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Mayo Clinic Medical Science Blog – an eclectic collection of research- and research education-related stories: feature stories, mini news bites, learning opportunities, profiles and more from Mayo Clinic.

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Nov 28, 2018 · Stem cells and chronic kidney disease

Millions of Americans have chronic kidney disease. Hundreds of thousands will progress to end stage kidney disease requiring either dialysis or kidney transplant. But research is underway to keep people from reaching that point.

“Our goal is to take a look at how we can repair the diabetic kidney in terms of delaying the rate of progression of kidney failure,” says LaTonya Hickson, M.D., a Mayo Clinic nephrologist.

Dr. Hickson is part of the research team looking at using stem cells to help regenerate failing kidneys.

“We take these cells from our abdominal fat and we can inject them back into the body for them to do good,” says Dr. Hickson. “They basically tell the kidney or other organ systems that are impaired to wake up and get back to work and help heal that organ system.”

While there’s a lot more research ahead, Dr. Hickson is excited about the possibilities.

Listen to the Mayo Clinic Radio Health Minute and learn more about stem cells and chronic kidney disease in the video below:

Nov 19, 2018 · Regenerative medicine is the future for Dr. Amy Lightner

Dr. Lightner was recently honored with the Sherman Emerging Leader Prize for making impressive contributions early in her career and demonstrating strong potential to make an even greater impact in the future. (Photo courtesy of Dark Spark Media.)

Volunteering for a reading program at a children’s hospital led to a career change for Amy Lightner, M.D. As a Stanford University undergraduate, she met a cardiac surgeon who turned her interest to medicine.

“I got to see how medicine and surgical intervention could change a child’s life forever,” says Dr. Lightner. “The research, in parallel, changed lives of thousands, and this combination of clinical practice and translational research was inspiring.”

Today Dr. Lightner is a Mayo Clinic colorectal surgeon, practicing minimally invasive surgical approaches and researching regenerative cellular therapies for some of the most difficult complications of inflammatory bowel disease (IBD). She is recognized as an emerging leader for her achievements in improving surgical outcomes for patients.

Dr. Lightner completed her general surgery residency and two years of full-time research studying stem cell biology in a liver regeneration and immunology laboratory. While she found liver transplantation fascinating, it was while she was finishing her clinical training she turned her focus to IBD and a career in colorectal surgery. IBD is an umbrella term used to describe disorders that involve chronic inflammation of the digestive tract, including ulcerative colitis and Crohn’s disease.

“I would see young patients suffering from chronic bowl disease coming to terms with suffering for the rest of their lives without a known cure,” says Dr. Lightner. “Surgery treated the symptoms but not the disease, and I thought we could do so much better than the current treatment options — we needed to find out how to treat the underlying disease.”

Dr. Lightner immersed herself in a Mayo Clinic fellowship for complex clinical care and stem cell research to treat perianal Crohn’s disease. It was during this time she started to see a connection to stem cells and the therapy for IBD. “This was the time that regenerative medicine was taking off at Mayo and becoming an institutional priority,” says Dr. Lightner. “The timing was perfect.”

For patients with ulcerative colitis, a proctocolectomy, or removal of the colon and rectum, is often a last resort. After a proctocolectomy, a J-pouch surgery is performed to create an internal pouch, eliminating the need for a permanent ostomy bag. However, in about 10 to 15 percent of patients the pouch will fail and they will need a permanent ostomy.

Dr. Lightner offers these patients an alternative. She is among a small group of surgeons across the country that is skilled at pouch reconstruction – giving patients a chance at an ostomy-free life.

While an innovative physician who is an expert at reconstructive pouch surgery, Dr. Lightner wants to see fewer IBD surgeries. She is currently researching innovative cellular, non-surgical therapies.

“Regenerative medicine with novel cellular and acellular therapies is the future of medicine,” says Dr. Lightner. “I want to change the way we think of treating IBD to focus on repair derived from our own cells.”

After seeing initial success in treating Crohn’s fistulas with adipose or bone marrow derived mesenchymal stem cells from healthy donors, Dr. Lightner is initiating a Phase 1 trial to explore a novel regenerative acellular therapy. The trial will be the first of its kind in perianal fistula, which affects approximately 25 percent of people with Crohn’s disease.

Translation into Practice Platforms
Dr. Lightner is the enterprise medical director for the Translation into Practice Platforms (TIPPs) for the Center for Regenerative Medicine. The platform accelerates regenerative medicine clinical trials and advancements into clinical practice. Teams provide help with protocol design and writing, and support in communicating with the U.S. Food and Drug Administration.

Unique Laboratory
Dr. Lightner’s research space is best described as a regenerative medicine surgical lab. This all-encompassing lab is working on optimizing regenerative products like extracellular vesicles and engineered mesenchymal stem cells.  The work in the lab can be applied to other disease states, allowing these products to cross disciplines. The goal is to bring multiple investigators together, and find novel regenerative therapies for the treatment of multiple diseases.

Oct 16, 2018 · Regenerative medicine facilities and expertise in Florida

The Regenerative Medicine Therapeutic Suites has seen 1,000 patients since opening on Mayo Clinic’s Florida campus almost two years ago. The model, which integrated patient care with laboratory functions, is enabling the development and use of regenerative medicine products.

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.

The Regenerative Medicine Therapeutic Suites have prompted — and been used in — several research projects in the areas of orthopedics, dermatology, reconstructive surgery and gastroenterology. From the treatment of pressure wounds to stroke to Alzheimer’s disease and beyond, the suites ensure a seamless pathway to translate emerging regenerative treatments to patient care.

In the video below, Shane Shapiro, M.D., program director for the Regenerative Medicine Therapeutic Suites and assistant professor of orthopedic surgery discusses the results of his randomized controlled trial of bone marrow aspiration and concentration for knee osteoarthritis.

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This story first appeared on the Center for Regenerative Medicine blog.

Oct 9, 2018 · Diabetes 3, 2, 1

Often people with diabetes have brain changes that are hallmarks of both Alzheimer’s disease and vascular dementia. Some researchers believe that each condition fuels the damage caused by the other. That link may occur as a result of the ways that type 2 diabetes affects the ability of the brain and other body tissues to use sugar (glucose) and respond to insulin.

“To function well, the neurons in your brain need fuel. If you don’t have a good blood supply to the brain, then you don’t get enough glucose,” says Guojun Bu, Ph.D., Mayo Clinic neuroscientist and associate director of the Center for Regenerative Medicine at Mayo Clinic in Jacksonville, Florida.

The reduction of blood flow to the brain caused by damaged blood vessels may be why those with diabetes have a higher risk of Alzheimer’s disease. Diabetes and Alzheimer’s disease are connected in ways that aren’t yet fully understood. Dr. Bu and other researchers are studying how insulin resistance may affect the brain and result in Alzheimer’s disease or other types of dementia.

The connection between diabetes and Alzheimer’s was recently discussed in the Everyday Health article, Why Some Researchers Are Calling Alzheimer’s Disease a ‘Type 3 Diabetes’. “It’s really more of a research term, rather than a medical term,” explains Dr. Bu. About 20 percent of the human population carries the riskier form of the gene APOE, called the E4. It is anticipated that more than 50 percent of Alzheimer’s cases can be linked to APOE4, according to the study, which was published in Neuron.

Learn more about Dr. Bu’s research in the video below:

Regenerative Endocrinology Research at Mayo Clinic

The endocrine cells of the pancreas are responsible for maintaining blood glucose levels. Glucose-responsive, insulin-secreting cells in the islets (beta cells) are dysfunctional in both type 1 and type 2 diabetes. In type 1 diabetes, beta cells are destroyed, while in type 2 diabetes, they may not produce enough insulin.

The Islet Regeneration Program at Mayo Clinic is poised to develop novel therapies for diabetes. The islet regeneration researchers are taking multiple approaches to restore, protect and replace pancreatic islets.

Mayo Clinic researchers are also investigating gene therapy as a potential means of enhancing the body’s natural ability to regenerate beta cells.

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This story first appeared on the Center for Regenerative Medicine Blog.

More Information

Neurobiology of Alzheimer’s Disease
Islet Regeneration
Stem Cell Differentiation for Diabetes

Sep 18, 2018 · Advancing regenerative medicine practice, science and technology - 2018 symposium

Recent practice advancements, scientific discoveries, product development and manufacturing are the topics featured at the 2018 Mayo Clinic Symposium on Regenerative Medicine and Surgery. Regenerative medicine leaders from around the globe will come together Nov. 29 – Dec. 1 at the Westin Kierland Resort in Scottsdale, AZ. This year’s symposium will emphasize clinical trial breakthroughs and therapeutic interventions that address diseases and conditions such as cancer, Alzheimer’s disease, spinal-cord injuries and diabetes. Updates on product development and manufacturing, CAR-T cell therapy, and the first patients treated at the Florida Regenerative Medicine Therapeutic Suites will also be presented.

“The symposium is a forum for the regenerative medicine community to engage and learn how science driven practice advancements in regenerative medicine are increasingly embedded in daily practice,” says Andre Terzic, M.D., Ph.D., the Michael S. and Mary Sue Shannon Family Director, Center for Regenerative Medicine, and the Marriott Family Director, Comprehensive Cardiac Regenerative Medicine. “We’re looking forward to hosting the growing regenerative medicine community to share experiences in bringing regenerative medicine breakthroughs to clinical trials and practice applications addressing patient needs.”

Keynote speakers, Richard McFarland, M.D., Ph.D., chief regulatory officer at Biofab (left), and Peter Marinkovich, M.D., associate professor of Dermatology at Stanford University (right).

Hear from keynote speakers on trends in clinical application, discovery science, regulatory science, cGMP manufacturing and quality assurance, and clinical trials. Sessions planned for the three-day symposium include:

  • Regenerative Medicine Breakthroughs
  • Intervention Spotlight
  • Clinical Trials & Clinical Experience
  • Regenerative Discoveries
  • Future of Regenerative Medicine
  • Accelerating Therapies to Application
  • Enterprise Translational Capabilities
  • Interactive Training and Education sessions

Keynote Speakers

Richard McFarland, M.D., Ph.D., chief regulatory officer at Biofab, will share best practices in industry partnerships to accelerate regenerative therapies into patient care. Peter Marinkovich, M.D., associate professor of Dermatology at Stanford University, will present the progress of skin regeneration in patients with Epidermolysis Bullosa, a genetic skin disease.

“Drs. McFarland and Marinkovich are renowned in the field of regenerative medicine, and we’re excited to have them present at the symposium,” says Richard Hayden, M.D., an otolaryngologist and director of education for the Center for Regenerative Medicine. “Additionally, the CAR-T cell therapy update and interactive education sessions will be highlights of the symposium.”

The symposium is open to everyone, including clinicians, researchers, educators, students, industry and the general public. Early registration discounts are available through Oct. 15 and student discounts apply.

Learn more about the 2018 Mayo Clinic Symposium on Regenerative Medicine and Surgery on the Center for Regenerative Medicine blog.

Aug 28, 2018 · Stem cell differentiation for diabetes: Alexander Revzin, Ph.D.

Alexander Revzin, Ph.D., is a professor of biomedical engineering at Mayo Clinic and recipient of a 2018 Regenerative Medicine Minnesota Research Award. He is developing microcapsules that could be used as cell carriers during differentiation of stem cells into pancreatic islets. These same capsules may also be useful as vehicles for islet transplantation. This technology will enable better scale-up of islet production from stem cells, may result in cost reduction, and may also alleviate problems associated with immune rejection of transplanted islets.

Learn more about Dr. Revzin’s research:

Aug 16, 2018 · Investigating gene therapy strategies for obesity: Ping Chen, Ph.D.

The major challenge in treating obesity is not just losing weight, but preventing weight from coming back. With a research grant form Regenerative Medicine Minnesota, Ping (Vicky) Chen, Ph.D., Mayo Clinic research associate,  is studying a new treatment strategy using gene transfer therapy to safely target brain mechanisms that control hunger and energy output.

Dr. Chen’s previous research in mice found that gene transfer of a well-tolerated liver enzyme could revolutionize clinical treatment of obesity, making it easier for obese patients to lose weight safely and minimize later weight gain. Her research on the mechanism behind weight control will further determine if this approach might succeed in humans. Researchers expect the effects to correlate positively with reduction in ailments such as diabetes, metabolic syndrome and fatty liver disease.

Learn more about Dr. Chen’s research:

Visit the Center for Regenerative Medicine Blog for more information on regenerative medicine at Mayo Clinic.

Aug 6, 2018 · Regenerative medicine in novel fetal therapy program

A life-saving procedure improves fetal lung health and reduces post-natal complications

Specialists at Mayo Clinic are pioneering a life-saving procedure as part of a clinical trial for babies with congenital diaphragmatic hernia (CDH).

When a baby’s lungs are not adequately developed at birth, severe complications and even death can result. Waiting for the baby to be born to intervene may be too late. A new study is underway to document fetal lung growth from early intervention before birth.

CDH develops when a baby’s chest cavity is compressed and lung development is restricted because the diaphragm does not close completely and the abdominal organs push into the chest cavity. Congenital diaphragmatic hernia is a life-threatening condition affecting 1 in 2,500 births.

The fetal endoscopic tracheal occlusion (FETO) procedure inserts an ultrasound-guided fetal endoscope through the mother’s abdomen or uterus into the amniotic cavity. The tiny scope advances to the fetal mouth and trachea, and a balloon is placed and inflated to close the trachea to promote lung growth.  The balloon is removed in a similar procedure a few weeks later and before delivery. The minimally invasive procedure is documented in the June issue of Mayo Clinic Proceedings. The study includes two cases of severe CDH and documents fetal lung growth and reversal of severe pulmonary hypoplasia.

 

 

 

One of those case studies is Xavier Sorying, now a healthy 7-month old baby. Before Xavier was born, his diaphragm — the muscle that separates the abdomen from the chest — developed a hole, resulting in CDH.

A team of physicians, including Rodrigo Ruano, M.D., Ph.D., fetal surgeon, and Denise Klinkner, M.D., pediatric surgeon, performed a FETO. The resulting pressure then expanded Xavier’s lungs and encouraged the growth of essential structures related to his lung function.

“The fact that we collaborated and had such a great outcome points to what Mayo Clinic strives to do, which is to put the patient first,” says Dr. Klinkner.

You can read Xavier’s full story on Sharing Mayo Clinic.

The study was funded by a grant from Regenerative Medicine Minnesota from the State of Minnesota. Dr. Ruano is the director of Mayo Clinic’s Fetal Diagnostic and Intervention Center and the division chair of Maternal and Fetal Medicine at Mayo Clinic.

For the latest on regenerative medicine at Mayo Clinic, visit the Center for Regenerative Medicine blog.

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Read related blog post on Dr. Ruano and his program.

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