• Biotherapeutics

    Science Saturday: Regenerative medicine on cusp of health care transformation

The evolution of regenerative medicine has reached a milestone, moving from exploratory pipelines to daily clinical care options. Mayo Clinic's Center for Regenerative Medicine, a driver of this innovation, explored the underpinnings needed to more broadly advance regenerative health care toward standardized practice. The conclusions of this study are outlined in a review published in Regenerative Medicine.

The vision of regenerative medicine is to move from treating symptoms of disease to targeting causes of disease, and from fighting disease to rebuilding health ― all while enriching health care by providing new cures. The aspirational goal is to increase health span — living a disease-free life for as long as possible.

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

"At the core of realizing an equitable regenerative medicine vision are team science research and technology development, scaled biomanufacturing and evidence-based practice implementation, collectively delivered by an educated, specialized workforce," says Andre Terzic, M.D., Ph.D., director of the Center for Regenerative Medicine and senior author of the study.

Need for new health care solutions

An aging global population is unleashing a tsunami of chronic conditions that don't always respond to conventional treatments. As an example, the Organ Procurement and Transplantation Network reported more than 118,000 patients were on its transplant waitlist in early March despite nearly 100 transplants performed daily. A new patient is added to the waitlist every nine minutes.

"The gap between organ procurement and patient demand underscores the necessity for alternatives," says Dr. Terzic. "Adoption of regenerative solutions is grounded on a value-added proposition, advancing intended outcomes beyond the extent of current management options."

Regenerative medicine options are expanding.

Satsuki Yamada, M.D., Ph.D.

"Cellular engineering and immunotherapies have opened curative perspectives in oncology for managing lymphoma or leukemia. Chondrocytes (cartilage cells) for cartilage repair and mesenchymal stem cells in graft‐versus‐host disease exemplify further progress. Cardioreparative biotherapies developed for mending failing hearts are another area of growth," says Satsuki Yamada, M.D., Ph.D., lead author of the study.

As a result, regenerative medicine is projected to account for 10% of all health care over the next decade.

"Armed with a healing toolbox, ranging from nanotherapies (small particles targeted to areas that need healing) to biopotentiated artificial organs, regenerative science is adept to augment health care systems. Regenerative technologies are practice-transformative, offering a disruptive armamentarium that targets normative organ restoration while furthering whole-person care," says Dr. Terzic.

Dramatic progress in scientific discoveries in recent decades and a deeper understanding of disease progression are triggering an increased readiness to integrate regenerative medicine into daily patient care, according to the research.

Atta Behfar, M.D., Ph.D.

"The deeper understanding of major disease causes has been a starting point for cures," says Atta Behfar, M.D., Ph.D., a co-author of the study and deputy director for Translation in the Center for Regenerative Medicine. "Science-driven responsible and regulated translation of innovative technology has enabled the launch of previously unimaginable care pathways adopted prudently for select complex diseases and serious disabilities."

A favorable environment for regenerative medicine

The regulatory environment in the U.S. is favorable for evaluating regenerative options for patient care. Notably, the 21st Century Cures Act is designed to accelerate new innovations from bench to bedside, particularly for serious conditions with limited therapeutics. The Food and Drug Administration has created the Breakthrough Therapy designation to speed development and review of new therapies and an Accelerated Approval Program that fast-tracks the approval of therapies that address unmet needs.

While the research outlines the increased readiness for regenerative medicine, much work is yet to be done. A review of the regenerative medicine landscape found more progress is needed to prepare the practice for integrating regenerative medicine across a broad range of medical specialties.

Supply chain readiness

An advanced supply chain is needed to ensure a compliant standardization and scale-up manufacturing of regenerative products and tools, such as stem cells, tissue engineering and gene editing.Accordingly, biomanufacturing facilities that meet strict quality control measures are designed to meet the demand of a maturing regenerative care practice.

Delivery readiness

Validated regenerative medicine products and procedures that address unmet patient needs are at the core of the regenerative medicine model of care. New regenerative medicine service lines delivered to the patient must demonstrate safety, effectiveness and long-term benefit.

"A compliant regenerative medicine-augmented model of care would entail a standardized delivery of clinical-grade biotherapies supported by supply capabilities that integrate comprehensive sourcing and manufacturing with scaled patient delivery," says Dr. Terzic.

Education readiness

A workforce specially trained in regenerative sciences is needed to deliver the newest innovations in regenerative care. New online learning platforms are making it possible to share regenerative science knowledge and evidence-based practices with learners. That opens a much-needed opportunity to engage a broader community of students, scientists, physicians and allied health care providers with regenerative medicine know-how.

Future of regenerative medicine

Regenerative medicine therapies don't work the same for everyone. Early adopters envision new tools that will help identify patients who are most likely to respond to targeted regenerative medicine procedures and therapies.

"Finding a means for matching regenerative treatments to the right patient would be the holy grail of modern health care," says Dr. Terzic.

The equitable vision of regenerative medicine must be built on strategies that offer broad and diverse access, including in underserved communities often deprived of innovation advances. The research found it is imperative to ensure that regenerative medicine is equitable and accessible for all.

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