Advancing the Science

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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1 day ago · 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.

Tue, Aug 28 6:00am · 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:

Thu, Aug 16 6:00am · 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.

Mon, Aug 6 6:00am · 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. CDH 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.

Thu, Jul 26 6:00am · Submit abstracts for 2018 Symposium on Regenerative Medicine and Surgery

Abstracts are now being accepted for the Mayo Clinic Symposium on Regenerative Medicine and Surgery. The symposium will be held Nov. 29-Dec. 1, 2018 at the Westin Kierland Resort & Spa in Scottsdale, Arizona.

Interested physicians, researchers, medical students, post-doctoral and pre-doctoral students, as well as allied health staff, are invited to submit abstracts for the poster session on Friday, Nov. 30. First place posters in each category will be awarded complimentary registration for the 2019 symposium.

The deadline to submit an abstract is August 31.

Priority will be given to posters that focus on:

  • Discovery, translation and application
  • Mayo Clinic’s unique capabilities in regenerative medicine
  • Transforming the practice
  • Leading innovation
  • Regulatory, FDA and manufacturing

A fee of $25 is required for each abstract submission. The abstract submission fee does not register you for the symposium and is nonrefundable. Those with accepted abstracts will be notified by September 30. If your abstract is selected, you are required to register to attend the symposium.

To submit an abstract, you must login to the Mayo Clinic School of Continuous Professional Development website. For assistance, please review the instructions for creating a user profile.

Save on conference fees by registering now. Early bird registration runs through Oct. 15.

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

Tue, Jul 17 6:00am · Researching gene therapy for liver diseases: Joseph Lillegard, M.D., Ph.D.

Joseph Lillegard, M.D., Ph.D., is a Regenerative Medicine Minnesota research grant recipient. He is researching gene therapy to fight inborn errors of metabolism of the liver, primarily Hereditary Tyrosinemia Type 1 (HT1), a metabolic disorder caused by an enzyme deficiency. HT1 is a genetic disorder that can lead to liver and kidney failure, and is fatal without treatment. There is no cure. This project aims to develop a lentivirus-mediated gene therapy to treat multiple inborn errors of liver metabolism, with the research in HT1 serving as a model for future studies of other rare liver diseases.

Dr. Lillegard is a supplemental consultant at Mayo Clinic and a pediatric general and thoracic surgeon at Children’s Minnesota.

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

 

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

Thu, Jun 21 6:00am · Cell therapy for cartilage defects: Aaron Krych, M.D.

Aaron Krych, M.D., Mayo Clinic orthopedic surgeon and recipient of a 2018 Regenerative Medicine Research Grant, is researching the initial safety and feasibility of RECLAIM, a single stage cartilage repair technique using the patient’s own cartilage cells and allogeneic mesenchymal stem cells.  The study will assess the patient’s improvement in pain and function following the procedure, while actively monitoring safety and adverse events.

Dr. Krych is a professor of orthopedics and a consultant in the Department of Orthopedic Surgery at Mayo Clinic. His clinical research interests include ACL and meniscus injuries, cartilage restoration and transplantation, hip labral tears and athletic injuries, and arthroscopic treatment of femeroacetabular impingement.

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

Thu, Jun 14 6:00am · Bridging the gap: Introducing the health care of tomorrow

Forward-thinking leadership, unparalleled expertise and a commitment to solving unmet patient needs allow Mayo Clinic to bridge the gap between the delivery of traditional health care today and the rapid advancement of revolutionary care in the near future.

Increasing Translational Capabilities

Traditionally, it could take years for physician-scientists to develop the essential expertise to create regenerative therapies and traverse the regulatory process. Today, experts across Mayo Clinic have access to resources that empower any specialty to advance new discoveries toward regenerative treatments. By seamlessly integrating manufacturing with regulatory and clinical trial competence, these translational capabilities collectively position the Center for Regenerative Medicine as an attractive partner and enabler in revolutionizing patient care. Continuing to build and enhance this work is pivotal to success.

The infrastructure required to achieve rapid translation of discoveries into transformative therapies for patients is essential to developing the future of medicine.

Together, several platforms make this translational work possible.

  • Early feasibility studies and first-in-human phase I and phase II clinical trials lay the groundwork for advancing discoveries.
  • Having a patient’s own cells readily available is critical, as they are often used as a starting point for regenerative therapies.
  • Off-the-shelf, cell-free regenerative therapies have the advantage of being immediately available, much like conventional medical therapies.
  • Manufacturing many stem cell doses at one time ensures that every patient can receive the same benefit.
  • Guidance on designing clinical trials, validating therapies and providing large-scale production of lifesaving treatments empower Mayo’s best and brightest to focus on innovation.

Artist’s rendering of building in Discovery Square.

Building Powerful Collaborations

Destination Medical Center (DMC) is a robust economic development initiative dedicated to creating a global destination for health and wellness. Within DMC, Discovery Square is being developed as a center for health care innovation where world-leading medical practitioners, educators, researchers and businesses come together as a thriving hub to develop powerful solutions.

Mayo Clinic serves as an anchor tenant in the first Discovery Square building, allowing for powerful collaboration to exist between Mayo and the world. The Center for Regenerative Medicine will use this newly dedicated space to create safe and effective products for therapeutic treatment.

Educating Future Health Care Leaders

Recognizing the importance of regenerative medicine to the future research and practice of health care, Mayo Clinic is committed to providing numerous opportunities for current health care practitioners and students to become educated in this new and evolving field – building the workforce of the future.

The first class of students in the Regenerative Sciences Training Program: Paige Arneson (biochemistry & molecular biology), Emma Goddery (neuroimmunology), and Christopher Paradise (molecular biology & experimental therapeutics).

Mayo Clinic Graduate School of Biomedical Sciences launched a new program of transdisciplinary  doctoral training in regenerative sciences. A first-of-its-kind program, this educational opportunity is designed to equip the next generation of leaders with the skills necessary for the discovery, translation and application of regenerative solutions. This advanced education is essential for students pursuing leadership in the regenerative health care space.

Creating New Patient Care Delivery Models

With the creation of revolutionary treatments, Mayo Clinic has also pioneered new ways to deliver care. Regenerative Medicine Therapeutic Suites provide a patient-focused space that integrates clinical functions and a laboratory where each patient’s own cells are processed for treatment. This could serve as a progressive point-of-care model for the future, and is currently being used while treating musculoskeletal conditions like osteoarthritis of the knee.

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

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