Phase I, dose-escalation, two-part trial of the PARP inhibitor talazoparib in patients with advanced germline BRCA1/2 mutations and selected sporadic cancers

Cancer Discovery

Volumn 7, Issue 6, 2017, Pages 620-629

  de Bono, Johann ^a   Ramanathan, Ramesh K ^b   Mina, Lida ^c   Chugh, Rashmi ^d   Glaspy, John ^e   Rafii, Saeed ^a   Kaye, Stan ^a   Sachdev, Jasgit ^b   Heymach, John ^f   Smith, David C ^d   Henshaw, Joshua W ^g   Herriott, Ashleigh ^h   Patterson, Miranda ^h   Curtin, Nicola J ^h   Byers, Lauren Averett ^f   Wainberg, Zev A ^e  

^a ROYAL MARSDEN HOSPITAL   (United Kingdom)

^b HonorHealth and the Scottsdale Lincoln Health Network   (United States)

^c INDIANA UNIVERSITY SCHOOL OF MEDICINE   (United States)

^d UNIVERSITY OF MICHIGAN   (United States)

^e UNIVERSITY OF CALIFORNIA   (United States)

^f UNIVERSITY OF TEXAS MD ANDERSON CANCER CENTER   (United States)

^g BIOMARIN PHARMACEUTICAL INC   (United States)

^h NEWCASTLE UNIVERSITY   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

TALAZOPARIB; ANTINEOPLASTIC AGENT; BRCA1 PROTEIN; BRCA1 PROTEIN, HUMAN; BRCA2 PROTEIN; BRCA2 PROTEIN, HUMAN; NICOTINAMIDE ADENINE DINUCLEOTIDE ADENOSINE DIPHOSPHATE RIBOSYLTRANSFERASE 1; NICOTINAMIDE ADENINE DINUCLEOTIDE ADENOSINE DIPHOSPHATE RIBOSYLTRANSFERASE INHIBITOR; PARP1 PROTEIN, HUMAN; PHTHALAZINE DERIVATIVE;

ADULT; AGED; ALOPECIA; ANEMIA; ANTINEOPLASTIC ACTIVITY; AREA UNDER THE CURVE; ARTICLE; BREAST CANCER; CANCER CHEMOTHERAPY; COLORECTAL CANCER; CONTROLLED STUDY; DOSE RESPONSE; DRUG CLEARANCE; DRUG DOSE ESCALATION; DRUG DOSE REDUCTION; DRUG EFFICACY; DRUG HALF LIFE; DRUG SAFETY; DRUG TOLERABILITY; DRUG WITHDRAWAL; EWING SARCOMA; FATIGUE; FEMALE; GERMLINE MUTATION; HUMAN; LIQUID CHROMATOGRAPHY-MASS SPECTROMETRY; MAJOR CLINICAL STUDY; MALE; MALIGNANT NEOPLASM; MAXIMUM PLASMA CONCENTRATION; MULTICENTER STUDY; MULTIPLE CYCLE TREATMENT; NAUSEA; NEUTROPENIA; OVARY CANCER; PANCREAS CANCER; PERIPHERAL BLOOD MONONUCLEAR CELL; PHARMACODYNAMICS; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; PROGRESSION FREE SURVIVAL; PROSTATE CANCER; RECOMMENDED DRUG DOSE; SINGLE DRUG DOSE; SMALL CELL LUNG CANCER; SPORADIC CANCER; THROMBOCYTOPENIA; TIME TO MAXIMUM PLASMA CONCENTRATION; TREATMENT DURATION; ADOLESCENT; BLOOD; CLINICAL TRIAL; ENZYMOLOGY; GENETICS; MAXIMUM TOLERATED DOSE; MIDDLE AGED; MONONUCLEAR CELL; NEOPLASM; TREATMENT OUTCOME; VERY ELDERLY; YOUNG ADULT;

ADOLESCENT; ADULT; AGED; AGED, 80 AND OVER; ANTINEOPLASTIC AGENTS; BRCA1 PROTEIN; BRCA2 PROTEIN; FEMALE; GERM-LINE MUTATION; HUMANS; LEUKOCYTES, MONONUCLEAR; MALE; MAXIMUM TOLERATED DOSE; MIDDLE AGED; NEOPLASMS; PHTHALAZINES; POLY (ADP-RIBOSE) POLYMERASE-1; POLY(ADP-RIBOSE) POLYMERASE INHIBITORS; TREATMENT OUTCOME; YOUNG ADULT;

EID: 85020427570     PISSN: 21598274     EISSN: 21598290     Source Type: Journal    
DOI: 10.1158/2159-8290.CD-16-1250     Document Type: Article

References (26)

1. Rouleau M, Patel A, Hendzel MJ, Kaufmann SH, Poirier GG. PARP inhibition: PARP1 and beyond. Nat Rev Cancer 2010; 10: 293-301.
2. Satoh MS, Lindahl T. Role of poly(ADP-ribose) formation in DNA repair. Nature 1992; 356: 356-8.
3. Ame JC, Rolli V, Schreiber V, Niedergang C, Apiou F, Decker P, et al. PARP-2, a novel mammalian DNA damage-dependent poly(ADPribose) polymerase. J Biol Chem 1999; 274: 17860-8.
4. Shall S, de Murcia G. Poly(ADP-ribose) polymerase-1: What have we learned from the deficient mouse model?. Mutat Res 2000; 460: 1-15.
5. Dantzer F, Schreiber V, Niedergang C, Trucco C, Flatter E, De La Rubia G, et al. Involvement of poly(ADP-ribose) polymerase in base excision repair. Biochimie 1999; 81: 69-75.
6. De Vos M, Schreiber V, Dantzer F. The diverse roles and clinical relevance of PARPs in DNA damage repair: Current state of the art. Biochem Pharmacol 2012; 84: 137-46.
7. Farmer H, McCabe N, Lord CJ, Tutt AN, Johnson DA, Richardson TB, et al. Targeting the DNA repair defect in BRCA mutant cells as a therapeutic strategy. Nature 2005; 434: 917-21.
8. Bryant HE, Schultz N, Thomas HD, Parker KM, Flower D, Lopez E, et al. Specific killing of BRCA2-deficient tumours with inhibitors of poly(ADP-ribose) polymerase. Nature 2005; 434: 913-7.
9. Lord CJ, Tutt AN, Ashworth A. Synthetic lethality and cancer therapy: Lessons learned from the development of PARP inhibitors. Annu Rev Med 2015; 66: 455-70.
10. Fong PC, Boss DS, Yap TA, Tutt A, Wu P, Mergui-Roelvink M, et al. Inhibition of poly(ADP-ribose) polymerase in tumors from BRCA mutation carriers. N Engl J Med 2009; 361: 123-34.
11. Fong PC, Yap TA, Boss DS, Carden CP, Mergui-Roelvink M, et al. Poly(ADP)-ribose polymerase inhibition: Frequent durable responses in BRCA carrier ovarian cancer correlating with platinum-free interval. J Clin Oncol 2010; 28: 2512-9.
12. Lee JM, Ledermann JA, Kohn EC. PARP Inhibitors for BRCA1/2 mutation- associated and BRCA-like malignancies. Ann Oncol 2014; 25: 32-40.
13. Mateo J, Carreira S, Sandhu S, Miranda S, Mossop H, Perez-Lopez R, et al. DNA-repair defects and olaparib in metastatic prostate cancer. N Engl J Med 2015; 373: 1697-708.
14. Shen Y, Rehman FL, Feng Y, Boshuizen J, Bajrami I, Elliott R, et al. BMN 673, a novel and highly potent PARP1/2 inhibitor for the treatment of human cancers with DNA repair deficiency. Clin Cancer Res 2013; 19: 5003-15.
15. Aoyagi-Scharber M, Gardberg AS, Yip BK, Wang B, Shen Y, Fitzpatrick PA. Structural basis for the inhibition of poly(ADP-ribose) polymerases 1 and 2 by BMN 673, a potent inhibitor derived from dihydropyridophthalazinone. Acta Crystallogr F Struct Biol Commun 2014; 70: 1143-9.
16. Murai J, Huang SY, Renaud A, Zhang Y, Ji J, Takeda S, et al. Stereospecific PARP trapping by BMN 673 and comparison with olaparib and rucaparib. Mol Cancer Ther 2014; 13: 433-43.
17. Garnett MJ, Edelman EJ, Heidorn SJ, Greenman CD, Dastur A, Lau KW, et al. Systematic identification of genomic markers of drug sensitivity in cancer cells. Nature 2012; 483: 570-5.
18. Lee HJ, Yoon C, Schmidt B, Park DJ, Zhang AY, Erkizan HV, et al. Combining PARP-1 inhibition and radiation in Ewing sarcoma results in lethal DNA damage. Mol Cancer Ther 2013; 12: 2591-600.
19. Byers LA, Wang J, Nilsson MB, Fujimoto J, Saintigny P, Yordy J, et al. Proteomic profiling identifies dysregulated pathways in small cell lung cancer and novel therapeutic targets including PARP1. Cancer Discov 2012; 2: 798-811.
20. Cardnell RJ, Feng Y, Diao L, Fan YH, Masrorpour F, Wang J, et al. Proteomic markers of DNA repair and PI3K pathway activation predict response to the PARP inhibitor BMN 673 in small cell lung cancer. Clin Cancer Res 2013; 19: 6322-28.
21. Waddell N, Pajic M, Patch AM, Chang DK, Kassahn KS, Bailey P, et al. Whole genomes redefine the mutational landscape of pancreatic cancer. Nature 2015; 518: 495-501.
22. Simon R, Freidlin B, Rubinstein L, Arbuck SG, Collins J, Christian MC. Accelerated titration designs for phase I clinical trials in oncology. J Natl Cancer Inst 1997; 89: 1138-47.
23. Gough K, Hutchison M, Keene O, Byrom B, Ellis S, Lacey L, et al. Assessment of dose proportionality: Report from the statisticians in the pharmaceutical industry/pharmacokinetics UK joint working party. Drug Inf J 1995; 29: 1039-48.
24. Rustin GJ, Quinn M, Thigpen T, du Bois A, Pujade-Lauraine E, Jakobsen A, et al. New guidelines to evaluate the response to treatment in solid tumors (ovarian cancer). J Natl Cancer Inst 2004; 96: 487-8.
25. Scher HI, Halabi S, Tannock I, Morris M, Sternberg CN, Carducci MA, et al. Design and end points of clinical trials for patients with progressive prostate cancer and castrate levels of testosterone: Recommendations of the Prostate Cancer Clinical Trials Working Group. J Clin Oncol 2008; 26: 1148-59.
26. Melnik MK, Webb CP, Richardson PJ, Luttenton CR, Campbell AD, Monroe TJ, et al. Phase II trial to evaluate gemcitabine and etoposide for locally advanced or metastatic pancreatic cancer. Mol Cancer Ther 2010; 9: 2423-9.