# Personalized vaccine for patients with AML

> **NIH NIH R01** · BETH ISRAEL DEACONESS MEDICAL CENTER · 2020 · $721,179

## Abstract

Abstract:
Acute Myeloid Leukemia (AML) is a lethal hematological malignancy for which standard
therapy is rarely curative. We have developed a personalized AML vaccine in which
patient derived leukemia cells are fused with autologous DCs such that a broad array of
antigens are presented in the context of DC mediated costimulation. The vaccine
effectively targets clonal diversity including the LSC compartment and induces
responses against neoantigen targets generated from the patient specific mutational
events. We are completing a phase II study in which patients with AML who achieve
remission following standard therapy undergo serial vaccination with DC/AML fusions.
Vaccination resulted in the dramatic expansion of AML specific lymphocytes in the
peripheral blood and bone marrow. Remarkably, despite a median age of 60 with
predominant intermediate and high risk disease, 75% of patients remain in remission at
a median of 4 years of follow up. The goals of the present study are to more fully
examine the DCAML fusion vaccine and confirm these dramatic clinical findings in the
context of a randomized trial design. A fundamental challenge to developing sustained
anti-leukemia immunity and durable disease response is overcoming the
immunosuppressive milieu by which tumor cells evade host immunity and re-introduce
tolerance. In the present study, we will also examine the potential synergy of vaccine
mediated T cell expansion with PDL1 blockade to prevent the reestablishment of
tolerance. Each of the treatment arms will be assessed with respect to the expansion of
T cells targeting whole AML cells, LSCs, and leukemia associated antigens. Based on
analysis of the AML mutational events, a neo-antigen profile will be generated for each
patient and their functional relevance will be interrogated by assessing the native
immune response following exposure to the whole cell vaccine. To define biomarkers
predictive of response, the immune landscape of the bone marrow microenvironment,
presence of AML driver mutations, gene signature for LSCs and immunoregulatory
pathways, and noncoding RNAs regulating antigen presentation and costimulation will
be interrogated with respect to their correlation with immune response and clinical
outcome.

## Key facts

- **NIH application ID:** 9829540
- **Project number:** 5R01CA212649-04
- **Recipient organization:** BETH ISRAEL DEACONESS MEDICAL CENTER
- **Principal Investigator:** David E. Avigan
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $721,179
- **Award type:** 5
- **Project period:** 2016-12-09 → 2021-11-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9829540

## Citation

> US National Institutes of Health, RePORTER application 9829540, Personalized vaccine for patients with AML (5R01CA212649-04). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/9829540. Licensed CC0.

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