Posts (150)

Fri, Apr 12 2:30pm · Hypoplastic Left Heart Syndrome Collaboration

Women kneeling with child dressed as doctor

Mayo Clinic and The Children’s Hospital at OU Medicine in Oklahoma City are collaborating within a consortium to help patients with hypoplastic left heart syndrome, also known as HLHS, a rare and complex form of congenital heart disease in which the left side of the heart is severely underdeveloped.

The collaboration is expanding the development of cell-based, innovative research opportunities to transform the lives of people living with hypoplastic left heart syndrome.

The Children’s Hospital at OU Medicine has been involved with the hypoplastic left heart syndrome program since 2015 and performed its first umbilical cord blood cell delivery in 2016, according to Timothy Nelson, M.D., Ph.D., director of Mayo Clinic’s Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome.

“We’re thrilled that they’ve joined the consortium because it means that individuals with hypoplastic left heart syndrome will now have more access to participating in groundbreaking clinical trials,” Dr. Nelson says.

This brings the total number of Hypoplastic Left Heart Syndrome Consortium members to six: Mayo Clinic, Children’s Hospital of Philadelphia, Children’s Hospital of Los Angeles, Children’s Minnesota, Children’s Hospital Colorado, and The Children’s Hospital at OU Medicine.

Harold Burkhart, M.D., who leads the pediatric cardiothoracic surgery team at OU Medicine, says being a member of the consortium establishes Oklahoma as a regional center for families to receive care closer to home.

“Families of children with heart anomalies want to know that we’re not just resting where we are,” Dr. Burkhart says. “We need to keep pushing forward to come up with newer treatments. This collaboration provides new hope for patients and it is the epitome of a bench-to-bedside research project.”

Read the full announcement on the Mayo Clinic News Network.

Thu, Mar 28 12:56pm · On Common Ground

picture of Nancy and Duane Baumann Shrill screams caused Berneice Baumann to stop what she was doing and rush into the kitchen. She found her 1-year-old son, Duane, sitting on the floor in tears. He’d pulled an electric coffeepot on top of himself, and the boiling liquid scalded him from the neck down.

A local doctor did his best to treat Duane in 1941, but despite his best efforts, raised scar tissue, known as keloids, began forming. Berneice knew that eventually the scar tissue would restrict Duane from turning his neck. At a follow-up appointment, the doctor recommended cutting the keloids off as a last resort. Berneice wanted a second opinion.

Seeing ‘Ghosts’

At the suggestion of her sister, Berneice packed up the family car and drove all night from their Illinois farm to reach Rochester, Minnesota. Upon arriving, she reached for Duane and entered Saint Marys Hospital, now known as Mayo Clinic Hospital — Rochester, Saint Marys Campus, where nuns greeted her and took young Duane down the hall to be evaluated by the doctors.

“At that time, doctors had bands with lights on their heads, white jackets, and they entered the room as a group. As a young boy, I thought they were ghosts floating into the room,” Duane says.

The doctors calmed Duane and immediately began working to stunt the keloids’ growth and shrink the newly developed tissue.

A week later, her son treated, Berneice approached the front desk to settle her bill. She cautiously made eye contact with the staff member and requested a payment plan. Money was scarce.

Seconds slogged by before the staff member returned, bill in hand. Glancing down at the paper, Berneice was filled with relief. “They told her she owed $10,” Duane says.

Just a Checkup

picture of Duane Baumann tending plants

Duane Baumann

In 1967, Duane earned his Ph.D. from Clark University and became a professor at Southern Illinois University in Carbondale, Illinois. Years later, he married his wife, Nancy, and started a water resource consulting firm. They had three daughters — Bridget, Rebecca and Brittany. Throughout the years Duane remained adamant everyone in his family have their medical needs treated at Mayo Clinic.

“My family would spend vacations in the Phoenix area to enjoy the warmer winter climate, play tennis and participate in other outdoor activities,” Rebecca says. “We would also have our checkups at Mayo Clinic in Scottsdale. It was an annual family outing for us.”

In 2004, the Baumanns visited Arizona during spring break. Since they were in the area, Nancy carved out time to stop by Mayo Clinic for her annual physical. Typically she would go in one day and receive a follow-up call the next to confirm everything looked normal.

This time was different.

A Swift Response

Nancy received a call requesting she come back to the clinic for a second mammogram.

“I thought nothing of it,” she says. The Baumanns had planned to travel back to Illinois that Saturday, but when Nancy went in for her follow-up results, predetermined plans became irrelevant.

“The doctor told me the tests revealed I had breast cancer,” Nancy says. “I asked them if I should just come back during the summer for treatment, but they told me it needed to be treated — now.”

The following week, surgical oncologist Barbara A. Pockaj, M.D., Michael M. Eisenberg Professor, conducted Nancy’s lumpectomy, and post-surgery tests determined Nancy was cancer-free.

“Every step of the way, from diagnosis through treatment, I was shown compassion for my situation and respect with all my questions,” Nancy says. “That gave me strength and courage to move through it.”

Family and Future

These days, Duane and Nancy find themselves enjoying short treks through the Sonoran desert that surrounds their Arizona home. Duane, true to his farming background, savors spending time in his backyard admiring and caring for the unique cactuses and plant life.

With a Mayo Clinic campus close to their home, they take time to stop by to hear about what is new in the world of medicine. Duane attended a Department of Development event where Wyatt W. Decker, M.D., vice president, Mayo Clinic, and CEO of Mayo Clinic in Arizona at that time, was speaking about regenerative medicine. Having recently experienced heart valve problems that required surgery — the same surgery Duane’s mother had undergone years earlier — the presentation on heart valve regeneration piqued Duane’s interest.

Mayo Clinic’s investment in its research and patients inspired the Baumanns to give to the Center for Regenerative Medicine. In honor of their generosity, Mayo Clinic recognizes the Baumann family as Major Benefactors.

“Mayo Clinic’s research in regenerative medicine is going to change the face of medicine,” says Duane, who credits Bruce K. Kimbel Jr., M.D.Patrick A. DeValeria, M.D., and Aleksandar Sekulic, M.D., Ph.D., for providing excellent care for his family.

Having grown up going to Mayo Clinic, the Baumanns’ daughters view the clinic as more than a place of health care.

“Mayo Clinic is unique in that doctors try to get to know their patients and engage in conversation,” Brittany says. With more than 60 years of interaction, the Baumann family is tied to Mayo Clinic in a special way. “Mayo Clinic is a legacy passed down from generation to generation,” says Bridget. “Mayo Clinic is part of our family.”

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This article originally appeared in Mayo Clinic Magazine. Read the original story in the Spring 2018 edition.

Mon, Mar 25 10:33am · Leading the Charge in Regenerative Medicine

Thank you for your interest. We would be happy to connect with you regarding regenerative medicine research, stem cell treatments and/or research at Mayo Clinic. Please call our Regenerative Medicine Consult Service at 844-276-2003 to schedule an appointment to speak with us. There is no charge for the appointment. We look forward to hearing from you.

Thu, Mar 14 6:53pm · Business Innovation with an Eye on Improving Vision

If eyes are the window to the world, Timothy W. Olsen, M.D. is building high performance window frames. With a passion for restoring vision, the ophthalmologist set sights on developing and bringing to market a first-of-its-kind device for treating age-related macular degeneration. The synergies around the Destination Medical Center economic initiative and Mayo Clinic’sresearch and practice community prompted him to move his business and clinical/surgical practice from Atlanta to Rochester, Minnesota.

Timothy W. Olsen, M.D.

“The business environment is second to none. Mayo has made a statement through Destination Medical Center that it wants to be an innovation center for medical technology,” says Dr. Olsen. “That combination of business, technology and connection to Medical Alley bio businesses in Minnesota makes this is a really good place to develop and commercialize medical devices.”

Destination Medical Center is a 20-year, multibillion dollar public-private partnership to position Rochester as a global destination for health care, biotechnology and life science discoveries. The money supports public infrastructure and does not go to Mayo Clinic. The convergence of entrepreneurship, medical expertise and regulatory support, Dr. Olsen says, is the perfect place for a successful product launch.

“Destination Medical Center is the city of Rochester, Olmsted County and the state of Minnesota. With those components, hopefully there will be private sector support as well as the opportunity for engaging with people involved in funding early stage start-up companies, including venture capital funding opportunities,” says Dr. Olsen.

A new device for age-related macular degeneration

Macular degeneration affects more than 3 million Americans and is the leading cause of vision loss for people over 50. There is no cure or treatment in the early stages. Patients with end stage macular degeneration may be suitable for monthly eye injections, but that is expensive and inconvenient. The disease process usually progresses despite the injections.

That’s where Dr. Olsen’s business comes in. His team is advancing research on a surgically-implantable device, using a technology that was first conceived at the University of Minnesota and patented through Emory University. That device acts as a window or picture frame holding regenerative tissue in place to support the macula at the back of the eye, potentially reversing vision loss and preserving the function of the macula.

The National Institutes of Health/National Eye Institute awarded a Small Business Technology Transfer Research grant for a one-year, phase I feasibility study. The grant goes directly to Dr. Olsen’s company, located in the Mayo Clinic Business Accelerator, with sub grant funding for preclinical research at Mayo.

“The goal of this research grant is to support scientists launching commercialization of a product rather than the researcher studying technology from an outside company, in an effort to speed the movement of a product from the research lab into the marketplace,” says Dr. Olsen.

After the feasibility study, there is a possibility of additional early-stage funding through a larger two-year, phase II National Institutes of Health research grant. Once that is completed, access to private capital would likely be needed to continue product development. Dr. Olsen believes the entrepreneurial environment created through the Destination Medical Center initiative will help attract that private investment. If the device passes regulatory approvals, it may take five to 10 years before it is offered in a clinical setting to address the unmet needs of millions of people suffering from age-related macular degeneration.

This article first appeared on the Advancing the Science blog.

Thu, Feb 28 2:52pm · A Regulator of Stem Cells

Ouch! The response is always the same to the sharp, sudden surprise of a paper cut. The body responds quickly to the tear in the skin and sends resources for protection. An abundant enzyme in the blood, thrombin, causes changes in other blood proteins to produce a gel like substance that forms the clot. It’s what causes the bleeding to stop and is the first stage of wound healing.

Thrombin is abundant in the blood and increases in the central nervous system with injury. If the body experiences trauma from a stroke, for example, this first responders rush in and start repairing the damage. While the enzyme, called thrombin, helps heal small cuts, it may cause problems when the body brings in the heavy hitters of healing: Stem cells.

Stem cells are a type of cell in the body that can become another cell. When damaged tissue needs to be replaced or expanded due to growth, stem cells expand or multiply to get it done.

“What we discovered is that thrombin activates a receptor on stem cells that actually blocks their ability to expand,” says discovery science researcher Isobel Scarisbrick, Ph.D.

Isobel Scarisbrick, Ph.D.

That means that when thrombin attaches to a stem cell, it is effectively turned off. So why does this happen? Thrombin is a regulator. It attaches to a cell membrane through a receptor called PAR1, and sends signals about the environment surrounding the cell. It is signaling information about what to do and what not to do, depending on the situation.

COMMON ENZYME IS A BODY BOSS
The thrombin receptor PAR1 is a newly identified fundamental regulator of stem cell biology.

“I like to think of this receptor like a switch,” says Dr. Scarisbrick. “When the switch is on, the cell does not mature, for example into a myelin producing cell. When the switch is off the stem cell can differentiate and expand.”

Dr. Scarisbrick explains that this information allows her team to look for ways the body can heal vital structures from within.

“It’s a new discovery that this common receptor plays a fundamental role in the biology of the human brain, and an important role in stem cell expansion and myelin production,” she says.

MYELIN REGENERATION
Dr. Scarisbrick and her research team are looking at the biology of how receptors regulate neural stem cells in the brain, the spinal cord, and the protective myelin sheath.

Myelin is a covering of the nerve cells that accelerates transmission of impulses, or instructions from the central nervous system, along the electrical cables knowns as axons. These impulses can slow or stop when the myelin sheath is damaged. Myelin regeneration occurs naturally in the body and there are many factors involved. On the cell wall is a receptor that allows a protein such as a growth factor, cytokine or even an enzyme such as thrombin to come along and attach to it and start sending out instructive signals for the cell to respond to.

“In our study the enzyme thrombin is directing the cell and it’s telling the cell what to do,” Dr. Scarisbrick says. “There are all kinds of things going on outside the cell and the cell machinery needs to respond.”

REBUILDING, REPAIRING MYELIN
Dr. Scarisbrick’s team has evidence in mouse models that when the thrombin receptor is switched off there is more of the myelin protein present at birth and even higher levels in adulthood. There is also evidence of better recovery after spinal cord injury.

In the adult mouse brain is a reservoir of neural stem cells that can differentiate into any cell type in the brain. These are the cells that migrate out to repair the area of myelin degeneration from stoke or other injury.

Generating myelin from adult neural stem cells, from left to right: Neural stem cells from the subventricular zone of the adult mouse brain, myelin producing oligodendrocytes from neural stem cells, and ultrastructure of the mature myelin sheath insulating axons

“The research shows that animals that do not have this receptor have an endogenous capacity to generate more stem cells,” she says.

FUNDAMENTAL BIOLOGY
Dr. Scarisbrick says she is particularly excited about this work because it is not often a research team can target just one factor and have such a big impact.

“I think we could focus exclusively just on the neural stem cells, but I think there’s an opportunity here to see just how fundamental this biology is,” she says. “My laboratory is particularly interested in the repair of the spinal cord and the myelinating regions, and we are excited our new discovery will translate into new strategies for regenerative repair.”

This article originally appeared in Mayo Clinic’s research magazine, Discovery’s Edge.

Fri, Feb 15 7:00am · Mayo Clinic Researchers Develop More Efficient System to Reprogram Stem Cells

Induced pluripotent stem cells, the workhorse of many regenerative medicine projects, start out as differentiated cells that are reprogrammed to pluripotent stem cells by exposure to a complex set of genetic cocktails. Mayo researchers now report that using the measles virus vector; they’ve trimmed that multi-vector process with four reprogramming factors down to a single “one cycle” vector process. They say the process is safe, stable, faster and usable for clinical translation. The findings appear in the journal Gene Therapy.

“If we’re going to successfully use reprogrammed stem cells to treat patients in the clinic, we need to ensure that they are safe and effective, that is, not prone to the risk of mutation and potential tumors,” says Patricia Devaux, Ph.D., Mayo Clinic molecular scientist and senior author of the article. “The measles virus vector has long been used safely at Mayo for treating cancer, so it is very safe. Now that we’ve combined a multiple-vectors process into one, it’s efficient as well.”

Previously, the four reprogramming factors – proteins OCT4, SOX2, KLF4 and cMYC – had to be introduced individually to the cells to induce them to change in the proper fashion for the desired outcome. That led to potential partially reprogrammed cells, as not all cells received the four factors required for reprogramming. The new Mayo process combines those factors within the measles virus vector so the process happens in one step and all targeted cells have the potential to reprogram. It should be noted that this measles virus is attenuated, that is all dangerous aspects of the virus have been removed, as they are in a vaccine, and the virus becomes a vector or carrier for other genetic material. The measles virus vaccine strain is often used today because it is safe, fast and targetable.

The researchers say a clinically applicable reprogramming system free from genomic modifications will go a long way to making widespread use of induced pluripotent stem cell therapies feasible. These are therapies in which an individual’s own cells are reprogrammed can then be use to work in a particular diseased organ, thus avoiding risk of cell rejection.

Additional co-authors of the article include first author Qi Wang, Alanna Vossen, and Yasuhiro Ikeda, D.V.M., Ph.D., all of Mayo Clinic. The research was supported by the National Institutes of Health, including the National Institute of Allergy and Infectious Diseases, National Center for Advancing Translational Sciences, Mayo Clinic Graduate School of Biomedical Sciences, and Mayo Clinic Center for Regenerative Medicine.

This article was first published on Mayo Clinic’s News Network.

Thu, Feb 7 2:52pm · Leading the Charge in Regenerative Medicine

Regenerative medicine therapies aim to rebuild and restore health to patients challenged by chronic conditions and degenerative diseases. Despite advances in the field, much of the science is still in early research phases, meaning that many treatments haven’t been proven safe and effective for humans as standard-of-care therapies yet. Because the scientific process is long, and there is great hope for regenerative therapies as treatments for a wide variety of diseases, the FDA has created streamlined pathways to help get regenerative options to patients more quickly. In fact, the FDA recently announced that it will greatly expand its regenerative therapies review process.

The hope in regenerative therapies has also led to “hype” surrounding this field of medicine. This is especially true for the case of stem cell therapies, which are often marketed to the public as cure-alls for a variety of medical conditions. Unfortunately, many of these for-profit clinics do not have scientific evidence to back up their claims, and many patients pay out-of-pocket for treatments that may not have any benefit (and, more concerning, may produce serious harms).

“It is our responsibility to make sure that we get safe and ethical products to our patients,” says Zubin Master, Ph.D., an associate consultant in the Biomedical Ethics Research Program at Mayo Clinic. “Our early experience in this process will pay off when more of these therapies become available through legal and approved pathways, and we translate them into the clinical practice for a number of specialties.”

In the Mayo Clinic Center for Regenerative Medicine, several steps have been taken to ensure that regenerative therapies are translated to the public responsibly. In a recently published paper, researchers outline three major ways in which they are innovating in order to best serve the needs of patients. The goals are to positively impact patient education and navigation, provide an example of an interdisciplinary clinical space that can be used for regenerative medicine research and treatment, and lastly, track the outcomes of patients.

The first area of innovation is the Regenerative Medicine Consult Service, a free service offered to patients who would like to know more about regenerative options for a particular medical condition. Patients can call in to speak with a consultant who can give information on the state of stem cell research, share potential research opportunities, and potentially recommend clinical services.

The  Regenerative Medicine Therapeutic Suites at Mayo Clinic’s Jacksonville campus is another advancement at Mayo Clinic. This clinical space is regulatory-compliant and integrated patient care with laboratory functions. The unique space serves both patients and clinicians, providing a place for multidisciplinary teams to expand current projects in order to deliver individualized regenerative therapies and procedures to patients.

Lastly, the Regenerative Evidence-Based Outcomes Registry was launched in November 2018 and has already logged nearly 200 surveys with information about patient treatments and outcomes. This “real world evidence” will be used in conjunction with other mechanisms, such as clinical trials, to validate therapies for patient use. The platform collects a variety of data, including information about ethical concerns related to patient understanding of stem cell therapies and the difference between research and therapy. This knowledge will provide a more robust source of information in order to advance regenerative therapies and education for patients around stem cells and regenerative medicine.

“Our program is about how we prepare our health care system to utilize these treatments in the future,” says Shane Shapiro, M.D., program director for the Regenerative Medicine Therapeutic Suites and assistant professor of orthopedic surgery. “Cell therapy will be a valuable tool for many conditions, and our health care providers have to be prepared to deploy them.”

— Cambray Smith, research assistant, Biomedical Ethics Research Program

Thu, Jan 31 10:08am · Apply for 2019 Regenerative Medicine Minnesota grants

Regenerative Medicine Minnesota is accepting proposals for grants relating to regenerative medicine education programs, clinical care, and biobusiness/biotechnology. Applicants must be based in Minnesota.

Important notes for Mayo Clinic investigators:

Application process and deadlines
Request for proposal (RFP) details:

The Office of Sponsored Projects Administration internal deadline for submitting budget requests is Feb. 15, 2019. Applications are due to Regenerative Medicine Minnesota by Feb. 25, 2019.

Since the Minnesota Legislature allocated resources to fund the program, Regenerative Medicine Minnesota has been working to improve the health of Minnesotans by advancing regenerative medicine therapies. Grants have been awarded to accelerate medical research; to develop biotechnology and biobusiness; and to recruit, train and retain the next generation of health care workers and researchers in the field of regenerative medicine.

More information
Visit the Regenerative Medicine Minnesota website or contact Beth Borg, operations administrator, Center for Regenerative Medicine Administration, at (77) 4-1075.

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