The Future of Regenerative Medicine
Janet Marchibroda, President, Alliance for Cell Therapy Now The human body itself is remarkable medicine. When we get a cut, our skin knits back together. When we break a bone, it mends. When we’re exposed to a pathogen, an army of cellular clones multiply and arm the immune system against it. The body still contains many more medical mysteries that, if harnessed, may lead to a host of new therapies. That’s the aim of the field of regenerative medicine: to uncover the body’s medical mysteries, and in doing so, use the body’s own tools to heal itself. There are thousands of ongoing clinical trials in regenerative medicine and cellular therapy around the world, including more than 800 active clinical trials in the U.S. that explore the use of these therapies for cancer, neurological conditions, musculoskeletal issues, cardiovascular disease, diabetes and even COVID-19. The Alliance for Regenerative Medicine noted that the sector raised a record $19.9 billion in funding in 2020. The interest in regenerative medicine is accelerating, and it’s becoming time to shape its future. As Dr. Peter Marks, director of the Center for Biologics Evaluation at the U.S. Food and Drug Administration (FDA), noted at the Fifth International Vatican Conference, having many contemporaneous studies on regenerative medicine approaches is key to pushing this field forward. “Doing the studies and doing them carefully and correctly and under IND (Investigational New Drug Application), is what needs to happen here,” he said. Sanford Health, Dr. Marks noted, is already doing this work. Sanford is one of only a handful of FDA-licensed health clinics
with a green light to study cutting-edge approaches to regenerative medicine, like the utilization of stem cells to address an injury. In 2017, for instance, Sanford Health and InGeneron, Inc., launched an FDA-approved clinical trial on the use of
adipose stem cells to treat rotator cuff injury. Early results from this trial have been promising, said Dr. David Pearce, the president of Innovation, Research, and World Clinic at Sanford Health. However, it’s the refinement of the clinical trial process for regenerative medicine that will have the biggest impact. “We have preliminary efficacy in some of our studies, but not the level in terms of multi- site clinical trials,” Dr. Pearce said. “And that’s what I’m looking forward to.” It’s clear that we may soon see more multi-site clinical trials for regenerative medicine-based therapies. That’s for a good reason: These studies help establish
both efficacy and safety. As Dr. Marks noted, any harm in the absence of therapeutic effect is problematic. Tiffany Facile, the clinical regenerative medicine director at Sanford Health, noted that there is some risk associated with procedures that involve an individual’s own or autologous cells, but ongoing studies should clarify what those risks may be and how to avoid them. “Studies that are currently running should reduce the safety concerns,” she continues. It’s possible we may soon see accelerated frameworks of these studies. Dr. Marks envisions a formalized pathway towards collaborative development for regenerative medicine. Manufacturers, following an approved protocol, could develop a product and participate in a coordinated multi-site clinical trial. “I think we need to think creatively like this in the field of regenerative medicine,” Dr. Marks says. “It isn’t the same as making a pill, so we’re not going to be able to export the pharmaceutical model in the same way that has been done for small molecule drugs or protein therapies.” Sanford Health and other institutions are working to anchor the study of this accelerating area of medicine in the U.S. Moving forward, we may start to see regulatory pathways change to accommodate and perhaps accelerate this burgeoning area of research.
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