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Pomalidomide is a substance being studied in the treatment of prostate cancer, multiple myeloma, and other types of cancer. Pomalidomide is a form of the drug thalidomide. It stops the growth of blood vessels, stimulates the immune system, and may kill cancer cells. Pomalidomide is a type of angiogenesis inhibitor and a type of immunomodulatory agent. Also called Actimid and CC-4047.  (from NCI's webpage).

Pomalidomide (originally CC-4047), is a derivative of thalidomide and acts as an immunomodulator. It can be taken orally. Phase I trial results showed neutropenia was a frequent side effect.Phase II clinical trials for multiple myeloma and myelofibrosis have reported 'promising results'.

Current developer:  Celgene Corporation.

 1: Lacy MQ, Rajkumar SV. Pomalidomide: a new IMiD with remarkable activity in both multiple myeloma and myelofibrosis. Am J Hematol. 2010 Feb;85(2):95-6. Review. PubMed PMID: 20095057.

2: Mesa RA, Pardanani AD, Hussein K, Wu W, Schwager S, Litzow MR, Hogan WJ, Tefferi A. Phase1/-2 study of Pomalidomide in myelofibrosis. Am J Hematol. 2010 Feb;85(2):129-30. PubMed PMID: 20052748.

3: Escoubet-Lozach L, Lin IL, Jensen-Pergakes K, Brady HA, Gandhi AK, Schafer PH, Muller GW, Worland PJ, Chan KW, Verhelle D. Pomalidomide and lenalidomide induce p21 WAF-1 expression in both lymphoma and multiple myeloma through a LSD1-mediated epigenetic mechanism. Cancer Res. 2009 Sep 15;69(18):7347-56. Epub 2009 Sep 8. PubMed PMID: 19738071.

4: Lacy MQ, Hayman SR, Gertz MA, Dispenzieri A, Buadi F, Kumar S, Greipp PR, Lust JA, Russell SJ, Dingli D, Kyle RA, Fonseca R, Bergsagel PL, Roy V, Mikhael JR, Stewart AK, Laumann K, Allred JB, Mandrekar SJ, Rajkumar SV. Pomalidomide (CC4047) plus low-dose dexamethasone as therapy for relapsed multiple myeloma. J Clin Oncol. 2009 Oct 20;27(30):5008-14. Epub 2009 Aug 31. PubMed PMID: 19720894.

5: Tefferi A, Verstovsek S, Barosi G, Passamonti F, Roboz GJ, Gisslinger H, Paquette RL, Cervantes F, Rivera CE, Deeg HJ, Thiele J, Kvasnicka HM, Vardiman JW, Zhang Y, Bekele BN, Mesa RA, Gale RP, Kantarjian HM. Pomalidomide is active in the treatment of anemia associated with myelofibrosis. J Clin Oncol. 2009 Sep 20;27(27):4563-9. Epub 2009 Aug 3. PubMed PMID: 19652059.

6: Galustian C, Meyer B, Labarthe MC, Dredge K, Klaschka D, Henry J, Todryk S, Chen R, Muller G, Stirling D, Schafer P, Bartlett JB, Dalgleish AG. The anti-cancer agents lenalidomide and pomalidomide inhibit the proliferation and function of T regulatory cells. Cancer Immunol Immunother. 2009 Jul;58(7):1033-45. Epub 2008 Nov 14. PubMed PMID: 19009291.

7: Streetly MJ, Gyertson K, Daniel Y, Zeldis JB, Kazmi M, Schey SA. Alternate day pomalidomide retains anti-myeloma effect with reduced adverse events and evidence of in vivo immunomodulation. Br J Haematol. 2008 Apr;141(1):41-51. PubMed PMID: 18324965.

8: Zhu D, Corral LG, Fleming YW, Stein B. Immunomodulatory drugs Revlimid (lenalidomide) and CC-4047 induce apoptosis of both hematological and solid tumor cells through NK cell activation. Cancer Immunol Immunother. 2008 Dec;57(12):1849-59. Epub 2008 Apr 8. PubMed PMID: 18392823.

9: Xu W, Celeridad M, Sankar S, Webb DR, Bennett BL. CC-4047 promotes Th1 cell differentiation and reprograms polarized human Th2 cells by enhancing transcription factor T-bet. Clin Immunol. 2008 Sep;128(3):392-9. Epub 2008 Jun 20. PubMed PMID: 18565795.

10: Moutouh-de Parseval LA, Verhelle D, Glezer E, Jensen-Pergakes K, Ferguson GD, Corral LG, Morris CL, Muller G, Brady H, Chan K. Pomalidomide and lenalidomide regulate erythropoiesis and fetal hemoglobin production in human CD34+ cells. J Clin Invest. 2008 Jan;118(1):248-58. PubMed PMID: 18064299; PubMed Central PMCID: PMC2117764.

11: Koh KR, Janz M, Mapara MY, Lemke B, Stirling D, Dörken B, Zenke M, Lentzsch S. Immunomodulatory derivative of thalidomide (IMiD CC-4047) induces a shift in lineage commitment by suppressing erythropoiesis and promoting myelopoiesis. Blood. 2005 May 15;105(10):3833-40. Epub 2004 Aug 3. PubMed PMID: 15292067.

12: Schey SA, Fields P, Bartlett JB, Clarke IA, Ashan G, Knight RD, Streetly M, Dalgleish AG. Phase I study of an immunomodulatory thalidomide analog, CC-4047, in relapsed or refractory multiple myeloma. J Clin Oncol. 2004 Aug 15;22(16):3269-76. Epub 2004 Jul 12. PubMed PMID: 15249589.

13: Teo SK, Chen Y, Muller GW, Chen RS, Thomas SD, Stirling DI, Chandula RS. Chiral inversion of the second generation IMiD CC-4047 (ACTIMID ) in human plasma and phosphate-buffered saline. Chirality. 2003 May 5;15(4):348-51. PubMed PMID: 12666243.

14: Ferguson GD, Jensen-Pergakes K, Wilkey C, Jhaveri U, Richard N, Verhelle D, De Parseval LM, Corral LG, Xie W, Morris CL, Brady H, Chan K. Immunomodulatory drug CC-4047 is a cell-type and stimulus-selective transcriptional inhibitor of cyclooxygenase 2. J Clin Immunol. 2007 Mar;27(2):210-20. Epub 2007 Feb 17. PubMed PMID: 17308870.

15: Verhelle D, Corral LG, Wong K, Mueller JH, Moutouh-de Parseval L, Jensen-Pergakes K, Schafer PH, Chen R, Glezer E, Ferguson GD, Lopez-Girona A, Muller GW, Brady HA, Chan KW. Lenalidomide and CC-4047 inhibit the proliferation of malignant B cells while expanding normal CD34+ progenitor cells. Cancer Res. 2007 Jan 15;67(2):746-55. PubMed PMID: 17234786.

16: Shalapour S, Zelmer A, Pfau M, Moderegger E, Costa-Blechschmidt C, van Landeghem FK, Taube T, Fichtner I, Bührer C, Henze G, Seeger K, Wellmann S. The thalidomide analogue, CC-4047, induces apoptosis signaling and growth arrest in childhood acute lymphoblastic leukemia cells in vitro and in vivo. Clin Cancer Res. 2006 Sep 15;12(18):5526-32. PubMed PMID: 17000689.

17: McCarthy DA, Macey MG, Streetly M, Schey SA, Brown KA. The neutropenia induced by the thalidomide analogue CC-4047 in patients with multiple myeloma is associated with an increased percentage of neutrophils bearing CD64. Int Immunopharmacol. 2006 Jul;6(7):1194-203. Epub 2006 Mar 29. PubMed PMID: 16714224.

18: Anderson G, Gries M, Kurihara N, Honjo T, Anderson J, Donnenberg V, Donnenberg A, Ghobrial I, Mapara MY, Stirling D, Roodman D, Lentzsch S. Thalidomide derivative CC-4047 inhibits osteoclast formation by down-regulation of PU.1. Blood. 2006 Apr 15;107(8):3098-105. Epub 2005 Dec 22. PubMed PMID: 16373662.

19: Hernandez-Ilizaliturri FJ, Reddy N, Holkova B, Ottman E, Czuczman MS. Immunomodulatory drug CC-5013 or CC-4047 and rituximab enhance antitumor activity in a severe combined immunodeficient mouse lymphoma model. Clin Cancer Res. 2005 Aug 15;11(16):5984-92. PubMed PMID: 16115943.

20: Streetly M, Hunt BJ, Parmar K, Jones R, Zeldis J, Schey S. Markers of endothelial and haemostatic function in the treatment of relapsed myeloma with the immunomodulatory agent Actimid (CC-4047) and their relationship with venous thrombosis. Eur J Haematol. 2005 Apr;74(4):293-6. PubMed PMID: 15777340.