“Regenerative medicine seeks ways to re-grow or engineer healthy tissue without the need for transplants,” Poss said. “On a global scale, there’s a tremendous organ shortage, and transplantation is an expensive and nonpermanent solution.
“Imagine the number of lives that could be improved if, for example, we could find ways to use the body’s innate healing mechanisms to regenerate heart muscle in patients that are spiraling toward heart failure after a heart attack.”
“Imagine how many lives could be improved if we could find interventions that restore functional spinal cord tissue and reverse paralysis.”
Ruffin of ARM sees a promising future for regenerative medicine.
“We will continue to see the development of additional regenerative medicine therapies for a broad number of acute and chronic, inherited and acquired diseases and disorders,” he said. “Therapies in this area will continue to advance along the regulatory pathway, many of which are entering phase III clinical trials this year.”
“In fact, in the next two years, we are anticipating a number of U.S. and E.U. approvals in the cell and gene therapy sector, including therapies that address certain types of cancers, debilitating retinal disorders, rare genetic diseases, and autoimmune conditions. We also expect to see sustained investment, which will help fuel growth and product development within this sector.”
A number of cell and gene therapies and technology platforms are demonstrating real potential to address areas of significant unmet medical need, Ruffin said.
These include cell therapies for blood cancers and solid tumors; gene therapies for rare genetic diseases as well as chronic conditions; and gene editing for the precise targeting and modification of genetic material of a patient’s cells to cure a broad range of diseases with a single treatment.
Poss at Duke talked about the ultimate quest.
Regenerative medicine has been most successful in restoring or replacing the hematopoietic tissue that creates blood, he said.
“We still lack successful regenerative therapies for most tissues,” Poss said. “The future of regenerative medicine — the holy grail — will be stimulating the regeneration of healthy tissue in patients without adding cells or manufactured tissue.”
Working out the details of innate mechanisms of regeneration in animals like salamanders, zebra fish, and mice, can inform this approach, he said. So can improvement in factor delivery and genome editing applications to encourage the regeneration of healthy tissue.
“Ultimately,” Poss said, “regenerative medicine will change the toolbox of physicians and surgeons, with major impact on outcomes of diabetes, spinal cord injuries, neurodegenerative disease, and heart failure.”
ARM says the public does not realize how far the field has progressed in recent years.
“Currently, there are more than 20 regenerative medicine products on the market,” Ruffin said, primarily in the therapeutic areas of oncology, musculoskeletal and cardiovascular repair, and wound healing.
More than 800 clinical trials are now underway to evaluate regenerative advanced therapies in a vast array of therapeutic categories, he said.
“We’re seeing a significant focus on oncology, cardiovascular disease, and neurodegenerative diseases, with more than 60 percent of trials falling into one of these three categories,” he added. “Even though the majority of people perceive regenerative medicine as something of the future, it’s actually here and now.”