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The Biotrust: Focusing the Power of Regenerative Medicine

Posted on April 10th, 2014 by Center for Regenerative Medicine

The stem cell approach is the heart of regenerative medicine. In the past few years its promise has become reality with Mayo Clinic delivering regenerative treatments for osteonecrosis, tendonitis and reconstructive surgery.

With the growing interest in regenerative medicine research and treatment, Mayo Clinic established the Regenerative Medicine Biotrust, which helps researchers more efficiently collect samples, process cells and share results.

“The process of harvesting a cell, differentiating it into specialized tissues, expanding it into big numbers, then using them for real applications in research, clinical applications, clinical trials, etc. – that’s complicated,” says Dennis Wigle, M.D., Ph.D., director of the Regenerative Medicine Biotrust. “You need an infrastructure to be able to do that.”

Building on Mayo Clinic’s expertise in biobanking, the Regenerative Medicine Biotrust gives researchers and clinicians access to living cells. The approach will fast-track discovery, accelerating the time-consuming processes of specimen collection and conversion. In this way, teams of scientists and clinicians will develop focused questions and go right to the prepared cells to find the answer.

 “The Biotrust puts Mayo at the cutting edge in investigating how fast we can make new technologies, apply new technologies and do it for real clinical care,” Dr. Wigle says. “This will advance regenerative solutions for our patients.”

To learn more, watch the video below:

Building a Better Liver

Posted on March 21st, 2014 by Center for Regenerative Medicine

From the pages of Mayo Clinic Magazine.

The spheroid liver cell (hepatocyte). Millions of these are needed to approximate the function of the natural human liver.

The spheroid liver cell (hepatocyte). Millions of these are needed to approximate the function of the natural human liver.

The Center for Regenerative Medicine Is Working on Treatments Unimaginable 10 Years Ago

A few years ago, nature dealt a fatal blow to Cassandra Rohrer, a 16-year-old patient of Scott Nyberg, M.D., Ph.D. She was fighting for her life with the assistance of an experimental artificial liver device, which was keeping her alive but couldn’t do so much longer. She needed a liver transplant.

After two days on the device, her family got the news that an organ was available and being flown from Michigan. The family and a team of caregivers jumped into action, preparing the girl for her lifesaving transplant. But the team delivering the liver got caught in a thunderstorm and didn’t make it in time. The girl became neurologically unstable and did not wake up after surgery.

“People with liver failure, one of the main causes of death is ammonia buildup in your blood,” Dr. Nyberg says. “Proteins break down and produce ammonia, essentially making oven cleaner, and it causes your brain to swell and you die from brain herniation. If any one of us overdoses from Tylenol and kills our liver, we’d die of brain herniation probably in two to three days if we didn’t get a liver transplant.”

Since then, Dr. Nyberg has kept a picture of Cassandra to remind him why he spends long hours searching for new treatments.

His search has taken him to places unimaginable 10 years ago. He began his work seeking to build a better artificial liver device, one that uses large doses of human liver cells and can treat patients for longer periods of time until they receive a transplant. It’s still a primary goal, but now he envisions also using a patient’s stem cells to bioengineer healthy liver cells, then transplanting them back to the patient to cure a diseased liver.

“Doctors are already doing liver cell transplants, but the supply of human cells is very limited, so there are only a few of them done a year,” Dr. Nyberg says. “Many diseases you can treat and cure with just the cells. There’s a large demand for cell transplants.”

He and colleagues in Mayo Clinic’s Center for Regenerative Medicine are pioneering a method to grow a large number of patient-specific liver cells to meet demand. They are also using these cells to try to create whole new livers, tailored to individual patients.

A New Liver of Your Own

Today, there are simply not enough livers available. About one in ten people on the liver transplant waiting list will die before an organ becomes available.

A contributing factor to the shortage is that sometimes a donated liver has a defect that makes it unusable. To save more lives, Dr. Nyberg is working with the regenerative medicine company Miromatrix to remove all the cells from these defective livers, leaving only a scaffold. He’s working with Mayo Clinic’s Allan Dietz, Ph.D., to populate the scaffold with stem cells taken from the patient, creating a whole new organ.

This tailor-made liver accomplishes two goals: It overcomes the hurdle of immunosuppressant medicines, and it makes more livers available.

A traditional organ transplant introduces foreign tissue into the body, which triggers the patient’s immune system to attack the new organ. To suppress the response, transplant patients take a cocktail of medicines for the rest of their lives. These medications are expensive and can have serious side effects. However, a liver built with the patient’s cells would end the need for immunosuppressants.

It would also help ensure that patients like Cassandra would have a liver when they needed one.

Read more about liver regeneration at Mayo Clinic.
Read more about Mayo Clinic's bioartificial liver in Discovery's Edge, Mayo's research magazine.

Regenerative Medicine Consult Service

Posted on March 19th, 2014 by Center for Regenerative Medicine

Worldwide, people are increasingly interested in regenerative medicine or stem cell therapies. To meet this interest, Mayo Clinic established the Regenerative Medicine Consult Service — the first consult service of its kind in the United States.

This service provides people with educational guidance and appropriate referrals to research studies and clinical trials in stem cell therapies. Indications for a regenerative medicine consult include, but are not limited to degenerative and congenital diseases of the heart, liver, pancreas and lungs in which the patient or clinician has questions about the value of stem cell-based therapies.

Patients and health care providers are informed of existing regenerative services at Mayo Clinic and, when appropriate, patients are referred to research studies at Mayo Clinic and elsewhere.

For more information, call 844-276-2003.

To learn more about regenerative medicine and the Consult Service, watch the video below.

Stem cells for treatment of ALS at Mayo Clinic

Posted on March 7th, 2014 by Center for Regenerative Medicine

Researchers in the Center for Regenerative Medicine are studying the use of intraspinal deliver of mesenchymal stem cells (MSCs) to the cerebral spinal fluid of patients with ALS using a dose-escalation study. Learn more below:

Stem Cells 101

Posted on February 14th, 2014 by Center for Regenerative Medicine

You've heard about stem cells in the news, and perhaps you've wondered if they might help you or a loved one with a serious disease. You may wonder what stem cells are, how they're being used to treat disease and injury, and why they're the subject of such vigorous debate.

Timothy Nelson, M.D., Ph.D., director of Mayo Clinic's Regenerative Medicine Consult Service, answers some of the most commonly asked questions about stem cells:

What are stem cells?

Stem cells are the body's raw materials. These cells have the ability to renew themselves or change to become specialized cells with a more specific function, such as blood cells, brain cells, heart muscle or bone.

Where do stem cells come from?

  • Adult stem cells are found in tissues of the body, such as bone marrow. Emerging evidence suggests that adult stem cells may be more versatile than previously thought and have the ability to create other types of cells.
  • Amniotic fluid stem cells come from the liquid that surrounds a developing fetus in the amniotic sac. More study of amniotic fluid stem cells is needed to understand their potential.
  • Embryonic stem cells are a general stem cell found in embryos. Use of this type of stem cell has significantly declined due to the discovery of induced pluripotent stem cells.
  • Induced pluripotent stem cells, commonly known as iPS cells, are derived from an adult stem cell that behaves like an embryonic stem cell. This new technique may help researchers avoid the controversy that comes with embryonic stem cells and prevent immune system rejection of the new stem cells.
  • Mesenchymal stem cells are adult stem cells found in tissues such as bone marrow, blood and the fallopian tube. They can become bone, cartilage, fat and possibly other cells.

How are stem cells being used to treat diseases?

Stem cell transplants, also known as bone marrow transplants, have been performed in the United States since the late 1960s. These transplants use adult stem cells.
Thanks to new technology, researchers are exploring the use of stem cells to treat a range of conditions. For example, teams at Mayo Clinic are investigating the use of adult stem cells to delay or eliminate the need for some hip replacements.
Adult stem cells are being tested to treat degenerative diseases such as heart failure. Stem cells from umbilical cord blood have been successfully used in clinical trials to treat cancer and blood-related diseases.

What does the future hold for stem cell therapy?

The use of adult stem cells continues to be refined and improved. Researchers are discovering that these cells may be more versatile than originally thought, which means they may be able to treat a wider variety of diseases, such as diabetes; heart, liver and lung diseases; neurological and bone disorders; hand, face and other injuries; and congenital anomalies.

Watch the video below for more information:

Wall Street Journal Article Discusses Umbilical Cord Blood Therapies

Posted on February 3rd, 2014 by Jennifer Schutz

From Mayo Clinic’s HLHS News and Views Blog.

HLHSA recent article in the Wall Street Journal discusses the therapeutic potential of umbilical cord blood stem cells, including the current clinical trial being conducted at Mayo Clinic using cord blood for Hypoplastic Left Heart Syndrome. The journal interviewed Tim Nelson, M.D., Ph.D, director of the Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome.

"Umbilical cord blood is the most common type of stem cells available and is commonly discarded," says Dr. Nelson. "It is important to systematically work with regulatory agencies to validate any new application. We hope that our focused efforts will indeed lead to new applications for children with congenital heart disease."

Read the full article here.

Mayo Clinic's Week at #WSCS13 in San Diego

Mayo Clinic’s Week at #WSCS13 in San Diego

Posted on January 22nd, 2014 by Jason Pratt

WSCS13 sunset banner

Mayo Clinic Discovery Leads to FDA Approval for Stem Cell Trial for Heart Failure Patients

Posted on January 21st, 2014 by Center for Regenerative Medicine

Cardio3 BioSciences, an international Mayo Clinic collaborator, has received FDA approval for a phase III pivotal clinical trial of its stem cell therapy. The trial will test the Mayo Clinic discovery of cardiopoietic (cardiogenically-instructed) stem cells designed to improve heart health in people suffering from heart failure. The multisite U.S. trial, called CHART-2, will aim to recruit 240 patients with chronic advanced symptomatic heart failure. Cardio3 BioSciences is a biotechnology company in Mont-Saint-Guibert, Belgium.

"Regenerative medicine is poised to transform the way we treat patients," says Andre Terzic, M.D., Ph.D., director of the Mayo Clinic Center for Regenerative Medicine. Watch the video below to see how stem cells are being used to treat people with heart failure.

Gene Therapy and Regenerative Medicine Lend Hope to Diabetes Treatment

Posted on January 10th, 2014 by Center for Regenerative Medicine

From Mayo Clinic's Discovery's Edge magazine.

Nearly everyone knows someone with diabetes — it's hard not to. In the United States, 1 in 3 adults and 1 in 6 children have high blood sugar, according to the National Institutes of Health.

After you eat, glucose is absorbed into your bloodstream and carried throughout your body. Insulin — a hormone made by beta cells in your pancreas — then signals your cells to take up glucose, helping your body turn the food into energy.

With diabetes, this process can go wrong in two basic ways: Type 1 diabetes results from the body's failure to produce insulin; type 2 diabetes occurs when there's plenty of insulin but the cells lose their ability to perceive its signal. In both cases, cells starve.

Living well with diabetes requires a lifelong commitment to monitoring blood sugar, eating properly, exercising regularly and maintaining a healthy weight. People with type 1 diabetes must also rely on insulin replacement therapy, usually through insulin injections. People with type 2 diabetes might need oral medication.

Still, every year, diabetes kills about 70,000 people in the United States and is a contributing cause in another 160,000 deaths each year, according to the Centers for Disease Control and Prevention.

Yasuhiro Ikeda, D.V.M., Ph.D., a molecular biologist at Mayo Clinic in Rochester, Minn., wants to change that.

After beginning his career as a veterinary feline specialist, Dr. Ikeda had to change course when he developed an allergy to his four-legged patients that made it impossible to be in a room with them. He turned his attention toward research and discovered that his interest in molecular virology had human as well as feline applications.

The story of one Mayo Clinic patient with diabetes turned Dr. Ikeda's attention back toward healing.

Photograph of Mayo Clinic stem cell researcher Yasuhiro Ikeda, D.V.M., Ph.D.

Yasuhiro Ikeda, D.V.M., Ph.D., is spearheading stem cell research in the Mayo Clinic Center for Regenerative Medicine.

"He had to have his legs amputated, then went blind, then developed kidney disease," Dr. Ikeda recalls. "He couldn't walk, couldn't see, and one day he decided not to come in. He refused treatment and died a few days later. That story stays in my mind. That's why I'm here now, because this new technology may really change things."

And that's why Dr. Ikeda and his research team are screening hundreds of thousands of compounds to discover potential drugs to treat type 2 diabetes — drugs that don't lead to crashing blood sugar levels.

Dr. Ikeda — known as Ike to friends and colleagues — is also working toward developing a cure for type 1 diabetes. He plans to use gene therapy to prevent pancreatic beta cell loss in patients newly diagnosed with type 1 diabetes.

"We are almost there," he says. Read the rest of this entry »

Stem Cells in Space: Testing Stroke Treatment

Posted on December 23rd, 2013 by Center for Regenerative Medicine

Abba Zubair, M.D., Ph.D, believes that cells grown in the International Space Station (ISS) could help patients recover from a stroke, and that it may even be possible to generate human tissues and organs in space. Dr. Zubair says, “On Earth, we face many challenges in trying to grow enough stem cells to treat patients. It takes a month to generate enough cells for a few patients. A clinical-grade laboratory in space could provide the answer we all have been seeking for regenerative medicine.”

Now, the Center for the Advancement of Science in Space (CASIS), a nonprofit organization that promotes research aboard the ISS, has awarded Dr. Zubair a $300,000 grant to send human stem cells into space to see if they grow more rapidly than stem cells grown on Earth.

Dr. Zubair, medical and scientific director of the cell therapy laboratory at Mayo Clinic in Florida, says the experiment will be the first one Mayo Clinic has conducted in space and the first to use these human stem cells, which are found in bone marrow.

To learn more, watch the video below.

Read the rest of this entry »

Enhancing Stem Cell Delivery to the Heart

Posted on December 16th, 2013 by Center for Regenerative Medicine

Mayo Clinic researchers and colleagues in Belgium have developed a specialized catheter for transplanting stem cells into the beating heart. This new catheter is being used in the European CHART-1 clinical trials, now underway. This is the first Phase III trial to regenerate hearts of patients who have suffered heart attack damage. The novel device includes a curved needle and graded openings along the needle shaft, allowing for increased distribution of cells. The result is maximized retention of stem cells to repair the heart. The findings appear in the journal Circulation: Cardiovascular Interventions.

Researchers from the Mayo Clinic Center for Regenerative Medicine in Rochester and Cardio3 Biosciences in Mont-Saint-Guibert, Belgium, collaborated to develop the device, beginning with computer modeling in Belgium. Once refined, the computer-based models were tested in North America for safety and retention efficiency.

To learn more, watch the video below. Read the full news release here.

Read the rest of this entry »

Printing Human Tissue

Posted on December 5th, 2013 by Center for Regenerative Medicine

Imagine lying in a hospital room after a heart attack. The doctor comes in with a small device that looks like a tiny apple corer and says, “You need a valve replacement. I just need a bit of your skin, and we’ll have a new one printed for you in no time.”

The lab of Mayo Clinic’s Robert Simari, M.D., recently acquired one of the very few bioprinters in the nation, a device that lets us imagine such a scenario. Wednesday, Dr. Simari told an audience at the World Stem Cell Summit that his lab is working toward such a goal. Dr. Simari is the director of Mayo Clinic’s Center for Regenerative Medicine’s Valvular and Vascular Repair and Regeneration Program.

The technology of 3D printing, which layers, or prints, material to make a three-dimensional shape, has been around for decades, but until recently, it has been confined to the mechanical world. Today, scientists are applying the technology to print human tissue, using as its ink living human cells. Dr. Simari referred to an article in The Economist, which stated, “It is impossible to foresee the long term impact of 3D printing... But the technology is coming and it is likely to disrupt every field it touches.”

Dr. Simari is testing if bioprinting can complement his work of applying stem cells to build biological heart valves. He is completing studies using animal valve scaffolds populated with stem cells. However, the process of populating these scaffolds with cells and waiting for them to grow is difficult and time-consuming. The bioprinter might cut down on that time by printing the valve on demand, microscopic cell layer by microscopic cell layer.

Dr. Simari hopes his work will give better options for patients with heart valves. Today, two main options exist for the 200,000 patients who need valve replacements each year, but both have drawbacks. One is a bioprosthetic valve, which fails 20 percent of the time within first six years of placement. The other is a mechanical valve, which requires patients take anticoagulant drugs for the rest of their lives. For many patients, both options carry even larger risks. For instance, neither valve can grow, so when children need a new valve, they face a series of operations as they age, replacing larger valves to keep up with their growing hearts.

Dr. Simari believes that a heart valve tailored to each patient will help such patients.

Mayo Clinic to co-host 2013 World Stem Cell Summit in San Diego

Posted on December 2nd, 2013 by Center for Regenerative Medicine

2013 WSCSMayo Clinic, California Institute for Regenerative Medicine, The Scripps Research Institute, Sanford-Burnham Medical Research Institute, and Institute for Integrated Cell-Material Sciences (iCeMS) at Kyoto University have joined Genetics Policy Institute to organize the 2013 World Stem Cell Summit — the largest and most comprehensive multi-track interdisciplinary stem cell conference aimed at uniting the global stem cell community and accelerating cures. The summit is this week — Dec. 4-6 — in San Diego, California.

Visit the 2013 World Stem Cell Summit website to learn more, and follow our blog to get the latest while we’re there!

“The summit is a great opportunity for the community of regenerative medicine to gather and share the advances of stem cell biology and regenerative medicine and surgery today,” says Andre Terzic, M.D., Ph.D., director of the Center for Regenerative Medicine at Mayo Clinic.

Regenerative Medicine in the Mayo Clinic Research Information Center

Posted on December 2nd, 2013 by Center for Regenerative Medicine



Several regenerative medicine activities at Mayo Clinic are currently displayed in the Research Information Center in Rochester, Minn.  The center features a new comprehensive video on regenerative medicine.


The Research Information Center is located in the Gonda Building, lobby level. Through interactive displays and scientific visualizations, visitors can learn how Mayo’s multidisciplinary research effort is changing the practice and delivery of medical care.


Mayo Clinic first in U.S. to test stem cell use in clinical trial for pediatric congenital heart disease

Posted on November 25th, 2013 by Center for Regenerative Medicine

Mayo Clinic recently announced the first stem cell-based clinical trial for congenital heart disease. The clinical trial aims to determine how stem cells derived from autologous umbilical cord blood can help children with Hypoplastic Left Heart Syndrome, a rare defect where the left side of the heart is critically underdeveloped.

Read the full news release and watch a video describing the research: