Posts (9)

5 days ago · Mayo Clinic Regenerative Medicine on the International Stage

Mayo Clinic experts are sharing their leadership and knowledge of regenerative medicine with an international audience at the World Stem Cell Summit in Miami. Every year more than 2,000 physicians, scientists, bioethicists, industry, government watchdogs and patient advocates from 44 countries convene at the World Stem Cell Summit to collaborate and focus on ways to advance emerging regenerative sciences. 

Shane Shapiro, M.D.

“This conference attracts some of the
most preeminent minds in regenerative medicine and is representative of the field’s
eco system.  It’s a chance to share our
latest research and newest applications of validated regenerative procedures,” says
Shane Shapiro, M.D., medical director of Mayo Clinic Center for Regenerative Medicine Therapeutic
at Mayo’s Jacksonville, Florida

Dr. Shapiro moderated three different
sessions at this year’s World Stem Cell Summit focusing on:

  • The latest 
    validated regenerative applications to patient care
  • Regulatory guidelines  and identifying safe and trusted procedures
  • Legal and ethical issues in regenerative

Regenerative medicine is redefining
health care with a focus on the body’s ability to heal itself.  Regenerative therapies aim to restore form
and function to diseased cells, tissues, or organs — and ultimately to the individual as a whole.  Mayo Clinic is a leader in bringing the
promise of regenerative medicine to patients everywhere through research,
clinical trials and application to patient care.

Mayo Clinic speakers share their
knowledge in the following presentations:

Carmen Terzic, M.D., Ph.D.

Carmen Terzic, M.D., Ph.D., a physiatrist and chair of Mayo Clinic
Physical Medicine and Rehabilitation leads research on directing stem cells
toward the formation of new heart tissue — known
as cardiogenesis  — to restore function lost to damaged tissue.  Dr. Terzic presented on her research in
cardiogenesis and its potential is to establish cardiovascular regenerative
medicine as the new standard of care for heart disease. She also spoke about
stem cells and exercise

Zubin Master, Ph.D.

Zubin Master, Ph.D., a bioethicist for Mayo Clinic Center for Regenerative Medicine, shared his research that reveals consumers are seeing a barrage of misinformation around stem cells and regenerative medicine.  Dr. Master presented data on the demographic groups that are most likely to be seeking regenerative medicine and how they are looking for credible information to make better decisions on their care.

Quinn Peterson, Ph.D.

Quinn Peterson, Ph.D.,  a scientist in Mayo Clinic’s Islet Regeneration Program, lectured on efforts to generate engineered islets that could be used as a therapy for type 1 diabetes. Islets are clusters of cells in the pancreas that produce hormones. Dr. Peterson’s research seeks to discover whether transplantation of engineered islets may restore the body’s ability to produce insulin and regulate blood glucose in patients with type 1 diabetes.

Wenchun Qu, M.D., Ph.D.

Wenchun Qu, M.D., Ph.D., a physiatrist and pain specialist with a special interest in regenerative medicine, leads research on mesenchymal stem cells (MSC) – adult stem cells that can produce several types of cells in the musculoskeletal system such as skeletal tissue, cartilage, bone and fat.  Dr. Qu’s lecture focused on the progress of MSC therapy trials at Mayo Clinic that seek to restore form and function.

Clinic Center for Regenerative Medicine
is a co-sponsor of this year’s
World Stem Cell Summit.


Fri, Jan 10 9:15am · Mayo Clinic Research Discovers a Molecular Switch for Repairing Central Nervous System Disorders

A molecular switch has the ability to turn on a
substance in animals that repairs neurological damage in disorders such as multiple
sclerosis (MS)
, Mayo Clinic
researchers discovered. The early research in animal models could advance an
already approved Food and Drug Administration therapy and also could lead to new
strategies for treating diseases of the central nervous system.

by Isobel
Scarisbrick, Ph.D.
, published in the Journal of Neuroscience
finds that by genetically switching off a receptor activated by blood proteins,
named Protease Activated Receptor 1 (PAR1), the body switches on regeneration
of myelin, a fatty substance that coats and protects nerves.

Isobel Scarisbrick, Ph.D.

regeneration holds tremendous potential to improve function. We showed when we
block the PAR1 receptor, neurological healing is much better and happens more
quickly. In many cases, the nervous system does have a good capacity for innate
repair,” says Dr.
Scarisbrick, principal investigator and senior author. “This sets the
stage for development of new clinically relevant myelin regeneration strategies.”

Thrombin and the Nervous System

Myelin acts like a wire
insulator that protects electrical signals sent through the nervous system.
Demyelination, or injury to the myelin, slows electrical signals between brain
cells, resulting in loss of sensory and motor function. Sometimes the damage is
permanent. Demyelination is found in disorders such as MS, Alzheimer’s disease, Huntington’s disease, schizophrenia and spinal cord injury.

Thrombin is a
protein in blood that aids in healing. However, too much thrombin triggers the
PAR1 receptor found on the surface of cells, and this blocks myelin production.
progenitor cells capable of myelin regeneration are often found at sites of
myelin injury, including demyelinating injuries in multiple sclerosis.

“These oligodendroglia fail to differentiate into mature
myelin regenerating cells for reasons that remain poorly understood,” says Dr. Scarisbrick. “Our research identifies PAR1 as a molecular switch of myelin
regeneration. In this
study, we demonstrate that blocking the function of the PAR1, also referred to
as the thrombin receptor, promotes myelin regeneration in two unique
experimental models of demyelinating disease.”

The Research

The research
focused on two mouse models. One was an acute model of myelin injury and the
other studied chronic demyelination, each modeling unique features of myelin
loss present in MS, Alzheimer’s disease and other neurological disorders.  Researchers genetically blocked PAR1 to block
the action of excess thrombin.

The research not
only discovered a new molecular switch that turns on myelin regeneration, but also
discovered a new interaction between the PAR1 receptor and a very powerful
growth system called brain derived neurotropic factor (BDNF). BDNF is like a
fertilizer for brain cells that keeps them healthy, functioning and growing.

Significantly, the
researchers found that a current Food and Drug Administration-approved drug
that inhibits the PAR1 receptor also showed ability to improve myelin production
in cells tested in the laboratory.

is important to say that we have not and are not advocating that patients take
this inhibitor at this time,”
says Dr. Scarisbrick. “We
have not used the drug in animals yet, and it is not ready to put in patients
for the purpose of myelin repair. Using cell culture systems, we are showing
that this has the potential to improve myelin regeneration.”

Additional research
is needed to verify and advance the findings toward clinical practice.

The study was made
possible by a grant from the National Multiple Sclerosis Society with support from the Mayo Clinic Rehabilitation Medicine
Research Center, the Center for Multiple Sclerosis and Autoimmune Neurology and
the Mayo Clinic Center for Regenerative Medicine.


Dec 26, 2019 · Top ways regenerative medicine is advancing the health care of tomorrow for patients today

Regenerative medicine accelerated from the bench into the practice in new ways in 2019, ushering in an era of care focused on the body’s amazing ability to heal itself. Bolstered by robust research, Mayo Clinic is at the forefront of delivering new therapies that restore form and function to diseased cells, tissues, or organs — and ultimately to the individual as a whole. 

Andre Terzic, M.D., Ph.D.

“The regenerative toolkit keeps on expanding concomitantly, and applications of regenerative medicine into practice is increasingly broadening to more conditions that benefit more patients. Across Mayo Clinic sites and specialties, from neurosurgery, neurology, otorhinolaryngology, pulmonary medicine, cardiology and cardiac surgery to cancer and musculoskeletal care, women’s health and plastic surgery, medicine, laboratory medicine and radiology, this year has seen remarkable achievements,” says Andre Terzic, M.D., Ph.D., director of Mayo Clinic Center for Regenerative Medicine. 

Redefining health care

Regenerative medicine is redefining clinical care, going beyond mitigating
disease symptoms to addressing the underlying cause. Mayo Clinic aspires to
cure, connect and transform through new regenerative therapies grounded in rigorous
science and in line with regulatory standards for quality and

Within the next decade, regenerative medicine is predicted to account
for 10% of all clinical care. As a prelude to the years to come, Dr. Terzic points
to inspiring examples that have highlighted regenerative care in 2019 at Mayo

immunotherapies are transforming cancer care

Mayo Clinic is one of a few select medical centers in the United States to provide chimeric antigen receptor (CAR) T-cell therapy  in a clinical setting.  CAR T-cell therapy unleashes the body’s immune system to go on search and destroy missions, targeting blood cancers, particularly B-cell leukemias and lymphomas.  In the past year, the number of clinical trials involving CAR T-cell therapy has doubled; there are two newly approved drug options offered for leukemia and lymphoma; and additional new therapies are expected to clear U.S. Food and Drug Administration approval in early 2020.

Neurosurgery is
offering new hope for spinal cord injury

at Mayo Clinic shows that stem cell intervention in the lumbar, or
lower back, offers hope for people paralyzed from spinal cord injury. A Mayo
Clinic case study found mesenchymal stem cells derived from a patient’s own fat
injected after standard surgery and physical and occupational therapy restored bodily
function in the first person tested. The patient, a 53-year-old man paralyzed
in a surfing accident, has shown significant gains in standing, walking and
gripping strength.

A second surgical procedure is referred to as spinal cord
bypass.  An implanted stimulator bypasses
the area of spinal injury, restoring the body’s ability to send messages to and
from the brain.  Mayo Clinic research has shown
this type of spinal cord stimulation helped a man paralyzed since 2013 regain ability
to stand and walk with assistance, regenerating information flow around the
severed lesion.

Larynx and lymph node
transplant: breakthroughs in regenerative surgery

Mayo Clinic launched a first of its kind transplant program
in its Arizona Otorhinolaryngology practice to restore function for people that
have had their larynx, or voice box, removed.  The United Network for Organ Sharing (UNOS)
has given its first approval for this procedure to Mayo Clinic. A successful
larynx transplant allows a patient to breathe through the mouth, swallow
normally and produce a human-sounding voice instead of breathing through an
opening in the neck and communicating by machine or special prosthesis.

“This formidable achievement underscores the collective
ability of Mayo Clinic to not only advance innovation but to do it in a way
that is systematic and meets the highest stringency for bringing a concept into
the practice,” says Dr. Terzic.

Concomitantly, in Plastic Surgery, lymph node transplants are showing promise as a therapy for lymphedema — a painful blockage of the lymph system that causes fluid buildup commonly seen in patients treated for breast cancer. Healthy lymph nodes are transplanted to replace the diseased counterparts, promoting regrowth of the lymph system.  

New procedures in
maternal fetal medicine enable correction before birth

Initially it was thought that regenerative medicine would
mainly address issues related to diseases of aging. However, research at Mayo
Clinic increasingly shows that regenerative medicine interventions can tap the
strong healing abilities of a young child or even be beneficial prior to birth.

Two recent examples of fetal surgeries led by a Gynecology
and Obstetrics specialist and performed at Mayo Clinic through U.S. Food &
Drug Administration-approved clinical trials include:

Fetal endoscopic trachea occlusion surgery  which seeks to correct underdeveloped lungs caused by severe congenital diaphragmatic hernia (CDH) — a condition in which internal organs push up against developing lungs, stunting growth. Without this surgery, 70% of infants born with severe CDH die. The surgeon places a balloon in the fetus’s trachea, causing the lungs to regrow and expand enough for the baby, at birth, to eventually breathe without assistance.

invasive fetoscopic repair of spina bifida
is another procedure under clinical assessment done in the second trimester
of pregnancy to repair a birth defect that occurs when the spine and spinal
cord don’t form properly.

Areas of advancements to watch in 2020

  • Regenerating organs for transplantation — Research at Mayo Clinic’s Florida campus will seek to address the shortage of donor organs by regenerating or rebuilding the health of organs previously considered not viable for transplantation.  
  • Biomanufacturing new products that promote healing — Mayo Clinic has a targeted investment in biomanufacturing of new cellular, acellular and tissue products that offer patients new options for healing. Opportunities to scale production for increased accessibility, affordability and cure rates are prioritized.
  • Educating the workforce of the future —Mayo Clinic is training the next generation of physician-scientists through regenerative medicine curricula that transcends the educational shield. Efforts are underway to develop a dedicated regenerative science track at the graduate school level and to train all medical students to become proficient in regenerative medicine.
  • Developing industry collaborations to speed new therapies to market  Mayo Clinic is forging new industry collaborations to bring to market new regenerative therapies for the benefit of patients around the world.

“It is an exciting time as regenerative medicine applications
continuously expand across disease conditions,” says Dr. Terzic.  “Regenerative medicine is driven by patients
seeking regenerative solutions. Therefore, we have a moral and societal mandate
to ensure the validity and ultimately the utility of these newest therapies.”

Mayo Clinic will continue to advance its regenerative
care  toolkit to address unmet needs of
the patient  and to advance Mayo Clinic’s
vision  to “cure, connect, and transform”
health care in 2020 and beyond.


Dr. Terzic is the Michael S. and Mary Sue Shannon Director, Mayo Clinic Center for Regenerative Medicine, and Marriott Family Professor in Cardiovascular Diseases Research.

Nov 27, 2019 · Mayo Clinic research is a step toward hope for spinal cord injuries

Early research published in Mayo Clinic Proceedings examines the first case at Mayo Clinic of stem cell therapy tested in humans for spinal cord injury. The case study found stem cell intervention, which took place after standard surgery, and physical and occupational therapy, restored some function in a patient with spinal cord injury. The report, “Celltop Clinical Trial: First Report From a Phase I Trial of Autologous Adipose-Derived Mesenchymal Stem Cells in the Treatment of Paralysis Due to Traumatic Spinal Cord Injury” is published in the Nov. 27, 2019 edition of Mayo Clinic Proceedings.

The research discusses the experience related to the first case in a phase I safety study of mesenchymal stem cell treatment for spinal cord injury. Mohamad Bydon, M.D., a Mayo Clinic neurologic surgeon and the lead author, cautions that each patient is different, so it’s too early to consider stem cell therapies as a treatment or cure for paralysis from spinal cord injury. Dr. Bydon adds that much like early trials in general, the stem cell trials are going to show variable response rates.

Mohamad Bydon, M.D.

in this case, the first subject was a superresponder, others may not respond in
the same manner. We do not yet understand all of the necessary biology needed
to achieve neurological recovery in paralyzed individuals,” says Dr.
Bydon. “One of our objectives in this study and future studies is to
better delineate who will be a responder and why patients respond differently.”

The research

The research centers on a 53-year-old man who suffered a spinal cord injury in a surfing accident that left him paralyzed below the neck. The patient had immediate improvements with standard therapy, but plateaued at six months post-injury. Researchers enrolled the patient in the study at Mayo Clinic nine months after the accident and injected the patient with stem cells 11 months after injury. After the stem cell injection, the patient significantly improved motor and sensory function.

case study focuses on feasibility, safety and dosing of stem cell therapy. The
study team derived mesenchymal stem cells from the patient’s fat cells and
injected them into the lower back in a procedure known as lumbar puncture.

Bydon; Wenchun Qu,
M.D., Ph.D.
a physical medicine and rehabilitation physician; and Allan Dietz,
a transfusion medicine physician, led the multidisciplinary research team at
Mayo Clinic.

Wenchun Qu, M.D., Ph.D.

spinal cord injury is a devastating condition for which scientists and
physicians are trying to find a cure. For the first time, we are inspiring hope
that people may receive better recovery in their function and quality of life,”
says Dr. Qu. “Mayo Clinic has been taking the lead in translating the
fruits of decades of research and treating neurological conditions, among which
have been very important clinical trials where we evaluate the safety,
feasibility and efficacy of adult stem cells for severe spinal cord injuries.”

Allan Dietz, Ph.D.

“This work both demonstrates the ability of cells to initiate repair and capitalizes on more than 10 years of work in the Immune, Progenitor and Cell Therapeutics Lab at Mayo Clinic. While there is still much to learn about the amazing ability of cells to heal tissue, this trial is an important step in advancing cell-based therapies toward clinical practice,” says Dr. Dietz.

collected cerebrospinal fluid to look for new biological markers that might
give clues to healing. Biological markers are important because they can help
identify the critical processes that lead to spinal cord injury at a cellular
level and could lead to new regenerative therapies.

study is needed to understand the effectiveness of stem cell lumbar injections
and why patients may respond differently.

there is no way to reverse the devastating life-changing effects of paralysis
from spinal cord injuries. Today, the only treatment is supportive care, such
as surgery and physical and occupational therapy.

Bydon says his early findings give hope that new regenerative therapies are on
the horizon for spinal cord injuries.

hope is that we will have novel treatments for spinal cord injuries in the coming
years that will be different from what we have today. These will be therapies that
do not rely upon supportive care, but therapies that rely on science to create
a regenerative process for the spinal cord,” says Dr. Bydon.

This research was made possible by funding from Mayo Clinic Transform the Practice Initiative and Regenerative Medicine Minnesota with support from the Mayo Clinic Center for Regenerative Medicine and the Department of Laboratory Medicine and Pathology Immune, Progenitor and Cell Therapeutics lab. The Transform the Practice Initiative aims to foster multidisciplinary teams of clinicians and researchers who align discovery and translational science, create new capacities and achieve solutions that improve the practice and address the unmet needs of patients.


Read the news release

Oct 1, 2019 · Zane's story: Fetal surgery to expand lungs

Zane Fouts with parents Trevor Fouts,
Alyse Ahern-Mittelsted

Baby Zane Fouts’ boundless curiosity starts at his feet, which
he grabs and plays with happily. The energetic boy who’s full of smiles is a
trailblazer for regenerative surgery performed in a clinical trial at Mayo Clinic even before birth.

“He’s our miracle baby,” says his mother, Alyse Ahern-Mittelsted.  “He’s a rock star.”

Ahern-Mittelsted was 20 weeks pregnant when an ultrasound showed Zane had severe congenital diaphragmatic hernia (CDH). This life-threatening condition blocks lungs from growing enough for babies to breathe on their own. Without intervention, 70% of infants born with severe CDH die. The bombshell news came less than a year after Ahern-Mittelsted unexpectedly lost a daughter at 31 weeks of gestation to a different condition — a failed placenta.

“We thought we were going to lose another baby. We were
really scared.”

CDH is a hole in the muscle separating the chest and abdomen.
That causes the spleen, stomach and bowels to push up into the chest cavity and
stunt lung growth. The result is small, underdeveloped lungs, known as pulmonary
.  It’s a rare condition that
affects 1 in 10,000 babies.

To try to save Zane’s life, Rodrigo Ruano, M.D., Ph.D., head of Maternal Fetal Medicine at Mayo Clinic, recommended fetal endoscopic trachea occlusion surgery while Zane was still in the womb.

Rodrigo Ruano, M.D., Ph.D.

“It’s a delicate procedure. We insert a 3-to-4 millimeter telescope through the mother and into the fetus. We advance a balloon into the baby’s mouth and detach it from a catheter placed inside the trachea, which is the airway of the fetus. The goal of this surgery is to regenerate and expand the lungs,” says Dr. Ruano.  “I feel so passionately about this surgery that I have dedicated my life to moving it toward standard of care treatment.”

When the balloon inside the fetus’ trachea inflates, it
fills the lungs with fluid, potentially causing the lungs to expand and grow. Because
a fetus breathes through the placenta, the balloon does not choke the baby.

Dr. Ruano is pioneering this procedure at Mayo Clinic
through U.S. Food and Drug Administration (FDA) -approved clinical trials with
support from the Mayo
Clinic Center for Regenerative Medicine
and a grant from Regenerative Medicine Minnesota.

While the surgery shows promise, it also comes with risk of preterm
labor and delivery. That meant the young couple from Cresco, Iowa, had a
decision to make about the health of their unborn child.

“We were told that without the surgery, our baby would only
have a 25% chance of ever coming home. With the surgery, the chances jumped to
75%,” says Ahern-Mittelsted. “We knew there was a chance this surgery might not
work. But, if this was going to give our son the best chance of survival, I
wasn’t going to second guess it.”

“After the doctor told us our options, I was looking for
more information (to help make a decision.) I looked online, but this procedure
is so new, there wasn’t a lot about it. I had to put my faith and trust in our
surgeon,” says Trevor Fouts, Zane’s father.

Surgery to place the balloon inside Baby Zane’s trachea was
performed at 27 weeks under local anesthesia and took only about 15 minutes.  

“When they were going to place the balloon, they had to move
the baby and place him in the right position. That was painful for me, but it
went fast,” she adds.

The balloon was removed at 34 weeks of pregnancy, and Zane
was born full-term at 39 weeks. How well a baby with Zane’s condition does at
birth depends on development of the lungs. Some babies whose lungs successfully
grow and develop may recover with few lingering medical issues. Others whose
lungs do not respond as well may have mild to long-term handicaps.

Immediately after birth, a breathing tube was placed in
Zane’s airway and he was connected to a ventilator. But, he was only on
machine-assisted breathing for a couple of weeks.

The balloon surgery expanded his lung capacity by about 60%.
After 52 days in the Neonatal Intensive Care Unit, he had improved enough to go

Zane Fouts

“Without this procedure he likely would not have been as
healthy as he is now.  He still has a
raspy voice and has a tough time with coughs. Eventually, we expect him to live
a normal life with normal activities.  We
think he’ll be able to participate in sports, although he may need an inhaler,”
says Ahern-Mittelsted.  “I believe this
surgery pretty much saved his life.”

Dr. Ruano has performed a total of five fetal endoscopic
trachea occlusion surgeries so far at Mayo Clinic. His research team is
compiling the data to establish whether this surgery improves chances for survival
and reduces recovery time. The long-term goal is to secure FDA approval of the
balloon used in the procedure so this surgery can be offered in daily clinical

Read about Dr. Ruano’s research on fetal endoscopic trachea occlusion surgery.

Sep 5, 2019 · Meet researcher Saad Kenderian: Using regenerative cell therapy to recognize and attack cancer

For as long as he can remember, Saad Kenderian, M.B., Ch.B., wanted to be a physician. Nothing could blunt his resolve –not even when exploding bombs and trappings of war forced the medical school in his hometown of Baghdad, Iraq, to close briefly. It is with that same determination he conducts Mayo Clinic research into chimeric antigen receptor (CAR) T-cell therapy, a regenerative therapy which unleashes the immune system to attack cancer.

Saad Kenderian, M.B., Ch.B.

“With CAR T, we are on the verge of discovering the potential of immune cells. The results that we are seeing are truly unprecedented, especially in B-cell leukemias and lymphomas. Some patients who really have no other hope are going into complete remission,” says Dr. Kenderian. “Through our research, we are discovering new CAR T products with fewer side effects. These new products may allow us to expand treatment to some blood cancers and solid tumors.”

The key hurdles to bringing CAR T-cell therapy to more patients are cost and access. It’s expensive and there are long waits for clinical trials. Dr. Kenderian’ s team is working collaboratively with Mayo Clinic Center for Regenerative Medicine,  Mayo Clinic Cancer Center  and the Mayo Clinic Department of Laboratory Medicine and Pathology’s Immune Progenitor and Cell Therapeutics Laboratory (IMPACT) lab to manufacture the cancer fighting cells in house and then ramp up production. Currently, Mayo uses commercial grade CAR T-cells engineered by an outside company.

“Making CAR T-cells in house could address issues of cost, access and innovation,” says Dr. Kenderian. “We can make the cells at a fraction of the cost, and we can increase production on our own timeline to increase availability and access to clinical trials. If an individual CAR T doesn’t work for a patient, we can go back and manufacture it according to each patient’s individual needs.”

Clinic Center for Individualized Medicine
supports research into new CAR T

Fighting cancer with
genetically engineered cells

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.

“This is a prime example of regenerative immune therapy.
Immune system T-cells are taken from each patient and engineered with an
artificial protein that supercharges them to recognize and attack cancer. A
large number of these cells are then injected back into the body. It’s a
therapy shaped to each patient,” Dr. Kenderian says.

CAR T-cell therapy may be used on lymphoma and leukemia patients whose cancer has returned twice and no longer responds to standard therapy.

Fascination with the
immune system

Part of Dr. Kenderian’s dream was to practice medicine in the United States. He never envisioned his career would put him on a circuitous path to harnessing the body’s ability to restore form and function.

 After completing his
residency at Michigan State University McLaren Hospital, he came to Mayo
for a fellowship in hematology and oncology. It was during that time
he grew fascinated with the power of the immune system and the potential that a
patient’s body could fight disease.

“Tapping the immune system is perhaps one of the only
therapeutic strategies that we can talk about as a potential cure (for cancer),”
he says.

As part of his fellowship, Dr. Kenderian studied under the
pioneers of CAR T-cell therapy at the University of Pennsylvania. He returned in
2016 to help establish the CAR T therapy program at Mayo Clinic.

Mayo Clinic is a
leader in advancing CAR T-cell therapy

Mayo Clinic, which specializes in treating rare and complex
conditions, is one of a select few medical centers in the United States to
offer CAR T-cell therapy in a clinical setting. Dr. Kenderian says research is
advancing this innovative treatment in the following ways:

  • The number of clinical trials involving CAR
    T-cell therapy has doubled in the past year.
  • There are two newly approved drug options for
    leukemia and lymphoma; a third product is expected to clear U.S. Food and Drug
    approval in early 2020.
  • Biomanufacturing of CAR T products is rapidly

Dr. Kenderian says there is still progress to be made in
increasing access and in getting insurance companies to pay. The collaborative
approach to scaling up CAR T cell manufacturing at Mayo, he believes, is an
important step toward solving those challenges.


Aug 6, 2019 · Mayo Clinic Startup Earns World Economic Forum Award

With the promise of potential lifesaving treatments like chimeric
antigen receptor (CAR) T-Cell therapy
comes complex challenges. For instance:
how can we be sure the right cells are going to the right patient? How can we communicate
problems with manufacturing that would affect a patient’s treatment schedule?

To address those concerns, Mayo Clinic startup company Vineti developed first-of-its-kind software package tracking for cell therapies. The software meticulously monitors quality control during every step of the cell’s journey from extraction to infusion. Vineti’s groundbreaking product captured a World Economic Forum Technology Pioneer Award in the field of health. Given to early- to growth-stage companies, this award recognizes new technologies and innovations poised for significant impact on business and society.

Andrew Danielsen

“Creating new products is an additional way that Mayo Clinic can improve patient care. It’s a way to amplify our staff’s talent in terms of meeting the needs of a large number of patients. A single drug, one diagnostic test or one piece of software could positively affect tens of millions of patients,” says Andrew Danielsen, chair of Mayo Clinic Ventures. “Having one of Mayo Clinic’s startups win this award is a validation of our staff’s work to bring a very innovative product to market.”

Vineti’s software reflects a new era of complex care delivered
at Mayo Clinic. Before CAR T-cell therapy, cell processing was typically done within
the treating institution. For CAR T, cells are shipped to an outside pharmaceutical
company where they are genetically modified with potential power to kill

Yi Lin, M.D., Ph.D.

“This adds a new layer of complexity to cell processing that we have not had in the past,” says Yi Lin, M.D., Ph.D., a hematologist who is the chair of the Cell Therapy Cross-Disciplinary Group at Mayo Clinic Cancer Center who collaborated on the Vineti software. “Mayo Clinic is a Center of Excellence in delivering CAR-T therapy. Our team is acutely aware of the impact of innovative software on improving patient care. We are happy to share our expertise with Vineti to enable the optimization of their software that brings together different groups to ensure safe and timely delivery of cells to the right patient.”

Mayo Clinic Cancer Center and the Department of Laboratory Medicine and Pathology  Immune, Progenitor, and Cell Therapeutics Lab (IMPACT) collaborated on the software.  Mayo Clinic Center for Regenerative Medicine provided support. Key innovators are Allan Dietz, Ph.D., Dr.Lin and Jeffrey Winters, M.D.

Allan Dietz, Ph.D.

“This award demonstrates the importance and transformative
nature of cell-based therapies for our patients. It is also a great example of
leadership role of innovation and application that the IMPACT lab and
transfusion medicine is having at Mayo and around the world,” says Dr. Dietz.

Jeffrey Winters, M.D.

“CAR T therapy is a rapidly growing treatment which is
critical for the treatment of a variety of patients who have no other options. The
Vineti software ensures that appropriate quality controls are in place for the
therapies to ensure patient safety. The award recognizes their critical role in
this area,” says Dr. Winters.

The software is also used to track cell processing for a range of other conditions, including rare genetic blood disorders. Cell therapy is being pursued in a wide range of serious disorders, including cardiac and inflammatory conditions.

Vineti began as a collaboration between Mayo Clinic and GE
Ventures. Mayo Clinic has retained an ownership stake in the company. Any revenue
generated is reinvested in Mayo Clinic research and education.


About GE Ventures
GE Ventures identifies, scales and accelerates ideas that will help make the world work better. Focused on the areas of software, advanced manufacturing, energy and health care, GE Ventures combines equity investing, new business creation, licensing and technology transfer to deliver an innovation platform designed to drive growth for partners and GE. For more information, visit, or follow on Twitter (@GE_Ventures) and LinkedIn.

About Mayo Clinic Ventures
Mayo Clinic Ventures serves Mayo Clinic by finding partners that can bring Mayo’s inventions and clinical knowledge to the marketplace to improve medicine everywhere. Mayo Clinic Ventures’ mission is straightforward: commercialize Mayo Clinic technologies for the benefit of patients worldwide, while generating revenue to support clinical practice, research and education at Mayo Clinic.

Jul 3, 2019 · Regenerative Medicine Minnesota: Executing Big Hairy Audacious Goals

excitement at the 2019 Regenerative Medicine Minnesota meeting was palpable. Health
care providers, scientists, educators, students, lawmakers and life sciences
entrepreneurs mobilized to celebrate the BHAG—big hairy audacious goals in
transforming health care. The BHAG challenge: develop new solutions and innovations
that establish Minnesota as a world class leader and destination for advancing
regenerative medicine into daily practice. Mayo Clinic hosted this year’s
Regenerative Medicine Minnesota meeting in Rochester.

Medicine Minnesota is a statewide bipartisan initiative aimed at
revolutionizing health care from a focus on treating disease to one of tapping
the body’s ability to heal itself. Regenerative Medicine Minnesota seeks to
build on the synergies of education, technology and research to create this new
landscape in health care.

Saranya Wyles, M.D., Ph.D., is one of many innovators who seized the BHAG challenge. Her “Mission to 2025” proposal is one of the nine Regenerative Medicine Minnesota education grant awards for 2019.

Saranya Wyles, M.D., Ph.D.

goal is to provide regenerative medicine education to all medical students who
train in Minnesota by 2025,” says Dr. Wyles. “We need to prepare the next-generation
physician-scientists so they can safely advance the latest regenerative innovations
into clinical practice. We also want them to have the fluency and resources to
answer questions as patients learn about and seek regenerative care. ”

Wyles’ grant will build on the Mayo Clinic Center for Regenerative Medicine
sponsorship and previous Regenerative Medicine Minnesota funding that provided
resources to establish the first patient-centered regenerative medicine medical
school curriculum. This course has trained more than 200 students to date.

“We highlight
ongoing clinical trials, pre-clinical models, bench-to-bedside translation, procedural
simulations, interactive patient experiences and basic science lectures,” says Dr.
Wyles. “The Regenerative Medicine Minnesota grant enables us to attract and
further retain medical trainees in this emerging field by providing travel
awards and research scholarships for summer internships in regenerative
sciences laboratories.”

Regenerative Medicine Minnesota annual meeting is a time for all innovators to
share their accomplishments and build strategic collaborations to accelerate
them. Regenerative Medicine Minnesota Board Co-Chairs
Andre Terzic, M.D., Ph.D., director
of Mayo Clinic Center for Regenerative Medicine
and Jakub Tolar, M.D., Ph.D., dean of
the Medical School and vice president for Clinical Affairs at the University of
Minnesota, outlined
the initiative’s major achievements. In five years of this legislative
initiative, Regenerative Medicine Minnesota has awarded 161 grants totaling
$21.9 million of dedicated investment. Grants fund education, research and
technology throughout the state.

year’s $4.35 million in awards will fund:

  • Eleven
    research grants
  • Nine
    education grants, including six grants to train k-12 students in regenerative
  • Eight
    biobusiness development awards

Andre Terzic, M.D., Ph.D.

“Minnesota’s investment in regenerative medicine has led to tangible advancements in regenerative sciences, translation of new knowledge and the rollout of clinical trials to offer patients hope of new, validated regenerative solutions to improve their health,” says Dr. Terzic.

Jakub Tolar, M.D., Ph.D.

are putting Minnesota at the forefront of introducing regenerative therapies
into clinical practice,” adds Dr. Tolar. “Regenerative Medicine
Minnesota has taken a novel approach to reaching these goals, developing a
pipeline that integrates everything from developing new therapies, to
recruiting and retaining the highly trained workforce of the future needed to
administer these therapies, to building the industry’s capacity to produce

of the research supported by Regenerative Medicine Minnesota grants at the University
of Minnesota and at Mayo Clinic include:

  • Performing fetal surgery to correct
    underdeveloped lungs before the baby is born.
  • Growing replacement blood vessels
    for coronary artery bypass.
  • Building an artificial liver to
    function until a human donor is located.
  • Developing a 4D printed system to
    guide delivery of stem cells: where, how, as what and when to deploy.

Three legislators who’ve been strong advocates,
namely — Rep. Tony Albright, R- Prior Lake, Sen.
Richard Cohen
D- St. Paul, and Senator
David Senjem
R-Rochester — appeared on a
legislative panel at the meeting where they affirmed their support and
direction of Regenerative Medicine Minnesota. With that support, the BHAG — big hairy
audacious goals — may
one day become reality in daily patient care.

Dr. Terzic is the Michael S. and Mary Sue Shannon Director, Mayo
Clinic Center for Regenerative Medicine, and Marriott Family Professor in
Cardiovascular Diseases Research.

Dr. Tolar is the Dean of the University of Minnesota Medical School and a Distinguished McKnight Professor in the Department of Pediatrics, Blood and Marrow Transplantation.

Grantees are recognized at the Regenerative Medicine Minnesota annual meeting

Watch a video overview of the 2019 Regenerative Medicine Minnesota grants


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