarcoma.

"I had dozens of surgeries and treatments that, over the years, took a toll on my larynx. After the last surgery, I woke up and my voice was gone," says Marty.

The larynx, also known as the voice box, is located in the throat. Its main functions are helping people to speak, swallow and breathe.

"I had to get a tracheostomy tube implanted to help me breathe, and since then, my quality of life went downhill. People would look at me like I was strange," says Marty. "I got to the point I just didn't want to leave the house anymore."

Determined to find a way to get her husband's voice back, Marty's wife Gina scoured the internet for solutions. Her search led her to Dr. David Lott and Mayo Clinic's Larynx and Trachea Transplant Program.

Dr. Lott and his team were conducting the first clinical trial in the U.S. on laryngeal transplantation. "I applied as a candidate for the program and got accepted. They told me it wouldn't be easy, but I was not going to give up," says Marty.

The groundbreaking larynx transplant

A team of multidisciplinary doctors, including six surgeons, performed the 21-hour transplant on Feb. 29. The first step was to remove Marty's larynx.

"Our first priority was to remove the cancer," explains Dr. Lott. "When we were confident the cancer was gone, we then focused on transplanting the donor larynx."

Laryngeal transplantation is a rare procedure that has been performed only a handful of times in the world. Marty's case was the third total larynx transplant in the U.S., and the first known case performed as part of a clinical trial. Dr. Lott hopes the clinical trial will allow his team to scientifically investigate the procedure to prove it is a safe and effective option for patients.

The Mayo transplant is also the first known case in the world of a larynx transplant performed on a patient with an active cancer. Doctors were able to proceed with the transplant because Marty was already on immunosuppressive therapy from a previous kidney transplant.

"Having a patient with an active cancer who already had his own immune suppression allowed us to do the transplant safely without introducing additional risk in a way that has rarely, if ever, been done before," says Girish Mour, M.B.B.S., medical director of the program. 

Currently there are tens of thousands of people worldwide who are living without a voice box, mostly due to trauma or cancer. According to the American Cancer Society, it is estimated there will be 12,650 new cases of laryngeal cancer in 2024. Dr. Lott says many of those patients will be among the highest at risk of losing their larynx.

Breaking the silence

In just over four months after his surgery, Marty had regained full ability to swallow foods, was making progress learning to breathe on his own again and speaking with his voice at 60%.

"I was able to talk to my 82-year-old mother on the phone," says Marty. She could hear me and understood every word I said. It is amazing."

"It is remarkable," says Dr. Lott. "Marty has exceeded all of our expectations. I didn't expect him to be eating a hamburger this soon after a larynx transplant, if ever. It is truly amazing."

Welcome home Marty

Marty and Gina celebrated the successful transplant with an extended road trip on their way home. "We wanted to take a moment and appreciate where this journey has led us and where we are today," says Marty.

The couple had already made a visit to the Grand Canyon. After leaving Phoenix, they stopped in San Diego to see the Pacific Ocean, visited the Golden Gate Bridge, and got a glimpse of Mount Rushmore and Niagara Falls.

"It feels so amazing to be able to talk to anyone I meet on the street," says Marty. I will be forever grateful to Dr. Lott and Mayo for giving me my life back. I am also deeply grateful for the generosity of my organ donor and the donor family. They are the true heroes of this story."

When the couple arrived home in Haverhill, they were greeted by a cheering crowd of family, friends and reporters all eager to hear his voice.

Marty Kedian talks with Boston Media on Aug. 1, 2024

"I will share my story with anyone who wants to hear it," says Marty. "My job right now is to heal up and get my voice back 100%. My next job will be to reach out to as many people as I can and let them know they can do it too."

The post Larynx transplant patient inspires hometown with his voice for hope appeared first on Mayo Clinic News Network.

Mayo Clinic Comprehensive Cancer Center and Columbia University recently announced being named the recipients of a $10.6 million grant from the National Cancer Institute to advance research in fighting glioblastoma - one of the deadliest and most aggressive forms of brain cancer.

Every year, thousands are diagnosed with the disease. Many are given little time to live. The prognosis is often poorer for older patients.

Researchers are searching for a cure. Meanwhile a team at Mayo Clinic is also looking for ways to help elderly patients diagnosed with glioblastoma live longer and maintain quality of life.

Now, an investigational study at Mayo Clinic using an innovative approach to treatment has shown promise in improving health outcomes for people like Richard Casper.

Watch: The fight against glioblastoma, one of the deadliest cancers

Journalists: Broadcast-quality video is in the downloads at the end of this post (3:52). Please courtesy: "Mayo Clinic News Network." Read the script.

Love at first sight

Richard and Carol Casper were in their early 20s when they met. Carol was smitten, but not sure Richard would call her again.

"So, anyway, he did," recalls Carol with a twinkle in her eye. "That's where it all started in 1962, and we got married in 1964."

Children soon followed, and their family began traveling the world with Richard's career in the U.S. Air Force. 

The day their world changed 

After nearly 30 years of military service, the couple was looking forward to retirement. Then suddenly, in 2019, Richard suffered a seizure and their world came crashing down. Testing began to identify the cause of his seizures.

Ultimately surgery confirmed the family's worst fear - brain cancer.

"That's when we got the definite diagnosis that it was glioblastoma, stage 4," says Carol. Doctors told the family Richard had less than a year to live. 

Glioblastoma: The deadliest form of brain cancer 

Glioblastoma is one of the most complex and fast-growing forms of brain cancer. It is also the most common primary malignant brain tumor in adults, though it can strike at any age. It can form in the spinal cord or the brain.

"It carries the worst prognosis, and it has to do with the aggressiveness of the cancer," says Sujay Vora, M.D., radiation oncologist with Mayo Clinic Comprehensive Cancer Center in Arizona. "Despite our best treatments, we see patients continue to recur, which requires additional treatments over time."  

According to the National Brain Tumor Society, more than 14,490 Americans are expected to be diagnosed in 2023. They estimate approximately 10,000 will die from the disease. 

Research to improve patient outcomes

Dr. Vora has been researching brain tumors and ways to improve patient outcomes for the past two decades. His team is now investigating an innovative treatment protocol for elderly patients, like Richard, who are often given less time than average to live.

"Most of these patients are in the six- to nine-month range," says Dr. Vora. "We're looking for ways of providing more effective and better treatments for patients in terms of both quality and quantity of life."

Richard sought treatment at Mayo Clinic in Arizona under the care of Alyx Porter, M.D., a neuro-oncologist. "When people come in, it is one of the scariest times in their life," says Dr. Porter. "My job is to provide them with comfort, reassurance and direction as to what the next steps will be."

Mayo Clinic's multidisciplinary approach

Mayo Clinic's healthcare model is based on a team approach. Physicians from a variety of disciplines work together to explore the best treatment options for a patient and deliver the highest level of care.

Richard's family was aware there was likely no cure. What they wanted was more time than his prognosis. His healthcare team agreed Richard was an ideal candidate for the clinical trial. Richard told doctors he was ready to be a participant.

Targeting cancer cells with advanced medical imaging

Radiation and chemotherapy are often used to try and slow progression of the cancer. The study is investigating an innovative approach researchers hope will help elderly glioblastoma patients live longer with fewer symptoms of the cancer and side effects from treatment.

It incorporates the use of an advanced medical imaging technique, called 18F-DOPA PET and MRI to target specific areas of the brain for treatment and spare healthy tissue.

"18F-DOPA is more or less a special type of sugar that cancer cells take at a faster rate compared to healthy cells that is very specific for glioblastoma. So, with this PET scan, we can actually now identify areas in the brain that are felt to be at highest risk for tumor growth," says Dr. Vora.

The other critical part of this regimen is that it's much more convenient for patients. "Instead of the traditional three to six weeks' worth of radiation, we're able to complete their entire treatment in one to two weeks, which allows more time for patients to stay with their family," says Dr. Vora.

The areas are then targeted with proton beam therapy to destroy cancer cells. Proton beam therapy is innovative technology that can painlessly deliver higher doses of radiation, in a shorter amount of time, with fewer side effects.

"I felt great," says Richard after completing his treatment. "I wouldn't even know I have a brain tumor right now if someone didn't tell me so."

Dr. Porter credits the study's treatment protocol. "With the shorter course of treatment, it saved him from many of the side effects that we know can be as devastating as the disease itself. He was able to finish the treatment, unscathed, walking and dancing into my office thrilled at how good he actually felt." 

Richard beats the odds 

Richard survived almost twice longer than his original prognosis while maintaining his quality of life. "My dad defied the odds and kept going," says Susan, Richard's daughter. "To be almost 2 ½ years in after being told you only have a few months left to live, is quite remarkable."

The Casper family says they're proud of Richard's courage to help in the fight against cancer. "What he went through, and even during the trial, made a big difference in helping other people," says Carol.  

Richard maintained his valiant battle till the end. He died on March 8, 2023. "All of us at Mayo feel very fortunate to have known Mr. Casper," says Dr. Vora. "He was a very special person."

Dr. Vora presented results of the trial at the 2023 American Society of Clinical Oncology.

"The results are quite promising," says Dr. Vora. "We're hoping to see these results carried on in a larger-scale study. If we continue to see the same types of results, we feel it could be a good option moving forward for all cancer patients." 

Related posts:

• Mayo Clinic and Columbia University receive $10.6 million grant from NCI to advance glioblastoma research with mathematical oncology
• Consumer Health: What is glioblastoma?
• Discovery’s Edge: Battling brain cancers through genomics

The post Fighting glioblastoma — one of the deadliest forms of brain cancer appeared first on Mayo Clinic News Network.

Dawn Botsford was sitting in a chair at the hair salon in 2011 when her hairdresser commented on a spot on the top of her head.

It was bug season in North Dakota, so Dawn didn’t think much of it. "When I told my hairdresser that, she said, ‘I really think you need to get it checked out.' She saved my life," Dawn recalls.

Dawn met with a local dermatologist to have the spot examined. A few hours after her biopsy, Dawn's dermatologist told her she had melanoma, a form of skin cancer.

Dawn's dermatologist wanted her to see a local oncologist as soon as possible, but there were no appointments available.

"With that kind of news, I got worried," Dawn says.

Dawn visited her local physician for another trusted opinion. His comment was even more unsettling: "This looks pretty serious."

He then picked up the phone and called Mayo Clinic to make a referral.

No. 1, and six hours away

After receiving news of availability and discussing it with her husband, John, and their son, Tom, Dawn made an appointment at Mayo Clinic in Rochester. Dawn and John made the trip to Mayo Clinic and met with Eric Moore, M.D., the next day.

Dr. Moore scheduled surgery for Dawn two days later.

"We're fortunate to have the No. 1 medical system in the world a six-hour drive away," Tom says. "We never really questioned that we were going to go to Mayo."

They really wanted to give me hope that this diagnosis was not fatal.

Dawn Botsford

In October 2011, after several appointments and the initial surgery to remove the spot, doctors confirmed that Dawn's melanoma was late-stage.

"The physicians at Mayo Clinic were all so kind and caring," Dawn says. "They really wanted to give me hope that this diagnosis was not fatal."

A month after surgery, Dawn developed an infection in the incision on the top of her head. She happened to be in Minneapolis with family, and they decided to head to Mayo Clinic in Rochester's emergency department, about 75 miles away. When they arrived, Dawn was amazed to see a doctor waiting for her who had read her chart and was up to speed on what was going on.

"That is remarkable patient care," she says.

Finding hope in a clinical trial

In early 2013, doctors performed a precautionary lung X-ray and found that melanoma had spread to Dawn's lungs.

Her oncologist, Robert McWilliams, M.D., suggested she enroll in a clinical trial with an immunotherapy drug, conducted by Svetomir Markovic, M.D., Ph.D., a medical oncologist and hematologist who specializes in the care of patients with advanced melanoma.

"Dr. Markovic said a clinical trial was her only option, and that the survival rate at the time was 2%," Tom says. "I still remember sitting in our home office, my dad and I Googling this stuff, looking for papers on outcomes and survival rates. That's what we found — 2%."

Dawn decided to take part in the study.

"It was always equally as important to her that she was participating in a clinical trial, not knowing if it would help her, but knowing that the results would eventually help others," John says.

Best bet: surgery

By the end of 2013, Dr. McWilliams and thoracic surgeon Stephen Cassivi, M.D., decided that surgery on both lungs was now Dawn's best bet.

The entire team of researchers, providers and staff who saved my life and always gave me hope is something the clinic can be very proud of.

Dawn Botsford

"I got back to the hospital, and in my room on the whiteboard there was a sign that said, 'Welcome back, Dawn!' I just felt like everybody took such good care of me," Dawn says. "They didn't know it, but they were challenging me, too. I had to get my lungs working again."

In July 2015, another spot was found on Dawn's right lung. She then had three stereotactic radiation treatments, a noninvasive radiosurgery technique. After her treatments, Dawn came back to Mayo Clinic every six months for routine testing.

10 years later

After numerous PET scans and appointments, and with the strong support of family and friends, Dawn officially completed her time with the oncology program at Mayo Clinic in July 2020 — 10 years after the first spotting of melanoma and five years since the last recurrence.

Following completion of her treatments, Dawn and her family decided to support Mayo Clinic through philanthropy. Mayo Clinic recognizes the Botsford Family Foundation as a Major Benefactor for the family's generosity.

"I can't say enough about the people and the facilities at Mayo Clinic," Dawn says. "The entire team of researchers, providers and staff who saved my life and always gave me hope is something the clinic can be very proud of."

For John and Tom, Mayo Clinic means even more.

"Not only did Dr. Markovic and the team at Mayo Clinic save my mom, but they allowed her to get to know her grandkids," Tom says. "That's something you can never put a price on."

This story originally appeared in Mayo Clinic Magazine.

The post Dawn’s Journey: Overcoming a near-zero survival rate appeared first on Mayo Clinic News Network.

The Reibels have relied on Mayo Clinic for their care for 25 years. That experience has inspired them to donate to Mayo Clinic's Center for Regenerative Medicine.

Even in retirement, Dr. and Mrs. Reibel — or Jay and Barbara as their friends at Mayo Clinic have come to know them — are always on the go. They travel the world and have been actively engaged in a host of civic and community organizations.

But chronic pain in both knees threatened Jay's ability to continue his active lifestyle. Over time, it became almost debilitating. He had trouble climbing stairs or even sitting for long periods. He couldn't make it through an opera — a favorite activity — without rubbing his knees in pain. It was a constant distraction and presented a serious challenge.

Doctors in New York City, close to the Reibels' home in Greenwich, Connecticut, were unable to find an answer or relieve the pain. Jay knew he needed to go to Mayo Clinic.

Transformative relief

The Reibels have a 25-year patient history with Mayo Clinic that began with Robert Frye, M.D., a Mayo Clinic cardiologist who led what was then the Division of Cardiovascular Diseases from 1974 to 1984, as well as the Department of Medicine from 1987 to 1999. Their Mayo experiences have been overwhelmingly positive — so much so that they eagerly anticipate their appointments even in challenging circumstances.

"We love Mayo Clinic because from the very beginning of our experience, it's been so welcoming," Barbara says. "We actually look forward to our visits. People usually don't look forward to going to hospitals or clinics."

Jay had heard about regenerative medicine techniques, including the use of a patient's own stem cells, to restore knee function and alleviate pain. Shane Shapiro, M.D., medical director of the Regenerative Medicine Therapeutics Suites at Mayo Clinic in Florida, was the first researcher in the emerging field to lead a study on the safety and efficacy of the technique. Jay inquired about the procedure and was referred for treatment.

"We love Mayo Clinic because from the very beginning of our experience, it's been so welcoming."

Barbara Reibel

The results were almost beyond Jay's belief. "It was — and I don't misuse the word or use it hyperbolically — transformative," Jay says. "Within a week, I had significant pain relief. And it continues now."

The effect of the procedure was immediately apparent to Jay's family and close friends. "It would be really difficult watching him while he was in pain," Jay's daughter, Kate, says. "Now he's able to do the things he loves to do again."

A unique perspective

As a result of their numerous outstanding interactions with Mayo, the couple decided to express their gratitude by becoming benefactors. Supporting Mayo Clinic's Center for Regenerative Medicine seemed like a natural fit. Before the Reibels committed to their philanthropic gift, however, they wanted to learn more.

Jay has a unique perspective based on his personal experience in medicine and business. Trained as a psychiatrist, Jay, early in his medical career, held several prominent positions on national and medical governing and advisory bodies, including the board of directors of Blue Cross-Blue Shield of Greater New York, the New York County Medical Society, and the American Psychiatric Association, as well as a presidential appointment to the advisory body for Medicare and Medicaid. Jay has applied his talent and experience to establish a system of high-quality private psychiatric hospitals. He also founded a publicly traded company that was the first managed care organization. His company grew to serve millions of people across the country.

Barbara also brought her unique perspective to studying the opportunity. During her career, she worked as a public health adviser for the former Department of Health, Education, and Welfare.

"The focus on getting the discoveries from the laboratory to application to the patient is wonderful. I'm an example of that."

Jay Reibel, M.D.

At Jay's request, the Reibels spent two days at Mayo Clinic in Florida studying the Center for Regenerative Medicine from a clinical, organizational and business standpoint. They met with the clinic's leaders, including Gianrico Farrugia, M.D., Mayo Clinic's president and CEO — who was CEO of Mayo Clinic in Florida at the time — and Andre Terzic, M.D., Ph.D., the Michael S. and Mary Sue Shannon Family Director for the Center for Regenerative Medicine.

The Reibels spent time with researchers, visited laboratories and learned about the science behind new and emerging techniques that harness the body's natural ability to heal. It was an introduction to what Mayo Clinic calls translational medicine — developments in the laboratory that are quickly translated to patient care improvements.

Something magical

"We were beyond impressed," Jay says. "The research that we saw being done was explained to us in a clear manner that we could understand. The teams of researchers with whom we met all evidenced their commitment to the ethos of Mayo Clinic and its profound commitment to excellence."

Perhaps most important of all, the Reibels saw the immense impact regenerative medicine will have in the future, spanning medical specialties to improve the lives of patients, as well as reducing the economic burden that many patients and society as a whole faces today.

"I appreciate the importance of what is happening in regenerative medicine, not only to the lives of people, but to the cost savings to the economy of the country," Jay says. "The focus on getting the discoveries from the laboratory to application to the patient is wonderful. I'm an example of that."

Through their experience becoming Mayo Clinic benefactors, the Reibels have forged new friendships with leaders and researchers they have met. "We value the personal relationships that we've developed," Jay says. "They're stimulating, meaningful and very enjoyable."

These relationships add a unique dimension to the Reibels' experiences at Mayo Clinic, and they reflect Mayo's commitment to values such as integrity, compassion and healing. It is apparent in every interaction, and it is what sets Mayo Clinic apart.

"There's something magical about Mayo Clinic," Barbara says. "It's the staff, whether it be the top physicians, the nurses and physical therapists, or the clerks at the desk. Everyone we've encountered in all of our experiences has been so sweet and lovely and helpful. We can't tell people enough how different Mayo Clinic is from any other medical institution with which we are familiar."

Note: A version of this story previously was published in Mayo Clinic Magazine.

HELPFUL LINKS

• Read more about regenerative medicine.
• Check out Mayo Clinic's Center for Regenerative Medicine.
• Learn about giving to Mayo Clinic.
• Explore Mayo Clinic.
• Request an appointment.

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Diagnosed with non-Hodgkin's lymphoma in her 40s, Tanis Milicevic tried a number of treatments to keep the disease in check. But the cancer came back. Then her Mayo Clinic care team offered Tanis a new option, and it made a world of difference.

At first, Tanis Milicevic's local doctor told her to wait and see. Pregnant with her second child, Tanis had been sensitive about changes in her body when she felt a worrisome bump on the back of her head.

The doctor's advice gave her temporary peace. The bump was small, and she could just monitor it. Then, after her baby's first birthday, Tanis felt a lump in her underarm. "That's when I knew that something was not right," she says.

Although she felt fine otherwise, her instincts about her health were correct. A biopsy showed she had non-Hodgkin's lymphoma. Tanis and her husband, Marko, learned that while her disease was slow growing at this stage, it also would be hard to cure.

"It was a difficult pill to swallow, but we remained optimistic. You live life and push forward," Marko says. They had to for their sons, 1-year-old Jonathon and 8-year-old Max.

Aggressive disease

Tanis, then 44, underwent radiation therapy  and then chemotherapy for two years. The treatments kept the cancer in remission for five years. That freed her to focus on her family and her business, a women's contemporary fashion boutique.

Then the cancer returned. That's when Tanis decided to go to Mayo Clinic. Under the care of Han Tun, M.D., a hematologist at Mayo Clinic in Florida, Tanis tried two new chemotherapy treatments to manage the recurrence, as well as a second relapse 18 months later. Her cancer was transforming and becoming more aggressive.

At the end of 2017, Tanis reviewed her options with Dr. Tun. It was just a matter of time before the next recurrence. The therapy for this type of cancer usually is a stem cell transplant, but Tanis had no suitable matching donor.

Yet there was still hope. A new treatment has just been approved by the Food and Drug Administration (FDA) for Tanis' type of lymphoma. Known as CAR-T cell therapy, it had been shown to improve remission and survival rates after other treatments had failed. Mayo Clinic had taken part in pivotal clinical trials that led to FDA approval of CAR-T cell therapy, and it was one of the first medical centers in the nation offering the treatment.

In 2018, Tanis became the first patient at Mayo Clinic in Florida to receive CAR-T cell therapy. "I don't think anything could come at a more perfect time for me," Tanis says.

Revolutionary treatment

CAR-T cell therapy, or chimeric antigen receptor-T cell therapy, is treatment that uses a patient's own cells to fight cancer. T cells are a type of white blood cell in the immune system that protect people from infection. In CAR-T cell therapy, T cells are collected from a patient's blood, genetically altered in a lab, multiplied and returned to the patient's bloodstream. The alteration gives T cells the chimeric antigen receptor, which helps T cells recognize, latch onto and kill the intended cancer cells.

"I think that in the past 10 years or so, probably this is the most innovative and revolutionary treatment that our field has seen," says hematologist and oncologist Mohamed Kharfan Dabaja, M.D., director of the Blood and Marrow Transplantation Program and the Human Cellular Therapy Laboratory at Mayo Clinic in Florida.

"(CAR-T cell therapy) has improved the complete remission rate by sevenfold."

Mohamed Kharfan Dabaja, M.D.

What has been exciting to cancer physicians and researchers is how CAR-T cell therapy is helping some patients achieve complete remission, even after other forms of treatments have failed. In complete remission, there are no symptoms or signs of cancer, and the disease is no longer detectable, although it could return later.

"It has improved the complete remission rate by sevenfold," Dr. Kharfan Dabaja says.

When patients achieve complete remission, they have a better chance of surviving. Survival rates two years after treatment are shown to have improved — from as low as 15% to over 50% with CAR-T cell therapy.

CAR-T cell therapy is fast-acting, showing evidence of response within one to three months. It stands out among similar cancer therapies in terms of how it overcomes obstacles — such as vast numbers of cancer cells and suppression of the immune system by cancer — quickly and on its own.

Expanding options

The FDA has approved two CAR-T cell therapies. Presently, both can treat adults with certain types of non-Hodgkin's lymphoma, but only one treats children and young adults, up to age 25, with B-cell acute lymphocytic leukemia. The therapies are used after at least two previous treatments have failed. They are not yet approved for use in earlier stages of the cancers.

Mayo Clinic is one of the few medical centers certified to offer the FDA-approved therapies. Mayo is also one of the few centers to have experience treating patients with the FDA-approved therapies in clinical trials. The treatments are available at Mayo Clinic in Arizona, Florida and Minnesota.

CAR-T cell therapy requires the specialty care that Mayo Clinic provides, especially once the CAR-T cells are infused back into a patient's body, where they release chemicals as they kill cancer cells. The resulting symptoms can be severe.

"These are living drugs," says Yi Lin, M.D., Ph.D., who leads the Cellular Therapeutics Cross-Disciplinary Group at Mayo Clinic Cancer Center. "Patients oftentimes need hospital, even ICU-level, support. They need to be at a very experienced center as they go through the treatment."

Mayo Clinic continues to drive forward new research in CAR-T cell therapy. Mayo is expanding clinical trials with a focus on using the treatment at earlier stages of cancer and in more types of cancer; countering the side effects and safely delivering treatment; and even using CAR-T cell therapy for conditions beyond cancer. Mayo Clinic in Arizona, Florida and Minnesota all will have trials open for various types of blood cancer this year.

Complete remission

Tanis met with Dr. Kharfan Dabaja in 2018, and in late spring, she began the CAR-T cell treatment. Unfortunately, Tanis experienced severe side effects and was hospitalized longer than she expected. That included spending time in the ICU. While it was difficult for Tanis, Marko and their family, the outcome was the result they had hoped for. Six weeks after treatment, Tanis was in complete remission.

"It's given me the chance to live, and I'm forever grateful."

Tanis Milicevic

"This is a patient that had not seen a complete response for many, many years," Dr. Kharfan Dabaja says. "Now she is experiencing a complete response, and with time, her condition has improved significantly, where she's almost back to her normal life."

Tanis reached an important landmark when she was still in complete remission 90 days after her treatment. More than half the patients who reach the 90-day mark will be alive two years later, Dr. Kharfan Dabaja says.

Now, for the first time in a decade, Tanis and her family are thinking of a future without cancer. "Tanis has gone through all the different treatments. This one definitely feels the most amazing," Marko says. "It has allowed me to have my wife and our kids to have their mom."

"It's given me the chance to live, and I'm forever grateful," Tanis says, "My advice for anybody else who has lymphoma is to know that there is hope."

Note: A version of this story previously was published in Mayo Clinic Magazine.

HELPFUL LINKS

• Learn about non-Hodgkin's lymphoma.
• Read more about CAR-T cell therapy.
• Connect with others talking about cancer on Mayo Clinic Connect.
• Explore Mayo Clinic Cancer Center.
• Request an appointment.

The post CAR-T Versus Cancer appeared first on Mayo Clinic News Network.

Mayo Clinic's 14-year partnership with Diné College has opened up opportunities for students like Corinna Sabaque to gain experience in fields where Native communities are often underrepresented and underserved. In Sabaque's case, those opportunities launched a career in cancer research.

In the summer of 2014, Corinna Sabaque moved more than 1,000 miles from her home in the Four Corners area of New Mexico to Rochester, Minnesota, to complete a 10-week internship in breast cancer research at Mayo Clinic.

"Coming to Minnesota was hard," Sabaque says. "I didn't know anybody. I didn't even know whether I wanted to do cancer research." Despite those difficulties, Sabaque came back that fall for another internship. This time she spent seven months studying pancreatic cancer alongside Gloria Petersen, Ph.D., an epidemiologist and deputy director for Population Sciences at Mayo Clinic Cancer Center.

A student from Diné College, a tribal college serving the Navajo Nation, Sabaque was pursuing a four-year degree in community health. Now she works at Mayo Clinic in Rochester as a health sciences researcher, dividing her time between pancreatic cancer research and health issues among Native populations.

Mayo Clinic's 14-year partnership with Diné College has opened up opportunities for students like Sabaque to gain experience in fields where Native communities often are underrepresented and underserved. In Sabaque's case, those opportunities launched a career in cancer research.

"Mayo opened a lot of doors for me to learn about cancer research, and that experience developed my interest," Sabaque says.

Seizing opportunities

A $75,000 grant supplement from the National Cancer Institute is helping Mayo Clinic and Diné College offer more Native American students like Sabaque opportunities to learn about cancer research. In April 2018, a group of Mayo representatives and educators, and leaders from five tribal colleges met in Shiprock, New Mexico. For two days, they shared ideas and discussed plans for a cancer research curriculum as part of a new four-year program in public health at Diné College to promote diversity in cancer research.

The meeting explored cultural perspectives and priorities in cancer research. "That meeting was critical," Dr. Gloria Petersen says. "It provided the time and space to develop culturally sensitive approaches to attract and interest a new generation of learners."

"(Mayo Clinic) Cancer Center really has something to offer with our longstanding relationship with Native tribes and our combination of resources, experience, scientists and investigators who can inspire young people and bring them along in a career in cancer research."

Gloria Petersen, Ph.D.

Funding for the two-day workshop came from a supplement to Mayo Clinic Cancer Center's National Cancer Institute's Cancer Center Support Grant. These supplements were offered to cancer centers that would promote cancer opportunities to underserved minorities.

"This was an opportunity we were not going to miss," Dr. Gloria Petersen says of the supplement. "The Cancer Center really has something to offer with our longstanding relationship with Native tribes and our combination of resources, experience, scientists and investigators who can inspire young people and bring them along in a career in cancer research."

Increasing involvement

With the support of researchers at Mayo Clinic, Diné College is preparing an application for a second grant. The grant would support the implementation of a Cancer 101 curriculum that was developed as a result of the meeting at Shiprock. Diné's curriculum will reach more than 50 students enrolled in advanced biology and public health courses annually.

The current vision of the curriculum also includes five 10-week cancer research internships at Mayo Clinic for Diné students. These are modeled after the opportunities Sabaque had as a student.

"Giving Native students the opportunity to come to Mayo Clinic would open doors for hands-on learning experiences in laboratory and clinical projects that they wouldn't normally have," Sabaque says. She credits time spent in cancer research labs at Mayo for piquing her interest in the field.

"This is really one step in a larger effort to help more Native students get involved in doing this kind of research," says Christi Patten, Ph.D., a professor of psychology in the Behavioral Health Research Program. Dr. Patten was principal investigator on the first grant Mayo and Diné College partnered on 14 years ago.

"These students would be doing research that comes from their community and is sensitive to their specific needs in the health care system."

Wesley Petersen, Ph.D.

Only about 4% of Native American and Alaskan Native populations have the advanced degrees necessary to be principal investigators in clinical research. Raising that number could reduce health disparities and bring valuable insights to cancer research.

"These students would be doing research that comes from their community and is sensitive to their specific needs in the health care system," says Wesley Petersen, Ph.D., director of Native American Research Outreach at Mayo Clinic. "By addressing research questions that are important to those patients, we're making steps toward improving their health."

Dr. Patten adds, "If we don't have Native voices at the table, we're missing a whole dimension of cancer research."

Note: A version of this story previously was published on the Advancing the Science blog.

HELPFUL LINKS

• Learn more about research at Mayo Clinic.
• Check out Mayo Clinic Cancer Center.
• Explore Mayo Clinic.
• Request an appointment.

The post Opening Doors for Cancer Research in Native American Communities appeared first on Mayo Clinic News Network.

When Stephanie Van Doren came to Mayo Clinic, she never expected to learn she had a genetic defect that raised her risk for a potentially fatal heart condition. But thanks to her physician's all-inclusive approach, Stephanie and her family now have information they need to live life to the fullest.

Abdominal issues and difficulty swallowing sent Stephanie Van Doren to a local hospital near her home in Debary, Florida. Unable to eat or drink, the 40-year-old mother of three had lost significant weight. At the hospital, she was diagnosed with gastritis — inflammation of the stomach lining — and sent home.

But when symptoms continued, including chest pain, Stephanie knew she needed another opinion. A friend recommended Mayo Clinic.

In early 2017, Stephanie traveled to Mayo Clinic's Florida campus and met with Timothy Woodward, M.D., a gastroenterologist. He diagnosed her with eosinophilic esophagitis — a disorder that causes buildup of white cells in the lining of the esophagus and is a major cause of digestive system illness.

"I liked him immediately because he took time to talk to me and listen to my answers," recalls Stephanie of her first encounter with Dr. Woodward.

Together, the two developed a care plan and, over time, Stephanie's condition improved. On a subsequent visit, though, Dr. Woodward recognized Stephanie might have another issue.

"It's important for me, when I'm taking care of someone, to look at them each time they come in and go over any concerns," Dr. Woodward says.

Seeing the whole person

Over the years, numerous people in Stephanie's extended family had died as a result of aortic dissections. An aortic dissection is a serious condition in which the inner layer of the large blood vessel that branches off from the heart, the aorta, tears. The condition is often fatal.

"Although we had settled her esophagus issues, I was concerned when we were talking about her family members and how many had passed suddenly from these heart issues," Dr. Woodward says.

"Most doctors talk to you about their one area. Dr. Woodward talked to me about me. He talked to me about my family, my life."

Stephanie Van Doren

In his physical exam, Dr. Woodward recognized Stephanie's long, thin limbs and hyperflexible joints as traits that can be associated with several genetic disorders. He knew if such a disorder was identified promptly, Stephanie may be a candidate for therapy to reduce her risk for future problems. He referred Stephanie to the Department of Clinical Genomics for evaluation.

"Most doctors talk to you about their one area. Dr. Woodward talked to me about me. He talked to me about my family, my life," Stephanie says. "He really cares about his patients."

Equipped to make informed decisions

Stephanie's evaluation in Clinical Genomics uncovered a significant issue. She learned she had a mutation in the ACTA2 gene that has been shown to cause thoracic aortic aneurysms and dissections. It is estimated that approximately 20,000 people in the U.S. die annually from these conditions.

"To some degree, I knew something was amiss, but never expected this," Stephanie says.

She was unnerved by the news. "It was definitely scary knowing what could happen to me," Stephanie says. "I felt like I had a ticking time bomb."

But once she met with Mayo Clinic experts in Cardiovascular Medicine to learn more about the condition and how to reduce her risk, Stephanie became more confident.

"I still have to live my life, but I know what I can and can't do," she says. "And I know my providers are watching me closely."

"One of the pleasures of being here at Mayo Clinic is having time to be proactive and pursue ideas that may positively impact our patients."

Timothy Woodward, M.D.

Based on the knowledge Stephanie gained, her family decided that her children also would be tested for the genetic variable. The oldest is not affected, but Stephanie's younger son and daughter tested positive. And though Stephanie admits she struggles some days knowing her children have more to worry about as they grow, she's glad her family has the information.

"They will become more educated as they age and be able to make proactive decisions to live their lives to the fullest," she says.

Thanks to the collaborative nature of Mayo Clinic, Dr. Woodward remains involved in Stephanie's ongoing care — even sharing updates on potential medication that could slow or prevent the progression of her disease.

"One of the pleasures of being here at Mayo Clinic is having time to be proactive and pursue ideas that may positively impact our patients," he says.

"I'm so thankful to Dr. Woodward and the team at Mayo Clinic," Stephanie says. "I feel good. I live every day, and I have hope for the future."

HELPFUL LINKS

• Learn more about genetic testing.
• Read about Mayo Clinic research into cardiovascular diseases.
• Explore Clinical Genomics and Gastroenterology and Hepatology at Mayo Clinic.
• Connect with others talking about heart conditions on Mayo Clinic Connect.
• Visit Mayo Clinic's Florida campus.
• Request an appointment.

The post Comprehensive Care Makes for a Brighter Future appeared first on Mayo Clinic News Network.

Just as things were settling down in the aftermath of a polar vortex in the Midwest, a new issue presented itself at Mayo Clinic's Rochester campus. And staff stepped up to meet the challenge.

When the sun rose on Thursday, Jan. 31, staff at Mayo Clinic's Rochester campus found themselves on the tail end of an arctic outbreak of historic proportions. The mercury had dipped to 30 degrees below zero Fahrenheit in Rochester, Minnesota, that morning, but an end to the bitter cold was in sight. The weather forecast predicted a significant warmup later in the day. A sigh of relief and a 70-degree temperature swing were on the horizon.

Members of the Healthcare Incident Command System — a multidisciplinary team that's mobilized when routine operations at Mayo Clinic are interrupted — thought everything would be back to normal by the afternoon.

Then an alarm sounded on the twelfth floor of the Guggenheim Building. The incident command group had another challenge coming its way.

No air exchange, no climate control

Guggenheim 12 houses mechanical systems for the building that is home to an array of laboratories. Hundreds of Mayo Clinic research staff work in Guggenheim, studying everything from mechanisms of stem cell aging to viral and gene therapies. All three of the building's air handling units had frozen in the extreme cold. That meant a controlled air supply and climate control no longer were possible on floors 13 through 20.

Joseph Schneider, unit head for mechanical systems in Facilities Operations, and Cameron Wood, heating, ventilation and air conditioning (HVAC) shop supervisor, went to assess the situation. What they found wasn't good.

The water coils in the air handling units had frozen. They had to be thawed out, and then checked for leaks and repaired. It wasn't going to be a quick fix. And because all three units were out of commission at the same time, the usual option of using a backup unit was not available.

That situation prompted a second activation of the Healthcare Incident Command System — this time with significant input from Hilary Blair, Research Operations administrator and Nicole Thome, biosafety coordinator.

They had to figure out the answers to two important questions: Are staff on the affected floors safe? Is any research in danger? Most of the affected floors contain eight to 10 laboratories. In all, more than 300 staff member were thrust into uncertainty that morning. "I needed to get over there to understand the impact," Blair says.

"Research typically isn't pulled into [the Healthcare Incident Command System], and we really appreciated the support structure they provided."

Nicole Thome

Together with Thome and colleagues from Facilities Operations and Environmental Services, Blair made the first of many rounds at Guggenheim, carefully assessing the situation on each of the upper floors.

"My concern was safety," Thome said. "If we have an HVAC impediment, is that going to change how we're working safely in the building?"

Even though chemical hoods and ducted biosafety cabinets were technically working, it was decided to post signs asking staff not to use them. "The hoods would not have been functioning in a way that would safely ventilate chemical and biological substances," Blair explains.

There also was concern about air pressure. Due to the unregulated airflow, hallway doors became hard to open or would slam shut, and elevator doors wouldn't completely close.

Freezers in jeopardy

Once the team established that staff members were not in any danger — though much of their work was halted — attention shifted to another concern. By noon, the focus was on the more than 100 freezers that contained samples and other substances for use in research experiments.

"The value of those assets can't be overstated," Blair says.

While the freezers kept running, the temperature in the rooms where the freezers were housed rose to over 100 degrees, due to the problems with the heating system. That put an enormous strain on the freezers' compressors, as they worked to keep their contents at 80 degrees below zero Fahrenheit.

"If the freezers fail, and we have to clean them up and move the contents out of the building, we'd have to follow Department of Transportation regulations, which would present a huge logistical challenge," Thome says.

Blair and Thome put their confidence in their colleagues from Facilities Operations, who, together with a team of local plumbers and sheet metal workers, worked feverishly to get the first air handling unit back up and running.

They succeeded. The first air handling unit came back online that evening. That seemed to stabilize the temperature in the freezer rooms. But it turned out that one revived air handling unit was not enough.

More work to be done

"My heart sunk when the freezer rooms were still over 80 degrees," Blair says. "We needed to make sure these freezers weren't going to fail."

Plumbers immediately began work on the second air handling unit. They worked through the night in an attempt to make the necessary repairs. But by Friday morning, Schneider had bad news.

"Unit two had so many breaks in the water coils, they were not repairable," he says. "At that time, we decided to run the unit without the coils and try to repair the third air handling unit."

After another morning of intense repair work, the picture looked much more hopeful. By noon, the second air handling unit was running again, albeit with reduced capacity. Nevertheless, the building was fully operational, and staff could resume their normal work.

Plumbers continued to work throughout Friday and much of Saturday to repair as many of the coils as they could on the third unit.

Teamwork shines through

"I really have to commend the entire team," Schneider says. "They had to deal with an enormous amount of breaks that had to be repaired."

Blair also has high praise for Schneider's team and the contractors who worked to get the upper floors back online. "They were very responsive and very communicative," she says. "We would not have gotten through it without them."

Blair adds that many research teams pitched in to monitor temperatures. One supervisor even volunteered to spend the night in Rochester, in case things went awry.

"The collaborative spirit from all involved, coupled with formal incident response processes, provided a foundation for effective coordination and communication, and successful recovery."

Byron Callies, Jr.

Thome also praises Blair for her efforts as a "boots on the ground administrator" and points to members of the staff who were willing to walk the floors to protect colleagues, equipment and assets. And she's grateful for the support her team received from the Healthcare Incident Command System.

"I was impressed," Thome says. "Research typically isn't pulled into that group, and we really appreciated the support structure they provided."

That's great feedback for Byron Callies, Jr., from Mayo's Clinical and Business Continuity/Emergency Management, who served as incident commander when the Healthcare Incident Command System was activated.

"A less-coordinated effort could have resulted in significant disruption or even loss of critical research," Callies says. "The collaborative spirit from all involved, coupled with formal incident response processes, provided a foundation for effective coordination and communication, and successful recovery."

Thome adds that she was impressed with the process from beginning to end. "We really didn't lose more than one workday, and credit goes to the massive team involved."

HELPFUL LINKS

• Check out Mayo Clinic's research laboratories.
• Learn more about research at Mayo Clinic.
• Explore Mayo Clinic's Rochester campus.
• Request an appointment.

The post Quick, Coordinated Action Solves a Polar Problem appeared first on Mayo Clinic News Network.

Mayo Clinic researcher John Fryer, Ph.D., is hopeful the work in his laboratory will yield insights into Alzheimer's disease, as well as sepsis — an often-deadly condition which claimed his father's life in 2016.

When he received a text message from his mother in Arizona telling him that his father had been diagnosed with sepsis, John Fryer, Ph.D., knew he had to move quickly.

Sepsis, a runaway inflammation response to infection, can be deadly. It strikes more than 1 million Americans every year, killing between 28 and 50 percent of those affected, according to the National Institutes of Health.

Dr. Fryer received the message about his father in October 2016. It came less than 48 hours after Dr. Fryer's laboratory at Mayo Clinic learned that its greatest discovery — about sepsis, no less — would be published in Molecular Psychiatry, one of the world's leading research journals. The afterglow from that achievement would disappear quickly.

"As soon as I saw the message, I called my wife, and within two hours, I was on a flight to Arizona," Dr. Fryer says.

By the time Dr. Fryer arrived in Arizona, the inflammatory response was unstoppable. His father, Ron Fryer, who was also battling end-stage renal disease, was receiving palliative care to make him as comfortable as possible. Over the next few hours, with Dr. Fryer in the hospital room, Ron Fryer slipped into unconsciousness. He died less than 48 hours later.

"I couldn't stop being a scientist and wishing I had been further along in my research because, maybe, it could have helped my father," Dr. Fryer says. "At the same time, I was glad I knew about sepsis and recognized that I had to leave immediately when I received my mother's message. That was very fortunate, because I got to see my dad and have a conversation with him before he lost consciousness."

An intriguing link

Dr. Fryer's ability to focus on the positive within the whirlwind of events that surrounded his father's death reflects a resiliency that has served him well.

"You have to be malleable — willing to adjust — if you want to have a successful research career," he says.

Dr. Fryer is a neuroscientist at Mayo Clinic's Florida campus whose primary focus is Alzheimer's disease. He began studying sepsis because people who survive sepsis often have a period of delirium, and they have significantly greater risk of cognitive impairment and dementia.

Dr. Fryer and his team had their resolve tested before their sepsis discovery. At an early point in the research, they reluctantly decided to put the project on hold due to lack of funding.

"It's the most important discovery I've made since I started my lab in 2011, and it wouldn't have happened without the Gilmers." — John Fryer, Ph.D.

"I told the team we had to put the project on pause. I didn't want to say 'stop,' so I just said 'pause,'" says Dr. Fryer, who is also assistant dean of Mayo Clinic Graduate School of Biomedical Sciences. "Everyone was surprised, and their shoulders just slumped. It was still bothering me when I got home."

A few days later, everything changed when Dr. Fryer's lab received a gift from Gary and Marilyn Gilmer, who are members of Mayo Clinic's Florida Leadership Council and recognized as major benefactors.

A crucial discovery

With the Gilmers' funding, Dr. Fryer's team renewed its efforts and discovered that a protein called lipocalin-2, or LCN2, may serve as a doorway for creating the first strategies to protect people from the cognitive effects of sepsis. The protein also may be useful for diagnosing sepsis earlier, which is essential to reduce the number of lives it claims.

"It's the most important discovery I've made since I started my lab in 2011, and it wouldn't have happened without the Gilmers," Dr. Fryer says. "We think we may be able to target LCN2, either directly or indirectly, to reduce inflammation during sepsis and protect the brain. We also think measuring LCN2 levels may be helpful for determining if a patient is developing sepsis."

For the Gilmers, the discovery had a double impact.

"We seized the opportunity to support Dr. Fryer's research because our family has firsthand experience with the devastating effects of dementia," Gary Gilmer says. "Little did we realize that he and his team would so quickly make a discovery that has potential for improving the treatment of two terrible conditions."

A connection to patient care

The next steps for Dr. Fryer and his team are to screen drugs that may work with LCN2 to protect the brain from sepsis and similar syndromes. They are already screening thousands of drugs that have approval from the Food and Drug Administration and are part of the National Institutes of Health's Clinical Collection. The team is also focused on better understanding the mechanisms that may connect sepsis with cognitive impairment and dementia.

At the same time, Dr. Fryer's team is working with clinical colleagues to develop other strategies to improve sepsis care. Mayo Clinic has a sepsis response team that defines best practices for preventing and treating sepsis in hospitalized patients. Dr. Fryer's laboratory is working with them to create new ways to identify sepsis earlier.

"The good news is that Mayo Clinic is the best place to take all of these next steps." — John Fryer, Ph.D.

For example, he thinks it may be possible to use genomic technologies to identify pathogens earlier and start a patient on antibiotics sooner. Dr. Fryer developed additional expertise in genomics through a Gerstner Family Career Development Award. This grant, created through philanthropy from the Gerstner Family Foundation, is awarded through the Mayo Clinic Center for Individualized Medicine. It gives seed funding to young investigators who are pursuing genomic approaches to improve prediction, prevention and treatment of diseases.

"The good news is that Mayo Clinic is the best place to take all of these next steps," Dr. Fryer says. "Collaborating with physicians and pushing the boundaries of translational science is our greatest strength, and that's where this research is headed. In fact, we've already started."

HELPFUL LINKS

• Read more about Fryer's laboratory.
• Learn about the Center for Individualized Medicine.
• Explore research at Mayo Clinic.
• Request an appointment.

The post Resiliency and Determination Propel a Researcher’s Discoveries appeared first on Mayo Clinic News Network.