Phase 1/2 open-label dose-escalation study of plasmid DNA expressing two isoforms of hepatocyte growth factor in patients with painful diabetic peripheral neuropathy

Molecular Therapy

Volumn 21, Issue 6, 2013, Pages 1279-1286

  Ajroud Driss, Senda ^a   Christiansen, Mark ^b   Allen, Jeffrey A ^a   Kessler, John A ^a  

^a NORTHWESTERN UNIVERSITY   (United States)

^b Diablo Clinical Research   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ISOPROTEIN; PLASMID DNA; SCATTER FACTOR; UNCLASSIFIED DRUG; VM 202;

ADULT; ANALGESIC ACTIVITY; ARTICLE; BONE PAIN; BRIEF PAIN INVENTORY; CLINICAL ARTICLE; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DIABETIC NEUROPATHY; DIARRHEA; DOSE RESPONSE; DRUG DOSE COMPARISON; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG MEGADOSE; DRUG SAFETY; DRUG TOLERABILITY; DRY EYE; FEMALE; FOOT FRACTURE; HEARTBURN; HUMAN; INGROWN NAIL; INJECTION SITE PAIN; LOW BACK PAIN; MALE; MCGILL PAIN QUESTIONNAIRE; NEUROPATHIC PAIN; PAIN ASSESSMENT; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; PROSTATE HYPERTROPHY; PROTEIN EXPRESSION; QUALITY OF LIFE; RETINA DISEASE; SINUSITIS; TREATMENT OUTCOME; VIRUS INFECTION; VISUAL ANALOG SCALE; XEROSTOMIA;

EID: 84878546514     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2013.69     Document Type: Article

References (41)

1. Callaghan, BC, Cheng, HT, Stables, CL, Smith, AL and Feldman, EL (2012). Diabetic neuropathy: clinical manifestations and current treatments. Lancet Neurol 11: 521-534.
2. Edwards, JL, Vincent, AM, Cheng, HT and Feldman, EL (2008). Diabetic neuropathy: mechanisms to management. Pharmacol Ther 120: 1-34.
3. Tesfaye, S and Selvarajah, D (2012). Advances in the epidemiology, pathogenesis and management of diabetic peripheral neuropathy. Diabetes Metab Res Rev 28 Suppl 1: 8-14.
4. Tesfaye, S, Vileikyte, L, Rayman, G, Sindrup, S, Perkins, B, Baconja, M et al.; on behalf of the Toronto Expert Panel on Diabetic Neuropathy*. (2011). Painful Diabetic Peripheral Neuropathy: Consensus Recommendations on Diagnosis, Assessment and Management. Diabetes Metab Res Rev 27: 629-638.
5. Davies, M, Brophy, S, Williams, R and Taylor, A (2006). The prevalence, severity, and impact of painful diabetic peripheral neuropathy in type 2 diabetes. Diabetes Care 29: 1518-1522.
6. Gore, M, Brandenburg, NA, Dukes, E, Hoffman, DL, Tai, KS and Stacey, B (2005). Pain severity in diabetic peripheral neuropathy is associated with patient functioning, symptom levels of anxiety and depression, and sleep. J Pain Symptom Manage 30: 374-385.
7. Hahn, W, Pyun, WB, Kim, DS, Yoo, WS, Lee, SD, Won, JH et al. (2011). Enhanced cardioprotective effects by coexpression of two isoforms of hepatocyte growth factor from naked plasmid DNA in a rat ischemic heart disease model. J Gene Med 13: 549-555.
8. Pyun, WB, Hahn, W, Kim, DS, Yoo, WS, Lee, SD, Won, JH et al. (2010). Naked DNA expressing two isoforms of hepatocyte growth factor induces collateral artery augmentation in a rabbit model of limb ischemia. Gene Ther 17: 1442-1452.
9. Carlsson, M, Osman, NF, Ursell, PC, Martin, AJ and Saeed, M (2008). Quantitative MR measurements of regional and global left ventricular function and strain after intramyocardial transfer of VM202 into infarcted swine myocardium. Am J Physiol Heart Circ Physiol 295: H522-H532.
10. Cho, KR, Choi, JS, Hahn, W, Kim, DS, Park, JS, Lee, DS et al. (2008). Therapeutic angiogenesis using naked DNA expressing two isoforms of the hepatocyte growth factor in a porcine acute myocardial infarction model. Eur J Cardiothorac Surg 34: 857-863.
11. Saeed, M, Martin, A, Ursell, P, Do, L, Bucknor, M, Higgins, CB et al. (2008). MR assessment of myocardial perfusion, viability, and function after intramyocardial transfer of VM202, a new plasmid human hepatocyte growth factor in ischemic swine myocardium. Radiology 249: 107-118.
12. Perin, EC, Silva, GV, Vela, DC, Zheng, Y, Baimbridge, F, Gahremanpour, A et al. (2011). Human hepatocyte growth factor (VM202) gene therapy via transendocardial injection in a pig model of chronic myocardial ischemia. J Card Fail 17: 601-611.
13. Saeed, M, Saloner, D, Do, L, Wilson, M and Martin, A (2011). Cardiovascular magnetic resonance imaging in delivering and evaluating the effcacy of hepatocyte growth factor gene in chronic infarct scar. Cardiovasc Revasc Med 12: 111-122.
14. Henry, TD, Hirsch, AT, Goldman, J, Wang, YL, Lips, DL, McMillan, WD et al. (2011). Safety of a non-viral plasmid-encoding dual isoforms of hepatocyte growth factor in critical limb ischemia patients: a phase I study. Gene Ther 18: 788-794.
15. Gu, Y, Zhang, J, Guo, L, Cui, S, Li, X, Ding, D et al. (2011). A phase I clinical study of naked DNA expressing two isoforms of hepatocyte growth factor to treat patients with critical limb ischemia. J Gene Med 13: 602-610.
16. Kim, JS, Hwang, HY, Cho, KR, Park, EA, Lee, W, Paeng, JC et al. (2012). Intramyocardial transfer of hepatocyte growth factor as an adjunct to CABG: phase I clinical study. Gene Ther (doi:10.1038/gt.2012.87).
17. Morishita, R, Aoki, M, Yo, Y and Ogihara, T (2002). Hepatocyte growth factor as cardiovascular hormone: role of HGF in the pathogenesis of cardiovascular disease. Endocr J 49: 273-284.
18. Gille, J, Khalik, M, König, V and Kaufmann, R (1998). Hepatocyte growth factor/scatter factor (HGF/SF) induces vascular permeability factor (VPF/VEGF) expression by cultured keratinocytes. J Invest Dermatol 111: 1160-1165.
19. Bussolino, F, Di Renzo, MF, Ziche, M, Bocchietto, E, Olivero, M, Naldini, L et al. (1992). Hepatocyte growth factor is a potent angiogenic factor which stimulates endothelial cell motility and growth. J Cell Biol 119: 629-641.
20. Nakagami, H, Kaneda, Y, Ogihara, T and Morishita, R (2005). Hepatocyte growth factor as potential cardiovascular therapy. Expert Rev Cardiovasc Ther 3: 513-519.
21. Matsumoto, K and Nakamura, T (1996). Emerging multipotent aspects of hepatocyte growth factor. J Biochem 119: 591-600.
22. Jayasankar, V, Woo, YJ, Pirolli, TJ, Bish, LT, Berry, M F, Burdick, J et al. (2005). Induction of angiogenesis and inhibition of apoptosis by hepatocyte growth factor effectively treats postischemic heart failure. J Card Surg 20: 93-101.
23. Maina, F, Hilton, MC, Ponzetto, C, Davies, AM and Klein, R (1997). Met receptor signaling is required for sensory nerve development and HGF promotes axonal growth and survival of sensory neurons. Genes Dev 11: 3341-3350.
24. Maina, F, Hilton, MC, Andres, R, Wyatt, S, Klein, R and Davies, AM (1998). Multiple roles for hepatocyte growth factor in sympathetic neuron development. Neuron 20: 835-846.
25. Gascon, E, Gaillard, S, Malapert, P, Liu, Y, Rodat-Despoix, L, Samokhvalov, IM et al. (2010). Hepatocyte growth factor-Met signaling is required for Runx1 extinction and peptidergic differentiation in primary nociceptive neurons. J Neurosci 30: 12414-12423.
26. Yang, XM, Toma, JG, Bamji, SX, Belliveau, DJ, Kohn, J, Park, M et al. (1998). Autocrine hepatocyte growth factor provides a local mechanism for promoting axonal growth. J Neurosci 18: 8369-8381.
27. Ebens, A, Brose, K, Leonardo, ED, Hanson, MG Jr, Bladt, F, Birchmeier, C et al. (1996). Hepatocyte growth factor/scatter factor is an axonal chemoattractant and a neurotrophic factor for spinal motor neurons. Neuron 17: 1157-1172.
28. Wong, V, Glass, DJ, Arriaga, R, Yancopoulos, GD, Lindsay, RM and Conn, G (1997). Hepatocyte growth factor promotes motor neuron survival and synergizes with ciliary neurotrophic factor. J Biol Chem 272: 5187-5191.
29. Hashimoto, N, Yamanaka, H, Fukuoka, T, Dai, Y, Obata, K, Mashimo, T et al. (2001). Expression of HGF and cMet in the peripheral nervous system of adult rats following sciatic nerve injury. Neuroreport 12: 1403-1407.
30. Tsuchihara, T, Ogata, S, Nemoto, K, Okabayashi, T, Nakanishi, K, Kato, N et al. (2009). Nonviral retrograde gene transfer of human hepatocyte growth factor improves neuropathic pain-related phenomena in rats. Mol Ther 17: 42-50.
31. Liu, ML, Mars, WM, Zarnegar, R and Michalopoulos, GK (1994). Uptake and distribution of hepatocyte growth factor in normal and regenerating adult rat liver. Am J Pathol 144: 129-140.
32. Cantón, A, Burgos, R, Hernández, C, Mateo, C, Segura, RM, Mesa, J et al. (2000). Hepatocyte growth factor in vitreous and serum from patients with proliferative diabetic retinopathy. Br J Ophthalmol 84: 732-735.
33. Apfel, SC, Schwartz, S, Adornato, BT, Freeman, R, Biton, V, Rendell, M et al. (2000). Effcacy and safety of recombinant human nerve growth factor in patients with diabetic polyneuropathy: A randomized controlled trial. rhNGF Clinical Investigator Group. JAMA 284: 2215-2221.
34. Ropper, AH, Gorson, KC, Gooch, CL, Weinberg, DH, Pieczek, A, Ware, JH et al. (2009). Vascular endothelial growth factor gene transfer for diabetic polyneuropathy: a randomized, double-blinded trial. Ann Neurol 65: 386-393.
35. Shakher, J and Stevens, MJ (2011). Update on the management of diabetic polyneuropathies. Diabetes Metab Syndr Obes 4: 289-305.
36. Said, G (2007). Diabetic neuropathy-a review. Nat Clin Pract Neurol 3: 331-340.
37. Cameron, NE, Eaton, SE, Cotter, MA and Tesfaye, S (2001). Vascular factors and metabolic interactions in the pathogenesis of diabetic neuropathy. Diabetologia 44: 1973-1988.
38. Dworkin, RH, Turk, DC, Wyrwich, KW, Beaton, D, Cleeland, CS, Farrar, JT et al. (2008). Interpreting the clinical importance of treatment outcomes in chronic pain clinical trials: IMMPACT recommendations. J Pain 9: 105-121.
39. Bril, V, England, J, Franklin, GM, Backonja, M, Cohen, J, Del Toro, D et al.; American Academy of Neurology; American Association of Neuromuscular and Electrodiagnostic Medicine; American Academy of Physical Medicine and Rehabilitation. (2011). Evidence-based guideline: Treatment of painful diabetic neuropathy: report of the American Academy of Neurology, the American Association of Neuromuscular and Electrodiagnostic Medicine, and the American Academy of Physical Medicine and Rehabilitation. Neurology 76: 1758-1765.
40. Zelman, DC, Gore, M, Dukes, E, Tai, KS and Brandenburg, N (2005). Validation of a modifed version of the brief pain inventory for painful diabetic peripheral neuropathy. J Pain Symptom Manage 29: 401-410.
41. Lee, Y, Park, EJ, Yu, SS, Kim, DK and Kim, S (2000). Improved expression of vascular endothelial growth factor by naked DNA in mouse skeletal muscles: implication for gene therapy of ischemic diseases. Biochem Biophys Res Commun 272: 230-235.