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【Science】2010转化医学的典范
Susan Desmond-Hellmann M.D., M.P.H.
S. Desmond-Hellmann, 2010: Awards Show What Translation Can Accomplish. Sci. Transl. Med. 2, 63ed9 (2010). http://stm.sciencemag.org/content/2/63/63ed9.full （原文见附件）
Those of us who share a passion for translational science often fear that the term “translation” could become overused or trite, thereby understating the case for increased investment and training in this area. The 2010 selection for the Nobel Prize in Physiology or Medicine as well as the three Lasker Awards brought welcome opportunities to celebrate truly groundbreaking translational research. These discoveries demonstrate why excellence in translational science matters so much.
More than baby steps. The translational science recognized by the 2010 Nobel Prize for Physiology or Medicine, awarded to Robert G. Edwards, addressed the inability to conceive a child—a profound reproductive defect that afflicts more than 10% of all couples worldwide. Dr. Edwards arduously translated his basic research on hormonal control of ovarian function, ovulation, and oocyte and embryonic maturation into the development of in vitro fertilization (IVF). After decades of this research, the first “test-tube baby” was born in 1978. Since then, ~4 million children have been delivered following IVF. In addition, this work has also had an immense impact on other medical fields, leading directly to such developments as preimplantation genetic diagnosis and stem cell research.
Metabolic malady. Douglas Coleman and Jeffrey M. Friedman received the 2010 Albert Lasker Basic Medical Research award for their work on leptin, a peptide hormone that functions in fat metabolism. These studies enhanced our understanding of the roles that genetics and brain chemistry play in obesity, which predisposes people to a variety of diseases including type 2 diabetes and hypertension. Coleman’s murine work identified a substance—leptin—in the bloodstream that restrained the appetite, and Friedman isolated the gene that encodes the protein and showed that fat cells release it. The identification and successful treatment of leptin deficiency, a rare human condition, not only changed the lives of those patients but, more fundamentally, advanced the concept that overweight people have metabolic disruptions. Given the many obesity-associated health disorders and the astonishing estimate by the U.S. Centers for Disease Control of 72.5 million obese adults in the United States alone (2007–2008), one cannot overstate the potential impact that improvements in our approaches to obesity can bring to society as a whole.
Sight for sore eyes. The 2010 Lasker-DeBakey Clinical Medical Research Award went to Napoleone Ferrara for his discovery of vascular endothelial growth factor (VEGF), a signaling protein that stimulates angiogenesis—the growth of new blood vessels. The work that began as a quest to understand the basic biology of angiogenesis then moved to the study of how perturbations of this process are involved in disease states. The link of abnormal angiogenesis to the wet form of age-related macular degeneration (AMD) spurred the development of a VEGF-blocking antibody—ranibizumab—for treatment of this debilitating disease. The findings of the ranibizumab clinical trials and subsequent work on other VEGF-blocking approaches have already enabled an estimated 1.2 million individuals in the United States alone to experience restoration of sight, which has a major beneficial impact on quality of life and helps patients to maintain their independence and avoid institutional care. I had the great honor of working with Dr. Ferrara and dozens of colleagues at Genentech and in academia to connect the seminal discovery of VEGF and its role in disease to a treatment that averted what had been an inevitable path to blindness in an aging population.
Think globally. The 2010 Lasker-Koshland Special Achievement in Medical Science Award is an inspiring example of how translational research can make an impact around the globe, not only in high-income countries but also in resource-limited settings. This award was given to David Weatherall for his multiple contributions to connecting the molecular understanding of thalassemia and related anomalies to advances in detection and clinical care. Thalassemia is a genetic disease of the blood in which a DNA defect causes the formation of aberrant hemoglobin and ultimately anemia. Dr. Weatherall’s work on thalassemia beautifully illustrates the impact of translational science in providing opportunities for scientists globally to use epidemiology, genetics, clinical care, and a commitment to training in an approach that is truly transformative.
As we celebrate the achievements of these very worthy individuals, we should recognize that translational science truly does “take a village” to achieve its ultimate success. Each member of the team can derive satisfaction in knowing that their contributions resulted in profound medical advances. But we must also have ways of acknowledging and rewarding those individuals who made the most substantive contributions to the team effort.
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作者:admin@医学,生命科学 2011-01-19 11:16
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