September marks the start of soybean harvest in Mankato, Minnesota, a busy time of year when farmers can't afford to be away from their fields for long. So, when Craig Smith, 66, began experiencing a burning sensation while urinating, he went to his family physician right away.

He was prescribed antibiotics, but his symptoms worsened. When Smith began to pass blood, his wife insisted that they drive straight to Mayo Clinic in Rochester — about an hour and a half away. There, Mayo Clinic physicians diagnosed him with metastatic urothelial cancer, or bladder cancer, which had spread to his spine.

Smith recalls receiving a phone call late that night from his Mayo Clinic doctor, who asked him if he wanted to just maintain his health for a few years or cure his cancer.

"I said, 'I'd like you to cure it,'" says Smith.

Seeking a bladder cancer cure

Smith's father had been a farmer, which was his dream too. But his father suggested he gain additional skills to supplement his farming income. Following his father's advice and encouragement from his high school welding teacher, Smith pursued his teaching certificate in welding. What he initially thought would be five or six years of teaching turned into a 45-year career developing welding programs at several local schools while also raising cattle and growing soybeans and corn on his 2,000-acre farm. Through his welding programs, he has trained several welders now employed by local manufacturing companies.

After his diagnosis in 2023, Smith took a hiatus from teaching and farming to focus on his cancer treatments at Mayo Clinic Health System in Mankato, which included chemotherapy, radiation and immunotherapy.

Metastatic urothelial cancer that has spread beyond the bladder usually is considered incurable and inoperable. However, Smith responded well to chemotherapy and radiation to his spine, which made his oncologist, Jacob Orme, M.D., Ph.D., and urologist, Paras Shah, M.D., consider Smith for a new, surgical approach to treatment.

Smith proceeded with the proposed surgery and had his bladder, prostate and 36 lymph nodes removed.

"In Mr. Smith's bladder, we found viable cancer cells that would have led to a relapse. Now, however, he is nearly two years from diagnosis and remains disease-free," says Dr. Shah.

Smith's positive response to treatment and surgery has spurred a clinical trial testing this aggressive approach in other bladder cancer patients. Currently, 17 participants are enrolled, and the results so far have been promising.

Advances in cancer treatment, such as immunotherapy that harnesses the body's immune system to fight cancer and the identification of biomarkers in the blood or urine that show how well a patient is responding to treatment, are helping the physicians select who will benefit most from surgery.

"The impetus for this study is to attack the cancer from multiple approaches, including treatments that cover head-to-toe and treatments that are directed right at the source tumor," says Stephen A. Boorjian, M.D., who is the David and Anne Luther Chair of Urology at Mayo Clinic and a lead proponent of the study.

"We want to remove the root of the cancer after we've burned off the leaves," adds Dr. Orme.

A team of researchers, physicians and clinical trials staff expedited the clinical trial through an accelerated pathway called a Rapid Activation Trial. It's part of a larger effort at Mayo Clinic to launch new clinical trials swiftly and effectively.

"Shortening activation timelines allows us to make a difference to more patients and their families," says Michelle Monosmith, Mayo Clinic Office of Clinical Trials operations administrator.

The study is supported by a generous donation by Ronald J. and Carol T. Beerman to Mayo Clinic and has been prioritized by Dr. Boorjian and Chair of Oncology Elisabeth Heath, M.D., to achieve more cures for men and women with bladder cancer.

"Our only goal is to help our patients live better and longer," says Dr. Orme.

That's what Smith plans to do as he continues to farm, teach and spend time with his family.

The post Farmer inspires new potential bladder cancer treatment appeared first on Mayo Clinic News Network.

For Stewart, the desire to become a scientist began at home. From a young age, she was aware of her father's diagnosis with cancer and the struggles he faced as his body became resistant to some treatments. Four years ago, when she became a predoctoral student at Mayo Clinic Graduate School of Biomedical Sciences, she chose a research project addressing blood cancers like his.

Working under the mentorship of Mayo Clinic hematologist oncologist Saad Kenderian, M.B., Ch.B., she has studied specially engineered immune cells, called chimeric antigen receptor-T cells or CAR-T cells, that are used to treat blood cancers like leukemia. She identified a signaling protein that, when neutralized, boosts the function of CAR-T cells by keeping them from reaching a state of "exhaustion." She and Dr. Kenderian's team published their findings in Nature Communications.

Stewart was delighted to learn that the journey of the new concept didn't stop there. "The approach she explored is novel, and her findings show there's a solid rationale to see whether it may help patients," says Dr. Kenderian.

'An incredible learning curve'

As the opportunity to advance the idea has emerged, Stewart has begun to learn about developing a clinical trial. "It's been an incredible learning curve," she says of taking a discovery beyond the lab space.

Close collaboration between researchers and clinicians at Mayo is what makes the steps of translation — from discovery to the clinic — happen efficiently, says Dr. Kenderian. Students like Stewart have opportunities to learn about the steps that shape clinical trials to bring new treatments to patients.

She and Dr. Kenderian met with a panel of Mayo physicians to get feedback about the design of a phase 1 clinical trial, which tests the safety of a new approach in a small cohort of patients who are interested in participating. "It was a great opportunity for me to hear how things are translated into the clinic, what questions and concerns clinicians may have, and how feasible our approach may be," she says.

"CAR-T cell therapy can be remarkable for some patients who have no other options, and the hope is to develop a more durable, lasting approach. This part of the research is still in early stages, but it's a very exciting project, and I feel lucky to be a part of it."

'Beyond what seemed possible'

Many Ph.D. students at Mayo Clinic are preparing for future careers in academia and industry. Another way they can learn about research that involves human participants is through an internship at Mayo Clinic's Office of Clinical Trials. Working with Mayo's clinical trials coordinators, students have followed a clinical trial underway and learned about designing a study that provides meaningful data and protects the rights, safety and welfare of patients who volunteer to participate.

But for some students, like Olivia Sirpilla, who is graduating in May from Mayo Clinic Graduate School of Biomedical Sciences, a long series of successful experiments in Dr. Kenderian's lab has opened the door to the next stages. Sirpilla's Ph.D. research looked at developing specially engineered stem cells from fat tissue as a treatment to calm the inflammation that occurs in immune diseases.

The team's results, published in Nature Biomedical Engineering, have set the stage for two potential clinical trials. One may be applicable to graft-versus-host-disease, an immune system complication that can occur after a stem cell transplant. The findings also may be applicable to the treatment of inflammatory bowel disease, particularly for patients who no longer respond to medications.

To design these clinical trials, Sirpilla and Dr. Kenderian are working with teams of Mayo clinicians who specialize in these disorders and their standard treatments. "I chose to come to Mayo Clinic for my Ph.D. to conduct lab research that would be highly translational," she says. "But to spend my thesis work developing a new cell therapy platform that is ready to be translated into a clinical trial is beyond what I thought possible in a Ph.D."

Research that reaches patients' daily lives  

Stephanie Zawada began her Ph.D. with an interest in computational approaches to research, but she has always strived to make advances that reach patients' daily lives.

Her thesis project aimed to help physicians assess people who have experienced a stroke and may be at risk for another.  She engaged two Ph.D. mentors who shared their expertise: Bart Demaerschalk, M.D.,  and  Bradley Erickson, M.D., Ph.D. Dr. Demaerschalk is a professor of neurology who treats patients with stroke at Mayo Clinic in Arizona and has a longstanding interest in digital health and using telemedicine to reach remote patients, and Dr. Erickson is a professor of radiology who leads artificial intelligence studies at his lab at Mayo Clinic in Minnesota.

Zawada "took a very innovative approach, exploring digital tools that may help a healthcare team continue to monitor and evaluate patients who have been discharged from the hospital but remain at risk for stroke recurrence," says Dr. Demaerschalk.

First, Zawada scoured available databases to identify attributes, including mood changes and sleep patterns, that can signal an oncoming stroke and can be captured by smartphone-based technology known as "wearables." Then she worked with her mentors and members of the Center for Digital Health to develop a pilot clinical trial with patients who volunteered to participate in a study from their homes.

Zawada designed a study that met the requirements of an institutional review board, or IRB, to address regulatory compliance. Aiming to get information from a "real-world" setting — where patients interact with app-based tools and the tools account for the range of daily activities — added logistical and mathematical complexity to the project. She worked with Dr. Demaerschalk and Mayo Clinic hospital-based stroke teams to recruit a cohort of 35 patients who gave consent to be part of the clinical trial.  

"Interacting with patients and hearing their concerns is the best way to advance useful technologies," Zawada says of designing the trial. "You want the clinical trial process to be as simple as possible for participants, and the only way to learn what tools and designs can make their lives better is to listen to them."

Her results showed that several behavioral and mood changes related to cerebrovascular disease can be captured with the use of wearable devices, even from patients going about their daily routines far from a hospital. The approach will need further investigation as a strategy to improve care and in the development of clinical trial measures, but the training to conduct a clinical trial is something that Zawada will take to the next stages of her career.

"Setting up a clinical trial is a complex process and really involves a team," says Zawada, who graduates in May and plans to continue researching wearables for patients who have other severe health conditions. "My thesis project was a multi-site collaborative effort — putting together the clinical questions, the new technology and the data analysis, the connection with patients — it's why I came to train at Mayo in the first place. As a student, I couldn't have done this project anywhere else."

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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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For Anthony Maita, 'Buddy' is not just any other dog.

"He's the best thing that's ever happened to me," says Anthony.

It's no wonder, considering Buddy was right by Anthony's side during one of the most challenging times of his life — when Anthony began having epileptic seizures.

Watch: When seizures don't stop: Anthony's battle against drug-resistant epilepsy

Journalists: Broadcast-quality video (2:38) is in the downloads at the end of this post. Please courtesy: "Mayo Clinic News Network." Read the script.

"I started having the seizures, noticeable seizures, and from there, it just started getting worse and worse," recalls Anthony.

It began after Anthony graduated from high school. He was making plans for his future and looking forward to attending college. That's when the seizures began.

Initially, the seizures were mild but quickly became more severe. "The experience (seizure) is like a loss of time, like a blank spot in your memory — like you're waking up without any recollection of what happened," says Anthony.

"The seizures were several times a week. His lips would be blue. His mouth would be blue," says Patricia Maita, Anthony's mother. "It so hard to see your child go through that and feel so helpless."

Doctors tried to manage Anthony's seizures with medication, but nothing worked. Eventually Anthony was diagnosed with drug-resistant epilepsy, or DRE.

In search of hope, Anthony's family turned to Mayo Clinic in Arizona.

"Up to a third of patients who develop epilepsy during their life will become resistant to medication," explains Jonathon J. Parker, M.D., Ph.D., a neurosurgeon at Mayo Clinic who specializes in treating the most serious and complex cases of epilepsy, including DRE.

"These patients have tried at least two medications, and they're still having seizures. At that point, we know the chances of seizure freedom unfortunately become very low, and that's when we start looking at other options," says Dr. Parker.

A battle for millions worldwide

Anthony is one of approximately 50 million people worldwide diagnosed with epilepsy. It is one of the most common neurological disorders globally. It is characterized by recurrent unprovoked seizures caused by abnormal electrical activity in the brain.

Of those diagnosed with epilepsy, approximately 30%, or 15 million people, are considered medication-resistant. Uncontrolled seizures often rob many people of their ability to live and function independently.

While it is rare, seizures can lead to sudden unexplained death in epilepsy, or SUDEP. "We know that more frequent seizures mean the patient is at higher risk of SUDEP, so that's why we are very aggressive about treating epilepsy with all the tools we have available," says Dr. Parker.

Current treatment options for patients with DRE include surgical procedures such as brain resection to remove a portion of the brain tissue responsible for generating seizures. A less invasive procedure involves laser ablation therapy that pinpoints and destroys abnormal brain tissue. While often effective, these surgical approaches carry the risk of possible side effects, such as memory impairment, motor deficits and speech difficulties. 

Neuromodulation is another surgical approach that uses electrical or magnetic stimulation to interrupt abnormal neural activity without removing brain tissue.

Unlocking new hope for patients

Now, a growing number of scientists across the globe are part of an innovative trend in research, investigating novel ways to treat DRE. It involves the use of regenerative medicine as a "reparative" approach to help the brain heal. 

Dr. Parker is the lead investigator of the first-in-human clinical trial at Mayo Clinic which studies the use of implanted specialized inhibitory brain cells as a potential reparative treatment for DRE. Dr. Parker's clinical trial is underway in Arizona.

Mayo Clinic in Arizona is one of 29 sites nationwide participating in the inhibitory brain cell implant clinical trial for patients with focal epilepsy, where seizures originate in a specific region of the brain. 

Anthony became Mayo Clinic's first patient to undergo the investigational brain cell implant. 

"We use a very minimally invasive technique where we inject the inhibitory cells through a pencil eraser-sized incision in the back of the head. Our hope is that, over time, these cells become part of the brain and help repair the neural circuitry, and reduce or prevent seizures without the side effects," says Dr. Parker. The cells are implanted in a one-time, single-dose procedure.

"Honestly, it was pretty easy," says Anthony. "I had no trouble with it." Anthony was discharged from the hospital the next day.

Doctors say it is still too early to determine whether the brain cell implant was effective, but they are hopeful.

"Anthony has been doing great since the procedure," says Dr. Amy Z. Crepeau, a neurologist at Mayo Clinic. "We have a great deal of optimism in regard to the potential of this brain cell therapy. Developing a safe and effective, minimally invasive treatment that does not carry the possible negative side effects could be a game changer in treating patients with DRE and improving their quality of life."

Tabitha's life-long struggle to control seizures

Tabitha Wilson lives in fear, never knowing when or where the next seizure will strike.

The Florida resident was diagnosed with epilepsy at the age of 2. She was placed on medication that adequately managed her seizures — until the week before her high school graduation. 

"I was 17 years old sitting in history class when the seizure happened," recalls Tabitha. "They had to load me up in an ambulance in front of the whole school."

Tabitha tried new types of medications, but the seizures only got worse.

"I fell down a flight of stairs, burned myself while cooking. I've completely blacked out and don't know where I am or who you are," says Tabitha. She was eventually diagnosed with drug-resistant epilepsy.

Tabitha underwent three brain surgeries to treat her DRE. Still, the seizures continued.

"I'll have good days and bad days. Some days, I'll have two, three, four seizures, back-to-back," says Tabitha.

Her uncontrolled seizures have robbed Tabitha of the ability to live independently. "I can't drive. I can't cook. I can't go swimming alone. I can't take a bath, only a shower and if someone is home with me," says Tabitha.

Watch: Tabitha Wilson shares what it's like to live with drug-resistant epilepsy.

Tabitha turned to Mayo Clinic in Florida where she learned about a clinical trial also investigating the potential of regenerative medicine as a possible treatment for DRE.

Dr. Sanjeet S. Grewal, director of stereotactic and functional neurosurgery at Mayo Clinic, is leading a team of researchers studying the use of implanted stem cells in conjunction with deep brain stimulation for patients like Tabitha.

Deep brain stimulation is one of the most recent FDA-approved methods of neuromodulation therapy for epilepsy. Studies show that patients who undergo deep brain stimulation experience median seizure reduction up to 70% after five years. However, Dr. Grewal says it is uncommon for patients to become seizure-free. 

"Unfortunately, neuromodulation doesn't give us the seizure freedom we want, and that's why we are trying to combine deep brain stimulation with stem cell therapy to see if we can increase the efficacy of neuromodulation," he says. 

Tabitha became the first patient to undergo the investigational treatment. Dr. Grewal says she is also the first person in the world to undergo surgery for deep brain stimulation and receive stem cell therapy in the thalamus in her brain as a potential treatment for DRE. 

Watch: Dr. Sanjeet Grewal, neurosurgeon, explains how Mayo researchers are leading a new trend in research for treating patients with drug-resistant epilepsy.

The clinical trial involves the use of mesenchymal stem cells, a type of adult stem cell that has anti-inflammatory properties. MSCs may also support tissue repair and healing. Further scientific research is needed to confirm their therapeutic potential in the field of regenerative medicine.

The MSCs used in the clinical trial are derived from fat tissue and created at the Human Cell Therapy Laboratory at Mayo Clinic in Jacksonville, Florida under the leadership of Abba Zubair, M.D., Ph.D., a pioneer in cell therapy.

Dr. Zubair's research teams have developed a cost-effective method of producing MSCs for use in potential treatments for conditions such as stroke.

Dr. Zubair has also led innovative research, including sending stem cells to the International Space Station to investigate how microgravity impacts their growth.

"My mission is to discover ways to address problems that patients have been struggling with and find a solution for them.
I believe the future is bright. "

Dr. Abba Zubair, Pioneer in Cell therapy, Mayo Clinic in Florida

Dr. Zubair has several research projects scheduled to launch into space in 2025.

"MSCs are what we call multipotent, meaning they can differentiate into different cell types based on where they're placed. If they are placed near blood vessels, they can become blood vessel types. If they're placed by heart cells, they can become heart cell types," explains Dr. Grewal.

The hope is the MSCs eventually become neural or brain cell types and interact in the part of the brain where the seizures occur. "It's called paracrine signaling, where they're releasing signals to the brain tissue around them and interacting in a way to try to repair that tissue."

Since undergoing the procedure, there has been an improvement in Tabitha's seizure management. However, Dr. Grewal says it is too early to know whether this is due to the deep brain stimulation, stem cells or both. 

Drs. Grewal and Parker say there is still a long road ahead to determine whether these cell therapies are proven safe and effective for patients with DRE. But they agree each day brings them one step closer to a potential treatment or cure for patients like Tabitha and Anthony.

"We've thought about this for generations, we just didn't have these technologies to enable it. Now we do," says Dr. Grewal. "So, whether it's wound healing, neurodegeneration, epilepsy or stroke, there are so many different studies going on investigating the potential of regenerative or reparative therapies."

Related articles

• Mayo Clinic researchers lead transformative shift toward neurorestorative treatment strategies for most severe forms of epilepsy
• Epilepsy Treatment
• Minimally invasive epilepsy treatment
• Mayo Clinic Minute: Demystifying epilepsy
• Epilepsy Surgery
• Laser ablation surgery helps treat young man’s epilepsy

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ROCHESTER, Minn. — Aortic valve stenosis (AVS) is a significant health concern affecting over 1.5 million Americans and millions more globally. Researchers at Mayo Clinic are exploring the use of a new drug called ataciguat to manage AVS. Results from preclinical and clinical studies, published in Circulation, show that ataciguat has the potential to significantly slow disease progression. The final step to establish the drug's long-term effectiveness and safety is a phase 3 trial, and efforts to launch that pivotal trial are soon to be underway with an industry partner.

In AVS, calcium deposits build up and narrow the aortic valve, forcing the heart to work harder to move blood. The condition typically progresses over time, with symptoms like chest pain, shortness of breath and fatigue affecting people over age 65. The current standard of care — watchful waiting — often leads to reduced quality of life before the condition is severe enough for the patient to have a surgical or interventional valve replacement.

"This research represents a significant advancement in the treatment of aortic valve stenosis," says Jordan Miller, Ph.D., director of the Cardiovascular Disease and Aging Laboratory at Mayo Clinic.  "Ataciguat has the potential to substantially delay or even prevent the need for valve replacement surgery, significantly improving the lives of millions."

Dr. Miller notes that the impact extends beyond simply delaying surgery. Younger patients with aggressive disease or congenital valve defects may develop symptoms in midlife. If a patient requires valve replacement before the age of 55, there is a more than 50% likelihood they will require multiple valve replacement surgeries over their lifetime due to recalcification of the implanted valve. Ataciguat, which slowed progression of native aortic valve calcification in the clinical trial, offers the potential for a once-in-a-lifetime procedure if they can reach the age of 65. The older a patient is, the less likely the implanted valve is to calcify.

Over the past decade, Mayo Clinic's research revealed that ataciguat reactivates a pathway crucial in preventing valvular calcification and stenosis. Preclinical studies in mice showed that this drug substantially slowed disease progression even when treatment began after the disease was established.  

Clinical trials in patients with moderate AVS demonstrated that once-daily ataciguat dosing was well tolerated, with minimal side effects compared to placebo. This latest phase 2 trial in 23 patients showed a 69.8% reduction in aortic valve calcification progression at six months compared to placebo, and patients receiving ataciguat tended to maintain better heart muscle function. Crucially, the research team confirmed that — despite its profound effect on slowing valve calcification — ataciguat did not negatively impact bone formation.

This important finding is the result of a collaborative effort between Mayo Clinic, the National Institutes of Health, the University of Minnesota, and Sanofi Pharmaceuticals. The research was conducted under an innovative academic-industry partnership grant administered by the National Center for Accelerating Translational Sciences and a Minnesota Biotechnology and Genomics Partnership grant.

Mayo Clinic and Dr. Miller have a financial interest in the intellectual property referenced in this news release. Mayo Clinic will use any revenue it receives to support its not-for-profit mission in patient care, education and research.  

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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:

• Terri Malloy, Mayo Clinic Communications, newsbureau@mayo.edu

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Billie, who has smoked most of his life, understands how using tobacco can negatively affect many aspects of a person's health. He is also a member of the Navajo Nation and has witnessed the challenges that family, friends and other members of his tribe have faced quitting tobacco. In recent years, he has watched the rising use of vaping among Navajo youth with concern. He wants to discourage young people from using tobacco except for ceremonial purposes.

"Science seeking to cure disease and transform the way we deliver healthcare requires a direct connection to the people we serve."

Annie Rusk, M.D.

Billie's experiences made him an ideal candidate to advise Dr. Rusk on her research through a Mayo Clinic program called Community Engagement (CE) Studios.

CE Studios is a consultation service that connects researchers to a group of "community experts" who represent the researcher's population of interest. These experts meet with the researcher, usually during a study's planning phase, to learn how the study is being designed, ask questions and provide feedback.

Since Mayo Clinic introduced CE Studios in 2021, Dr. Rusk has used it numerous times. She believes community input is essential for all research projects, regardless of their focus. 

"By involving people with lived experiences, we can gain valuable insights and ensure our research is relevant and impactful," says Dr. Rusk. "Even discovery science projects taking place in a lab can benefit from community perspectives."

Lived experiences provide key insights

For the CE Studios session with Dr. Rusk, Billie joined three other community experts from tribes in the Midwest, Alaska and Canada in a virtual meeting. The four community experts were there to advise Dr. Rusk on a new clinical trial.

Kory Billie, a member of the Navajo Nation from Phoenix, wants to discourage youth in his community from using tobacco, except for ceremonial purposes.

Dr. Rusk's research focuses on understanding barriers to smoking cessation among Native Americans. She felt it was important to consult with people from a variety of Indigenous communities to ensure her research aligned with their community health needs and priorities.

Billie says he enjoyed this opportunity to connect with like-minded people from similar backgrounds, and to speak with Dr. Rusk about how tobacco use was affecting communities like his. He shared his experiences with homelessness and addiction, as well as the difficulty of accessing smoking cessation resources in Indian Country.

What Dr. Rusk learned from Billie and the other community experts during the CE Studios session motivated her to make an important change to her study design.

The elements of the study that resonated best with the community experts were those geared toward developing practical behavioral health interventions. Billie and the other experts emphasized that understanding the social and cultural drivers of smoking would be key to achieving meaningful outcomes.  "You have to understand a person's environment and the way they're brought up," says Billie.

The community experts were less keen on a genetic analysis component Dr. Rusk had planned to include. They felt this aspect did not align meaningfully with community health priorities. Based on this feedback, Dr. Rusk eliminated this component from her study. 

While incorporating this kind of feedback can be challenging, Dr. Rusk says the long-term benefits are worth it for all.

"Science seeking to cure disease and transform the way we deliver healthcare requires a direct connection to the people we serve," she says.

Health is a blessing

Billie says he appreciated this chance to participate in CE Studios. He hopes that the knowledge he and the other experts shared will give Dr. Rusk the context she needs to develop a tobacco intervention that will have a lasting impact. Upon completion of the study, he looks forward to hearing from her to see the results.

Billie encourages other people to consider participating in CE Studios to help advance research to improve the health of their communities. He connects his motivation for this work to a Navajo proverb that reminds his people to live their lives with purpose: "Remember to walk in beauty. Beauty before you and beauty behind you."

If the community can improve the health of its people today, Billie says, they will pass that blessing on to future generations.

Get involved in CE Studios

A CE Studios community expert can be a person with any type of lived experience. Experts receive a small honorarium in exchange for their time. People interested in joining Mayo Clinic’s database of community experts should complete a Community Expert Enrollment Form.  

Mayo Clinic Community Engaged Research

Learn more about community outreach and engagement and community-engaged research at Mayo Clinic.

This work is supported by Mayo Clinic Center for Clinical and Translational Science and by Mayo Clinic Comprehensive Cancer Center.

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Most patients with Alzheimer's disease and Alzheimer's Disease Related Dementias (ADRD) experience the gradual onset and progression of cognitive symptoms, leading to decline over years or decades. However, in a small subset of patients, symptoms begin rapidly, leading to dementia within one year and complete incapacitation within two years of symptom onset. A new study at Mayo Clinic aims to determine why patients with Alzheimer’s disease and ADRD develop this rapidly progressive dementia (RPD).

"The factors that give rise to extreme, rapidly progressive clinical traits are unknown," says Gregg Day, M.D., a neurologist and clinical researcher at Mayo Clinic in Florida. "These cases are challenging to treat in practice because there are many possible causes and diseases to consider, many tests that can be done and a clear need to coordinate evaluations rapidly."

Dr. Day will lead a team of researchers from Mayo Clinic in Florida and Rochester, Minnesota, to study the biology of RPD through a project funded by the National Institute on Aging of the National Institutes of Health (NIA/NIH).

Specifically, the research team and collaborators aim to:

• Determine the factors that make patients with Alzheimer's disease and ADRD susceptible to RPD.
• Study the contributions of amyloid and tau toxic proteins and vascular changes in the brain to rates of progression in patients with Alzheimer's disease and ADRD.
• Identify cellular pathways that contribute to rapid declines in patients with Alzheimer's disease and ADRD.

The researchers plan to collect clinical and genomic information from 120 diverse patients with rapid progressive Alzheimer's disease and ADRD over the next three years. Findings in patients with RPD, identified through Alzheimer's Disease Research Centers studies nationally, will be compared with data from participants with typical progressive Alzheimer's disease and ADRD enrolled in studies at the Alzheimer's Disease Research Center at Mayo Clinic.  

The team hopes to learn how factors such as age, sex, medical history, structural and social determinants of health, genetic variants and other brain changes may make some patients more susceptible to rapid decline. Findings will be validated through expansive protein analyses in cerebrospinal fluid from an independent group of patients with autopsy-confirmed rapid progressive Alzheimer's disease and ADRD. Results will be extended to identify biomarkers and disease-modifying targets that may improve diagnosis and treatment of patients with Alzheimer's disease and ADRD.

"This project represents a substantial investment from NIH to study patients with RPD," says Dr. Day. "We hope the results of our research will inform new approaches, diagnostic tests and treatment targets that will improve outcomes in patients with AD/ADRD. The ultimate goal is to slow down the pathologic progression of disease in these patients, independent of their rate of decline."

The research will combine Mayo Clinic's expertise in digital innovation and telemedicine to engage patients across the United States. This study will also leverage Mayo's Clinical Trials Beyond Walls program, which allows patients to complete some, if not all assessments from the comfort of their own homes or local community facilities. The decentralized clinical trials initiative is designed to remove barriers to clinical trial participation by providing digital solutions and remote services to reimagine the trial experience for all involved, including participants, investigators, study teams and clinical care providers. Decentralized research ― studies conducted outside the walls of traditional research facilities ― may use a wide range of technologies and services such as telehealth, remote monitoring, mobile phlebotomy, retail pharmacy and home healthcare.

Other Mayo Clinic researchers working on this project include:

• Christian Lachner, M.D.
• Neill Graff-Radford, M.D.
• Leonard Petrucelli, M.D., Ph.D.
• Tania Gendron, Ph.D.
• Manoj Jain, M.D.
• Owen Ross, Ph.D.
• Nilufer Ertekin-Taner, M.D., Ph.D.
• Dennis Dickson, Ph.D.
• Melissa Murray, Ph.D.
• Vijay Ramanan, M.D., Ph.D.
• David T. Jones, M.D.
• Prashanthi Vemuri, Ph.D.
• Alicia Algeciras-Schimnich, Ph.D.
• John R. Mills, Ph.D.
• Rickey Carter, Ph.D.
• Lauren Haydu, Ph.D.

The research will be made possible through NIH grant award number R01 AG089380.

Related:

Researchers identify new criteria to detect rapidly progressive dementia

Researchers find other diseases may mimic rare brain disorder linked to dementia

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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

Journalists: Broadcast-quality video (2:45) is in the downloads at the end of this post. Please courtesy: "Mayo Clinic News Network." Read the script.

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.

Related articles

• Breakthrough in treatment approach showing promise in the fight against glioblastoma, the deadliest and most aggressive type of brain cancer
• (VIDEO) Focus on hope: Brescia’s Story
• All-Star pitcher Liam Hendriks shares how he closed out cancer at Mayo Clinic in Arizona
• Cancer is tough. Evelyn Owens is tougher
• 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/

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ROCHESTER, Minn. — A study at Mayo Clinic suggests that an hourglass-shaped stent could improve blood flow and ease severe and reoccurring chest pain in people with microvascular disease. Of 30 participants in a phase 2 clinical trial, 76% saw improvement in their day-to-day life. For example, some participants who reported not being able to walk around the block or up a flight of stairs without chest pain were able to do these ordinary physical activities at the end of a 120-day period. Clinical measures of blood flow related to the microvasculature of the heart significantly improved during follow-up, according to findings published in the Journal of the American College of Cardiology: Cardiovascular Interventions.

Microvascular disease is a condition in which tiny blood vessels in the heart are not working properly, resulting in reduced blood flow to the heart. The resulting chest pains, or angina, can be debilitating, limiting a person's ability to exercise, do household chores or even walk to the mailbox. About 40% of patients receiving a diagnostic coronary angiogram for chest pain do not have blocked arteries that also can cause angina. However, up to 66% of these patients do have coronary microvascular disease, which is more common in women overall and found in people with conditions such as diabetes, high blood pressure and obesity.

For decades, there have been few viable treatment options to improve blood flow through the tiny vessels of the heart. At most, doctors have treated symptoms of angina with several medications and cardiovascular disease prevention methods, including healthy eating, weight loss and regular exercise. The use of a stent could target the issue behind the chest pain — the severe reduction in blood flow affecting the heart muscle.

Unlike tube-shaped stents used to open clogged arteries, the hourglass-shaped stent narrows in the middle. The different design is thought to increase back pressure, redistributing blood flow more fully through small vessels in the heart that were not working at capacity.

"The patients with heart-related microvascular dysfunction in this study had little ability to control their chronic angina, which severely limited their day-to-day activities," says Amir Lerman, M.D., a cardiologist at Mayo Clinic and senior author of the study. "Beyond reductions in chest pain and being able to comfortably handle more physical activity, the majority of patients in the study also showed a connection between the changes in their coronary flow reserve, which is a measure of maximum blood flow, and changes in their quality-of-life responses on the survey. This points to the link between the physiological measurement and angina symptoms."

Dr. Lerman notes that more studies are needed to better understand how the reducer stent works and its long-term effects on blood flow. The stent did not improve chest pain symptoms in 20%-30% of the participants, so future research studies will need to better identify which patients respond best to this therapy.   

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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:

• Terri Malloy, Mayo Clinic Communications, newsbureau@mayo.edu

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Rural adults are more likely to smoke than their urban counterparts. Enhancing digital literacy and improving access to the internet and digital devices may make it easier for rural smokers to quit. These are the findings of a randomized, controlled pilot clinical trial Mayo Clinic researchers published in Nature Communications Medicine.

Digital tools offer a convenient way for rural residents to access support to quit smoking. However, limited digital literacy and technology access can be obstacles. The study tested a program to equip rural residents to take full advantage of these tools.

"Interventions in previous studies had a sole focus, targeting one of three things: device access, internet access or digital literacy," says Christi Patten, Ph.D., a Mayo Clinic behavioral health researcher and a senior author of the study. "We were curious if bundling those resources would be more effective."

The clinical trial included 90 adults living in rural areas of Minnesota, Wisconsin and Iowa and was co-designed with the collaboration of a rural health community advisory board. Prior to the trial, participants smoked an average of 16-17 cigarettes per day and 83% indicated a high readiness to quit. Each person was enrolled in an online smoking cessation program and randomly assigned to one of three study groups:

• Group 1 participants (the control) received a guide to accessing local computer and internet resources.
• Group 2 participants received a loaner computer tablet with internet connectivity and the guide.
• Group 3 participants received weekly telephone coaching to support their individual technology needs, a loaner computer tablet and the guide.

The researchers evaluated participant engagement in the program to gauge the effectiveness of the intervention. They also closely monitored smoking-related outcomes to evaluate the program’s ability to help participants quit smoking.

Coaching provides digital support on the quitting journey

The study revealed a trend toward higher engagement among participants who received additional support, particularly among those in the coaching group.

Overall, 42% of participants completed a survey at the end of the study, with the coaching group at 57%, the loaner computer tablet group at 43%, and the control group at 27%. This pattern held true for other study activities as well, such as the completion of remote testing to track smoking cessation progress.  Although the researchers acknowledged engagement could have been improved, they found the response to the interventions encouraging, particularly the response to coaching.

The researchers observed that coaching not only improved participant engagement but also had an unexpected, positive "spillover effect" on smoking-related outcomes. Participants who received the digital literacy coaching were more likely to report quitting smoking, abstaining from tobacco overall and using online evidence-based cessation resources. These findings, they say, point to a possible smoking-related treatment response that should be tested in a larger clinical trial.

"Changing behavior is incredibly hard," says Andrea Cheville, M.D., a Mayo Clinic physical medicine and rehabilitation physician and a senior author of the study. "The fact that they responded to the coaching was quite remarkable."

In post-trial interviews, participants expressed appreciation for the personalized coaching support for their technology needs, saying it "gave them a feeling of being supported on their quitting journey."

Mary Anne Wolesky, a member of the community advisory board that co-designed the study, says studies like this tell rural residents that Mayo researchers are "in their corner" and want to help.

"There's still a lot of fear of technology in our rural communities," observes Wolesky. "It sends a real message that Mayo would send participants iPads and invest the time to teach them how to use them to improve their health."

Dr. Patten notes that overcoming barriers to improve digital equity is often less about the technology itself than it is about empowering people and giving them confidence to use it. 

Improving remote healthcare delivery

The researchers note that few people who smoke can quit without help; however, people who use smoking cessation resources such as counseling tend to be more successful. For this reason, the researchers say it is vital to improve access to smoking cessation services in rural communities.

"Rural residents have potentially the most to gain from geographically neutral remote delivery systems, such as digital cessation programs," says Dr. Cheville.

The researchers are encouraged by the results of their study, which demonstrate that digital access interventions, particularly digital literacy coaching, can help bridge the digital divide for rural communities. These findings are especially important in the current era of rapidly evolving digital healthcare.

"If we don’t develop strategies to broadly enfranchise rural dwellers, we risk aggravating disparities in smoking and in all aspects of health," says Dr. Cheville.

Looking to the future

The researchers are committed to advancing digital health interventions through ongoing, community-engaged research. In future studies they plan to focus on refining their digital access and literacy interventions, validating the smoking-related treatment response, and exploring ways to target digital behavior and smoking behavior change. They also plan to study the use of digital literacy coaching to individualize pain management for rural patients and to improve remotely delivered cancer care. 

The first author of this study is Sydney Kelpin, Ph.D., who is now a licensed clinical psychologist at Spectrum Health Medical Group in Grand Rapids, Michigan. Review the study for a complete list of authors, disclosures and funding.

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