Hypoplastic Left Heart Syndrome (HLHS) is a rare disease that affects approximately 1 of every 4,300 babies every year. HLHS is a congenital heart defect in which the left side of the heart is underdeveloped. It occurs during fetal growth when the baby’s heart is developing. Without immediate intervention after birth, 95% of infants with HLHS will die within a few weeks.[i]
Karen Wanek Family Program for HLHS at Mayo Clinic is the catalyst that brings
together hospitals to address this serious health condition. Together they’ve
formed a national consortium to give patients more options when it comes to
participating in innovative clinical trials and other HLHS research. This consortium, consisting of eight members, aligns
regional centers and an advocacy group into a collaboration to accelerate
innovation and discovery sciences, as well as bring clinical trials and
expertise to patients across the country. The Ochsner Hospital for Children in New
Orleans, Louisiana, is the latest to join the HLHS Consortium.
All consortium members are participating in a phase II clinical trial using stem cells from a baby’s own umbilical cord blood in regenerative therapy. During the second of three surgeries to repair the heart, stem cells are injected into the heart muscle to help it grow stronger, with the goal to delay or prevent the need for transplant. The trial is open at all consortium sites, and Ochsner Hospital for Children already has collected cord blood with stem cells waiting to be used for a patient when the trial opens there later this year. Read more information and the full HLHS Consortium announcement on the Mayo Clinic News Network.
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.
Regenerative medicine is redefining clinical care—going
beyond disease symptom management to addressing the underlying cause of disease
to restore health. Regenerative care is expected to represent 10% of all health
care in the next decade. Yet, education in regenerative medicine lags
behind scientific and clinical advances. This threatens to leave physicians-in-training
ill-equipped to address changing needs in patient care.
In a recently
published paper, Mayo Clinic educators lay out the Mayo Clinic’s
Regenerative Medicine and Surgery course, supported in part by Regenerative Medicine Minnesota, as a
solution to bridge this recognized knowledge gap through an innovative
curriculum and transdisciplinary training. The course serves an educational
imperative to enhance the practitioners’ literacy, competency and overall
proficiency in new knowledge and specialized skillsets mandated by the rapidly
evolving regenerative era.
“This curriculum was collectively developed by the Center
for Regenerative Medicine experts in order to build the foundation for a
specialized workforce equipped with skills to distinguish safe and valid
regenerative options, as well as carry out regenerative care,” says Saranya
Wyles, M.D., Ph.D., course director. “It emphasizes the need for regenerative
medicine to be introduced early and longitudinally during medical education in
order to develop a proficient physician cohort that can advise patients to distinguish
validated and robustly regulated treatments.”
This curriculum offers a comprehensive educational experience that encompasses discovery, development and delivery of next-generation patient management, while spanning technologies across relevant medical and surgical specialties. It is offered across medical training as a first-year medical student elective, fourth-year medical student clinical elective and residency selective.
“The Regenerative Medicine and Surgery course offers a patient-centric
curriculum that involves patients previously enrolled in clinical trials, as
well as simulated patient experiences,” says Richard Hayden, M.D., director of
education for the Center for Regenerative Medicine. “This educational format is
unique to Mayo Clinic, providing an educational prototype in regenerative
Educators at the Center for Regenerative Medicine are continuously
evolving this ‘from the patient to the patient’ training platform sharing globally
the growing experience.
“Over the five-year developmental period of this course, the
curriculum increased student literacy in regenerative medicine and inspired a
sizeable percentage of participants to pursue expanded degree programs in the
area of regenerative science and associated medical applications,” says Dr.
In addition to the Regenerative Medicine and Surgery course,
offered twice yearly, the Center for Regenerative Medicine offers education
and training on regenerative medicine techniques, advances and
application for learners at all levels of expertise, from online modules
available to the general public to specialized training for current and future
medical professionals. A recent blog
post highlighted the 2019 Regenerative Medicine and Surgery course.
science has advanced next-generation technologies from the research bench to potential
clinical care options. But in the rigorous development of patient therapies, it
is critical to validate the safety and efficacy of regenerative solutions.
Shane Shapiro, M.D., medical director of the Regenerative Medicine Therapeutics Suites on Mayo Clinic’s Florida campus, encourages patients to educate themselves to make informed choices about their care. The Suites deliver regenerative therapies for a variety of musculoskeletal injuries and conditions and for dermatologic applications.
“In general, regenerative medicine is regarded with much hope, but with that has come much hype,” says Dr. Shapiro. “Treatments should always emphasize the evidenced-based standard of care predicated on best practices and validated science.”
Shapiro offers three considerations for musculoskeletal regenerative care:
1. Orthobiologics are a new regenerative
patients with torn ligaments, pulled muscles, joint pain and injured tendons
heal with standard therapies. However routine treatment with gentle pain
relievers and physical therapy is not effective for all patients with chronic injuries.
And for patients with degenerative diseases, such as arthritis, only palliative
care is available.
these cases, orthobiologics can bridge the treatment gap for patients who do
not respond to standard treatment but are not ready for joint replacement
surgery. Orthobiologics use biological agents with growth factors to activate
healing. For example, there are therapies that use platelets that then are injected
into the area of the patient’s injury.
people who need regular treatment, orthobiologics, like platelet-rich plasma spun
from a patient’s own blood, often enable people to live active lives until
there is another option for them like joint replacement,” says Dr.
A second option that is relatively new to clinical practice is bone marrow aspirate concentrate injections. Bone marrow aspirate is concentrated bone marrow that contains stem cell and many other cells that assist with healing.
2. Regenerative therapies are not a
therapies are increasingly being considered to supplement the spectrum of traditional
treatments,” says Dr. Shapiro.
Mayo Clinic patients receive any regenerative treatments, they are referred to
Medicine Consult Service, where they receive
education and are offered access to the appropriate Mayo Clinic specialist team.
3. Regenerative medicine is still
medicine is an emerging field. Physician-scientists are learning and advancing
the science every day. Mayo Clinic performs regenerative therapies that have
been studied rigorously over many years.
are 10 randomized controlled trials over 10 years to support the use of
platelet-rich plasma in arthritis to relieve pain. There are 18 randomized
controlled trials that overall provide scientific evidence supporting the use
of platelet-rich plasma in rotator cuff surgery to help with healing,”
says Dr. Shapiro
addition, Mayo Clinic is the first academic medical center to conduct a
randomized controlled trial on bone marrow aspirate concentrate.
is still much to learn, Dr. Shapiro says, and physicians are continually performing
research and applying knowledge to refine the procedures.
continue to study and refine our experience,” says Dr. Shapiro.
more about Dr. Shapiro’s research in these journal articles:
Bringing regenerative medicine to more patients requires highly skilled medical providers. During a recent weeklong educational course, 42 medical and graduate students were immersed in regenerative medicine at Mayo Clinic. The Regenerative Medicine and Surgery Selective introduces students to regenerative medicine and surgery by engaging students in the fundamental principles and cutting-edge applications of regenerative medicine within clinical practice. This knowledge will enable them to advance the use of regenerative therapies in all areas of medical practice.
“The Regenerative Medicine and Surgery Selective gives us the opportunity to provide students with a hands-on experience of regenerative medicine at Mayo Clinic,” says Saranya Wyles, M.D., Ph.D., course director. “The students participate in interactive lectures, laboratory demonstrations, clinical highlights, and patient interactions during the course.”
Learners of all levels of expertise and education came to enhance
their knowledge of regenerative medicine and to experience Mayo’s patient-centered,
team-based approach that spans the spectrum of discovery science, translational
research, and clinical application.
For Mayo Clinic
Alix School of Medicine first-year medical student, Eric Grewal,
who is studying immunology, experiencing the translation from research in the
lab to the application of regenerative medicine in a clinical setting was the highpoint
of the week.
“We’re just beginning to understand the capabilities of
immunotherapy and how a patient’s own cells can be used to heal them,” says
Grewal. “I hope to one day apply what I’ve learned during this course, and
throughout my training, to develop the next generation of immunotherapies for
Immunotherapy is just one area using regenerative medicine. The field spans all medical specialties, from prenatal surgery to cardiology to neurology, and beyond.
Maria Astudillo Potes, in Mayo Clinic’s Postbaccalaureate
Program, had a personal reason for attending the Regenerative Medicine and
Surgery Selective course. She became interested in regenerative medicine for
neurological disorders after her grandfather suffered a spinal cord injury in
injury sparked my interest in stem cells and regenerative medicine,” says Astudillo
Potes. “This course helped me to understand how the work in the lab is being
translated and applied to patient care across disciplines.”
Astudillo Potes is starting her M.D., Ph.D., training this
summer, where she hopes to study the role of stem cell therapies for spinal
Medicine Education at Mayo Clinic The Center for Regenerative Medicine offers education
and training on regenerative medicine techniques, advances and application
for learners at all levels of expertise, from online modules available to the
general public to specialized training for current and future medical professionals.
Additionally, the center disseminates knowledge about regenerative medicine
through conferences and symposiums and expands learning opportunities through
strategic education collaborations. Richard Hayden, M.D., is the director of
education for the Center for Regenerative Medicine.
The soft glow of Amy Alexander’s alarm clock in the darkness
toyed with her attempt to fall back to sleep. Her thoughts wandered to the job
she left on the printer the night before. Knowing she wouldn’t rest until she
knew it was running smoothly, Amy headed into work early.
As part of the Anatomic Modeling Lab at Mayo Clinic, Amy, a biomedical engineer, pressed print the day before on a life-sized 3D model of a 35-year-old man’s face for a surgeon preparing to repair a misaligned jaw.
The model, which took about 12 hours to complete, ran through
the night to be ready in time. It held the key to the surgeon’s preparation to
restore the man’s ability to perform seemingly simple tasks such as eating
For this particular craniofacial surgery, the surgeon planned to
take three pieces of the patient’s leg bone and attach it to the jaw — a
procedure he had done many times before. But, because he had a model of the
patient’s exact facial anatomy, he was able to rehearse the surgery and knew in
advance precisely where to make the cuts and what to expect during the
operation, making the procedure quicker and less invasive. This surgical
simulation supports better outcomes and faster recovery times.
“Medicine is very visual, and 3D models represent another way to look inside a patient, look at the disease,” says Jonathan M. Morris, M.D., a neuroradiologist and co-director of Mayo Clinic’s 3D modeling lab. “Surgeons can hold, manipulate and see a specific patient’s anatomy with a clarity that cannot be replicated in two dimensions on a computer.”
Mayo Clinic’s journey into 3D anatomical modeling began in 2006 when planning for the complex and critical separation of conjoined twins who shared a liver. The success of the surgery and the usefulness of the anatomical models spurred additional requests for 3D printing. As a result, Mayo Clinic has become a leader in anatomical modeling and 3D printing.
“It’s an educational tool we can offer to provide next-level visualization to contribute to precision surgery,” says Jane Matsumoto, M.D., radiologist and co-director of Mayo Clinic’s 3D Anatomic Modeling Lab.
Mayo Clinic uses 3D models for planning and practicing procedures in all medical specialties. Many surgeons also use the models as educational tools for current and future physicians, and as a visual to explain diagnoses to patients and families and talk through treatment options.
Physician-scientists are also building off of this foundation
in regenerative medicine to recreate living
tissue in a process called bioprinting. This experimental technology uses a
scaffold seeded with cells to mimic tissues such as skin, bone, muscles and
valves so patients might someday have a nonmechanical replacement for their
damaged tissue. For example, cardiologist Amir Lerman, M.D., Barbara Woodward Lips
Professor, is researching bioprinting heart valves to free patients from the
need for multiple surgeries or the fear of blood clots.
From tumor resections to facial reconstructions and heart surgeries, 3D models take away guesswork and facilitate communication among physicians and scientists.
“It’s a tool we can use to offer individualized care,” Dr. Matsumoto says. “The demand for 3D printing at Mayo Clinic is really a reflection of the high level of surgery our surgeons do here.”
This article originally appeared in Mayo Clinic Magazine. Read the
full story in the Fall 2018 edition.
In 2014, the Minnesota Legislature allocated resources to
fund Regenerative Medicine Minnesota
to improve the health of Minnesotans by advancing regenerative medicine
therapies. Grants are awarded to accelerate medical research and to developers
of biotechnology and biobusinesses across the state. Education grants, the
focus of this announcement, are awarded to recruit, train and retain the next
generation of health care workers and researchers in the field of regenerative
“With our colleagues in Education, the Center for
Regenerative Medicine is investing in talent and education to develop the
regenerative medicine workforce of the future,” says Andre Terzic, M.D., Ph.D.,
the Michael S. and Mary Sue Shannon Director, Mayo Clinic Center for
Regenerative Medicine. “This new funding helps us bring regenerative medicine
education across the state.”
Four Mayo Clinic teams were selected to receive 2019 Education Program Awards from Regenerative Medicine Minnesota.
Bruce Horazdovsky, Ph.D. — Education Program Award (K-12)
Women in Science and Engineering Research to promote young scientists through community engaged STEM activities in Minnesota
Project Summary: The Women in Science and Engineering Research (WiSER) group at Mayo Clinic will host a monthly series of STEM-education courses through collaborations with the Rochester Public Library and Minnesota Children’s Museum. These events will focus on engaging K-12 students in Science, Technology, Engineering, and Math (STEM) fields. The goal is for these activities to spark enthusiasm and awareness of STEM-related careers, provide strong role models as women in science, and promote a healthy relationship between scientists and the community. WiSER team members include: Amanda Huff, Crystal Mendoza, Elisabeth Shanblatt, Ph.D., Ribu Goyal and Ashley Krull, Ph.D.
Rosalie Sterner — Education Program Award (Post-Secondary)
Postdoctoral Training in Regenerative T Cell Immunotherapy and CART Cell
Chimeric antigen receptor T cell therapy (CAR-T) is a new, potentially curative pillar in the treatment of cancer that was FDA approved in 2017 for the treatment of B-cell leukemia and lymphoma malignancies. CAR-T cell therapy seeks to harness the power of the immune system by genetically modifying cells, equipping them with power to kill cancer. These synthetic cells act like a living drug that uses the body’s defense system to fight disease. The processes of engineering, generating, and administering CAR-T cells are very complex and require expertise across multiple fields of science and medicine. This grant will fund an innovative interdisciplinary educational program to train Minnesotan graduate students, medical students, residents, fellows, and post-doctoral trainee in the foundations of CAR-T cell therapy and regenerative immunotherapy and increase their interests in the field. Team members include: Saad Kenderian, M.B., Ch.B., Andre Terzic, M.D., Ph.D., Richard Hayden, M.D., Karen Hedin, Ph.D., Tammy Hughes, Andrea Oevering and Jilian Foxen.
Mark Wehde — Education Program Award (K-12)
The Mayo Clinic Career Awareness in Regenerative Medicine, Science, and Engineering Program Project Summary: The Mayo Clinic Division of Engineering, the Center for Regenerative Medicine, and the Human Resources Career Awareness team have successfully partnered for several years to create a STEM career development program aimed at informing local area high school STEM instructors of the opportunities for their students in health care. This year the focus will be additive manufacturing and tissue engineering. Among other activities, participants will have firsthand exposure to 3D printing during the day. At the end of the day they will take the 3D printers with them to their schools, thanks to the grant provided by Regenerative Medicine Minnesota. Team members include: Jilian Foxen, Jon Ninas, Kari Grapp, Alissa Cornell, Henry Walker, Brady Olson, Claire Eggleston, John Angeli and Amy Alexander.
Saranya Wyles, M.D.,
Ph.D. — Education Program Award (Post-Secondary)
Building and Retaining Next-Generation Physician Workforce in Regenerative Medicine and Surgery in Minnesota: Mission to 2025
Project Summary: Over the past six years, Dr. Wyles led an interdisciplinary student and faculty team to build a patient-centric regenerative medicine curriculum embedded into medical school and residency core learning. This program presents an innovative curriculum that addresses this recognized knowledge gap by educating next-generation learners. Support from Regenerative Medicine Minnesota allows for regenerative medicine principles to be longitudinally integrated across all Minnesota medical school curricula by 2025 with the goal of training over 1000 medical students to address unmet patient needs through regenerative sciences. This transdisciplinary training is a prototype that can promote education-driven practice globally. Team memberes include: Richard Hayden, M.D., Andre Terzic, M.D., Ph.D., Fredric Meyer, M.D., Christopher Paradise, Ph.D., Dileep Monie, Raquel Harguth and Courtney Paradise.
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.”