National heart, lung, and blood institute and the translation of cardiovascular discoveries into therapeutic approaches

Circulation Research

Volumn 112, Issue 9, 2013, Pages 1212-1218

  Galis, Zorina S ^a   Black, Jodi B ^b   Skarlatos, Sonia I ^a  

^a NATIONAL HEART LUNG AND BLOOD INSTITUTE   (United States)

^b Review Branch   (United States)

Author keywords

cardiovascular; National Heart, Lung, and Blood Institute; National Institutes of Health; programs resources; translational

Indexed keywords

ALPHA ANTITRYPSIN DEFICIENCY; ARTICLE; CARDIOVASCULAR DISEASE; CARDIOVASCULAR SYSTEM; COMMERCIAL PHENOMENA; CYSTIC FIBROSIS; DRUG INDUSTRY; DUCHENNE MUSCULAR DYSTROPHY; FABRY DISEASE; FOOD AND DRUG ADMINISTRATION; GENE THERAPY; GLYCOGEN STORAGE DISEASE TYPE 2; HEALTH CARE COST; HUMAN; INVESTMENT; MEDICAL RESEARCH; NATIONAL HEALTH ORGANIZATION; ORGANIZATION AND MANAGEMENT; PNEUMOCYSTIS PNEUMONIA; PRIORITY JOURNAL; PRODUCT DEVELOPMENT; PUBLIC HEALTH; PUBLIC-PRIVATE PARTNERSHIP; RISK; TRANSLATIONAL RESEARCH; UNITED STATES; WISKOTT ALDRICH SYNDROME;

ACADEMIES AND INSTITUTES; ANIMALS; CARDIOVASCULAR DISEASES; COOPERATIVE BEHAVIOR; DIFFUSION OF INNOVATION; GOVERNMENT REGULATION; HEALTH CARE SECTOR; HUMANS; INTERDISCIPLINARY COMMUNICATION; INTERINSTITUTIONAL RELATIONS; NATIONAL HEART, LUNG, AND BLOOD INSTITUTE (U.S.); PROGRAM DEVELOPMENT; RESEARCH SUPPORT AS TOPIC; TRANSLATIONAL MEDICAL RESEARCH; UNITED STATES;

EID: 84877260000     PISSN: 00097330     EISSN: 15244571     Source Type: Journal    
DOI: 10.1161/CIRCRESAHA.113.301100     Document Type: Article

References (24)

1. Munos B. Lessons from 60 years of pharmaceutical innovation. Nat Rev Drug Discov. 2009;8:959-968.
2. Raben N, Nagaraju K, Lee E, Kessler P, Byrne B, Lee L, LaMarca M, King C, Ward J, Sauer B, Plotz P. Targeted disruption of the acid alphaglucosidase gene in mice causes an illness with critical features of both infantile and adult human glycogen storage disease type II. J Biol Chem. 1998;273:19086-19092.
3. Raben N, Danon M, Gilbert AL, Dwivedi S, Collins B, Thurberg BL, Mattaliano RJ, Nagaraju K, Plotz PH. Enzyme replacement therapy in the mouse model of Pompe disease. Mol Genet Metab. 2003;80:159-169.
4. Kishnani PS, Corzo D, Nicolino M, et al. Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease. Neurology. 2007;68:99-109.
5. Byrne BJ, Falk DJ, Clément N, Mah CS. Gene therapy approaches for lysosomal storage disease: next-generation treatment. Hum Gene Ther. 2012;23:808-815.
6. Galis ZS, Hoots WK, Kiley JP, Lauer MS. On the value of portfolio diversity in heart, lung, and blood research. Circ Res. 2012;111:833-836.
7. Stevens AJ, Jensen JJ, Wyller K, Kilgore PC, Chatterjee S, Rohrbaugh ML. The role of public-sector research in the discovery of drugs and vaccines. N Engl J Med. 2011;364:535-541.
8. Nobelprize.org. The nobel prize in chemistry 2012. http://www.nobelprize. org/nobel-prizes/chemistry/laureates/2012. Accessed April 2, 2013.
9. NIH. NIH grantees win 2012 nobel prize in chemistry. 2012; http://www. nhlbi.nih.gov/news/press-releases/2012/nih-grantees-win-2012-nobelprize-in- chemistry.html. Accessed April 2, 2013.
10. Dorey E. Merck ditches biogeneric. Nat Biotechnol. 2010;28:636.
11. Barter PJ, Caulfield M, Eriksson M, et al; ILLUMINATE Investigators. Effects of torcetrapib in patients at high risk for coronary events. N Engl J Med. 2007;357:2109-2122.
12. David M. The demise of the blockbuster. N Engl J Med. 2007;356: 1292-1293.
13. Research America. 2010 US investment in health research. 2010 http://www.researchamerica.org/uploads/healthdollar10.pdf. Accessed April 2, 2013.
14. Coller BS, Califf RM. Traversing the valley of death: A guide to assessing prospects for translational success. Sci Transl Med. 2009;1:10cm19.
15. FDA. Developing products for rare diseases & conditions. http://www.fda. gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/ default. htm. Accessed April 2, 2013.
16. Mullard A. 2011 FDA drug approvals. Nat Rev Drug Discov. 2012;11: 91-94.
17. Kaiser J. A present for NIH: president signs law creating new translational center. Science News. December 27, 2011.
18. Collins FS. Reengineering translational science: the time is right. Sci Transl Med. 2011;3:90cm17.
19. Entrepreneurship NACOIA. Recommendations to facilitate universitybased technology commercialization. 2011; http://center.ncet2.org/images/files/nacie- letter-university-commercialization.pdf. Accessed April 2, 2013.
20. Carmichael M. BS. "Desperately seeking cures: How the road from promising scientific breakthrough to real-world remedy has become all but a dead end. Newsweek, Vol. 155, No. 22.
21. Lauer MS, Skarlatos S. Translational research for cardiovascular diseases at the National Heart, Lung, and Blood Institute: moving from bench to bedside and from bedside to community. Circulation. 2010;121:929-933.
22. Woo SL, Skarlatos SI, Joyce MM, Croxton TL, Qasba P; Heart, Lung, and Blood Diseases Working Group. Critical resources for gene therapy in Heart, Lung, and Blood Diseases Working Group. Mol Ther. 2006;13:641-643.
23. Nielsen report. Secret to successful new product innovation: Keep the boss out of it. 2010; http://www.nielsen.com/us/en/insights/press-room/2010/ secret-to-successful.html. Accessed April 2, 2013.
24. Lauer MS. Thought exercises on accountability and performance measures at the National Heart, Lung, and Blood Institute (NHLBI): an invited commentary for circulation research. Circ Res. 2011;108:405-409.