By Juliana L. Blum, Ph.D., co-founder and EVP, Humacyte
The future is arriving now with regenerative medicine, and it’s more amazing than we ever imagined. Current advances in true regenerative medicine have the potential to completely reshape how we think about treating, or healing, chronic disease and trauma. This creates enormous possibilities, including the opportunity to rethink the entire innovation spectrum in medicine: from product development to clinical practice.
Regenerative medicine is a rapidly advancing field where the promise is centered on restoring function and biological activity of tissue damaged by age, disease or trauma. In short, it is healing or replacing areas in the body that do not function properly.
The scope of what is becoming possible is broad – and transformative. Consider how medicine would change if we could replace tissues structures, including missing or damaged organs, with new bioengineered parts that regenerate and behave as if it they were part of the body all along. Chronic disease accounts for at least 75 percent of all U.S. healthcare costs, well over $1 trillion. In addition, trauma or injuries account for $327 billion in healthcare costs and nearly $4 trillion in life or quality of life economic losses, according to the Centers for Disease Control and Prevention.
Regenerative medicine solutions to these crushing healthcare needs have historically sounded more like imaginative science fiction than fact. But that is quickly changing. Recent scientific advancements in gene and cell therapies have opened the door to a myriad of opportunities to reprogram how the human body operates to heal itself. And we are now able to bioengineer human tissue and grow it industrially in a manufacturing facility to potentially repair, replace and reverse disease from damaged, failing or missing body parts.
But these rapid, seismic advances also highlight how industry and medicine will also require profound shifts to reap the most value from the regenerative medicine revolution. It will require interdisciplinary innovation and collaboration throughout drug development, regulation reimbursement and medical practice as we approach this new reality where innovations like bioengineered tissue and organs are science fact, not fiction.
These three imperatives will help make regenerative medicine part of everyday medicine.
A New Kind of Cross-Discipline Innovation
Off-the-shelf bioengineered products open enormous possibilities and require a cross-disciplinary approach with core expertise in biology, chemistry, physics, engineering, design, informatics, and more. Broad core teams will need ingenuity to foster innovation and the development of engineered tissues, implants and regenerative cell and gene products. For instance, when we began our company almost two decades ago, we had to find a local glassblower to help us design key elements of a prototype bioreactor to manufacture bioengineered tissue because what we needed simply did not yet exist. Future innovation in regenerative medicine will leverage ideas from a multitude of core disciplines and thinking, including accessibility to critical components and system equipment.
The future will also require diverse teams to enable fresh ways to problem solve, create, and collaborate. More than just social imperatives for diversity, scientific problem-solving and creativity will require diverse perspectives and fresh views on approaches. Regenerative medicine’s promise will only be fully realized with strong and diverse teams that can build upon existing approaches and unveil new strategies and modalities by bringing fresh perspectives to solve problems. A significant amount of research points to diversity improving innovation. We must fully harness that potential within our companies and regenerative medicine research.
Developing Capability to Manufacture Tissues / Regenerative Medicine at Scale
Manufacturing regenerative medicine products for widespread commercial use is – and will continue to be – crucial to making this an everyday part of medicine. We have seen the inherent limitations in some first-generation regenerative medicine products tailored to each individual patient. They can be cost prohibitive, and the personalized manufacturing required can be a significant barrier to widespread use. Cell therapy – and gene therapy too – continues to grapple with creating and manufacturing off-the-shelf products. And commercial-scale manufacturing is essential for bioengineered tissue products to reach a viable market opportunity.
Manufacturing process innovation will also be essential to address the emerging technologies. Solving for scalable, cost-efficient manufacturing processes is critical for the development of regenerative medicine products at commercial scale. And each successive bioengineered implant/product innovation could start with unique white space – a blank sheet of paper – requiring novel manufacturing set-ups. This will require blue-sky thinking, a form of creative brainstorming and entrepreneurial tinkering that isn’t limited by practical constraints, as there is no template or defined process for the development and manufacture of these products.
For instance, the production of our investigational, bioengineered human blood vessels required the construction of a bioreactor that created the physiologic environment of a blood vessel, including the right biochemical cues to help cells grown and mechanical cues that mimic the actions of the heart. The whole manufacturing process has a “pulse.” Defining needs for the vessels led to the decision of in-house manufacturing of the product.
The future routine integration of regenerative medicine products could also bring evolutions in logistics and supply. These include shelf life, cold chain, and supply chain. Challenges with short shelf-life and limited supply create major difficulties with widespread use, including coordinating timing of delivery of the product and surgery for the patient. Products that are stored in existing medical-grade refrigerators and freezers and are available off-the-shelf may help overcome these limitations. Changing medical practice with regenerative medicine will depend in large measure on how we innovate in manufacturing.
Acceleration of Adoption of New Science / Practices in Medicine
Regenerative medicine has the potential to offer significantly new ways of treating conditions or injuries. However, ensuring patient access will be challenging with current regulatory procedures and payment models. Creating new regulatory and reimbursement paths for regenerative medicine products could accelerate patient access to new treatments.
Regenerative Medicine Advanced Therapy (RMAT) Designation by the U.S. Food and Drug Administration (FDA) certainly streamlines an expedited path for development and review. But most of the designated programs are still in clinical trials. A true test will be during the review and approval process.
In addition, this new wave of regenerative medicine products could bring more promise of healing, as opposed to chronic treatment. Surgeons could have a manufactured, living alternative for repairing or replacing damaged or diseased blood vessels, tissue or even organs. From our own work, we know future blood vessel repair or replacement in patients could be possible with off-the-shelf vessels, which can become part of person’s own tissue, grown in a manufacturing site and implanted into a patient.
This raises the prospect of finding new reimbursement approaches that simultaneously incentivize innovation while allowing for broad use. Alternative reimbursement and financing structures such as annuity models and annuity funds to cover the inevitable patient migration to different health plans are likely only the start of such new thinking. As more regenerative medicine products approach the market, patient access may hinge on payment solutions, including the adoption of novel, new reimbursement models.
Integration into medical practice will be the next challenge, as medicine can be reticent to change longstanding and reliable, but dated, approaches. As highlighted in multiple studies, new scientific discoveries can take 17 years to be widely incorporated in medical practice. The transformation of medical care will likely require instilling new mindsets and collaborative demonstrations, training and education with physicians, underscoring the need for clinical socialization of these technologies early and often, beginning with the development stage all the way through FDA approval and commercial availability.
Moreover, to be integrated with everyday medical practice, products will also have to fit with the day-to-day inventory structures of medical practices and hospitals. And industry must provide a compelling reason to switch to new, potentially healing technologies.
But speeding the adoption of new regenerative products into medical practice is an imperative if we are to eventually lift the burden of chronic disease.
Regenerative Medicine: Challenges and Opportunities
We are living in an era that will see real progress in regenerative medicine. While the prospects of healing with cell and gene therapy or by implanting lab-grown tissue and organs into the human body may sound futuristic, these products are here already or are just around the corner. Maximizing the unprecedented potential will demand new ways of collaborating and innovating, manufacturing and evolving medical practice and its payment structures.
If we work on these the three imperatives, we can work toward ensuring that regenerative medicine is an everyday reality in medicine. Only then can we fully realize its potential to solve some of the most persistent unmet needs for patients.
Dr. Blum is the co-founder and executive vice president of Humacyte, a clinical-stage biotechnology platform company developing universally implantable bioengineered human tissue at commercial scale.