Phase 2 trial of a multivalent WT1 peptide vaccine (galinpepimut-S) in acute myeloid leukemia

Peter G. Maslak*, Tao Dao, Yvette Bernal, Suzanne M. Chanel, Rong Zhang, Mark Frattini, Todd Rosenblat, Joseph G. Jurcic, Renier J. Brentjens, Maria E. Arcila, Raajit Rampal, Jae H. Park, Dan Douer, Laura Katz, Nicholas Sarlis, Martin S. Tallman, David A. Scheinberg

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

111 Scopus citations


A National Cancer Institute consensus study on prioritization of cancer antigens ranked the Wilms tumor 1 (WT1) protein as the top immunotherapy target in cancer. We previously reported a pilot study of a multivalentWT1 peptide vaccine (galinpepimut-S) in acutemyeloid leukemia (AML) patients. We have now conducted a phase 2 study investigating this vaccine in adults with AML in first complete remission (CR1). Patients received 6 vaccinations administered over 10 weeks with the potential to receive 6 additional monthly doses if they remained in CR1. Immune responses (IRs) were evaluated after the 6th and 12th vaccinations by CD4+ T-cell proliferation, CD8+ T-cell interferon-g secretion (enzyme-linked immunospot), or the CD8- relevant WT1 peptide major histocompatibility complex tetramer assay (HLA-A∗02 patients only). Twenty-two patients (7 males; median age, 64 years) were treated. Fourteen patients (64%) completed ≥6 vaccinations, and 9 (41%) received all 12 vaccine doses. Fifteen patients (68%) relapsed, and 10 (46%) died. The vaccine was well tolerated, with the most common toxicities being grade 1/2 injection site reactions (46%), fatigue (32%), and skin induration (32%). Median disease-free survival from CR1was 16.9months, whereas the overall survival from diagnosis has not yet been reached but is estimated to be ≥67.6months. Nine of 14 tested patients (64%) had an IR in ≥1 assay (CD4 or CD8). These results indicated that the WT1 vaccine was well tolerated, stimulated a specific IR, and was associated with survival in excess of 5 years in this cohort of patients.

Original languageEnglish (US)
Pages (from-to)224-234
Number of pages11
JournalBlood Advances
Issue number3
StatePublished - Feb 14 2018

ASJC Scopus subject areas

  • Hematology


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