• Research

    Aspiring to deliver new cures for complex conditions

Investigating cell therapies

Mayo Clinic is building toward a future when biologics can cure cancer, kidney disease and diabetes. Mayo marks 2022 as a year of significant strides in accelerating science to make and deliver regenerative biotherapeutics. The goal is to offer new options, based on rigorous research in cell and gene therapies, for disorders with few available treatments.

The Mayo Clinic Center for Regenerative Biotherapeutics has a new strategy of advancing regenerative discoveries toward early-stage clinical trials and industry collaborations.

Julie Allickson, Ph.D.

"Pharmaceuticals at the drugstore often only address the symptoms. With biotherapeutics, we are looking for cures," says Julie Allickson, Ph.D., the Michael S. and Mary Sue Shannon Family Director of Mayo Clinic's Center for Regenerative Biotherapeutics. "We are building an advanced ecosystem of expert physicians, scientists, industry collaborators and advanced facilities to lead the way in providing a new class of medicines, especially for unmet clinical needs."

Dr. Allickson is also the Otto Bremer Trust Director, Biomanufacturing and Product Development, Center for Regenerative Biotherapeutics.

Regenerative biotherapeutics are aimed at restoring damaged tissues, cells and organs that are at the root of most diseases. Mayo's strategy emphasizes therapeutics known as biologics that are derived from human sources, such as blood, tissue, cells, enzymes, genes or genetically engineered cells. Biologics show potential for more targeted healing with fewer side effects than standard pharmaceuticals.

Becoming a biomanufacturing hub

In August, Center for Regenerative Biotherapeutics announced a strategic collaboration with National Resilience Inc. to establish a biomanufacturing hub for regenerative technologies in Rochester, Minnesota. Their work will focus on process development and quality control to expedite discoveries from the lab to patient care.

"Working with industry and their expertise gives us the opportunity to streamline and validate the approval process and move technologies successfully toward commercialization," says Dr. Allickson.

Center for Regenerative Biotherapeutics will focus on discoveries in technologies that:

  • Provide new treatments for serious and complex illnesses.
  • Show potential for commercial viability.

Center for Regenerative Biotherapeutics and collaborators are investigating several technologies in phase 1 clinical trials using:

  • A patient's biomanufactured T cells to unleash the body's immune response against multiple myeloma tumor cells.
  • 3D facial scanning to create tissue engineered-cartilage molds for nasal restoration after cancer surgery.
  • Chimeric antigen receptor-T cells (CAR-T  cells), manufactured on-site, for B-cell malignancies.
  • A personalized vaccine to see if it triggers the body's immune system against solid, metastasized tumors.
  • Mesenchymal stem cells as a possible therapeutic against organ rejection and tissue damage.
  • Stromal vascular fraction stem cells to regenerate tissue and repair vocal cord injury.

Advancing the practice

The year 2022 is also marked by the new Mayo Clinic Regenerative Medicine Therapeutic Suites in Arizona for patients seeking enhanced care when standard procedures don't meet their needs. The suites connect clinical practice, research and lab space where on-site patient cell processing is available for immediate application to their condition.

The Arizona suites are modeled after Mayo's first Regenerative Medicine Therapeutic Suites in Jacksonville, Florida. Eight different clinical departments, including dermatology, gynecology, urology and orthopedics, collaborate to provide regenerative services for conditions such as osteoarthritis of the knee and female-pattern baldness.

Research highlights

Several peer-reviewed journals published these Mayo Clinic regenerative medicine findings:

  • A Mayo Clinic study of real-world evidence published in the Orthopaedic Journal of Sports Medicine suggests adding bone marrow aspirate concentrate at the time of shoulder surgery could assist healing and prevent the need for revision surgery. Bone marrow aspirate is fluid taken from bone marrow that contains concentrated growth factors, stem cells and other specialized cells that may regenerate tissue and cartilage.
  • A preclinical study published in NPJ Regenerative Medicine suggests a substance known as purified exosome product regenerated skeletal muscles and eased symptoms of stress urinary incontinence. Exosomes act like a delivery service by moving cargo from one cell to another with instructions for targeting exact tissues that need repair.
  • A proof-of-concept, preclinical study published in Scientific Reports suggests heart muscle changes detected from blood samples can reveal who may be susceptible to heart disease later in life.

Training the workforce of the future

A well-trained workforce is needed to integrate validated regenerative technologies into patient care. This year, Mayo Clinic approved the first master's degree in Regenerative Sciences in Medicine within the Mayo Clinic Graduate School of Biomedical Sciences. The curriculum covers a variety of topics, including regenerative technologies, bioethics, biomanufacturing, regulatory considerations and clinical trials.

Also in 2022, Mayo welcomed the second class of students into its Regenerative Sciences Ph.D. track, one of the first doctoral programs of its kind in the nation.

Looking ahead to 2023

A new Skoll Foundation grant will fund research in inflammation with a focus on gastroenterology, rheumatology and dermatology. Research teams will work with partners in the practice to investigate the immunology of cells and autoimmune and inflammatory conditions

The Mayo Clinic Symposium on Regenerative Medicine and Surgery 23 will be held April 3–5 in Scottsdale, Arizona. Several nationally- and internationally-renowned speakers will share the latest regenerative medicine knowledge, including Shinya Yamanaka, M.D., Ph.D., the Nobel laureate who discovered how to reprogram human cells, and Peter Marks, M.D., Ph.D., director of the Center for Biologics Evaluation and Research at the Food and Drug Administration.

The new year is shaping up to be equally as exciting as 2022, with potential for new milestones in advancing new biologically based medicines that address the unmet needs of patients.

###

Note that this article is meant to be an overview, but not a comprehensive list, of regenerative biotherapeutics progress in 2022.

Related Articles