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Fri, Jul 31 · Leave a Comment

Meet the Researcher: Abba Zubair, M.D., Ph.D., Talks Stem Cells in Space

By Center for Regenerative Medicine Center for Regenerative Medicine

Abba Zubair, M.D., Ph.D.

Abba Zubair, M.D., Ph.D.

Abba C. Zubair, M.D., Ph.D., medical and scientific director of the Cell Therapy Laboratory at Mayo Clinic in Florida will present "Growing Stem Cells in Space: Medicine’s Next Big thing?” All are welcome to the Research Information Center in Rochester, Minn. (Gonda Building, Lobby Level) Tuesday August 4, at 1:30 p.m.

Dr. Zubair will discuss the possibility of growing stem cells aboard the International Space Station, a strategy that may accelerate the growth of human tissues and organs which currently require a substantial amount of time to develop due to gravity. This experiment breaks new ground as the first of its kind to be conducted in orbit.

Join Dr. Zubair to learn about this approach and its potential role in regenerative medicine, the implications of which range far, from the study of neuroregeneration, to organ transplantation, to the treatment of stroke patients. Read more about Dr. Zubair's study: Stem Cells in Space: Testing Stroke Treatment.

The presentation is open to the public.

To learn more about the Mayo Clinic Research Information Center and to view past “Meet the Researchers,” please visit:


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Tags: Abba Zubair, Mayo Clinic, Meet the Researcher, Regenerative Medicine, Research, space, stem cells, stroke

Fri, Jul 31 · Leave a Comment

Researchers Test Bioartificial Liver Device to Treat Acute Liver Failure

By Center for Regenerative Medicine Center for Regenerative Medicine

This story first appeared on the Mayo Clinic News Network.

Approximately 30,000–40,000 people die from liver disease each year, according to theAmerican Liver Foundation. For people who experience acute liver failure, the only proven treatment has been liver transplantation. Researchers at Mayo Clinic have developed and are testing an alternative to liver transplantation called the Spheroid Reservoir Bioartificial Liver that can support healing and regeneration of the injured liver, and improve outcomes and reduce mortality rates for patients with acute liver failure --without requiring a transplant.

bioliver slideDeveloped by Scott Nyberg, M.D., Ph.D., principal investigator in theArtificial Liver and Liver Transplantation Laboratory at Mayo Clinic, and liver transplant surgeon, the device uses healthy hepatocytes, or liver cells, from pigs to do the job of a normal, healthy liver, which aids in digestion and the removal of waste and toxins from the bloodstream. Treatment with the Spheroid Reservoir Bioartificial Liver (SRBAL) has been shown to reduce the severity of liver disease and improve survival in pigs. Future clinical studies are planned to assess the SRBAL as a less-invasive, long-term treatment option to liver transplantation. Results from a study using the device in a pivotal preclinical trial were published today in the Journal of Hepatology.


“Acute liver failure claims the lives of over 30 percent of people who are diagnosed with this condition. Liver transplantation has been the go-to option for treating acute liver failure, but it also comes with many risks and isn’t always an option, due to compatibility and availability of donor livers,” says Dr. Nyberg. “A bioartificial liver device could allow physicians to treat and extend the lives of more patients, safely and cost-effectively, with fewer risks.”

The study conducted by Dr. Nyberg was designed to serve as a preclinical trial on pigs with drug-induced acute liver failure. The animals were treated using the Spheroid Reservoir Bioartificial Liver and were injected with healthy donor hepatocytes to determine if this treatment method could reverse the severity of their disease.

“This study demonstrated that animals treated using the bioartificial liver responded to the healthy hepatocytes and reached the study endpoint with less disease severity than animals that received other forms of treatment,” said Dr. Nyberg. “Although the artificial liver is not yet cleared for use on humans, these findings show promise as an effective treatment option for diseases like liver cancer and hepatitis, which is becoming an increasingly common diagnosis.”

The rights to the SRBAL have been exclusively optioned to Liver Cell Technologies for commercial development. Mayo Clinic and Dr. Nyberg have a financial interest in the product and Liver Cell Technologies.

According to the American Liver Foundation, there are more than 100 different types of liver disease that can compromise liver function and lead to chronic and life-threatening conditions such as hepatitis, non-alcoholic fatty liver disease, and liver cancer.


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Tags: Bioartificial Liver, Liver Transplantation, Mayo Clinic, Regenerative Medicine, scott nyberg, stem cells

Mon, Jul 27 · Leave a Comment

Human Cell Therapy Laboratory: Developing Drugs from the Patient, for the Patient

By Center for Regenerative Medicine Center for Regenerative Medicine

This story originally appeared on the Mayo Medical Laboratories Blog.

It’s almost a crime the outside world doesn’t know about the human clinical trials occurring at Mayo Clinic’s Human Cell Therapy Laboratory (HCTL) in Rochester. Currently, there are eleven (phase I) trials underway (or just finishing), investigating the use of cellular therapies. Developed in HCTL, these therapies are designed to treat chronic wounds—particularly patients with anal fistulas secondary to Crohn’s disease—renal stenosis, and fatal neurological diseases such as amyotrophic lateral sclerosis (ALS) and multiple-system atrophy (MSA).


Allan Dietz, Ph.D.

All of these trials are using autologous (derived from the patient’s own stem cells and delivered back to the patient) cell therapies. The stem cells originate from adipose (fat), bone marrow, or umbilical cord blood. Hence, rather than something synthetic and pharmaceutically manufactured, the stem cells act as “a drug from the patient, for the patient,” says Allan Dietz, Ph.D., biochemist and Director of HCTL. “We’re really driven by the fact that there are a lot of unmet patient needs out there, and lots of indications where [pharmaceutical] drugs have either continually failed or the whole patient need has been abandoned by the pharmaceutical industry. We need to come up with something else. So that’s the foundation we work off of, based in transfusion medicine.”

HCTL’s cancer vaccine platforms—currently focused on boosting the immune system against non-Hodgkin lymphoma and ovarian and brain cancers—are also showing promise in human trials. The vaccine process involves taking autologous monocytes from a patient’s blood and converting them into active, potent, mature dendritic cells. These cells “flip on a switch that really cranks up your immune system,” says Dr. Dietz. “We started this work on immune-based cancer therapies over fifteen years ago. I think the greatest change that’s occurred is that you can now affect the patient’s tumor by working on the patient—not the tumor. And I think it’s going to change how we treat cancer. It’s cheap, feasible, and extremely safe. It’s early, but we’re seeing very good evidence of improvement in cancer patients’ immune systems and evidence of long-term stable disease.”

The Human Cell Therapy Laboratory maximizes its efficiency by trying to identify broadly applicable platforms that have the most impact on the highest number of diseases. Another factor that amps up efficiency is its “one-stop shop” approach: The laboratory provides both the platforms and the research that goes into each one. It provides regulatory advice to physicians, helping to design the clinical trial and move things quickly through the U.S. Food and Drug Administration. It manufactures cells and, in some cases, helps monitor patient outcomes, performing some of the ancillary studies that happen during those trials.

Efficiency is of utmost importance because by the time patients enter a trial, they are in the eleventh hour of their disease or condition, having no other treatment options, which is why close, long-term collaborations between HCTL and Mayo physicians are paramount.

“We have really excellent, dedicated physicians who are not satisfied with what they can offer their patients,” says Dr. Dietz. “These are the men and women who are driven to provide something different for their patients. It makes us very efficient because we combine our expertise and develop the kind of teamwork that’s needed to move these cell therapies into the clinic as fast as possible. I have no way to prove it, but I think I have the best team at Mayo, period.

“We try and stay ahead of everybody by constantly improving on cell-based therapies. As our team collaborators move through phase I of trials, it becomes clearer to them how to modify these platforms for the next generation of trials. Then it comes back to our lab to work on those modifications to specialize a platform into something specific to those patient indications.”


Dr. Dietz likens his team’s work to providing Mayo physicians with a “toolbox.” And physicians who use these platform tools are beginning to combine them in innovative ways. In doing so, they’re bringing new options to the clinic. “We have one trial in which the measles virus—which is being used as an exciting technology here at the clinic—is being used to infect stem cells that home in on the tumor and deliver the measles virus to ovarian cancer,” says Dr. Dietz. “So you don’t get a measles virus/stem cell combination unless you have both tools in the toolbox.”

Meanwhile, HCTL continues to innovate cell matrix combinations, such as the recellularization of whole organs for transplant—with a goal of being the first to transplant a fully functional, recellularized liver in a human.

All of this begs the question: How soon will human cell therapies alter the practice of medicine as we know it?

“Cell-based therapies are already in the clinic now, with bone marrow transplant, for example. That’s still the foundation of our work,” say Dr. Dietz. “But I think we’re going to see these therapies creep into different parts of medicine at different rates, depending on how quickly we can demonstrate their usefulness. There will be places where it will be implemented relatively quickly, as with wound healing, because the cells seem to have a very unique and powerful capacity to heal wounds. I think in the next ten years, you’re going to be really stunned at some of the applications as cells find their place in therapy. I think it’s the next big phase of medicine.”

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Tags: Allan Dietz, Neurology, Oncology, Regenerative Medicine, Research, stem cells

Wed, Jul 15 · Leave a Comment

Repairing Mitochondrial Disease

By Center for Regenerative Medicine Center for Regenerative Medicine

A multidisciplinary team  has successfully  eliminated fatal mitochondrial DNA mutations in stem cells from patients with mitochondrial diseases. The study is published in the current issue of Nature as a collaboration between some of the top research institutions and Mayo Clinic's Center for Regenerative Medicine.

Mitochondrial diseases are a particular struggle for patients and their families as treatment options are limited, something made even more dire as many of those affected are children. Andre Terzic, M.D., Ph.D., Director of Mayo Clinic's Center for Regenerative Medicine, explains: "these are life threatening conditions where standard care is limited to alleviating  symptoms of disease. Our proof-of-concept study shows that functionally corrected stem cells can be generated from these patients, providing initial steps towards regenerative therapy for mitochondrial disease.”

Mitochondrial DNA defects impair the ability of patient's mitochondria, the body's cellular metabolic engine, to generate energy . Once the mitochondria begin to malfunction, energy production becomes insufficient to support normal functions, and the patient experiences a variety of debilitating symptoms particularly in tissues with high energy requirements.

Now, researchers at Oregon Health & Science University in collaboration with Salk Institute, Sanford Consortium for Regenerative Medicine, University of Oxford, Cincinnati Children’s Hospital Medical Center, and Mayo Clinic's Center for Regenerative Medicine have applied  methods to restore healthy mitochondria in cells derived from patients with mitochondrial disease. Through generation of pluripotent stem cells they were able to either select stem cells containing healthy mitochondria or to replace the dysfunctional mitochondria with healthy counterparts from a donor cell. “Restoration of mitochondrial function in stem cells from patients with mitochondrial disease is thus achievable” explains co-author Clifford Folmes, Ph.D.

This technique  restored  mitochondrial function in different forms of mitochondrial disease, including Leigh Syndrome and Mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes (MELAS). Dr. Terzic concludes: "This is a transformative moment where deadly diseases are targeted and potential solutions are offered."


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Tags: Andre Terzic, Clifford Folmes, Mayo Clinic, mitochondrial disease, Oregon Health and Science University, Regenerative Medicine

Fri, Jul 10 · Leave a Comment

New Lung Restoration Center to Bolster Lung Transplants

By Center for Regenerative Medicine Center for Regenerative Medicine

A rendering of the lung restoration center on the Mayo Clinic campus in Jacksonville, Florida.

A rendering of the lung restoration center on the Mayo Clinic campus in Jacksonville, Florida.

Mayo Clinic, in collaboration with United Therapeutics Corporation, plans to change the landscape of lung transplantation with the construction of the new Lung Restoration Center in Jacksonville, Florida. The new center will use a process called ex vivo lung perfusion (EVLP) to revitalize donated lungs, allowing doctors to utilize organs that would otherwise not be viable for transplantation.

The U.S. Department of Health and Human Services states that over 100,000 patients in the United States are waiting for an organ donation, 1,600 of whom need lungs.  The problem facing these patients is that lung tissue itself is very fragile, leaving very few of the donated organs in good enough condition for transplantation. It is the goal of the new Lung Restoration Center to remedy this issue through EVLP which takes donated lungs in marginal condition and inundates them with nutrients and oxygen, restoring them. The center also has interest in exploring the possibilities of regenerative medicine in the lung restoration process.

As part of the collaboration, the two organizations also plan to research ways in which regenerative medicine may aid in treatment of damaged tissues and organs. Mayo Clinic has already made leaps in this particular field of study and the inclusion of United Therapeutics Corporation will provide new avenues for progress.

Construction of the new center is set to be complete by late 2017 and the two organizations are considering plans to create similar centers in both Minnesota and Arizona. Organs treated at the Florida center will be made available to transplant centers across the country.

Read the full announcement on the Mayo Clinic News Network.

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Tags: Dr Gianrico Farrugia, Florida, Lung Restoration, Lung Transplant, Mayo Clinic, Mayo Clinic in Florida, Medical Research, News Release, Regenerative Medicine, Research, stem cell, Transplantation

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