Phase I Study of Irofulven (MGI 114), an Acylfulvene Illudin Analog, in Patients with Acute Leukemia

Estey E, deLima M, Strom S, Pierce S, Freireich EJ, Keating MJ: Long-term follow-up of patients with newly diagnosed acute myeloid leukemia treated at the University of Texas M.D. Anderson Cancer Center. Cancer 80: 2176–2180, 1997 Freireich EJ: Four decades of therapy for AML. Leukemia, 12 Suppl 1: S54–56, 1998 Cortes JE, Kantarjian H, Freireich EJ: Acute lymphocytic leukemia: a comprehensive review with emphasis on biology and therapy. Cancer Treat Res 84: 291–323, 1996 Freireich EJ, Keating MJ: Conclusions regarding leukemia long-term survival. Cancer 80: 2215–2217, 1997 Freireich EJ: The road to the cure of acute lymphoblastic leukemia: a personal perspective. Oncology 54: 265–269, 1997 Estey E, Thall P, Beran M, Kantarjian H, Pierce S, Keating M: Effect of diagnosis (refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, or acute myeloid leukemia {AML}) on outcome of AML-type chemotherapy. Blood 90: 2969–2977, 1997 Cheson BD: Standard and low-dose chemotherapy for the treatment of myelodysplastic syndromes. Leuk Res 22Suppl 1: S17–21, 1998 Sacchi S, Kantarjian HM, O'Brien S, Cortes J, Rios MB, Giles FJ, Beran M, Koller CA, Keating MJ, Talpaz M: Chronic myelogenous leukemia in nonlymphoid blastic phase: analysis of the results of first salvage therapy with three different treatment approaches for 162 patients. Cancer 86: 2632–2641, 1999 Stahnke K, Boos J, Bender-Gotze C, Ritter J, Zimmermann M, Creutzig U: Duration of first remission predicts remission rates and long-term survival in children with relapsed acute myelogenous leukemia. Leukemia 12: 1534–1538, 1998 Chessels JM, Swansbury GJ, Reeves B, Bailey CC, Richards SM: Cytogenetics and prognosis in childhood lymphoblastic leukaemia: results of MRC UKALL X. Medical Research Council Working Party in Childhood Leukaemia. Br J Haematol 99: 93–100, 1997 Anchel M, Hervey A, Robbins WJ: Antibiotic substances from Basidomycetes. VII. Clitocybe iludens. Proc Natl Acad Sci USA 36: 30–36, 1950 Kelner MJ, McMorris TC, Beck WT, Zamora JM, Taetle R: Preclinical evaluation of illudins as anticancer agents. Cancer Res 47: 3186–3189, 1987 Kelner MJ, McMorris TC, Taetle R: Preclinical evaluation of illudins as anticancer agents: basis for selective cytotoxicity. J Natl Cancer Inst 82: 1562–1565, 1990 Woynarowska BA, Woynarowski JM, Herzig MC, Roberts K, Higdon AL, MacDonald JR: Differential cytotoxicity and induction of apoptosis in tumor and normal cells by hydroxymethylacylfulvene (HMAF). Biochem Pharmacol 59: 1217–1226, 2000 Kelner MJ, McMorris TC, Estes L, Starr RJ, Rutherford M, Montoya M, Samson KM, Taetle R: Efficacy of Acylfulvene Illudin analogues against a metastatic lung carcinoma MV522 xenograft nonresponsive to traditional anticancer agents: retention of activity against various mdr phenotypes and unusual cytotoxicity against ERCC2 and ERCC3 DNA helicasedeficient cells. Cancer Res 55: 4936–4940, 1995 Murgo A, Cannon DJ, Blatner G, Cheson BD: Clinical trials referral resource. Clinical trials of MGI-114. Oncology (Huntingt) 13: 233, 237-238, 1999 Kelner MJ, McMorris TC, Estes L, Rutherford M, Montoya M, Goldstein J, Samson K, Starr R, Taetle R: Characterization of illudin S sensitivity in DNA repair-deficient Chinese hamster cells. Unusually high sensitivity of ERCC2 and ERCC3 DNA helicase-deficient mutants in comparison to other chemotherapeutic agents. Biochem Pharmacol 48: 403–409, 1994 Woynarowski JM, Napier C, Koester SK, Chen SF, Troyer D, Chapman W, MacDonald JR: Effects on DNA integrity and apoptosis induction by a novel antitumor sesquiterpene drug, 6-hydroxymethylacylfulvene (HMAF, MGI 114). Biochem Pharmacol 54: 1181–1193, 1997 McMorris TC: Discovery and development of sesquiterpenoid derived hydroxymethylacylfulvene: a new anticancer drug. Bioorg Med Chem 7: 881–886, 1999 Bennett JM, Catovsky D, Daniel MT, Flandrin G, Galton DA, Gralnick HR, Sultan C: Proposals for the classification of the acute leukaemias. French-American-British (FAB) co-operative group. Br J Haematol 33: 451–458, 1976 Cortes J, Estey E, Beran M, O'Brien S, Giles FJ, Koller C, Keating MJ, Kantarjian HM: Cyclophosphamide, ara-C, and topotecan (CAT) for patients with refractory or relapsed acute leukemia. Leuk Lymph 36: 479–484, 2000 McMorris TC, Anchel M: Fungal metabolites. The structure of the novel sesquiterpenoids Illudin-S and-M. J Am Chem 87: 1594–1600, 1965 Kelner MJ, McMorris TC, Estes L, Wang W, Samson KM, Taetle R: Efficacy of HMAF (MGI-114) in the MV522 metastatic lung carcinoma xenograft model nonresponsive to traditional anticancer agents. Invest New Drugs 14: 161–167, 1996 MacDonald JR, Muscoplat CC, Dexter DL, Mangold GL, Chen SF, Kelner MJ, McMorris TC, Von Hoff DD: Preclinical antitumor activity of 6-hydroxymethylacylfulvene, a semisynthetic derivative of the mushroom toxin illudin S. Cancer Res 57: 279–283, 1997 Kelner MJ, McMorris TC, Estes L, Samson KM, Bagnell RD, Taetle R: Efficacy of MGI 114 (6-hydroxymethylacylfulvene, HMAF) against the mdr1/gp170 metastatic MV522 lung carcinoma xenograft. Eur J Cancer 34: 908–913, 1998 Reed E: Platinum-DNA adduct, nucleotide excision repair and platinum based anti-cancer chemotherapy. Cancer Treat Rev 24: 331–344, 1998 Sancar A: DNA repair in humans. Annu Rev Genet 29: 69–105, 1995 Dabholkar M, Bostick-Bruton F, Weber C, Bohr VA, Egwuagu C, Reed E: ERCC1 and ERCC2 expression in malignant tissues from ovarian cancer patients. J Natl Cancer Inst 84: 1512–1517, 1992 Dabholkar M, Vionnet J, Bostick-Bruton F, Yu JJ, Reed E: Messenger RNA levels of XPAC and ERCC1 in ovarian cancer tissue correlate with response to platinum-based chemotherapy. J Clin Invest 94: 703–708, 1994 Dabholkar MD, Berger MS, Vionnet JA, Egwuagu C, Silber JR, Yu JJ, Reed E: Malignant and nonmalignant brain tissues differ in their messenger RNA expression patterns for ERCC1 and ERCC2. Cancer Res 55: 1261–1266, 1995 Lee KB, Parker RJ, Bohr V, Cornelison T, Reed E: Cisplatin sensitivity/resistance in UV repair-deficient Chinese hamster ovary cells of complementation groups 1 and 3. Carcinogenesis 14: 2177–2180, 1993 Megason G, Weitman S, Blaney S, Winick N, Kuhn J, Pullen J, Bernstein M: A trial of 6-Hydroxymetylacylfulvene (MGI-114) in children with solid tumors, a Pediatric Oncology Group Phase I Cooperative Agreement Study. J Clin Oncol, Proc ASCO 19: 201a, 2000 Shehab TM, Kaminski MS, Lok AS: Acute liver failure due to hepatic involvement by hematologic malignancy. Dig Dis Sci 42: 1400–1405, 1997 Kelner MJ, McMorris TC, Estes L, Samson KM, Trani NA, MacDonald JR: Anti-leukemic action of the novel agent MGI 114 (HMAF) and synergistic action with topotecan. Leukemia 14: 136–141, 2000 Beran M, Estey E, O'Brien SM, Giles FJ, Koller CA, Kornblau S, Keating M, Kantarjian HM: Results of topotecan singleagent therapy in patients with myelodysplastic syndromes and chronic myelomonocytic leukemia. Leuk Lymphoma 31: 521–531, 1998 Beran M, Kantarjian H: Topotecan in the treatment of hematologic malignancies. Semin Hematol 35: 26–31, 1998 Beran M, Kantarjian H, O'Brien S, Koller C, al-Bitar M, Arbuck S, Pierce S, Moore M, Abbruzzese JL, Andreeff M, Keating M, Estey E: Topotecan, a topoisomerase I inhibitor, is active in the treatment of myelodysplastic syndrome and chronic myelomonocytic leukemia. Blood 88: 2473–2479, 1996 Kantarjian HM, Beran M, Ellis A, Zwelling L, O'Brien S, Cazenave L, Koller C, Rios MB, Plunkett W, Keating MJ et al.: Phase I study of Topotecan, a new topoisomerase I inhibitor, in patients with refractory or relapsed acute leukemia. Blood 81: 1146–1151, 1993 Rowinsky EK, Adjei A, Donehower RC, Gore SD, Jones RJ, Burke PJ, Cheng YC, Grochow LB, Kaufmann SH: Phase I and pharmacodynamic study of the topoisomerase I-inhibitor topotecan in patients with refractory acute leukemia. J Clin Oncol 12: 2193–2203, 1994 Rowinsky EK, Kaufmann SH, Baker SD, Miller CB, Sartorius SE, Bowling MK, Chen TL, Donehower RC, Gore SD: A Phase I and pharmacological study of topotecan infused over 30 minutes for five days in patients with refractory acute leukemia. Clin Cancer Res 2: 1921–1930, 1996 Beran M, Kantarjian H: Results of topotecan-based combination therapy in patients with myelodysplastic syndromes and chronic myelomonocytic leukemia. Semin Hematol 36: 3–10, 1999 Beran M, Estey E, O'Brien S, Cortes J, Koller CA, Giles FJ, Kornblau S, Andreeff M, Vey N, Pierce SR, Hayes K, Wong GC, Keating M, Kantarjian H: Topotecan and cytarabine is an active combination regimen in myelodysplastic syndromes and chronic myelomonocytic leukemia. J Clin Oncol 17: 2819–2830, 1999 Vey N, Kantarjian H, Beran M, O'Brien S, Cortes J, Koller C, Estey E: Combination of topotecan with cytarabine or etoposide in patients with refractory or relapsed acute myeloid leukemia: results of a randomized Phase I/II study. Invest New Drugs 17: 89–95, 1999 Mitsui I, Kumazawa E, Hirota Y, Aonuma M, Sugimori M, Ohsuki S, Uoto K, Ejima A, Terasawa H, Sato K: A new watersoluble camptothecin derivative, DX-8951f, exhibits potent antitumor activity against human tumors in vitro and in vivo. Jpn J Cancer Res 86: 776–782, 1995 Vey N, Giles F, Kantarjian H, Smith TL, Beran M, Jeha S: The topoisomerase I inhibitor DX-8951f is active in a severe combined immunodeficient mouse model of human acute myelogenous leukemia. Clin Cancer Res 6: 731–736, 2000 Estey E, Kornblau S, Pierce S, Kantarjian H, Beran M, Keating M: A stratification system for evaluating and selecting therapies in patients with relapsed or primary refractory acute myelogenous leukemia. Blood 88: 756, 1996 Address for offprints: Francis J. Giles, Department of Leukemia, Box 61, The University of Texas M.D. Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX 77030–4095, USA; E-mail: [email protected]