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Friday, July 30, 2010 |
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Article ArchiveTranslational Research: Bridging Science For Better Patient OutcomesCover Story Issue: November 2009 By: Juliet Farmer
In its most basic definition, translational science is the transformation of basic scientific research into clinical applications for patient care. Yet, anyone involved in any of the steps from laboratory to bedside knows there’s a lot more to it. Translational research brings together experts in basic science and clinical science, as well as leaders in business and industry—all with the goal of getting an idea to market quickly and efficiently. “From a research perspective, translational research is saying to the scientific community, ‘Let’s figure out how to direct your unique and exciting work to directly benefit the patient,’ and plan out research in patient terms,” he continues. “Basic scientists think about fundamental questions, and clinicians think about the patient. There was no reliable bridge between the two until translational research.” Dr. Vail knows of what he speaks, having been in the trenches researching hip and knee joint biomechanics, biomaterials, articular cartilage injury and repair, and clinical outcomes after joint replacement. Complementing that, Dr. Vail’s clinical interests include conservative joint reconstruction options for younger patients, hip and knee joint replacement, hip resurfacing, less invasive approaches, management of bone loss, and treatment of avascular necrosis. Concept to Implementation UCSF’s was the only orthopaedic team competing for $20 million in 2008 from the California Institute of Regenerative Medicine (CIRM) to create cartilage from stem cells that can be delivered to the patient (essentially using a patient’s own cells, combining them with a matrix, then transplanting them back onto the patient’s injury). The department has orthopaedic research laboratories operating at Parnassus Heights, San Francisco General Hospital, and the VA Medical Center. These facilities’ major areas of expertise and investigation include fracture repair; biomechanics and bioengineering of musculoskeletal tissues; mesenchymal stem cell biology; cartilage, bone, tendon and intervertebral disc regeneration; and molecular and cellular mechanisms of musculoskeletal development. From Petri to Product This translational research has been part of the university’s focus for years, although it’s not required of university or academic centers. It has also aided in bringing together the right team—basic scientists; clinicians; researchers; experts from the Haas School of Business, University of California Berkeley; Martin Chemers, social psychologist at University of California, Santa Cruz; and industry leaders in polymer manufacturing, tissue engineering, and cell processing—to apply for a CIRM Disease Team grant to use stem cells to repair articular cartilage. The idea behind the CIRM grant is to accelerate clinical translation of new stem cell discoveries by refining the basic scientific concept, clarifying the specific clinical application, then creating the mechanism to bring the idea/device to the marketplace and patient care arena, allowing basic science to translate into something real for patients. “This grant reaches from basic science to bringing a product to clinical trials in a four-year time frame,” explains Dr. Vail. UCSF’s goal, according to their own CIRM grant application, is “to develop a clinically successful stem cell based therapy for focal cartilage defects through the open advancement of knowledge and pursuit of creativity, research excellence, teamwork, high ethical standards, and the rapid transfer of discoveries for societal benefit and commercial relevance." According to Dr. Vail, there are approximately 20 grant proposals covering many areas of medicine, but UCSF’s is the only musculoskeletal applicant in this stage of consideration. Approximately 10 of the 20 applicants will be approved, and the grant, which is renewable each year based on last year’s progress, is to be awarded soon. “The CIRM grant process has been very beneficial for our department, and whether we are awarded it or not, we will pursue these ideas,” he notes. “We’re very hopeful that the work is funded, but if not we’re looking for other ways to carry it forward.” Roadblocks to Market One hurdle to getting a product to market is naïveté, says Professor Jeffrey Lotz, Ph.D., Director of the Orthopaedic Bioengineering Laboratory at UCSF. “Say we discover something in the laboratory, and get the scientific details worked out. We assume that a therapy evolves from that naturally. But for treatment to be clinically viable, it needs to be FDA approved and insurance company reimbursed. There’s a big gap between expertise required to do the basic and pre-clinical research and that necessary to navigate the bureaucracy that comes with developing a new therapy. There’s a different skill set required to take data and put together a program for FDA approval.” Dr. Lotz’s expertise in spine biomechanics and intervertebral disc biology, as well as his laboratory work focusing on identifying mechanisms of intervertebral disc degeneration, exploring tissue engineering approaches for treating low back pain, and the biomechanics of spinal instrumentation, has been a training ground for identifying what obstacles might appear in the road. “If you anticipate hurdles to market, you can be much more efficient in what you do, honing trials and experiments to get a product where it needs to be. It gets you to the end zone more quickly and efficiently,” he explains. On the flip side, being in a rush to get the information out can also backfire. Dr. Lotz cites an example of a new drug discovery that could have helped many. The researchers, with all the best intentions, decided to publish for the world to see without filing any patents. Once it was out in the public domain, industry wanted nothing to do with it because they couldn’t get sole ownership or rights over the end product, and consequently couldn’t justify incurring the significant costs needed for developing a new drug. Performing Partnerships In the hopes of fostering the spirit of translational research, Dr. Lotz says the National Institutes of Health (NIH) is emphasizing clinical relevance in their grant review process. NIH offers a small business initiative that calls for collaboration between university and industry, but the challenge, of course, is in how to facilitate it. At UCSF, they developed an Industry Research Center, complete with a group of technical staff, which provides infrastructure to help industry-driven projects or prototypes for product development. The Industry Research Center brings together industry sponsors, clinicians and engineers to more efficiently match new technologies to unmet clinical needs. Building these in-house, cross-platform teams has been a model for bringing clinical and research faculty together. Transparency of information and relationships also allows for an open discussion about sharing responsibilities and credit. “There are plenty of pieces of the pie for people to benefit from, and [what we’re doing] takes into consideration where people are in career trajectory and creates a climate where people can learn to get along and trust each other,” Dr. Lotz notes. Of course, the goal of any new treatment is to be safe and better than current gold standard, so it’s important to understand early on how to focus one’s efforts. |
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