Sodium Phenylbutyrate (4PBA): A Definitive Collection of Scientific Evidence for Different Cancer Types

Evidence Library: Sodium Phenylbutyrate (4PBA) and Cancer

This page is a curated evidence library focused on sodium phenylbutyrate, often shortened to 4PBA, and how it has been studied in different cancer settings. It is designed to help you quickly locate published research that is relevant to a specific cancer type or a specific treatment combination.

The library is organized in two main ways. First, you will find sections that group studies by context, such as clinical trials and real patient reports, and studies where 4PBA is explored alongside chemotherapy, radiation therapy, or targeted therapies. Second, you will find cancer specific sections, where studies are grouped by tumor type so you can scan only what applies to you.

To find what you need, start with the table of contents and click the section that matches your situation. If you are looking for a combination approach, choose the therapy category first. If you are looking for a specific diagnosis, jump directly to your cancer type and expand the list of studies underneath it. Each entry includes citation details so you can look up the original publication.

Clinical Trials and Real Patient Data on Cancer

  1. Complete response of a recurrent, multicentric malignant glioma in a patient treated with phenylbutyrate.
    2002. Baker MJ, Brem S, Daniels S, Sherman B, Phuphanich S. J Neurooncol. Sep;59(3):239-42. doi: 10.1023/a:1019905127442. PMID: 12241121.

  2. Long-Term Survival and Complete Response of a Patient With Recurrent Diffuse Intrinsic Brainstem Glioblastoma Multiforme.
    2004. Burzynski, SR, Lewy, RI, Weaver, R, Janicki, T, Jurida, G, Khan, M, Larisma, CB, Paszkowiak, J, Szymkowski, B. Integrative Cancer Therapies, 3(3); pp. 257-261.

  3. Preliminary Findings on the Use of Targeted Therapy in Combination with Sodium Phenylbutyrate in Recurrent Advanced Pancreatic Cancer—A Potential Strategy for Improved Survival.
    2014. Burzynski, S., Janicki, T., Burzynski, G., and Brookman, S. Journal of Cancer Therapy; 5, 1072-1091.

  4. Preliminary Findings on the Use of Targeted Therapy in Combination with Sodium Phenylbutyrate in Colorectal Cancer after Failure of Second-Line Therapy—A Potential Strategy for Improved Survival.
    2014. Burzynski, S., Janicki, T., Burzynski, G., and Brookman, S. Journal of Cancer Therapy; 5, 1270-1288.

  5. Phase I dose escalation clinical trial of phenylbutyrate sodium administered twice daily to patients with advanced solid tumors. 2006. Camacho, L. H., Olson, J., Tong, W. P., Young, C. W., Spriggs, D. R., & Malkin, M. G. Investigational New Drugs, 25(2), 131–138.
  6. Pilot study of combination transcriptional modulation therapy with sodium phenylbutyrate and 5-azacytidine in patients with acute myeloid leukemia or myelodysplastic syndrome.
    2006. P. Malask, S. Chanel, L.H. Camacho, S. Soignet, P.O. Dandolfi, I. Guernah, R. Warrell, S. Nimer. Leukemia, 20, 212-217.

  7. Combination of cytotoxic-differentiation therapy with 5-fluoruracil and phenylbutyrate in patients with advanced colorectal cancer.
    2007. Sung, MW; Waxman, S. Anticancer Res; 27(2):995-1001

  8. Long-Term Survival (27.7 Years) Following IV Antineoplaston Therapy (ANP) in a 36-Year-Old Female with a Progressive Diffuse Intrinsic Pontine Glioma (DIPG).
    2021. Burzynski SR, Janicki T, Burzynski GS, Beenken S. Int J Radiol Imaging Technol; 7(1): 073. doi: 10.23937/2572-3235.1510073.

  9. Impact of prolonged infusions of the putative differentiating agent sodium phenylbutyrate on myelodysplastic syndromes and acute myeloid leukemia.
    2002. Gore SD, Weng LJ, Figg WD, Zhai S, Donehower RC, Dover G, Grever MR, Griffin C, Grochow LB, Hawkins A, Burks K, Zabelena Y, Miller CB. Clin Cancer Res. Apr;8(4):963-70. PMID: 11948101.

  10. Oral sodium phenylbutyrate in patients with recurrent malignant gliomas: a dose escalation and pharmacologic study.
    2005. Phuphanich S, Baker SD, Grossman SA, Carson KA, Gilbert MR, Fisher JD, Carducci MA. Neuro Oncol. Apr;7(2):177-82. doi: 10.1215/S1152851704000183. PMID: 15831235; PMCID: PMC1871887.

  11. A Phase I clinical and pharmacological evaluation of sodium phenylbutyrate on a 120-hour infusion schedule.
    2001. Carducci MA, Gilbert J, Bowling MK, Noe D, Eisenberger MA, Sinibaldi V, Zabelina Y, Chen TL, Grochow LB, Donehower RC. Clin Cancer Res; 7(10):3047-55. PMID: 11595694.

  12. A phase I dose escalation and bioavailability study of oral sodium phenylbutyrate in patients with refractory solid tumor malignancies.
    2001. Gilbert J, Baker SD, Bowling MK, Grochow L, Figg WD, Zabelina Y, Donehower RC, Carducci MA. Clin Cancer Res; 7(8):2292-300. PMID: 11489804.

  13. Treatment of Esthesioneuroblastoma and Nonsmall Cell Lung Cancer with Phenylbutyrate.
    2011. S. Burzynski and E. Nagy-Kubove. Journal of Cancer Therapy; Vol. 2 No. 4, pp. 518-522.

  14. Long-term survival over 21 years and pathologically confirmed complete response in pediatric anaplastic astrocytoma: A case report.
    2015. Burzynski, S.R., Burzynski, G.S., Marszalek, A., Janicki, T.J., Martinez-Canca, J.F. J Neurol Stroke 2(6):00072.

  15. A phase II study of antineoplastons A10 and AS2-1 in adult patients with newly-diagnosed anaplastic astrocytoma.
    2015. Burzynski, S.R., Janicki, T.J., Burzynski, G.S., Marszalek, A. Cancer Clin Oncol 4(1):28-38.

  16. Treatment of recurrent glioblastoma multiforme (rGBM) with antineoplaston AS2-1 in combination with targeted therapy.
    2019. Burzynski, S.R., Janicki, T.J., Beenken, S. Cancer Clin Oncol 8(1):1-10.

  17. Long-term survival (over 20 years), complete response and normal childhood development in medulloblastoma treated with antineoplastons A10 and AS2-1. 2015. Burzynski, S.R., Burzynski, G.S., Marszalek, A., Janicki, T.J., Martinez-Canca, J.F. J Neurol Stroke; 2(3):00054.
  18. Long-term survival following treatment with antineoplastons for colon cancer with unresectable multiple liver metastasis: report of a case.
    2003. Ogata Y, Tsuda H, Matono K, Kumabe T, Saitsu H, Hara H, Akagi Y, Araki Y, Sata M and Shirouzu K. Surg Today; 33:448–453.

  19. Oral sodium phenylbutyrate in patients with recurrent malignant gliomas: a dose escalation and pharmacologic study.
    2005. Phuphanich S, Baker SD, Grossman SA, Carson KA, Gilbert MR, Fisher JD, Carducci MA. Neuro Oncol; 7(2):177-82.

  20. Combined DNA methyltransferase and histone deacetylase inhibition in the treatment of myeloid neoplasms.
    2006. Gore SD, Baylin S, Sugar E, Carraway H, Miller CB, Carducci M, Grever M, Galm O, Dauses T, Karp JE, Rudek MA, Zhao M, Smith BD, Manning J, Jiemjit A, Dover G, Mays A, Zwiebel J, Murgo A, Weng LJ, Herman JG. Cancer Res; 66(12):6361-9. doi: 10.1158/0008-5472.CAN-06-0080. PMID: 16778214.

  21. Pharmacokinetics of 5-Azacitidine Administered With Phenylbutyrate in Patients With Refractory Solid Tumors or Hematologic Malignancies.
    2005. Rudek MA, Zhao M, He P, Hartkle C, Gilbert J, Gore SD, Carducci MA, Baker SD. Journal of Clinical Oncology; Vol 23 #17, June 10.

  22. Therapeutic targeting of transcription in acute promyelocytic leukemia by use of an inhibitor of histone deacetylase.
    1998. Warrell RP Jr, He LZ, Richon V, Calleja E, Pandolfi PP. J Natl Cancer Inst; 90(21):1621-5. doi: 10.1093/jnci/90.21.

  23. The preventive effect of antineoplaston AS2-1 on HCC recurrence.
    2003. Tsuda H, Sata M, Kumabe T, Uchida M, Hara H. Oncol Rep; 10(2):391-7.

  24. Antineoplaston AS2-1 for maintenance therapy in liver cancer.
    1997. Tsuda H, Sata M, Saitsu H, Yamana K, Hara H, Yamada S, and Kumabe T. Oncol Rep; 4:1213-1216.

  25. Phase I clinical studies of antineoplaston A5 injections.
    1987. Burzynski SR, Kubove E, Burzynski B. Drugs Exp Clin Res; 13 Suppl 1:37-43.

  26. Treatment of hormonally refractory cancer of the prostate with antineoplaston AS2-1.
    1990. Burzynski SR, Kubove E, Burzynski B. Drugs Exp Clin Res; 16(7):361-9.

  27. Initial clinical study with antineoplaston A2 injections in cancer patients with five years’ follow-up.
    1987. Burzynski SR, Kubove E. Drugs Exp Clin Res; 13 Suppl 1:1-11.

  28. Personalized cancer treatment for ovarian cancer.
    2013. Chumworathayi B. Asian Pac J Cancer Prev; 14(3):1661-4. doi: 10.7314/apjcp.2013.14.3.1661. PMID: 23679252.

Boosts Chemotherapy Effectiveness

  1. Effect of sodium phenylbutyrate on the sensitivity of PC3/DTX-resistant prostate cancer cells to docetaxel.
    2017. Xu YW, Xheng SB, Chen BS, Wen Y, Zhu SW. Nan Fang Yi Ke Da Xue Xue Bao; 37(1): 130-134.

  2. Combination Phenylbutyrate/gemcitabine therapy effectively inhibits in vitro and in vivo growth of NSCLC by intrinsic apoptotic pathways.
    2006. Schniewind B, Heintz K, Kurdow R, Ammerpohl O, Trauzold A, Emme D, Dohrmann P, and Kalthoff H. Journal of Carcinogenesis; 1477-3163-5-25.

  3. Quick response of advanced cancer to chemoradiation therapy with antineoplastons.
    1998. Tsuda H, Sata M, Kumabe T, Hara H, Eriguchi N, Sugita Y, and Nagamatsu H. Oncol Rep; 5: 597-1197.

  4. Personalized cancer treatment for ovarian cancer.
    2013. Chumworathayi B. Asian Pac J Cancer Prev; 14(3): 1661-4. doi: 10.7314/apjcp.2013.14.3.1661. PMID: 23679252.

  5. Antineoplastic action of 5-aza-2’-deoxycytidine and phenylbutyrate on human lung carcinoma cells.
    2002. Boivin A-J, Momparler LF, Hurtubise A, and Momparler RL. Anti-Cancer Drugs; Vol 13, 869-874.

  6. Pilot study of combination transcriptional modulation therapy with sodium phenylbutyrate and 5-azacytidine in patients with acute myeloid leukemia or myelodysplastic syndrome.
    2006. P. Malask, S. Chanel, L.H. Camacho, S. Soignet, P.O. Dandolfi, I. Guernah, R. Warrell, and S. Nimer. Leukemia; 20, 212-217.

  7. Sodium phenylbutyrate induces apoptosis in human retinoblastoma Y79 cells: The effect of combined treatment with the topoisomerase I-inhibitor topotecan.
    2001. Calvaruso G, Carabillo M, Guiliano M, Lauricella M, D’Anneo A, Vento R, and Tesoriere G. International Journal of Oncology; 18: 1233-1237.

  8. ER stress and autophagy are involved in the apoptosis induced by cisplatin in human lung cancer cells.
    2016. Shi S, Tan P, Yan B, Gao R, Zhao J, Wang J, Gup J, Li N, Ma Z. Oncol Rep; 35(5): 2606-14.

  9. Preclinical evaluation of the antineoplastic action of 5-aza-2’-deoxycytidine and different histone deacetylase inhibitors on human Ewing’s sarcoma cells.
    2008. Hurtubise, A, Bernstein, ML, and Momparler, RL. Cancer Cell Int; 8:16.

  10. Addition of histone deacetylase inhibitors in combination therapy.
    2007. Caraway, HE, Gore, SD. J Clin Oncol; 25(15): 1955-1956.

  11. Sequence-dependent antitumor effects of differentiation agents in combination with cell cycle-dependent cytotoxic drugs.
    2007. Verheul, HMW, Qian, DZ, Carducci, MA, and Pili, R. Cancer Chemother Pharmacol; 60(3): 329-339.

  12. Combined DNA methyltransferase and histone deacetylase inhibition in the treatment of myeloid neoplasms.
    2006. Gore SD, Baylin S, Sugar E, Carraway H, Miller CB, Carducci M, Grever M, Galm O, Dauses T, Karp JE, Rudek MA, Zhao M, Smith BD, Manning J, Jiemjit A, Dover G, Mays A, Zwiebel J, Murgo A, Weng LJ, and Herman JG. Cancer Res; 66(12): 6361-9. doi: 10.1158/0008-5472.CAN-06-0080. PMID: 16778214.

  13. Modulation of 5-fluorouracil cytotoxicity through thymidylate synthase and NF-kappaB down-regulation and its application on the radiolabelled iododeoxyuridine therapy on human hepatoma cell.
    2005. Wang, HE, Wu, HC, Kao, SJ, Tseng, FW, Wang, YS, Yu, HM, Chou, SL, Yen, SH, and Chi, KH. Biochem Pharmacol; 69(4): 617-626.

  14. Complementary effects of HDAC inhibitor 4-PB on gap junction communication and cellular export mechanisms support restoration of chemosensitivity of PDAC cells.
    2007. Ammerpohl O, Trauzold A, Schniewind B, Griep U, Pilarsky C, Grutzmann R, Saeger HD, Janssen O, Sipos B, Kloppel G, and Kalthoff H. Br J Cancer; 96(1): 73-81. doi: 10.1038/sj.bjc.6603511. PMID: 17164759; PMCID: PMC2360208.

  15. Pharmacokinetics of 5-Azacitidine Administered With Phenylbutyrate in Patients With Refractory Solid Tumors or Hematologic Malignancies.
    2005. Rudek MA, Zhao M, He P, Hartkle C, Gilbert J, Gore SD, Carducci MA, and Baker SD. Journal of Clinical Oncology; Vol 23 #17, June 10.

  16. Phenylbutyrate interferes with the Fanconi anemia and BRCA pathway and sensitizes head and neck cancer cells to cisplatin.
    2008. Burkitt K, Ljungman M. Mol Cancer; 7, 24.

  17. Apoptosis and Re-expression of p16 Gene in the Myeloma Cell Line U266 Induced by Synergy of Histone Deacetylase Inhibitor and Demethylating Agent.
    2002. Du HL, Qi Y, Shi YJ, Bu DF, Wu SL. Chinese Journal of Cancer; 21(10): 1057-1061.

  18. The differentiation inducers phenylacetate and phenylbutyrate modulate camptothecin sensitivity in colon carcinoma cells in vitro by intracellular acidification.
    2001. Cosentini E, Haberl I, Pertschy P, Teleky B, Mallinger R, Baumgartner G, Wenzl E, and Hamilton G. International Journal of Oncology; 19: 1069-1074.

Boosts Radiotherapy Effectiveness

  1. Phenylbutyrate Sensitizes Human Glioblastoma Cells Lacking Wild-Type P53 Function To Ionizing Radiation.
    2007. Lopez, CA, Feng, FY, Herman, JM, Nyati, MK, Lawrence, TS, and Ljungman, M. Int J Radiation Oncology Biol Phys; Vol. 69, No. 1, pp. 214-220.

  2. Antitumor Histone deacetylase inhibitors suppress cutaneous radiation syndrome: Implications for increasing therapeutic gain in cancer radiotherapy.
    2004. Chung, YL, Wang, AJ, and Yao, LF. Mol Cancer Ther; 3(3): 317-325.

Boosts Targeted Therapy Effectiveness

  1. Preliminary Findings on the Use of Targeted Therapy in Combination with Sodium Phenylbutyrate in Colorectal Cancer after Failure of Second-Line Therapy—A Potential Strategy for Improved Survival.
    2014. Burzynski, S., Janicki, T., Burzynski, G., and Brookman, S. Journal of Cancer Therapy; 5, 1270-1288.

  2. The effect of combined treatment with sodium phenylbutyrate and cisplatin, erlotinib or gefitinib on resistant NSCLC cells.
    2018. Al-Keilani MS, Alzoubi KH, Jaradat SA. Clin Pharmacol; 10: 135-140.

  3. Successful Treatment of Recurrent Triple-Negative Breast Cancer with Combination of Targeted Therapies.
    2011. S. Burzynski, A. Marquis, E. Nagy-Kubove, and T. Janicki. Journal of Cancer Therapy; Vol. 2 No. 3, pp. 372-376.

  4. Preliminary Findings on the Use of Targeted Therapy in Combination with Sodium Phenylbutyrate in Recurrent Advanced Pancreatic Cancer—A Potential Strategy for Improved Survival.
    2014. Burzynski, S, Janicki, T, Burzynski, G, and Brookman, S. Journal of Cancer Therapy; 5, 1072-1091.

  5. Phenylbutyrate Attenuates the Expression of Bcl-XL, DNA-PK, Caveolin-1, and VEGF in Prostate Cancer Cells.
    2001. Goh M, Chen F, Paulsen MT, Yeager AM, Dyer ES, and Ljungman M. Neoplasia; Vol. 3, No. 4, pp. 331-338.

  6. Enhanced differentiation of colon cancer cells induced by combinations of inhibitors of kinases and of histone deacetylases.
    2006. Lea, MA, Ibeh, C, Shah, N, and Moyer, MP. Proceedings of the American Association for Cancer Research Annual Meeting; 47:1184.

How 4PBA Works Across a Wide Range of Cancers

Brain Cancer

  1. Complete response of a recurrent, multicentric malignant glioma in a patient treated with phenylbutyrate.
    2002. Baker MJ, Brem S, Daniels S, Sherman B, Phuphanich S. J Neurooncol; 59(3): 239-42. doi: 10.1023/a:1019905127442. PMID: 12241121.

  2. Oral sodium phenylbutyrate in patients with recurrent malignant gliomas: a dose escalation and pharmacologic study.
    2005. Phuphanich S, Baker SD, Grossman SA, Carson KA, Gilbert MR, Fisher JD, Carducci MA. Neuro Oncol; 7(2): 177-82. doi: 10.1215/S1152851704000183. PMID: 15831235; PMCID: PMC1871887.
  3. Long-term survival over 21 years and pathologically confirmed complete response in pediatric anaplastic astrocytoma: A case report.
    2015. Burzynski, SR, Burzynski, GS, Marszalek, A, Janicki, TJ, Martinez-Canca, JF. J Neurol Stroke; 2(6): 00072.

  4. Long-term Survival (>13 Years) in a Child With Recurrent Diffuse Pontine Gliosarcoma: A Case Report.
    2014. Burzynski SR, Janicki TJ, Burzynski GS, Marszalek A. J Pediatr Hematol Oncol; 36(7): e433–e439. doi: 10.1097/MPH.0000000000000020.

  5. A phase II study of antineoplastons A10 and AS2-1 in adult patients with newly-diagnosed anaplastic astrocytoma.
    2015. Burzynski, SR, Janicki, TJ, Burzynski, GS, Marszalek, A. Cancer Clin Oncol; 4(1): 28-38.

  6. Treatment of recurrent glioblastoma multiforme (rGBM) with antineoplaston AS2-1 in combination with targeted therapy.
    2019. Burzynski, SR, Janicki, TJ, Beenken, S. Cancer Clin Oncol; 8(1): 1-10.

  7. Phenylbutyrate sensitizes human glioblastoma cells lacking wild-type P53 function to ionizing radiation.
    2007. Lopez, CA, Feng, FY, Herman, JM, Nyati, MK, Lawrence, TS, Ljungman, M. Int J Radiation Oncology Biol Phys; Vol. 69, No. 1, pp. 214-220.

  8. Epigenetic cancer therapy makes headway.
    2006. Mack, GS. Journal of the National Cancer Institute; 98(20): 1443-1444.

  9. HDAC inhibitors effectively induce cell type-specific differentiation in human glioblastoma cell lines of different origin.
    2008. Svechnikova I, Almqvist PM, Ekström TJ. Int J Oncol; 32(4): 821-7. PMID: 18360709.

  10. Participation of the chaperone Hsc70 in the trafficking and functional expression of ASIC2 in glioma cells.
    2007. Vila-Carriles WH, Zhou ZH, Bubien JK, Fuller CM, Benos DJ. J Biol Chem; 282(47): 34381-91. doi: 10.1074/jbc.M705354200. PMID: 17878160.

  11. AN-113, a novel prodrug of 4-phenylbutyrate with increased anti-neoplastic activity in glioma cell lines.
    2007. Entin-Meer M, Rephaeli A, Yang X, Nudelman A, Haas-Kogan DA. Cancer Lett; 253(2): 205-214.

  12. Inhibitory effects of phenylbutyrate on the proliferation, morphology, migration and invasiveness of malignant glioma cells.
    1998. Engelhard HH, Homer RJ, Duncan HA, Rozental J. J Neurooncol; 37(2): 97-108. doi: 10.1023/a:1005865125588. PMID: 9524087.

  13. Surface expression of ASIC2 inhibits the amiloride-sensitive current and migration of glioma cells.
    2006. Vila-Carriles WH, Kovacs GG, Jovov B, Zhou ZH, Pahwa AK, Colby G, Esimai O, Gillespie GY, Mapstone TB, Markert JM, Fuller CM, Bubien JK, Benos DJ. J Biol Chem; 281(28): 19220-32. doi: 10.1074/jbc.M603100200. PMID: 16704974.

  14. Histone deacetylase inhibitor 4-phenylbutyrate suppresses GAPDH mRNA expression in glioma cells.
    2004. Appelskog IB, Ammerpohl O, Svechnikova IG, Lui WO, Almqvist PM, Ekström TJ. International Journal of Oncology; 24: 1419-1425.

  15. Histone deacetylase inhibitor 4-phenylbutyrate modulates glial fibrillary acidic protein and connexin 43 expression, and enhances gap-junction communication in human glioblastoma cells.
    2004. Asklune, T, Appelskog IB, Ammerpohl O, Ekström TJ, Almqvist, PM. European Journal of Cancer; Vol. 40, No. 7.

  16. Gene expression profiling of phenylbutyrate-induced differentiation of glioma cells by cDNA array.
    2003. Sun Li-Jun, Huang Qiang, Lan Qing, Du Zi-wei, Hu Geng-xi, Wang Ai-dong. Chinese Journal of Cancer Research; 15(1): 38-42.

  17. Experimental study of combination therapy against human glioma xenograft by differentiation-inducing agent and cytotoxic chemotherapeutic drug.
    2002. Shi MG, Huang Q, Dong J, Sun ZF, Lan Q. Chinese Journal of Cancer; 21(10): 190-1094.

  18. Long-Term Survival (27.7 Years) Following IV Antineoplaston Therapy (ANP) in a 36-Year-Old Female with a Progressive Diffuse Intrinsic Pontine Glioma (DIPG).
    2021. Burzynski SR, Janicki T, Burzynski GS, Beenken S. Int J Radiol Imaging Technol; 7(1): 073. doi: 10.23937/2572-3235.1510073.

  19. Long-term survival and complete response of a patient with recurrent diffuse intrinsic brainstem glioblastoma multiforme.
    2004. Burzynski, SR, Lewy, RI, Weaver, R, Janicki, T, Jurida, G, Khan, M, Larisma, CB, Paszkowiak, J, Szymkowski, B. Integrative Cancer Therapies; 3(3): 257-261.

Breast Cancer

  1. Successful Treatment of Recurrent Triple-Negative Breast Cancer with Combination of Targeted Therapies.
    2011. Burzynski S, Marquis A, Nagy-Kubove E, Janicki T. Journal of Cancer Therapy; Vol. 2 No. 3, pp. 372-376.

  2. Epigenetic regulation of ZEB1-RAB25/ESRP1 axis plays a critical role in phenylbutyrate treatment-resistant breast cancer. 2016. Kikuchi M, Yamashita K, Waraya N, Ushiku H, Kojo K, Ema A, Kosaka Y, Katoh H, Sengoku N, Enomoto T, Tanino H, Sawanobori M, Watanabe T. Oncotarget; 7(2): 1741-1753.
  3. Short-term modulation of cell proliferation and apoptosis and preventive/therapeutic efficacy of various agents in a mammary cancer model.
    2007. Christov K, Grubbs CJ, Shilkaitis A, Juliana MM, Lubet RA. Clin Cancer Res; 13(18 Part 1): 5488-5496.

  4. Phenylbutyrate inhibits the invasive properties of prostate and breast cancer cell lines in the sea urchin embryo basement membrane invasion assay.
    2002. Dyer ES, Paulsen MT, Markwart SM, Goh M, Livant DL, Ljungman M. Int J Cancer; 101: 496-499.

  5. Polypeptides that inhibit human breast cancer cell division.
    1979. Beall PT, Szopa B, Burzynski SR, Hazlewood CF. Cancer Biochem Biophys; 3(2): 93-6.

  6. Potential utility of antineoplaston A-10 levels in breast cancer.
    2000. Badria F, Mabed M, Khafagy W, Abou-Zeid L. Cancer Lett; 155(1): 67-70. doi: 10.1016/s0304-3835(00)00408-0. PMID: 10814881.

Blood Cancer

  1. Pilot study of combination transcriptional modulation therapy with sodium phenylbutyrate and 5-azacytidine in patients with acute myeloid leukemia or myelodysplastic syndrome.
    2006. Malask P, Chanel S, Camacho LH, Soignet S, Dandolfi PO, Guernah I, Warrell R, Nimer S. Leukemia; 20: 212-217.

  2. Impact of prolonged infusions of the putative differentiating agent sodium phenylbutyrate on myelodysplastic syndromes and acute myeloid leukemia.
    2002. Gore SD, Weng LJ, Figg WD, Zhai S, Donehower RC, Dover G, Grever MR, Griffin C, Grochow LB, Hawkins A, Burks K, Zabelena Y, Miller CB. Clin Cancer Res; 8(4): 963-70. PMID: 11948101.

  3. Impact of the putative differentiating agents sodium phenylbutyrate and sodium phenylacetate on proliferation, differentiation, and apoptosis of primary neoplastic myeloid cells.
    1997. Gore SD, Samid D, Weng LJ. Clin Cancer Res; 3(10): 1755-62. PMID: 9815560.
  1. Pharmacokinetics of 5-Azacitidine Administered With Phenylbutyrate in Patients With Refractory Solid Tumors or Hematologic Malignancies.
    2005. Rudek MA, Zhao M, He P, Hartkle C, Gilbert J, Gore SD, Carducci MA, Baker SD. J Clin Oncol; Vol 23 #17, June 10.

  2. New drugs in acute myeloid leukemia.
    2008. Estey E. Seminars in Oncology; 35(4): 439-448.

  3. Targets of epigenetics therapy—Gene reactivation as a novel approach in MDS treatment.
    2007. Claus R, Router B, Loubbert M. Cancer Treat Rec; 33(Suppl. 1): S47-S52.

  4. Therapeutic targeting of transcription in acute promyelocytic leukemia by use of an inhibitor of histone deacetylase.
    1998. Warrell RP Jr, He LZ, Richon V, Calleja E, Pandolfi PP. J Natl Cancer Inst; 90(21): 1621-5. doi: 10.1093/jnci/90.21.

  5. Butyrate and phenylacetate as differentiating agents: practical problems and opportunities.
    1995. Newmark HL, Young CW. J Cell Biochem Suppl; 22: 247-53. doi: 10.1002/jcb.240590831. PMID: 8538206.

  6. Phenylbutyrate-induced G1 arrest and apoptosis in myeloid leukemia cells: structure-function analysis.
    1999. DiGiuseppe JA, Weng LJ, Yu KH, Fu S, Kastan MB, Samid D, Gore SD. Leukemia; 13(8): 1243-53. doi: 10.1038/sj.leu.2401471. PMID: 10450753.

  7. Do histone deacetylase inhibitors have a place in the treatment of myelodysplastic syndromes?
    2007. Gore SD. Leukemia Research; 31(Suppl. 1): S21.

  8. Combined DNA methyltransferase and histone deacetylase inhibition in the treatment of myeloid neoplasms.
    2006. Gore SD, Baylin S, Sugar E, Carraway H, Miller CB, Carducci M, Grever M, Galm O, Dauses T, Karp JE, Rudek MA, Zhao M, Smith BD, Manning J, Jiemjit A, Dover G, Mays A, Zwiebel J, Murgo A, Weng LJ, Herman JG. Cancer Res; 66(12): 6361-9. doi: 10.1158/0008-5472.CAN-06-0080. PMID: 16778214.

  9. The novel histone deacetylase inhibitor OSU-HDAC42 has class I and II histone deacetylase (HDAC) inhibitory activity and represents a novel therapy for chronic lymphocytic leukemia.
    2006. West DA, Lucas DM, Davis ME, De lay MD, Johnson AJ, Guster SE, Freitas MA, Parthun MR, Wang D, Kulp SK, Grever MR, Chen CS, Byrd JC. Blood; 108(11 Part 1): 794A-795A.

  10. Combined targeting of the epigenetic silence in leukemia: Cooperating activities of DNA methylation and histone deacetylation inhibitors.
    2005. Luebbert M. Leukemia Research; 29(7): 727-728.

  11. Apoptosis and Re-expression of p16 Gene in the Myeloma Cell Line U266 Induced by Synergy of Histone Deacetylase Inhibitor and Demethylating Agent.
    2002. Du HL, Qi Y, Shi YJ, Bu DF, Wu SL. Chinese Journal of Cancer; 21(10): 1057-1061.

Bladder Cancer

  1. Inhibition of cell growth and induction of apoptosis in bladder cancer cell lines by a novel histone deacetylase inhibitor derived from phenylbutyrate.
    2005. Mortazavi A, Hoot DR, Carlton PS, Wang S, Degroff VL, Lu Q, Kulp S, Chen CS, Clinton SK. Proceedings of the American Association for Cancer Research Annual Meeting; 46(Suppl S): 422.

  2. Inhibition of bladder tumour growth by histone deacetylase inhibitor.
    2010. Ozawa A, Tanji N, Kikugawa T, Sasaki T, Yanagihara Y, Miura N, Yokoyama M. BJU Int; 105(8): 1181-6. doi: 10.1111/j.1464-410X.2009.08795.x. Epub 2009 Aug 13. PMID: 19681894.

  3. Initial clinical study with antineoplaston A2 injections in cancer patients with five years’ follow-up.
    1987. Burzynski SR, Kubove E. Drugs Exp Clin Res; 13(Suppl 1): 1-11.

  4. MAGE-A9 mRNA and protein expression in bladder cancer.
    2007. Picard V, Bergeron A, Larue H, Fradet Y. Int J Cancer; 120(10): 2170-2177.

Cervical Cancer

  1. Phenylbutyrate inhibits growth of cervical carcinoma cells independent of HPV type and copy number.
    2003. Finzer P., Stöhr M., Seibert N., Rösl F. J. Cancer Res. Clin. Oncol.; 129: 107-113.

Colon Cancer

  1. Preliminary Findings on the Use of Targeted Therapy in Combination with Sodium Phenylbutyrate in Colorectal Cancer after Failure of Second-Line Therapy—A Potential Strategy for Improved Survival.
    2014. Burzynski S., Janicki T., Burzynski G., Brookman S. Journal of Cancer Therapy; 5: 1270-1288.

  2. Long-term survival following treatment with antineoplastons for colon cancer with unresectable multiple liver metastasis: report of a case.
    2003. Ogata Y, Tsuda H, Matono K, Kumabe T, Saitsu H, Hara H, Akagi Y, Araki Y, Sata M, Shirouzu K. Surg Today; 33: 448–453.

  3. Combination of cytotoxic-differentiation therapy with 5-fluorourcail and phenylbutyrate in patients with advanced colorectal cancer.
    2007. Sung MW, Waxman S. Anticancer Res; 27(2): 995-1001.

  4. The effects of short-chain fatty acids on human colon cancer cell phenotype are associated with histone hyperacetylation.
    2002. Hinnebusch BF, Meng S, Wu JT, Archer SY, Hodin RA. J Nutr; 132(5): 1012-1017. doi: 10.1093/jn/132.5.1012. PMID: 11983830.

  5. Enhanced differentiation of colon cancer cells induced by combinations of inhibitors of kinases and of histone deacetylases.
    2006. Lea MA, Ibeh C, Shah N, Moyer MP. Proceedings of the American Association for Cancer Research Annual Meeting; 47: 1184.

  6. Sodium Phenylbutyrate sensitizes TRAIL-mediated apoptosis by induction of transcription from the DR5 gene promoter through Ap1 sites in colon cancer cells. 2001. Kim YH, Park JW, Lee JY, et al. Carcinogenesis; 25: 1813-1820.
  7. Phenylbutyrate-Induced Apoptosis is Associated with Inactivation of NF-кB in HT-29 colon cancer cells.
    2002. Feinman R, Clarke KO, Harrison LE. Cancer Chemother Pharmacol; 49: 27-34.

  8. The differentiation inducers phenylacetate and phenylbutyrate modulate camptothecin sensitivity in colon carcinoma cells in vitro by intracellular acidification.
    2001. Cosentini E, Haberl I, Pertschy P, Teleky B, Mallinger R, Baumgartner G, Wenzl E, Hamilton G. International Journal of Oncology; 19: 1069-1074.

Esophageal Cancer

  1. Clinical aspects of molecular biology for the diagnosis and treatment of esophageal cancer.
    2006. Brock MV. Esophagus; 3(3): 91-94.

Lymphoma

  1. Pilot study of sodium phenylbutyrate as adjuvant in cyclophosphamide-resistant endemic Burkitt’s lymphoma.
    2007. Phillips JA, Griffin BE. Trans R Soc Trop Med Hyg; 101(12): 1265-1269.

Liver Cancer

  1. Apoptosis and tumor remission in liver tumor xenografts by 4-phenylbutyrate.
    2003. Svechnikova I, Gray SG, Kundrotiene J, Ponthan F, Kogner P, Ekström T. Int J Oncol; 22: 579-588.

  2. Antineoplaston treatment for advanced hepatocellular carcinoma.
    1998. Kumabe T, Tsuda H, Uchida M, Ogoh Y, Hayabuchi N, Sata M, Nakashima O, Hara H. Oncol Rep; 5(6): 1363-1367. doi: 10.3892/or.5.6.1363.

  3. Antineoplaston AS2-1 for maintenance therapy in liver cancer.
    1997. Tsuda H, Sata M, Saitsu H, Yamana K, Hara H, Yamada S, Kumabe T. Oncol Rep; 4: 1213-1216.

  4. The preventive effect of antineoplaston AS2-1 on HCC recurrence.
    2003. Tsuda H, Sata M, Kumabe T, Uchida M, Hara H. Oncol Rep; 10(2): 391-397.

  5. Inhibitory effect of antineoplaston A10 and AS2-1 on human hepatocellular carcinoma.
    1996. Tsuda H, Iemura A, Sata M, Uchida M, Yamana K, Hara H. Kurume Med J; 43(2): 137-147. doi: 10.2739/kurumemedj.43.137.

  6. Expression of histone deacetylase 4 in human liver carcinoma cell line Bel-7402 and its significance.
    2007. Meng M, Wang CT, Jiang JM, Zhang JC, Jiang JJ, Jin CJ. Chin J Cancer Biother; 14(2): 153-157.

  7. Efficacy of a novel histone deacetylase inhibitor in murine models of hepatocellular carcinoma.
    2007. Lu YS, Kashida Y, Kulp SK, Wang YC, Wang D, Hung JH, Tang M, Lin ZZ, Chen TJ, Cheng AL, Chen CS. Hepatology; 46(4): 1119-1130. doi: 10.1002/hep.21804. Erratum in: Hepatology; 2019 Jul;70(1): 454. doi: 10.1002/hep.30802. PMID: 17654699.

  8. p21waf1/Cip1 partially mediates apoptosis in hepatocellular carcinoma cells.
    2007. Svechnikova I, Ammerpohl O, Ekström TJ. Biochem Biophys Res Commun; 354(2): 466-471. doi: 10.1016/j.bbrc.2006.12.222. Epub 2007 Jan 10. PMID: 17239344.

  9. Modulation of 5-fluorouracil cytotoxicity through thymidylate synthase and NF-kappa B down-regulation and its application on the radiolabelled iododeoxyuridine therapy on human hepatoma cell.
    2005. Wang HE, Wu HC, Kao SJ, Tseng FW, Wang YS, Yu HM, Chou SL, Yen SH, Chi KH. Biochem Pharmacol; 69(4): 617-626.

Lung Cancer

  1. Treatment of Esthesioneuroblastoma and Non-small Cell Lung Cancer with Phenylbutyrate.
    2011. Burzynski S., Nagy-Kubove E. Journal of Cancer Therapy; 2(4): 518-522.

  2. Combination Phenylbutyrate/gemcitabine therapy effectively inhibits in vitro and in vivo growth of NSCLC by intrinsic apoptotic pathways.
    2006. Schniewind B., Heintz K., Kurdow R., Ammerpohl O., Trauzold A., Emme D., Dohrmann P., Kalthoff H. Journal of Carcinogenesis; 5: 25. doi: 10.1186/1477-3163-5-25.

  3. The effect of combined treatment with sodium phenylbutyrate and cisplatin, erlotinib, or gefitinib on resistant NSCLC cells.
    2018. Al-Keilani MS, Alzoubi KH, Jaradat SA. Clin Pharmacol; 10: 135-140.

  4. Sodium 4-Phenylbutyrate Induces Apoptosis of Human Lung Carcinoma Cells Through Activating JNK Pathway.
    2004. Zhang X., Wei L., Yang Y., Yu Q. Journal of Cellular Biochemistry; 93: 819-829.

  5. Inhibition of DNA methylation and histone deacetylation prevents murine lung cancer.
    2003. Belinsky SA, Klinge DM, Stidley CA, Issa JP, Herman JG, March TH, Baylin SB. Cancer Res; 63(21): 7089-7093. PMID: 14612500.

  6. Silencing of genes by promoter hypermethylation: key event in rodent and human lung cancer.
    2005. Belinsky SA. Carcinogenesis; 26(9): 1481-1487.

  7. Antineoplastic action of 5-aza-2’-deoxycytidine and phenylbutyrate on human lung carcinoma cells.
    2002. Boivin A-J., Momparler LF, Hurtubise A., Momparler RL. Anti-Cancer Drugs; 13: 869-874.

  8. Enhanced Growth Inhibition by Combination Differentiation Therapy with Ligands of Peroxisome Proliferator-activated Receptor-γ and Inhibitors of Histone Deacetylase in Adenocarcinoma of the Lung.
    2002. Chang T-H, Szabo E. Clinical Cancer Research; 8: 1206-1212.

Gastric (Stomach) Cancer

  1. Growth inhibitory effect of 4-phenyl butyric acid on human gastric cancer cells is associated with cell cycle arrest.
    2012. Li LZ, Deng HX, Lou WZ, Sun XY, Song MW, Tao J, Xiao BX, Guo JM. World J Gastroenterol; 18(1): 79-83. doi: 10.3748/wjg.v18.i1.79.

Head and Neck Cancer

  1. Sodium Phenylbutyrate Inhibits Tumor Growth and the Epithelial-Mesenchymal Transition of Oral Squamous Cell Carcinoma In Vitro and In Vivo.
    2018. Qian K, Sun L, Zhou G, Ge H, Meng Y, Li J, Li X, Fang X. Cancer Biother Radiopharm; 33(4): 139-145.

  2. Non-viral delivery of the connexin 43 gene with histone deacetylase inhibitor to human nasopharyngeal tumor cells enhances gene expression and inhibits in vivo tumor growth.
    2007. Hattori Y, Fukushima M, Maitani Y. Int J Oncol; 30(6): 1427-1439. PMID: 17487363.

  3. Phenylbutyrate interferes with the Fanconi anemia and BRCA pathway and sensitizes head and neck cancer cells to cisplatin.
    2008. Burkitt K., Ljungman M. Mol Cancer; 7: 24.

Medulloblastoma

  1. Long-term survival (over 20 years), complete response and normal childhood development in medulloblastoma treated with antineoplastons A10 and AS2-1. 2015. Burzynski SR, Burzynski GS, Marszalek A, Janicki TJ, Martinez-Canca JF. J Neurol Stroke; 2(3): 00054.
  2. Phenylbutyrate and Phenylacetate Induce Differentiation and Inhibit Proliferation of Human Medulloblastoma Cells.
    2004. Li XN, Parikh S, Shu Q, Jung HL, Chow CW, Perlaky L, Leung HCE, Su J, Blaney S, Lau CC. Clin Cancer Res; 10(3): 1150-1159.

Melanoma

  1. Histone deacetylase inhibitors radiosensitize human melanoma cells by suppressing DNA repair activity.
    2005. Munshi A, Kurland JF, Nishikawa T, Tanaka T, Hobbs ML, Tucker SL, Ismail S, Stevens C, Meyn RE. Clin Cancer Res; 11(13): 4912-49122.

  2. Transcriptional upregulation of TGF-alpha by phenylacetate and phenylbutyrate is associated with differentiation of human melanoma cells.
    1995. Liu L, Hudgins WR, Miller AC, Chen LC, Samid D. Cytokine; 7(5): 449-456. doi: 10.1006/cyto.1995.0061. PMID: 7578983.

  3. 14-3-3sigma gene silencing during melanoma progression and its role in cell cycle control and cellular senescence.
    2009. Schultz J, Ibrahim SM, Vera J, Kunz M. Mol Cancer; 8: 53. doi: 10.1186/1476-4598-8-53. PMID: 19642975; PMCID: PMC2723074.

Neuroblastoma

  1. Effect of butyrate analogues on proliferation and differentiation in human neuroblastoma cell lines.
    1998. Rocchi P, Ferreri AM, Magrini E, Perocco P. Anticancer Res; 18(2A): 1099-1103. PMID: 9615772.

Ovarian Cancer

  1. Personalized cancer treatment for ovarian cancer.
    2013. Chumworathayi B. Asian Pac J Cancer Prev; 14(3): 1661-1664. doi: 10.7314/apjcp.2013.14.3.1661. PMID: 23679252.

  2. Growth inhibitory effects of aromatic fatty acids on ovarian tumor cell lines.
    1998. Melichar B, Ferrandina G, Verschraegen CF, Loercher A, Abbruzzese JL, Freedman RS. Clin Cancer Res; 4(12): 3069-3076.

  3. Histone deacetylase inhibitors and aspirin interact synergistically to induce cell death in ovarian cancer cells.
    2008. Sonnemann J, Hüls I, Sigler M, Palani CD, Hong LT, Völker U, Kroemer HK, Beck JF. Oncology Reports; 20(1): 219-224.

Pancreatic Cancer

  1. Preliminary Findings on the Use of Targeted Therapy in Combination with Sodium Phenylbutyrate in Recurrent Advanced Pancreatic Cancer—A Potential Strategy for Improved Survival.
    2014. Burzynski S., Janicki T., Burzynski G., Brookman S. Journal of Cancer Therapy; 5: 1072-1091.

  2. Antiproliferative Effect of Phenylbutyrate in AsPC-1 Pancreatic Cancer Cell Line.
    2006. Jin C., Park JW, Choi JW, Kang H, Jin GB, Choi SM, Park SS, Ryu D, Jang LC. Korean J Hepatobiliary Pancreat Surg; 10(1): 1-9.

  3. Impact of the histone deacetylase inhibitor 4-phenylbutyrate on the clearance of apoptotic pancreatic carcinoma cells by human macrophages.
    2012. Welsch L, Welsch T, Dovzhanskiy DI, Felix K, Giese NA, Krysko DV, Werner J. Int J Oncol; 40(2): 427-435. doi: 10.3892/ijo.2011.1239. PMID: 22020984.

  4. Complementary effects of HDAC inhibitor 4-PB on gap junction communication and cellular export mechanisms support restoration of chemosensitivity of PDAC cells.
    2007. Ammerpohl O, Trauzold A, Schniewind B, Griep U, Pilarsky C, Grutzmann R, Saeger HD, Janssen O, Sipos B, Kloppel G, Kalthoff H. Br J Cancer; 96(1): 73-81. doi: 10.1038/sj.bjc.6603511. PMID: 17164759; PMCID: PMC2360208.

Prostate Cancer

  1. Treatment of hormonally refractory cancer of the prostate with antineoplaston AS2-1.
    1990. Burzynski SR, Kubove E, Burzynski B. Drugs Exp Clin Res; 16(7): 361-369.

  2. Sodium phenylbutyrate antagonizes prostate cancer through the induction of apoptosis and attenuation of cell viability and migration.
    2016. Xu Y, Zheng S, Chen B, et al. Onco Targets Ther; 9: 2825.

  3. Phenylbutyrate induces apoptosis in human prostate cancer and is more potent than phenylacetate.
    1996. Carducci MA, Nelson JB, Chan-Tack KM, Ayyagari SR, Sweatt WH, Campbell PA, Nelson WG, Simons JW. Clin Cancer Res; 2(2): 379-387. PMID: 9816181.

  4. Effect of sodium phenylbutyrate on the sensitivity of PC3/DTX-resistant prostate cancer cells to docetaxel.
    2017. Xu YW, Zheng SB, Chen BS, Wen Y, Zhu SW. Nan Fang Yi Ke Da Xue Xue Bao; 37(1): 130-134.

  5. Antitumor effects of a novel phenylbutyrate-based histone deacetylase inhibitor, (S)-HDAC-42, in prostate cancer.
    2006. Kulp SK, Chen CS, Wang DS, Chen CY, Chen CS. Clin Cancer Res; 12(17): 5199-5206. doi: 10.1158/1078-0432.CCR-06-0429. Retraction in: Clin Cancer Res; 2019 May 1;25(9): 2940. doi: 10.1158/1078-0432.CCR-19-0731. PMID: 16951239.

  6. Increases in NMR-visible lipid and glycerophosphocholine during phenylbutyrate-induced apoptosis in human prostate cancer cells.
    2005. Milkevitch M, Shim H, Pilatus U, Pickup S, Wehrle JP, Samid D, Poptani H, Glickson JD, Delikatny EJ. Biochim Biophys Acta; 1734(1): 1-12.

  7. Phenylbutyrate attenuates the expression of Bcl-XL, DNA-PK, Caveolin-1, and VEGF in prostate cancer cells.
    2001. Goh M, Chen F, Paulsen MT, Yeager AM, Dyer ES, Ljungman M. Neoplasia; 3(4): 331-338.

  8. Phenylbutyrate inhibits the invasive properties of prostate and breast cancer cell lines in the sea urchin embryo basement membrane invasion assay.
    2002. Dyer ES, Paulsen MT, Markwart SM, Goh M, Livant DL, Ljungman M. Int J Cancer; 101: 496-499.

Retinoblastoma Cancer

  1. Sodium phenylbutyrate induces apoptosis in human retinoblastoma Y79 cells: The effect of combined treatment with the topoisomerase I-inhibitor topotecan.
    2001. Calvaruso G, Carabillo M, Guiliano M, Lauricella M, D’Anneo A, Vento R, Tesoriere G. Int J Oncol; 18: 1233-1237.

Thyroid Cancer

  1. Effects of sodium phenylbutyrate on invasive ability of human thyroid follicular carcinoma cell line and expression of MMP-9 and TIMP-1.
    1994. Sun K, Liu C. Chinese Journal of Cancer Biotherapy; 6.
Scroll to Top