Pilot trial of K562/GM-CSF whole-cell vaccination in MDS patients

Tara M. Robinson; Gabrielle T. Prince; Chris Thoburn; Erica Warlick; Anna Ferguson; Yvette L. Kasamon; Ivan M. Borrello; Allan Hess; B. Douglas Smith

doi:10.1080/10428194.2018.1443449

Pilot trial of K562/GM-CSF whole-cell vaccination in MDS patients

Tara M. Robinson
, Gabrielle T. Prince
, Chris Thoburn
, Erica Warlick
, Anna Ferguson
, Yvette L. Kasamon
, Ivan M. Borrello
, Allan Hess
, B. Douglas Smith

Medicine - Hematology, Oncology, Transplant

Research output: Contribution to journal › Article › peer-review

10 Scopus citations

Abstract

Myelodysplastic syndromes (MDS) are a heterogeneous group of clonal hematopoietic stem cell malignancies. Currently, approved drugs are given with non-curative intent as the only known cure is allogeneic bone marrow transplantation, which relies on the donor’s immune system driving an allogeneic effect. Previous efforts to harness the endogenous immune system have been less successful. We present the results of a pilot study of K562/GM-CSF (GVAX) whole-cell vaccination in MDS patients. The primary objective of safety was met as there were no serious adverse events. One patient had a decrease in transfusion requirements and another demonstrated hematologic improvement suggesting a signal for clinical activity. In vitro correlative studies indicated biological effects on immune cells following vaccination. Although only a pilot study, results are encouraging that an immunotherapeutic approach with a whole-cell vaccine may be feasible in MDS patients.

Original language	English (US)
Pages (from-to)	2801-2811
Number of pages	11
Journal	Leukemia and Lymphoma
Volume	59
Issue number	12
DOIs	https://doi.org/10.1080/10428194.2018.1443449
State	Published - Dec 2 2018

Bibliographical note

Funding Information:
We kindly thank the research support staff at SKCCC and the patients who enrolled in the study. We also thank Dr. Hao Zhang at the Johns Hopkins School of Public Health Flow Cytometry Core Facility for assistance with cell sorting and Dr. Ian Kaplan at Adaptive Biotechnologies for assistance with TCRseq analysis. Experiments done though the human immunology core facility were supported by National Institutes of Health Grant [P30 CA006973].

Publisher Copyright:
© 2018, © 2018 Informa UK Limited, trading as Taylor & Francis Group.

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

SDG 3 Good Health and Well-being

Keywords

Cancer vaccine
immunotherapy
myelodysplastic syndrome
phase 1 clinical trial

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10.1080/10428194.2018.1443449

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6352988

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title = "Pilot trial of K562/GM-CSF whole-cell vaccination in MDS patients",

abstract = "Myelodysplastic syndromes (MDS) are a heterogeneous group of clonal hematopoietic stem cell malignancies. Currently, approved drugs are given with non-curative intent as the only known cure is allogeneic bone marrow transplantation, which relies on the donor{\textquoteright}s immune system driving an allogeneic effect. Previous efforts to harness the endogenous immune system have been less successful. We present the results of a pilot study of K562/GM-CSF (GVAX) whole-cell vaccination in MDS patients. The primary objective of safety was met as there were no serious adverse events. One patient had a decrease in transfusion requirements and another demonstrated hematologic improvement suggesting a signal for clinical activity. In vitro correlative studies indicated biological effects on immune cells following vaccination. Although only a pilot study, results are encouraging that an immunotherapeutic approach with a whole-cell vaccine may be feasible in MDS patients.",

keywords = "Cancer vaccine, immunotherapy, myelodysplastic syndrome, phase 1 clinical trial",

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note = "Funding Information: We kindly thank the research support staff at SKCCC and the patients who enrolled in the study. We also thank Dr. Hao Zhang at the Johns Hopkins School of Public Health Flow Cytometry Core Facility for assistance with cell sorting and Dr. Ian Kaplan at Adaptive Biotechnologies for assistance with TCRseq analysis. Experiments done though the human immunology core facility were supported by National Institutes of Health Grant [P30 CA006973]. Publisher Copyright: {\textcopyright} 2018, {\textcopyright} 2018 Informa UK Limited, trading as Taylor \& Francis Group.",

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AU - Kasamon, Yvette L.

AU - Borrello, Ivan M.

AU - Hess, Allan

AU - Smith, B. Douglas

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PY - 2018/12/2

Y1 - 2018/12/2

N2 - Myelodysplastic syndromes (MDS) are a heterogeneous group of clonal hematopoietic stem cell malignancies. Currently, approved drugs are given with non-curative intent as the only known cure is allogeneic bone marrow transplantation, which relies on the donor’s immune system driving an allogeneic effect. Previous efforts to harness the endogenous immune system have been less successful. We present the results of a pilot study of K562/GM-CSF (GVAX) whole-cell vaccination in MDS patients. The primary objective of safety was met as there were no serious adverse events. One patient had a decrease in transfusion requirements and another demonstrated hematologic improvement suggesting a signal for clinical activity. In vitro correlative studies indicated biological effects on immune cells following vaccination. Although only a pilot study, results are encouraging that an immunotherapeutic approach with a whole-cell vaccine may be feasible in MDS patients.

AB - Myelodysplastic syndromes (MDS) are a heterogeneous group of clonal hematopoietic stem cell malignancies. Currently, approved drugs are given with non-curative intent as the only known cure is allogeneic bone marrow transplantation, which relies on the donor’s immune system driving an allogeneic effect. Previous efforts to harness the endogenous immune system have been less successful. We present the results of a pilot study of K562/GM-CSF (GVAX) whole-cell vaccination in MDS patients. The primary objective of safety was met as there were no serious adverse events. One patient had a decrease in transfusion requirements and another demonstrated hematologic improvement suggesting a signal for clinical activity. In vitro correlative studies indicated biological effects on immune cells following vaccination. Although only a pilot study, results are encouraging that an immunotherapeutic approach with a whole-cell vaccine may be feasible in MDS patients.

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Pilot trial of K562/GM-CSF whole-cell vaccination in MDS patients

Abstract

Bibliographical note

UN SDGs

Keywords

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