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

The post (VIDEO) When seizures don’t stop: The battle against drug-resistant epilepsy appeared first on Mayo Clinic News Network.

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

The post New drug may reduce need for aortic valve surgeries by slowing disease progression appeared first on Mayo Clinic News Network.

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.

The post Native community experts guide tobacco cessation research appeared first on Mayo Clinic News Network.

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

The post Mayo Clinic researchers to study causes of rapidly progressive dementia appeared first on Mayo Clinic News Network.

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/

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

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

The post Hourglass-shaped stent could ease severe chest pain from microvascular disease appeared first on Mayo Clinic News Network.

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.

The post Bridging the digital divide to help rural smokers quit appeared first on Mayo Clinic News Network.

Clinical research coordinators manage the day-to-day activities of clinical trials, from recruiting and obtaining consent from participants, to collecting data and tracking adverse events. Known as "CRCs," their job is one of the most vital in research.

"The work of CRCs is highly valued and positions Mayo Clinic as a leader in clinical trials and at the forefront of providing hope and innovative treatments to patients," says Naveen Pereira, M.D., director of the Office of Clinical Trials in the Center for Clinical and Translational Science.

At Mayo Clinic, CRCs support thousands of studies across the enterprise. The role is dynamic, full of opportunities for growth, and much more.

Discovering the unexpected

Carl Griffin II is a lead CRC in Cardiovascular Research. When he first began working as a CRC, he was prepared for a challenge but did not anticipate how absorbing and complex the job would be.

"The intricacy of the work — how you take a participant from start to finish and all of the different pieces of study management involved — surprised me," he says.

A few years into the role, Griffin highlights the job's multifaceted nature as one of its best features. Every day brings new challenges and learning opportunities.

Patsy Caceres Figueroa, a senior CRC in Cardiovascular Research, says that before taking on the role, she did not realize how much work went into producing research publications. She also didn't anticipate how much she would love working with study participants. For her, one of the most rewarding perks of the job has been the long-term relationships she has built with people who volunteer to participate in research.

"We work with participants, sometimes for years, and get to know them well," she says, noting that this personal connection has helped her find added fulfillment in her work.

A strong, supportive team

At Mayo Clinic, the sense of community among CRCs is important to their success. This supportive environment ensures smooth operations and fosters an environment where CRC team members feel valued and have a sense of belonging among team members.

Caceres Figueroa recalls when her team rallied around her after the loss of her grandmother.

"I was coordinating a complex industry study during a particularly busy time," she recalls. "But my team was ready to help me, taking over my duties so that I could be with my family."

Griffin, who balances work with raising young children, says he also values his team's supportive spirit.

"Knowing that my colleagues are there to cover responsibilities when needed—and that I’ll do the same for them—makes all the difference," he says.

Professional growth opportunities

As Griffin reflects on his time as a CRC, he notes how the job has given him opportunities to enhance his skills and boost his confidence. He says he has particularly benefited from training new colleagues.

"I don’t think three years ago I would have felt confident doing that," he says. "I would have been quietly panicking about it. But now it just feels relaxed."

Caceres Figueroa also appreciates how Mayo Clinic has supported her professional growth, particularly through training resources designed exclusively for clinical trials staff. She's pursuing a certification course to improve her knowledge of clinical research.

Beyond their core roles, Griffin and Caceres Figueroa participate in broader aspects of research, which enriches their careers. Griffin is working with a team to enhance scheduling procedures, and Caceres Figueroa has begun learning the ropes of auditing. Both are enthusiastic about contributing to continuous improvement in their work unit.

"I feel so fortunate to be in this position and in this job," says Caceres Figueroa.

Learn more

• Read more about the essential role of clinical research coordinators in clinical trials: Clinical research coordinators: Advancing research, improving care.
• Explore a life-changing career as a Clinic Research Coordinator.

The post The dynamic role of clinical research coordinators appeared first on Mayo Clinic News Network.

Research teams from Mayo Clinic in Florida and Carnegie Mellon University will work on a federally funded project to accelerate the development and testing of a new device to treat patients with type 2 diabetes and obesity.

The bioelectrical device, known as Rx On-site Generation Using Electronics, or ROGUE, is being developed at Carnegie Mellon University. It will act as a "living pharmacy" using engineered cells capable of producing therapy to treat patients with both conditions. At the conclusion of device development, Mayo Clinic will conduct a first-in-human clinical trial to test the viability of ROGUE in humans for wider applicability.

The goal for the device is to produce regulated levels of glucagon-like peptide 1 (GLP-1)-based therapies, which can improve glucose levels in patients with diabetes and also promote weight loss.

The Advanced Research Projects Agency for Health, or ARPA-H, made the award to Carnegie Mellon and the project's principal investigator, Itzhaq Cohen-Karni, Ph.D. The ROGUE collaboration also includes bioengineering faculty from Boston University, Georgia Institute of Technology, Northwestern University, Rice University, University of California Berkeley and Bruder Consulting and Venture Group.

"There is great potential for ROGUE to expand access and decrease the costs of these medical therapies for at-risk patients who may not otherwise be able to use these agents for diabetes and obesity," says Susan Samson, M.D., Ph.D., chair of the Division of Endocrinology, Diabetes and Metabolism, Department of Medicine, Mayo Clinic in Florida. Dr. Samson has a background in preclinical models and translational research in the use of GLP-1 receptor agonists for metabolic disorders and is the clinical team lead and principal investigator of the project at Mayo.

ROGUE is an implanted device designed to make biological drugs more accessible to patients by reducing development and manufacturing costs while making it easier for patients to follow their treatment plan. The device, implanted during a minimally invasive outpatient procedure, will be designed to deliver continuous therapeutic peptides.

"Our team will be engaged throughout the project to provide expertise from the clinical side to ensure that the device is usable, safe and comfortable for patients down the line," says Mayo physician-scientist and co-principal investigator Maria Daniela Hurtado Andrade, M.D., Ph.D. Dr. Hurtado Andrade brings basic science and clinical trial expertise to the project and also is an expert in the field of weight management.

A phase one clinical trial will be conducted at Mayo Clinic in Florida, the sole clinical site for testing the device in humans. The trial requires the recruitment of a diverse group of patients, including those most at risk for diabetes and obesity.

"Mayo Clinic in Florida, which is embedded within the Jacksonville community, is strategically positioned to recruit this diverse study population," says Dr. Hurtado Andrade. As part of the project, Mayo Clinic will provide diabetes educators to help educate patients on the device's use.

The post Mayo Clinic, Carnegie Mellon researchers secure ARPA-H award for multiyear implantable device research for diabetes and obesity appeared first on Mayo Clinic News Network.

CAR-T cell therapy is a treatment that harnesses the power of a person's immune system to combat cancer cells. In this regenerative immunotherapy, a person's T cells, or white blood cells known as lymphocytes that are involved in the immune system response — are collected and genetically modified to produce chimeric antigen receptors or CARs. They are then infused back into the patient's blood stream, where they target and destroy cancer cells. Mayo Clinic researchers are advancing and improving CAR-T cell therapy to expand its capabilities to treat more conditions. Read more about the latest advances in CAR-T at Mayo Clinic. 

CAR-T cell researchers at Mayo Clinic optimistic about future of treating blood cancers 

While many CAR-T cell therapies use a patient's own cells, Mayo Clinic is also exploring the applications of allogeneic, or "off-the-shelf," CAR-T therapies. These are generated by healthy donors or gene-edited sources that have been genetically altered to reduce the possibility of rejection by the patient's immune system. 

"This can provide a faster turnaround time for manufacturing, expand patient access and allow patients to receive CAR-T cell therapy at the time of need," says Yi Lin, M.D., Ph.D., a hematologist and oncologist at Mayo Clinic Comprehensive Cancer Center. 

CAR-T cell therapy is usually offered as a later-stage therapy for various blood cancers, but recent research has shown it may be useful in some blood cancer cases earlier in a patient's treatment plan.

"CAR-T cell therapy in earlier lines of treatment has reported superior outcomes as compared to using it in later lines of therapy," says Rafael Fonseca, M.D., hematologist at Mayo Clinic Comprehensive Cancer Center. 

Unleashing CAR-T cell therapy to destroy solid tumors in thyroid cancer 

Mayo Clinic researchers are working to apply CAR-T cell therapy to solid tumors in thyroid cancer. CAR-T has shown promising results in blood cancers, and new research is focused on using this treatment on more types of malignancies.

"CAR-T cell therapy is unlike other therapeutics," says Saad Kenderian, M.B., Ch.B., a hematologist and cancer researcher. "Other therapies may slow down cancer. CAR-T cell therapy has shown great promise in stopping B-cell lymphomas and leukemias. Some of my patients have gone into complete remission that has lasted for years after just one treatment." 

Could CAR-T cell therapy improve kidney transplants? 

CAR-T cell therapy could provide a revolutionary approach to organ transplantation for "sensitized patients" who are hard to match and susceptible to rejection, Mayo Clinic researchers discovered. 

Their pioneering research focuses on using CAR-T cells derived from the patient's own immune system to prevent rejection of donated organs. Sensitized patients are those who have high levels of antibodies that cause their immune systems to react negatively to potential donor organs. These patients often face extended waiting periods for a transplant. 
 
Research from this proof-of-concept study is published in Kidney International. 
 
"This research is one of the first steps toward applying CAR-T cells in the field of transplantation to try to make more donor organs available for transplant and reduce the wait for patients who need a new kidney," says Tambi Jarmi, M.D., first author on the study and a transplant nephrologist at Mayo Clinic in Florida. 

Mayo Clinic scientists pioneer immunotherapy technique for autoimmune diseases

Mayo Clinic scientists have developed an immunotherapy strategy that potentially lays the groundwork for treating a spectrum of autoimmune diseases.

The new technique, detailed in a preclinical study published in Nature Biomedical Engineering, involves combining chimeric antigen receptors (CARs) with mesenchymal stromal cells (MSCs), which are found in various tissues in the body and are known for calming down the immune system, controlling inflammation and promoting immune tolerance to prevent the body's own tissues from being attacked. 

"The pioneering approach shows potential in targeting inflammatory disease sites more precisely and improving immunosuppression and healing outcomes," says Dr. Kenderian. "We're planning to study interventions that minimize the need for long-term medications for autoimmune diseases." 

Unleashing viruses aimed at killing cancer 

Mayo Clinic cancer researcher Richard Vile, Ph.D., is leading research into genetically engineered viruses aimed at unleashing a two-pronged attack on cancer. One part of this technology, known as an oncolytic virus, is designed to infect, break open and destroy cancer cells while sparing healthy tissue. Dr. Vile's preclinical studies have shown that oncolytic viruses replicated in cancer cells and cascaded to kill other diseased cells. That, in turn, triggered an immune response in which the patient's own T cells, stimulated by the virus, recognized and targeted metastasized tumors for a second wave of cancer destruction.

"Oncolytic viruses are a way to alert the immune system and mobilize it to kill cells infected with cancer," says Dr. Vile. "There's the direct killing of cancer cells with the virus, and then there's the major effect of immune activation. The immune system is incredibly well evolved to recognize infection, clear infection and kill all the cells around it that could be harboring infection." 

Dr. Vile's team is combining oncolytic viruses with CAR-T cell therapy to target solid tumors from liver cancer. This experimental approach of loading CAR-T cells with oncolytic virus is a new way to expand CAR-T cell therapy beyond treatment for blood cancers into treatment for solid tumors. 

Preparing to biomanufacture a new CAR-T cell therapy for B-cell cancers 

Mayo Clinic research has developed a new type of CAR-T cell therapy aimed at killing B-cell blood cancers that have returned and are no longer responding to treatment. This pioneering technology, designed and developed in the lab of Hong Qin, M.D., Ph.D., killed B-cell tumors grown in the laboratory and tumors implanted in mouse models. The preclinical findings are published in Cancer Immunology, Immunotherapy. 

"This study shows our experimental CAR-T cell therapy targets several blood cancers, specifically chronic lymphocytic leukemia," says Dr. Qin. "Currently there are six different CAR-T cell therapies approved for treatment of relapsed blood cancers. While the results are impressive, not everyone responds to this treatment. Our goal is to provide novel cell therapies shaped to each patient's individual need."

Dr Qin's team developed a cell therapy to target a protein known as B-cell activating factor receptor (BAFF-R) found in patients with B-cell cancers, particularly those with chronic lymphocytic leukemia. The BAFF-R protein is linked to tumor growth. The cell therapy under investigation allows the immune system to quash cancer and target tumors that have returned or have resisted available CAR-T cell therapies. 

Using a molecular scissors to improve CAR-T cell therapy 

Mayo Clinic researchers who mined the molecular foundations of cancer have uncovered a new reason CAR-T cell therapy fails in some patients. This discovery has fueled new strategies that incorporate antibodies and gene editing to improve the outcome of this breakthrough treatment.  

"This is a very exciting discovery that offers new hope of overcoming challenges of CAR-T cell therapy that many cancer patients experience," says Dr. Kenderian, a hematologist and senior author on this research, published in Nature Communications. 

"We describe for the first time a mechanism causing the resistance and failure of CAR-T cells, which lies within a protein routinely made by the engineered cells. This research puts us on a new path for improving the longevity of CAR-T cell therapy." 

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