Galinpepimut-S (GPS): an investigational agent for the treatment of acute myeloid leukemia

References

• Patel SA, Gerber JM, User’s A. Guide to novel therapies for acute myeloid leukemia. Clin Lymphoma Myeloma Leuk. 2020 May;20(5):277–288.
   PubMed Web of Science ®Google Scholar
• Lin C, Li Y. The role of peptide and DNA vaccines in myeloid leukemia immunotherapy. Cancer Cell Int. 2013 Feb;13(1):13.
   PubMedGoogle Scholar
• Curran KJ, Brentjens RJ. Chimeric antigen receptor T cells for cancer immunotherapy. J Clin Oncol. 2015 May;33(15):1703–1706.
   PubMed Web of Science ®Google Scholar
• O’Hear C, Heiber JF, Schubert I, et al. Anti-CD33 chimeric antigen receptor targeting of acute myeloid leukemia. Haematologica. 2015 Mar;100(3):336–344.
   PubMed Web of Science ®Google Scholar
• Thomas ED, Buckner CD, Banaji M, et al. One hundred patients with acute leukemia treated by chemotherapy, total body irradiation, and allogeneic marrow transplantation. Blood. 1977 Apr;49(4):511–533.
   PubMed Web of Science ®Google Scholar
• Melief CJ, Kast WM. T-cell immunotherapy of tumors by adoptive transfer of cytotoxic T lymphocytes and by vaccination with minimal essential epitopes. Immunol Rev. 1995 June;145:167–177.
   PubMed Web of Science ®Google Scholar
• Rosenberg SA. Progress in human tumour immunology and immunotherapy. Nature. 2001 May;411(6835):380–384.
   PubMed Web of Science ®Google Scholar
• Ritz J. Tumor immunity: will new keys unlock the door? J Clin Oncol. 1994 Feb;12(2):237–238.
   PubMed Web of Science ®Google Scholar
• Miwa H, Beran M, Saunders GF. Expression of the Wilms’ tumor gene (WT1) in human leukemias. Leukemia. 1992 May;6(5):405–409.
   PubMed Web of Science ®Google Scholar
• Miyagi T, Ahuja H, Kubota T, et al. Expression of the candidate Wilm’s tumor gene, WT1, in human leukemia cells. Leukemia. 1993 Jul;7(7):970–977.
   PubMed Web of Science ®Google Scholar
• Inoue K, Sugiyama H, Ogawa H, et al. WT1 as a new prognostic factor and a new marker for the detection of minimal residual disease in acute leukemia. Blood. 1994 Nov;84(9):3071–3079.
   PubMed Web of Science ®Google Scholar
• Brieger J, Weidmann E, Fenchel K, et al. The expression of the Wilms’ tumor gene in acute myelocytic leukemias as a possible marker for leukemic blast cells. Leukemia. 1994 Dec;8(12):2138–2143.
   PubMed Web of Science ®Google Scholar
• Menssen HD, Renkl HJ, Rodeck U, et al. Presence of Wilms’ tumor gene (wt1) transcripts and the WT1 nuclear protein in the majority of human acute leukemias. Leukemia. 1995 Jun;9(6):1060–1067.
   PubMed Web of Science ®Google Scholar
• Bergmann L, Miething C, Maurer U, et al. High levels of Wilms’ tumor gene (wt1) mRNA in acute myeloid leukemias are associated with a worse long-term outcome. Blood. 1997 Aug;90(3):1217–1225.
   PubMed Web of Science ®Google Scholar
• Menssen HD, Renkl HJ, Rodeck U, et al. Detection by monoclonal antibodies of the Wilms’ tumor (WT1) nuclear protein in patients with acute leukemia. Int J Cancer. 1997 Mar;70(5):518–523.
   PubMed Web of Science ®Google Scholar
• Tamaki H, Ogawa H, Ohyashiki K, et al. The Wilms’ tumor gene WT1 is a good marker for diagnosis of disease progression of myelodysplastic syndromes. Leukemia. 1999 Mar;13(3):393–399.
   PubMed Web of Science ®Google Scholar
• Ogawa H, Tamaki H, Ikegame K, et al. The usefulness of monitoring WT1 gene transcripts for the prediction and management of relapse following allogeneic stem cell transplantation in acute type leukemia. Blood. 2003 Mar;101(5):1698–1704.
   PubMed Web of Science ®Google Scholar
• Call KM, Glaser T, Ito CY, et al. Isolation and characterization of a zinc finger polypeptide gene at the human chromosome 11 Wilms’ tumor locus. Cell. 1990 Feb;60(3):509–520.
   PubMed Web of Science ®Google Scholar
• Gessler M, Poustka A, Cavenee W, et al. Homozygous deletion in Wilms tumours of a zinc-finger gene identified by chromosome jumping. Nature. 1990 Feb;343(6260):774–778.
   PubMed Web of Science ®Google Scholar
• Drummond IA, Madden SL, Rohwer-Nutter P, et al. Repression of the insulin-like growth factor II gene by the Wilms tumor suppressor WT1. Science. 1992 Jul;257(5070):674–678.
   PubMed Web of Science ®Google Scholar
• Englert C, Hou X, Maheswaran S, et al. WT1 suppresses synthesis of the epidermal growth factor receptor and induces apoptosis. EMBO J. 1995 Oct;14(19):4662–4675.
   PubMed Web of Science ®Google Scholar
• Goodyer P, Dehbi M, Torban E, et al. Repression of the retinoic acid receptor-alpha gene by the Wilms’ tumor suppressor gene product, wt1. Oncogene. 1995 Mar;10(6):1125–1129.
   PubMed Web of Science ®Google Scholar
• Hewitt SM, Hamada S, McDonnell TJ, et al. Regulation of the proto-oncogenes bcl-2 and c-myc by the Wilms’ tumor suppressor gene WT1. Cancer Res. 1995 Nov;55(22):5386–5389.
   PubMed Web of Science ®Google Scholar
• Kim J, Prawitt D, Bardeesy N, et al. The Wilms’ tumor suppressor gene (wt1) product regulates Dax-1 gene expression during gonadal differentiation. Mol Cell Biol. 1999 Mar;19(3):2289–2299.
   PubMed Web of Science ®Google Scholar
• Oka Y, Tsuboi A, Kawakami M, et al. Development of WT1 peptide cancer vaccine against hematopoietic malignancies and solid cancers. Curr Med Chem. 2006;13(20):2345–2352.
   PubMed Web of Science ®Google Scholar
• Oka Y, Tsuboi A, Elisseeva OA, et al. WT1 peptide cancer vaccine for patients with hematopoietic malignancies and solid cancers. ScientificWorldJournal. 2007 May;7:649–665.
   PubMedGoogle Scholar
• Oji Y, Ogawa H, Tamaki H, et al. Expression of the Wilms’ tumor gene WT1 in solid tumors and its involvement in tumor cell growth. Jpn J Cancer Res. 1999 Feb;90(2):194–204.
   PubMedGoogle Scholar
• Oji Y, Miyoshi S, Maeda H, et al. Overexpression of the Wilms’ tumor gene WT1 in de novo lung cancers. Int J Cancer. 2002 Jul;100(3):297–303.
   PubMed Web of Science ®Google Scholar
• Miyoshi Y, Ando A, Egawa C, et al. High expression of Wilms’ tumor suppressor gene predicts poor prognosis in breast cancer patients. Clin Cancer Res. 2002 May;8(5):1167–1171.
   PubMed Web of Science ®Google Scholar
• Oji Y, Miyoshi Y, Koga S, et al. Overexpression of the Wilms’ tumor gene WT1 in primary thyroid cancer. Cancer Sci. 2003 Jul;94(7):606–611.
   PubMed Web of Science ®Google Scholar
• Oji Y, Yamamoto H, Nomura M, et al. Overexpression of the Wilms’ tumor gene WT1 in colorectal adenocarcinoma. Cancer Sci. 2003 Aug;94(8):712–717.
   PubMed Web of Science ®Google Scholar
• Yamagami T, Sugiyama H, Inoue K, et al. Growth inhibition of human leukemic cells by WT1 (Wilms tumor gene) antisense oligodeoxynucleotides: implications for the involvement of WT1 in leukemogenesis. Blood. 1996 Apr;87(7):2878–2884.
   PubMed Web of Science ®Google Scholar
• Hosen N, Sonoda Y, Oji Y, et al. Very low frequencies of human normal CD34+ haematopoietic progenitor cells express the Wilms’ tumour gene WT1 at levels similar to those in leukaemia cells. Br J Haematol. 2002 Feb;116(2):409–420.
   PubMed Web of Science ®Google Scholar
• Baird PN, Simmons PJ. Expression of the Wilms’ tumor gene (WT1) in normal hemopoiesis. Exp Hematol. 1997 Apr;25(4):312–320.
   PubMed Web of Science ®Google Scholar
• Menssen HD, Renkl HJ, Entezami M, et al. Wilms’ tumor gene expression in human CD34+ hematopoietic progenitors during fetal development and early clonogenic growth. Blood. 1997 May;89(9):3486–3487.
   PubMed Web of Science ®Google Scholar
• Tsuboi A, Oka Y, Ogawa H, et al. Constitutive expression of the Wilms’ tumor gene WT1 inhibits the differentiation of myeloid progenitor cells but promotes their proliferation in response to granulocyte-colony stimulating factor (G-CSF). Leuk Res. 1999 May;23(5):499–505.
   PubMed Web of Science ®Google Scholar
• Kim SC, Yoo NC, Hahn JS, et al. Monitoring of WT-1 gene expression in peripheral blood of patients with acute leukemia by semiquantitative RT-PCR; possible marker for detection of minimal residual leukemia. Yonsei Med J. 1997 Aug;38(4):212–219.
   PubMedGoogle Scholar
• Maslak PG, Dao T, Bernal Y, et al. Phase 2 trial of a multivalent WT1 peptide vaccine (galinpepimut-S) in acute myeloid leukemia. Blood Adv. 2018 2;2(3):224–234.
   PubMed Web of Science ®Google Scholar
• Oka Y, Udaka K, Tsuboi A, et al. Cancer immunotherapy targeting Wilms’ tumor gene WT1 product. J Immunol. 2000 Feb;164(4):1873–1880.
   PubMed Web of Science ®Google Scholar
• Tsuboi A, Oka Y, Ogawa H, et al. Cytotoxic T-lymphocyte responses elicited to Wilms’ tumor gene WT1 product by DNA vaccination. J Clin Immunol. 2000 May;20(3):195–202.
   PubMed Web of Science ®Google Scholar
• Oka Y, Tsuboi A, Taguchi T, et al. Induction of WT1 (Wilms’ tumor gene)-specific cytotoxic T lymphocytes by WT1 peptide vaccine and the resultant cancer regression. Proc Natl Acad Sci U S A. 2004 Sep;101(38):13885–13890.
   PubMed Web of Science ®Google Scholar
• Rezvani K, Yong AS, Mielke S, et al. Leukemia-associated antigen-specific T-cell responses following combined PR1 and WT1 peptide vaccination in patients with myeloid malignancies. Blood. 2008 Jan;111(1):236–242.
   PubMed Web of Science ®Google Scholar
• Rezvani K, Yong AS, Mielke S, et al. Repeated PR1 and WT1 peptide vaccination in Montanide-adjuvant fails to induce sustained high-avidity, epitope-specific CD8+ T cells in myeloid malignancies. Haematologica. 2011 Mar;96(3):432–440.
   PubMed Web of Science ®Google Scholar
• Pinilla-Ibarz J, May RJ, Korontsvit T, et al. Improved human T-cell responses against synthetic HLA-0201 analog peptides derived from the WT1 oncoprotein. Leukemia. 2006 Nov;20(11):2025–2033.
   PubMed Web of Science ®Google Scholar
• May RJ, Dao T, Pinilla-Ibarz J, et al. Peptide epitopes from the Wilms’ tumor 1 oncoprotein stimulate CD4+ and CD8+ T cells that recognize and kill human malignant mesothelioma tumor cells. Clin Cancer Res. 2007 Aug;13(15 Pt 1):4547–4555.
   PubMed Web of Science ®Google Scholar
• Brayer J, Lancet JE, Powers J, et al. WT1 vaccination in AML and MDS: a pilot trial with synthetic analog peptides. Am J Hematol. 2015 Jul;90(7):602–607.
   PubMed Web of Science ®Google Scholar
• Mailänder V, Scheibenbogen C, Thiel E, et al. Complete remission in a patient with recurrent acute myeloid leukemia induced by vaccination with WT1 peptide in the absence of hematological or renal toxicity. Leukemia. 2004 Jan;18(1):165–166.
   PubMed Web of Science ®Google Scholar
• Maslak PG, Dao T, Krug LM, et al. Vaccination with synthetic analog peptides derived from WT1 oncoprotein induces T-cell responses in patients with complete remission from acute myeloid leukemia. Blood. 2010 Jul;116(2):171–179.
   PubMed Web of Science ®Google Scholar
• Keilholz U, Letsch A, Busse A, et al. A clinical and immunologic phase 2 trial of Wilms tumor gene product 1 (WT1) peptide vaccination in patients with AML and MDS. Blood. 2009 Jun;113(26):6541–6548.
   PubMed Web of Science ®Google Scholar
• Melamed D. Sellas begins screening AML patients for phase 3 trial testing cancer vaccine Galinpepimut-S. 2020. GPS compared with BAT in AML CR2/CR2p. Phase III; 2020.
   Google Scholar
• Galinpepimut-S in combination with pembrolizumab in patients with selected advanced cancers. Phase 1/2. [cited 2020 Dec 20]. Available from: https://clinicaltrials.gov/ct2/show/NCT03761914
   Google Scholar