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  Eflornithine

Eflornithine is a difluoromethylated ornithine compound with antineoplastic activity. Eflornithine irreversibly inhibits ornithine decarboxylase, an enzyme required for polyamine biosynthesis, thereby inhibiting the formation and proliferation of tumor cells. Polyamines are involved in nucleosome oligomerization and DNA conformation, creating a chromatin environment that stimulates neoplastic transformation of cells. This agent has been shown to induce apoptosis in leiomyoma cells. Check for active clinical trials or closed clinical trials using this agent. (NCI Thesaurus).

Eflornithine (α-difluoromethylornithine or DFMO) is a drug found to be effective in the treatment of facial hirsutism  (excessive hair growth) as well as in African trypanosomiasis (sleeping sickness). Eflornithine hydrochloride cream, which is for topical administration in women suffering from facial hirsutism, is marketed under the brand name Vaniqa by Almirall in Europe, CSL in Australia, Triton in Canada, Medison in Israel and SkinMedica in the USA.  Eflornithine for injection against sleeping sickness is manufactured by Sanofi Aventis and sold under the brand name Ornidyl in the USA. Both are prescription drugs.  Eflornithine was initially developed for cancer treatment at Merrell Dow Research Institute in the late 1970s, but, while having little use in treating malignancies, it was found to be highly effective in reducing hair growth, as well as in treatment of African trypanosomiasis (sleeping sickness), especially the West African form (Trypanosoma brucei gambiense). (source: http://en.wikipedia.org/wiki/Eflornithine).  

Current developer:    Cancer Prevention Pharmaceuticals.

1: Cloete TT, Johansson CC, N'Da DD, Vodnala SK, Rottenberg ME, Breytenbach JC, Ashton M. Mono-, di- and trisubstituted derivatives of eflornithine: synthesis for in vivo delivery of DL-alpha-difluoromethylornithine in plasma. Arzneimittelforschung. 2011;61(5):317-25. PubMed PMID: 21755816.

2: Helena KJ, N'Da DD, Johansson CC, Breytenbach JC, Ashton M. Effects of oral administration of synthesized delta-amides of eflornithine in the rat. Arzneimittelforschung. 2010;60(11):682-8. PubMed PMID: 21175041.

3: Vincent IM, Creek D, Watson DG, Kamleh MA, Woods DJ, Wong PE, Burchmore RJ, Barrett MP. A molecular mechanism for eflornithine resistance in African trypanosomes. PLoS Pathog. 2010 Nov 24;6(11):e1001204. PubMed PMID: 21124824; PubMed Central PMCID: PMC2991269.

4: Baker N, Alsford S, Horn D. Genome-wide RNAi screens in African trypanosomes identify the nifurtimox activator NTR and the eflornithine transporter AAT6. Mol Biochem Parasitol. 2011 Mar;176(1):55-7. Epub 2010 Nov 18. PubMed PMID: 21093499; PubMed Central PMCID: PMC3032052.

5: Zell JA, McLaren CE, Chen WP, Thompson PA, Gerner EW, Meyskens FL. Ornithine decarboxylase-1 polymorphism, chemoprevention with eflornithine and sulindac, and outcomes among colorectal adenoma patients. J Natl Cancer Inst. 2010 Oct 6;102(19):1513-6. Epub 2010 Aug 26. PubMed PMID: 20798393; PubMed Central PMCID: PMC2950167.

6: Jansson-Löfmark R, Römsing S, Albers E, Ashton M. Determination of eflornithine enantiomers in plasma by precolumn derivatization with o-phthalaldehyde-N-acetyl-L-cysteine and liquid chromatography with UV detection. Biomed Chromatogr. 2010 Jul;24(7):768-73. PubMed PMID: 20020417.

7: Priotto G, Kasparian S, Mutombo W, Ngouama D, Ghorashian S, Arnold U, Ghabri S, Baudin E, Buard V, Kazadi-Kyanza S, Ilunga M, Mutangala W, Pohlig G, Schmid C, Karunakara U, Torreele E, Kande V. Nifurtimox-eflornithine combination therapy for second-stage African Trypanosoma brucei gambiense trypanosomiasis: a multicentre, randomised, phase III, non-inferiority trial. Lancet. 2009 Jul 4;374(9683):56-64. Epub 2009 Jun 24. PubMed PMID: 19559476.

8: Balasegaram M, Young H, Chappuis F, Priotto G, Raguenaud ME, Checchi F. Effectiveness of melarsoprol and eflornithine as first-line regimens for gambiense sleeping sickness in nine Médecins Sans Frontières programmes. Trans R Soc Trop Med Hyg. 2009 Mar;103(3):280-90. Epub 2008 Oct 22. PubMed PMID: 18947846.

9: Sanderson L, Dogruel M, Rodgers J, Bradley B, Thomas SA. The blood-brain barrier significantly limits eflornithine entry into Trypanosoma brucei brucei infected mouse brain. J Neurochem. 2008 Nov;107(4):1136-46. Epub 2008 Sep 24. PubMed PMID: 18823367; PubMed Central PMCID: PMC2695853.

10: Jansson R, Malm M, Roth C, Ashton M. Enantioselective and nonlinear intestinal absorption of eflornithine in the rat. Antimicrob Agents Chemother. 2008 Aug;52(8):2842-8. Epub 2008 Jun 2. PubMed PMID: 18519728; PubMed Central PMCID: PMC2493103.

11: Namazi MR. Hypothesis: the potential utility of topical eflornithine against cutaneous leishmaniasis. Indian J Dermatol Venereol Leprol. 2008 Mar-Apr;74(2):158-9. PubMed PMID: 18388383.

12: Priotto G, Pinoges L, Fursa IB, Burke B, Nicolay N, Grillet G, Hewison C, Balasegaram M. Safety and effectiveness of first line eflornithine for Trypanosoma brucei gambiense sleeping sickness in Sudan: cohort study. BMJ. 2008 Mar 29;336(7646):705-8. Epub 2008 Mar 5. PubMed PMID: 18321960; PubMed Central PMCID: PMC2276259.

13: Robays J, Raguenaud ME, Josenando T, Boelaert M. Eflornithine is a cost-effective alternative to melarsoprol for the treatment of second-stage human West African trypanosomiasis in Caxito, Angola. Trop Med Int Health. 2008 Feb;13(2):265-71. PubMed PMID: 18304274.

14: Hoffmann R. A 4-month, open-label study evaluating the efficacy of eflornithine 11.5% cream in the treatment of unwanted facial hair in women using TrichoScan. Eur J Dermatol. 2008 Jan-Feb;18(1):65-70. Epub 2007 Dec 18. PubMed PMID: 18086592.

15: Checchi F, Piola P, Ayikoru H, Thomas F, Legros D, Priotto G. Nifurtimox plus Eflornithine for late-stage sleeping sickness in Uganda: a case series. PLoS Negl Trop Dis. 2007 Nov 7;1(2):e64. PubMed PMID: 18060083; PubMed Central PMCID: PMC2100371.

16: Priotto G, Kasparian S, Ngouama D, Ghorashian S, Arnold U, Ghabri S, Karunakara U. Nifurtimox-eflornithine combination therapy for second-stage Trypanosoma brucei gambiense sleeping sickness: a randomized clinical trial in Congo. Clin Infect Dis. 2007 Dec 1;45(11):1435-42. Epub 2007 Oct 22. PubMed PMID: 17990225.

17: Jobanputra KS, Rajpal AV, Nagpur NG. Eflornithine. Indian J Dermatol Venereol Leprol. 2007 Sep-Oct;73(5):365-6. Review. PubMed PMID: 17921631.

18: Jackson J, Caro JJ, Caro G, Garfield F, Huber F, Zhou W, Lin CS, Shander D, Schrode K; Eflornithine HCl Study Group. The effect of eflornithine 13.9% cream on the bother and discomfort due to hirsutism. Int J Dermatol. 2007 Sep;46(9):976-81. PubMed PMID: 17822506.

19: Does eflornithine help women face hirsutism? Drug Ther Bull. 2007 Aug;45(8):62-4. Review. PubMed PMID: 17718095.

20: Hamzavi I, Tan E, Shapiro J, Lui H. A randomized bilateral vehicle-controlled study of eflornithine cream combined with laser treatment versus laser treatment alone for facial hirsutism in women. J Am Acad Dermatol. 2007 Jul;57(1):54-9. Epub 2007 Jan 30. PubMed PMID: 17270315.

21: Wolf JE Jr, Shander D, Huber F, Jackson J, Lin CS, Mathes BM, Schrode K; Eflornithine HCl Study Group. Randomized, double-blind clinical evaluation of the efficacy and safety of topical eflornithine HCl 13.9% cream in the treatment of women with facial hair. Int J Dermatol. 2007 Jan;46(1):94-8. PubMed PMID: 17214730.

22: Balasegaram M, Harris S, Checchi F, Ghorashian S, Hamel C, Karunakara U. Melarsoprol versus eflornithine for treating late-stage Gambian trypanosomiasis in the Republic of the Congo. Bull World Health Organ. 2006 Oct;84(10):783-91. PubMed PMID: 17128358; PubMed Central PMCID: PMC2627491.

23: Smith SR, Piacquadio DJ, Beger B, Littler C. Eflornithine cream combined with laser therapy in the management of unwanted facial hair growth in women: a randomized trial. Dermatol Surg. 2006 Oct;32(10):1237-43. PubMed PMID: 17034372.

24: Malm M, Bergqvist Y. Determination of eflornithine enantiomers in plasma, by solid-phase extraction and liquid chromatography with evaporative light-scattering detection. J Chromatogr B Analyt Technol Biomed Life Sci. 2007 Feb 1;846(1-2):98-104. Epub 2006 Sep 22. PubMed PMID: 16996325.

25: Ganger LK, Hamzavi IH. Excess salt and pepper hair treated with a combination of laser hair removal and topical eflornithine HCl. J Drugs Dermatol. 2006 Jun;5(6):544-5. PubMed PMID: 16774107.

26: Jouanique C. [Eflornithine: evidence of efficacy]. Ann Dermatol Venereol. 2005 Jun-Jul;132(6-7 Pt 2):3S11-3S13. French. PubMed PMID: 16223125.

27: Chappuis F, Udayraj N, Stietenroth K, Meussen A, Bovier PA. Eflornithine is safer than melarsoprol for the treatment of second-stage Trypanosoma brucei gambiense human African trypanosomiasis. Clin Infect Dis. 2005 Sep 1;41(5):748-51. Epub 2005 Jul 19. PubMed PMID: 16080099.

28: Na-Bangchang K, Doua F, Konsil J, Hanpitakpong W, Kamanikom B, Kuzoe F. The pharmacokinetics of eflornithine (alpha-difluoromethylornithine) in patients with late-stage T.b. gambiense sleeping sickness. Eur J Clin Pharmacol. 2004 Jun;60(4):269-78. Epub 2004 May 13. PubMed PMID: 15141331.

29: Louis FJ, Keiser J, Simarro PP, Schmid C, Jannin J. [Eflornithine in the treatment of African trypanosomiasis]. Med Trop (Mars). 2003;63(6):559-63. Review. French. PubMed PMID: 15077414.

30: Burri C, Brun R. Eflornithine for the treatment of human African trypanosomiasis. Parasitol Res. 2003 Jun;90 Supp 1:S49-52. Epub 2002 Dec 10. Review. PubMed PMID: 12811548.

31: Eflornithine trial results. TDR News. 1998 Oct;(57):1-2. PubMed PMID: 12294755.

32: Mpia B, Pépin J. Combination of eflornithine and melarsoprol for melarsoprol-resistant Gambian trypanosomiasis. Trop Med Int Health. 2002 Sep;7(9):775-9. PubMed PMID: 12225509.

33: Eflornithine. Am J Health Syst Pharm. 2001 Dec 1;58(23):2244. PubMed PMID: 11763800.

34: Balfour JA, McClellan K. Topical eflornithine. Am J Clin Dermatol. 2001;2(3):197-201; discussion 202. Review. PubMed PMID: 11705097.

35: Coyne PE Jr. The eflornithine story. J Am Acad Dermatol. 2001 Nov;45(5):784-6. PubMed PMID: 11606936.

36: Malhotra B, Noveck R, Behr D, Palmisano M. Percutaneous absorption and pharmacokinetics of eflornithine HCl 13.9% cream in women with unwanted facial hair. J Clin Pharmacol. 2001 Sep;41(9):972-8. PubMed PMID: 11549102.

37: Shapiro J, Lui H. Vaniqa--eflornithine 13.9% cream. Skin Therapy Lett. 2001 Apr;6(7):1-3, 5. Erratum in: Skin Therapy Lett 2001 May;6(8):5. PubMed PMID: 11376395.

38: Hickman JG, Huber F, Palmisano M. Human dermal safety studies with eflornithine HCl 13.9% cream (Vaniqa), a novel treatment for excessive facial hair. Curr Med Res Opin. 2001;16(4):235-44. PubMed PMID: 11268707.

39: Hennemann A. [Eflornithine for hair removal. Topical application for hirsutism]. Med Monatsschr Pharm. 2001 Feb;24(2):38-9. German. PubMed PMID: 11255983.

40: Pépin J, Khonde N, Maiso F, Doua F, Jaffar S, Ngampo S, Mpia B, Mbulamberi D, Kuzoe F. Short-course eflornithine in Gambian trypanosomiasis: a multicentre randomized controlled trial. Bull World Health Organ. 2000;78(11):1284-95. PubMed PMID: 11143188; PubMed Central PMCID: PMC2560627.

41: Eflornithine cream for facial hair reduction. Med Lett Drugs Ther. 2000 Oct 2;42(1089):96. PubMed PMID: 11015161.

42: Sjoerdsma A, Schechter PJ. Eflornithine for African sleeping sickness. Lancet. 1999 Jul 17;354(9174):254. PubMed PMID: 10421331.

43: Clerinx J, Taelman H, Bogaerts J, Vervoort T. Treatment of late stage rhodesiense trypanosomiasis using suramin and eflornithine: report of six cases. Trans R Soc Trop Med Hyg. 1998 Jul-Aug;92(4):449-50. PubMed PMID: 9850406.

44: Buckner JC, Burch PA, Cascino TL, O'Fallon JR, Scheithauer BW. Phase II trial of recombinant interferon-alpha-2a and eflornithine in patients with recurrent glioma. J Neurooncol. 1998 Jan;36(1):65-70. PubMed PMID: 9525827.

45: Jennings FW, Gichuki CW, Kennedy PG, Rodgers J, Hunter CA, Murray M, Burke JM. The role of the polyamine inhibitor eflornithine in the neuropathogenesis of experimental murine African trypanosomiasis. Neuropathol Appl Neurobiol. 1997 Jun;23(3):225-34. PubMed PMID: 9223132.

46: Lipke DW, Newman PS, Tofiq S, Aziz SM, Soltis EE. Eflornithine alters changes in vascular responsiveness associated with coarctation hypertension. Clin Exp Hypertens. 1997 Apr;19(3):297-312. PubMed PMID: 9107438.

47: Khonde N, Pépin J, Mpia B. A seven days course of eflornithine for relapsing Trypanosoma brucei gambiense sleeping sickness. Trans R Soc Trop Med Hyg. 1997 Mar-Apr;91(2):212-3. PubMed PMID: 9196773.

48: Taelman H, Clerinx J, Bogaerts J, Vervoort T. Combination treatment with suramin and eflornithine in late stage rhodesian trypanosomiasis: case report. Trans R Soc Trop Med Hyg. 1996 Sep-Oct;90(5):572-3. PubMed PMID: 8944277.

49: Zeller W, Garbrecht M, Dierlamm J, Vogel I, Fiedler W, Hossfeld D. Phase I-II study of interferon-gamma and eflornithine (DFMO) in patients with advanced renal cell carcinoma, malignant melanoma and colorectal carcinoma. Oncol Rep. 1996 May;3(3):447-51. PubMed PMID: 21594390.

50: Simarro PP, Asumu PN. Gambian trypanosomiasis and synergism between melarsoprol and eflornithine: first case report. Trans R Soc Trop Med Hyg. 1996 May-Jun;90(3):315. PubMed PMID: 8758090.

51: Soltis EE, Newman PS, Olson JW. Eflornithine treatment in SHR: potential role of vascular polyamines and ornithine decarboxylase in hypertension. Clin Exp Hypertens. 1994 Sep;16(5):595-610. PubMed PMID: 7951165.

52: Harvengt C. Drugs recently released in Belgium. Eflornithine--finasteride. Acta Clin Belg. 1994;49(1):42-6. PubMed PMID: 7514829.

53: Milord F, Loko L, Ethier L, Mpia B, Pépin J. Eflornithine concentrations in serum and cerebrospinal fluid of 63 patients treated for Trypanosoma brucei gambiense sleeping sickness. Trans R Soc Trop Med Hyg. 1993 Jul-Aug;87(4):473-7. PubMed PMID: 8249087.

54: Smith DE, Davies S, Smithson J, Harding I, Gazzard BG. Eflornithine versus cotrimoxazole in the treatment of Pneumocystis carinii pneumonia in AIDS patients. AIDS. 1992 Dec;6(12):1489-93. PubMed PMID: 1492932.

55: Levin VA, Prados MD, Yung WK, Gleason MJ, Ictech S, Malec M. Treatment of recurrent gliomas with eflornithine. J Natl Cancer Inst. 1992 Sep 16;84(18):1432-7. PubMed PMID: 1380989.

56: Milord F, Pépin J, Loko L, Ethier L, Mpia B. Efficacy and toxicity of eflornithine for treatment of Trypanosoma brucei gambiense sleeping sickness. Lancet. 1992 Sep 12;340(8820):652-5. PubMed PMID: 1355219.

57: Paulson YJ, Gilman TM, Heseltine PN, Sharma OP, Boylen CT. Eflornithine treatment of refractory Pneumocystis carinii pneumonia in patients with acquired immunodeficiency syndrome. Chest. 1992 Jan;101(1):67-74. PubMed PMID: 1729113.

58: Smith D, Davies S, Nelson M, Youle M, Gleeson J, Gazzard B. Pneumocystis carinii pneumonia treated with eflornithine in AIDS patients resistant to conventional therapy. AIDS. 1990 Oct;4(10):1019-21. PubMed PMID: 2261116.

59: Clarkson AB Jr, Sarić M, Grady RW. Deferoxamine and eflornithine (DL-alpha-difluoromethylornithine) in a rat model of Pneumocystis carinii pneumonia. Antimicrob Agents Chemother. 1990 Sep;34(9):1833-5. PubMed PMID: 2285303; PubMed Central PMCID: PMC171942.

60: Gradoni L, Iorio MA, Gramiccia M, Orsini S. In vivo effect of eflornithine (DFMO) and some related compounds on Leishmania infantum preliminary communication. Farmaco. 1989 Dec;44(12):1157-66. PubMed PMID: 2517472.

61: Van Bogaert I, Haemers A. Eflornithine. A new drug in the treatment of sleeping sickness. Pharm Weekbl Sci. 1989 Jun 23;11(3):69-75. Review. PubMed PMID: 2505225.

62: Benhamou PH, Chandenier J, Schechter PJ, Epelbaum S, Tell GP, Haegele KD, Pautard JC, Piussan C. [African trypanosomiasis in children treated with eflornithine. A case]. Presse Med. 1989 Jun 17;18(24):1199-202. French. PubMed PMID: 2525760.

63: Eozenou P, Jannin J, Ngampo S, Carme B, Tell GP, Schechter PJ. [A trial treatment with eflornithine of trypanosomiasis caused by Trypanosoma brucei gambiense in the Peoples Republic of the Congo]. Med Trop (Mars). 1989 Apr-Jun;49(2):149-54. French. PubMed PMID: 2507863.

64: Cohen JL, Ko RJ, Lo AT, Shields MD, Gilman TM. High-pressure liquid chromatographic analysis of eflornithine in serum. J Pharm Sci. 1989 Feb;78(2):114-6. PubMed PMID: 2497239.

65: O'Toole BA, Huffman KW, Gibson JP. Effects of eflornithine hydrochloride (DFMO) on fetal development in rats and rabbits. Teratology. 1989 Feb;39(2):103-13. PubMed PMID: 2494758.

66: Rolston KV, Fainstein V, Bodey GP. Intestinal crytosporidiosis treated with eflornithine: a prospective study among patients with AIDS. J Acquir Immune Defic Syndr. 1989;2(5):426-30. PubMed PMID: 2507771.

67: Sahai J, Berry AJ. Eflornithine for the treatment of Pneumocystis carinii pneumonia in patients with the acquired immunodeficiency syndrome: a preliminary review. Pharmacotherapy. 1989;9(1):29-33. Review. PubMed PMID: 2493638.

68: Petru AM, Azimi PH, Cummins SK, Sjoerdsma A. African sleeping sickness in the United States. Successful treatment with eflornithine. Am J Dis Child. 1988 Feb;142(2):224-8. PubMed PMID: 3124603.

69: Doua F, Boa FY, Schechter PJ, Miézan TW, Diai D, Sanon SR, De Raadt P, Haegele KD, Sjoerdsma A, Konian K. Treatment of human late stage gambiense trypanosomiasis with alpha-difluoromethylornithine (eflornithine): efficacy and tolerance in 14 cases in Côte d'Ivoire. Am J Trop Med Hyg. 1987 Nov;37(3):525-33. PubMed PMID: 3120607.

70: Levin VA, Chamberlain MC, Prados MD, Choucair AK, Berger MS, Silver P, Seager M, Gutin PH, Davis RL, Wilson CB. Phase I-II study of eflornithine and mitoguazone combined in the treatment of recurrent primary brain tumors. Cancer Treat Rep. 1987 May;71(5):459-64. PubMed PMID: 3105881.

71: McCann PP, Bitonti AJ, Bacchi CJ, Clarkson AB Jr. Use of difluoromethylornithine (DFMO, eflornithine) for late-stage African trypanosomiasis. Trans R Soc Trop Med Hyg. 1987;81(4):701-2. PubMed PMID: 3127968.

72: Gastaut JA, Tell G, Schechter PJ, Maraninchi D, Mascret B, Carcassonne Y. Treatment of acute myeloid leukemia and blastic phase of chronic myeloid leukemia with combined eflornithine (alpha difluoromethylornithine) and methylglyoxal-bis-guanyl hydrazone (methyl-GAG). Cancer Chemother Pharmacol. 1987;20(4):344-8. PubMed PMID: 3121203.

73: di Bari C, Pastore G, Roscigno G, Schechter PJ, Sjoerdsma A. Late-stage African trypanosomiasis and eflornithine. Ann Intern Med. 1986 Nov;105(5):803-4. PubMed PMID: 3094420.

74: Gilman TM, Paulson YJ, Boylen CT, Heseltine PN, Sharma OP. Eflornithine treatment of Pneumocystis carinii pneumonia in AIDS. JAMA. 1986 Oct 24-31;256(16):2197-8. PubMed PMID: 3093697.