THE STATE OF REGENERATIVE MEDICINE

By Ed FieldTHE STATE OF REGENERATIVE MEDICINENorth Carolina has the resources, but needs a plan to advance. Ed Field is president and CEO of Aldagen, a clinical stage, regenerative medicine company in Durham, NC JASON VARNEY | VARNEYPHOTO.COM One of the most promising forms of emerging therapy is the use of a person's own or a donor's cells to treat a variety of diseases. This approach, often called cell therapy and more recently regenerative medicine, attracts a great deal of attention because the therapeutic use of stem cells falls into this category. Many US states and other countries developed strategic plans to attract the top researchers, clinicians, and companies competing in this space. North Carolina does not have a specific plan to support this effort, despite already having many of the key elements that other regions covet. This article outlines action items that might keep North Carolina in a leading position in this emerging therapeutic field. The idea of using cells as therapeutic agents is not new. Stems cells have been infused into cancer patients for nearly 40 years to reconstitute the blood and immune systems of patients undergoing bone-marrow transplants. More recently - with the discovery of different types of stems cells in several tissue sources, including bone marrow, cord blood, and adipose tissue - researchers began exploring the use of these cells for replacing or repairing damaged tissue. For instance, clinicians have injected stem cells into the hearts of heart-attack patients, hoping to reverse the ischemic damage. Are initiatives in place to attract leading researchers and clinicians in this field? It is precisely the notion of repairing or regenerating damaged tissue that excites so many people. The potential applications for cell therapy include heart disease, peripheral vascular disease, stroke, diabetes, Parkinson's and Alzheimer's disease, and others. These diseases affect millions of US patients, and billions of dollars are spent treating these diseases. Fortunately, progress toward cell therapies continues, with clinical trials being performed in several of these disease areas.GOLD RUSH?Due to the vast promise of regenerative medicine, many states and countries identified it for strategic investments with the goal of attracting leading researchers and clinicians who might ultimately discover new technologies and therapies that will spawn new companies and create new jobs. At least 20 states - including Maryland, Connecticut, and California - have or are considering programs. The support ranges from research grants to academic researchers, grants to support clinical trials, and incentives to build facilities and attract federal funding and venture investment.California has the most ambitious initiative. Proposition 71 provides $3 billion to be invested in regenerative medicine over the next 10 years in California through an organization called the California Institute of Regenerative Medicine (CIRM). This institute expects that this funding will result in human clinical studies for cell therapies for three to five diseases. The funding is earmarked for several key areas including basic research, disease-directed research, development of tools and technology, and new facilities.NORTH CAROLINA'S POSITIONNorth Carolina is uniquely positioned to be a leader in regenerative medicine despite lacking a formal initiative. North Carolina's distinctive set of elements that make it ideally positioned includes: world-class researchers and clinicians, leading clinical-stage regenerative-medicine companies, and active venture investors in cell therapy.This state hosts a variety of researchers in regenerative medicine. For example, Tony Atala, director of the Institute for Regenerative Medicine in Winston-Salem, successfully grew artificial organs. Joanne Kurtzberg, director of the Pediatric Bone Marrow Transplant Program at Duke University, has performed more cord-blood transplants than any other clinician. Recently, she showed that cord-blood transplants are promising therapies for various genetic disorders. Regenerative medicine could also boost cardiovascular therapies, which are being explored at Eastern Carolina University, Duke University, and the University of North Carolina at Chapel Hill.North Carolina also has companies that work on clinical aspects of regenerative medicine. The list includes my company, Aldagen, and Argos Therapeutics, both of which have cell therapies in clinical studies for various diseases, such as chronic heart disease, critical limb ischemia, HIV, and cancer. In addition, Tengion in Winston-Salem has an artificial bladder in clinical trials. These companies also built a base of employees who understand the regulatory, clinical, and commercial complexities of developing cell therapies.Pushing ahead cell therapy also depends on financing. North Carolina is home to several venture funds, including Aurora Funds and Intersouth Partners, which have invested in cell-therapy companies. These two firms have more than a billion dollars in committed capital. This funding expertise is crucial because many investors shy away from cell-therapy investments.TO CELL OR NOT TO CELLIn January 2004, North Carolina developed a plan to build on its leading position in biotechnology. This plan included providing support for building new companies, growing existing companies, attracting companies from out of the state, and training a workforce to meet the needs of the biotechnology economy. While these pillars can support an overall biotechnology sector, it is imperative that each initiative is reviewed to be sure it also enhances key areas, such as regenerative medicine, where North Carolina is or can be a leader.For instance, are initiatives in place to attract leading researchers and clinicians in this field? Do biomanufacturing training programs include courses related to cell manufacturing and the unique set of regulatory and logistical challenges associated with this effort? Are there incentives to build the unique clean space required to perform cell research and manufacturing? And finally, can North Carolina continue to keep or attract regenerative-medicine companies?For North Carolina, thoughtful actions to address these questions would not only be worthwhile for economic development but could also benefit its citizens. Unfortunately, North Carolina ranks high in disease prevalence in the areas where regenerative medicine holds its greatest promise, such as in treating cardiovascular disease, diabetes, and stroke. With continued support and attention, North Carolina could build on its unique position and become a leader in regenerative medicine. Many states are trying to attain such a position with far less to build on.

THE STATE OF REGENERATIVE MEDICINE

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