Orphan/rare drug discovery through drug repositioning

Drug Discovery Today: Therapeutic Strategies

Volumn 8, Issue 3-4, 2011, Pages 71-76

  Muthyala, Ramaiah ^a  

^a UNIVERSITY OF MINNESOTA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

AMPHOTERICIN B; ANTIBIOTIC AGENT; ARBACLOFEN; ARSENIC TRIOXIDE; ASTEMIZOLE; CEFTRIAXONE; CLOSANTEL; CYPROHEPTADINE; DULOXETINE; EFLORNITHINE; GENTAMICIN; HYDROXYUREA; IVERMECTIN; LOSARTAN POTASSIUM; MEMANTINE; MILTEFOSINE; MINOCYCLINE; MIRTAZAPINE; ORPHAN DRUG; RETINOIC ACID; RITUXIMAB; SILDENAFIL; SULINDAC; TAMOXIFEN; THALIDOMIDE; VESICULAR GLUTAMATE TRANSPORTER 1;

AFRICAN TRYPANOSOMIASIS; ARTICLE; AWARENESS; BIPOLAR DISORDER; BRAIN ISCHEMIA; DOWN SYNDROME; DRUG APPROVAL; DRUG INDUSTRY; DRUG REPOSITIONING; DUCHENNE MUSCULAR DYSTROPHY; FOOD AND DRUG ADMINISTRATION; FRAGILE X SYNDROME; GOVERNMENT; HUMAN; LEISHMANIASIS; LEPROSY; MALARIA; MARFAN SYNDROME; MULTIPLE MYELOMA; ONCHOCERCIASIS; PROGRESSIVE MULTIFOCAL LEUKOENCEPHALOPATHY; PROMYELOCYTIC LEUKEMIA; PUBLIC-PRIVATE PARTNERSHIP; PULMONARY HYPERTENSION; RHEUMATOID ARTHRITIS; SICKLE CELL ANEMIA; URINE INCONTINENCE;

EID: 84862165688     PISSN: None     EISSN: 17406773     Source Type: Journal    
DOI: 10.1016/j.ddstr.2011.10.003     Document Type: Review

References (33)

1. Ellen, H.M. and Moors, J.F. (2007) Orphan drugs: unmet societal need for non-profitable privately supplied new products. Res. Policy 36, 19
2. Ashburn, T.T. and Thor, K.B. (2004) Drug repositioning: identifying and developing new uses for existing drugs. Nat. Rev. Drug Discov. 3, 673-683 (Pubitemid 39173508)
3. Sharma, A.J.A. et al. (2010) Orphan drug: development trends and strategies. J. Pharm. Bioall. Sci. 2, 290-299
4. Tambuyzer, E. (2010) Rare diseases, orphan drugs and their regulation: questions and misconceptions. Nat. Rev. Drug Discov. 9, 921-929
5. Dunoyer, M. (2011) Accelerating access to treatments for rare diseases. Nat. Rev. Drug Discov. 10, 475-476
6. Padhy, B.M. and Gupta, Y.K. (2011) Drug repositioning: re-investigating existing drugs for new therapeutic indications. J. Postgrad. Med. 57, 153-160
7. Clark, J.E. et al. (2002) Novel trends in orphan market drug discovery: amyotrophic laterral sclerosis as a case study. Front. Biosci. 7, c83-c96
8. Seoane-Vazquez, E. et al. (2008) Incentives for orphan drug research and development in the United States. Orphanet J. Rare Dis. 3, 7
9. Grau, M.D. and Serbedzija, G. (2005) Innovative strategies for drug repurposing. Drug Discov. Dev. 8
10. Sakharkar, M.K. et al. (2008) Quantitative analysis on the characteristics of targets with FDA approved drugs. Int. J. Biol. Sci. 4, 15-22
11. Stavrovskaya, I.G. et al. (2004) Clinically approved heterocyclics act on a mitochondrial target and reduce stroke-induced pathology. J. Exp. Med. 200, 211-222 (Pubitemid 38982180)
12. Cereghino, J.J. et al. (2002) Rectal diazepam gel for treatment of acute repetitive seizures in adults. Arch. Neurol. 59, 1915-1920 (Pubitemid 35424650)
13. Brewer, G.J. (2006) Fundamental problems lie ahead in the drug discovery and commercialization process: restructuring of the pharmaceutical industry and an improved partnership with academia are required. J. Invest. Med. 54, 314-322
14. Boguski, M.S. et al. (2009) Drug discovery. Repurposing with a difference. Science 324, 1394-1395
15. Lussier, Y.A. and Chen, J.L. (2011) The emergence of genome-based drug repositioning. Sci. Transl. Med. 3 96ps35
16. Sirota, M. et al. (2011) Discovery and preclinical validation of drug indications using compendia of public gene expression data. Sci. Transl. Med. 3 96ra77
17. Dudley, J.T. et al. (2011) Computational repositioning of the anticonvulsant topiramate for inflammatory bowel disease. Sci. Transl. Med. 3 96ra76
18. Chong, C.R. et al. (2006) A clinical drug library screen identifies astemizole as an antimalarial agent. Nat. Chem. Biol. 2, 415-416 (Pubitemid 44114918)
19. Rothstein, J.D. et al. (2005) [beta]-Lactam antibiotics offer neuroprotection by increasing glutamate transporter expression. Nature 433, 73-77 (Pubitemid 40101145)
20. Kerry, A. and O'Connor, B.L.R. (2005) Finding new tricks for old drugs: an efficient route for public sector drug discovery. Nat. Rev. Drug Discov. 4, 1005-1014
21. Chopra, S. et al. (2010) Repurposing FDA-approved drugs to combat drugresistant Acinetobacter baumannii. J. Antimicrob. Chemother. 65, 2598-2601
22. Ejim, L. et al. (2011) Combinations of antibiotics and nonantibiotic drugs enhance antimicrobial efficacy. Nat. Chem. Biol. 7, 348-350 doi:10.1038/NCHEMBIO559
23. Ha, S. et al. (2008) IDMap: facilitating the detection of potential leads with therapeutic targets. Bioinformatics 24, 1413-1415 (Pubitemid 351737154)
24. Heemskerk, J. (2005) Screening existing drugs for neurodegeneration: The National Institute of Neurologic Disorders and Stroke (NINDS) model. Retina 25 (Suppl.), S56-S57 (Pubitemid 44726526)
25. Haupt, V.J. and Schroeder, M. (2011) Old friends in new guise: repositioning of known drugs with structural bioinformatics. Brief. Bioinformatics 12, 5
26. Duenas-Gonzalez, A. et al. (2008) The prince and the pauper. A tale of anticancer targeted agents. Mol. Cancer 7, 1-32
27. Collins, F.S. (2011) Mining for therapeutic gold. Nat. Rev. Drug Discov. 10, 397
28. Huang, R. et al. (2011) The NCGC Pharmaceutical Collection: a comprehensive resource of clinically approved drugs enabling repurposing and chemical genomics. Sci. Transl. Med. 3 80ps16
29. Partnerships. http://www.4cures.org/, http://ctsapharmaportal.org/
30. Mullard, A. (2011) Could pharma open its drug freezers? Nat. Rev. Drug Discov. 10, 399-400
31. Frye, S. et al. (2011) US academic drug discovery. Nat. Rev. Drug Discov. 10, 409-410
32. Xu, K. and Coté, T.R. (2011) Database identifies FDA-approved drugs with potential to be repurposed for treatment of orphan diseases. Brief. Bioinformatics 12, 341-345
33. Goldberg, N.H. et al. (2011) Availability of comparative efficacy data at the time of drug approval in the United States. J. Am. Med. Assoc. 305, 1786-1789