Linked clinical trials-the development of new clinical learning studies in Parkinson's disease using screening of multiple prospective new treatments

Journal of Parkinson's Disease

Volumn 3, Issue 3, 2013, Pages 231-239

  Brundin, Patrik ^a   Barkerb, Roger A ^b   Connc, P Jeffrey ^c   Dawsond, Ted M ^d   Kieburtze, Karl ^e   Lees, Andrew J ^f   Schwarzschild, Michael A ^g   Tanner, Caroline M ^h   Isaacs, Tom ⁱ   Duffen, Joy ⁱ   Matthews, Helen ⁱ   Wyse, Richard K H ⁱ  

^a VAN ANDEL RESEARCH INSTITUTE   (United States)

^b UNIVERSITY OF CAMBRIDGE   (United Kingdom)

^c VANDERBILT UNIVERSITY SCHOOL OF MEDICINE   (United States)

^d JOHNS HOPKINS UNIVERSITY   (United States)

^e UNIVERSITY OF ROCHESTER MEDICAL CENTER   (United States)

^f UNIVERSITY COLLEGE LONDON   (United Kingdom)

^g MASSACHUSETTS GENERAL HOSPITAL   (United States)

^h PARKINSON S INSTITUTE   (United States)

ⁱ Cure Parkinsons Trust UK   (United Kingdom)

Author keywords

Disease modification; Drug repositioning; Neuroprotection

Indexed keywords

ANTIPARKINSON AGENT; BEXAROTENE; CARBENOXOLONE; DEFERASIROX; DEFERIPRONE; DIMEBON; EPITHILONE D; EXENDIN 4; FENOFIBRATE; GENISTEIN; LIRAGLUTIDE; MERCAPTAMINE; METFORMIN; NABIXIMOLS; NILOTINIB; OLAPARIB; PERINDOPRIL; PROMETHAZINE; RAPAMYCIN; RESVERATROL; SIMVASTATIN; SITAGLIPTIN; TELMISARTAN; TOPOTECAN; TREHALOSE; UNCLASSIFIED DRUG; VELIPARIB;

CLINICAL RESEARCH; CLINICAL STUDY; DRUG EFFICACY; DRUG RESEARCH; DRUG SCREENING; INTERVENTION STUDY; PARKINSON DISEASE; PRIORITY JOURNAL; PROSPECTIVE STUDY; RESEARCH PRIORITY; REVIEW;

ANTIPARKINSON AGENTS; CLINICAL TRIALS AS TOPIC; HUMANS; PARKINSON DISEASE; PROSPECTIVE STUDIES;

EID: 84888869235     PISSN: 18777171     EISSN: 1877718X     Source Type: Journal    
DOI: 10.3233/JPD-139000     Document Type: Review

References (40)

1. Ashburn TT,&Thor KB (2004) Drug repositioning: Identifying and developing new uses for existing drugs. Nat Rev Drug Discov, 3(8), 673-683
2. Keiser MJ, Setola V, Irwin JJ, Laggner C, Abbas AI, Hufeisen SJ, Jensen NH, Kuijer MB, Matos RC, Tran TB, Whaley R, Glennon RA, Hert J, Thomas KL, Edwards DD, Shoichet BK, & Roth BL (2009) Predicting new molecular targets for known drugs. Nature 462(7270), 175-181
3. Keiser MJ, Irwin JJ, & Shoichet BK (2010) The chemical basis of pharmacology. Biochemistry, 49(48), 10267-10276
4. Peters J-U, Schnider P, Mattei P, & Kansy M (2009) Pharmacological promiscuity: Dependence on compound properties and target specificity in a set of recent Roche compounds. Chem Med Chem, 4, 680-686
5. Swinney DC, & Anthony J (2011) How were new medicines discovered? Nat Rev Drug Discov, 10(7), 507-519
6. Williams M (2011) Productivity shortfalls in drug discovery: Contributions from the preclinical sciences? J Pharmacol Exp Ther, 336, 3-8
7. Lounkine E, Keiser MJ,Whitebread S, Mikhailov D, Hamon J, Jenkins JL, Lavan P, Weber E, Doak AK, Ĉot́e S, Shoichet BK, & Urban L (2012) Large-scale prediction and testing of drug activity on side-effect targets. Nature, 486(7403), 361-367
8. Gregori-Puigjańe E, Setola V, Hert J, Crews BA, Irwin JJ, Lounkine E, Marnett L, Roth BL, & Shoichet BK (2012) Identifying mechanism-of-Action targets for drugs and probes. Proc Natl Acad Sci U S A, 109(28), 11178-11183
9. Oprea TI, Bauman JE, Bologa CG, Buranda T, Chigaev A, Edwards BS, Jarvik JW, Gresham HD, Haynes MK, Hjelle B, Hromas R, Hudson L, Mackenzie DA, Muller CY, Reed JC, Simons PC, Smagley Y, Strouse J, Surviladze Z, Thompson T, Ursu O, Waller A, Wandinger-Ness A, Winter SS, Wu Y, Young SM, Larson RS,Willman C, & Sklar LA (2011) Drug repurposing from an academic perspective. Drug Discov Today Ther Strateg, 8, 61-69
10. O'Connor KA, Roth BL (2005) Finding new tricks for old drugs: An efficient route for public-sector drug discovery. Nat Rev Drug Discov, 4(12), 1005-1014
11. Reaume AG (2011) Drug repurposing through nonhypothesis driven phenotypic screening. Drug Discovery Today: Therapeutic Strategies 8, 85-88
12. Kola I, & Landis J (2004) Can the pharmaceutical industry reduce attrition rates? Nat Rev Drug Discov, 3(8), 711-715
13. Exenatide, a GLP-1 agonist originally approved for use in Diabetes Type II http://www.clinicaltrials.gov/ct2/show/NCT01 174810
14. Pioglitazone, a glitazone originally approved for use in Diabetes Type II. http://www.clinicaltrials.gov/ct2/show/NCT01 280123
15. Isradipine, a calcium channel blocker originally approved for use in hypertension. http://www.clinicaltrials.gov/ct2/show/ NCT00909545 http://www.clinicaltrials.gov/ct2/show/NCT 00753636
16. Deferiprone, an iron chelator originally approved for use in beta-Thalassaemia. http://www.clinicaltrials.gov/ct2/show/ NCT01539837 http://www.clinicaltrials.gov/ct2/show/NCT 00943748
17. Inosine, used in the SURE-PD trial to raise serum and CSF urate levels. http://clinicaltrials.gov/ct2/show/NCT00833690
18. Aviles-Olmos I, Dickson J, Kefalopoulou Z, Djamshidian A, Ell P, Soderlund T, Whitton P, Wyse R, Isaacs T, Lees A, Limousin P, & Foltynie T (2013) Exenatide and the treatment of patients with Parkinson's disease. J Clin Invest, 123(6), 2730-2736
19. Barker RA, Stacy M, Brundin P (2013) A new approach to disease-modifying drug trials in Parkinson's disease. J Clin Invest, 123(6), 2364-2365
20. Perry T, Haughey NJ, Mattson MP, Egan JM, & Greig NH (2002) Protection and reversal of excitotoxic neuronal damage by glucagon-like peptide-1 & exendin-4. J Pharmacol Exp Ther, 302(3), 881-888
21. Harkavyi A, Abuirmeileh A, Lever R, Kingsbury AE, Biggs CS, & Whitton PS (2008) Glucagon-like peptide 1 receptor stimulation reverses key deficits in distinct rodent models of Parkinson's disease. J Neuroinflammation, 5, 5-19
22. Bertilsson G, Patrone C, Zachrisson O, Andersson A, Dannaeus K, Heidrich J, Kortesmaa J, Mercer A, Nielsen E, Rönnholm H, & Wikström L (2008) Peptide hormone exendin-4 stimulates subventricular zone neurogenesis in the adult rodent brain and induces recovery in an animal model of Parkinson's disease. J Neurosci Res, 86(2), 326-338
23. Li Y, Perry T, Kindy MS, Harvey BK, Tweedie D, Holloway HW, Powers K, Shen H, Egan JM, Sambamurti K, Brossi A, Lahiri DK, Mattson MP, Hoffer BJ, Wang Y, & Greig NH (2009) GLP-1 receptor stimulation preserves primary cortical and dopaminergic neurons in cellular and rodent models of stroke and Parkinsonism. Proc Natl Acad Sci U S A, 106(4), 1285-1290
24. Kim S, Moon M, & Park S (2009) Exendin-4 protects dopaminergic neurons by inhibition of microglial activation and matrix metalloproteinase-3 expression in an animal model of Parkinson's disease. J Endocrinol, 202(3), 431-439
25. Fan R, Li X, Gu X, Chan JC, & Xu G (2010) Exendin-4 protects pancreatic beta cells from human islet amyloid polypeptide-induced cell damage: Potential involvement of AKT and mitochondria biogenesis. Diabetes Obes Metab, 12(9), 815-824
26. Elm JJ, Goetz CG, Ravina B, Shannon K, Wooten GF, Tanner CM, Palesch YY, Huang P, Guimaraes P, Kamp C, Tilley BC, Kieburtz K, & Investigators NET-PD (2005) A responsive outcome for Parkinson's disease neuroprotection futility studies. Ann Neurol, 57(2), 197-203
27. NINDS NET-PD Investigators (2006) A randomized, doubleblind, futility clinical trial of creatine and minocycline in early Parkinson disease. Neurology, 66(5), 664-671
28. NINDS NET-PD Investigators (2008) A pilot clinical trial of creatine and minocycline in early Parkinson disease: 18-month results. Clin Neuropharmacol 31(3), 141-150
29. Tilley BC, Palesch YY, Kieburtz K, Ravina B, Huang P, Elm JJ, Shannon K, Wooten GF, Tanner CM, & Goetz GC; NETPD Investigators (2006) Optimizing the ongoing search for new treatments for Parkinson disease: Using futility designs. Neurology 66(5), 628-633
30. Tilley BC, & Galpern WR (2007) Screening potential therapies: Lessons learned from new paradigms used in Parkinson disease. Stroke, 38(2 Suppl), 800-803
31. CINAPS http://www.ninds.nih.gov/research/parkinsonsweb/cinaps/Compound dossiers.htm
32. Arrowsmith J (2011) Trial watch: Phase II failures: 2008-2010. Nature Reviews Drug Discovery, 10, 328-329
33. Prinz F, Schlange T, & Asadullah K (2011) Believe it or not: How much can we rely on published data on potential drug targets? Nature Reviews Drug Discovery, 10(9), 712
34. Sherer TB, Chowdhury S, Peabody K, & Brooks DW (2012) Overcoming obstacles in Parkinson's disease. Mov Disord, (13), 1606-1611
35. Getz KA, & Kaitin KI (2012) Open innovation: The new face of pharmaceutical research and development. Expert Rev Clin Pharmacol, 5(5), 481-483
36. Parkinson Progression Marker Initiative (2011) The Parkinson Progression Marker Initiative (PPMI). Prog Neurobiol 95(4), 629-635
37. Enna SJ, & Williams M (2009) Challenges in the search for drugs to treat central nervous system disorders. J Pharmacol Exp Ther, 329, 404-411
38. Hellerstein MK (2008) A critique of the molecular targetbased drug discovery paradigm based on principles of metabolic control: Advantages of pathway-based discovery. Metab Eng, 10, 1-9
39. HellersteinM K (2008) Exploiting complexity and the robustness of network architecture for drug discovery. J Pharmacol Exp Ther, 325, 1-9
40. Sydes MR, Parmar MK, Mason MD, Clarke NW, Amos C, Anderson J, de Bono J, Dearnaley DP, Dwyer J, Green C, Jovic G, Ritchie AW, Russell JM, Sanders K, Thalmann G, & James ND (2012) Flexible trial design in practice-stopping arms for lack-of-benefit and adding research arms mid-Trial in STAMPEDE:Amulti-Arm multi-stage randomized controlled trial. Trials, 13, 168