Vaccine Therapy in Treating Patients With Myelodysplastic Syndromes

Early Phase 1

Terminated

Brief Summary: RATIONALE: Vaccines made from cancer cells may help the body build an effective immune response to kill abnormal cells.

PURPOSE: This clinical trial is studying how well vaccine therapy works in treating patients with myelodysplastic syndromes (MDS).

Detailed Description: OBJECTIVES:

Primary

* Determine the safety of GM-K562 cell vaccine in patients with myelodysplastic syndromes.

* Determine the hematologic and cytogenetic response in patients treated with this vaccine.

Secondary

* Determine if vaccination with GM-K562 cell vaccine can induce an immune response to common myeloid antigens (e.g., Wilms' tumor-1 \[WT-1\], survivin, or proteinase-3), as defined by a 30% increase from baseline in specific cytotoxic T-cells measured by Elispot assay, in patients with myelodysplastic syndromes.

* Determine if immune response correlates with any clinical responses (e.g., hematologic response, resolution of cytogenetic abnormalities, or decrease in other parameters, such as WT-1 mRNA levels).

OUTLINE: This is an open-label study.

Patients receive GM-K562 cell vaccine subcutaneously once in weeks 0, 3, 6, 9, and 17 in the absence of disease progression or unacceptable toxicity.

Blood and tissue samples are collected periodically for correlative and biomarker studies. Samples are analyzed by cytogenetic studies, fluorescent in situ hybridization (FISH), and flow cytometry. Elispot is used to quantify cellular cytotoxic T-cell response to Wilms' tumor-1 (WT-1), survivin, and proteinase 3.

After completion of study treatment, patients are followed every 3 months for 1 year.

PROJECTED ACCRUAL: A total of 15 patients will be accrued for this study.

Recruitment & Eligibility

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Arm && Interventions

Group	Intervention	Description
K562/GM-CSF cell vaccine	K562/GM-CSF cell vaccine	Vaccinations of 1x10\^8 cells are given to participants at weeks 0, 3, 6, 9, and 17.

Primary Outcome Measures

Name	Time	Method
Hematologic Response Rate as Assessed by Number of Participants Achieving a Major Hematologic Response	Baseline, week 21 post-intervention	A major hematologic response is defined as any of the following: hemoglobin increase \>= 2 g/dL from baseline; platelet increase \>= 30k/mcL from baseline; or neutrophil increase \>= 100% or \>= 500/mcL from baseline.
Cytogenetic Response Rate as Assessed by Number of Participants Achieving a Cytogenetic Response	Week 21	Cytogenetic response is defined as normalization of pretreatment cytogenetic abnormalities.

Secondary Outcome Measures

Name	Time	Method
Immune Response Rate as Assessed by Number of Participants Who Exhibit Induced Immune Response to WT-1, Survivin, or Proteinase-3	Baseline, week 21 post-intervention	Immune response to WT-1, survivin, or proteinase-3 as defined by a 30% increase from baseline in cytotoxic T cells measured by Elispot analysis.
Combined Immune and Clinical Response Rate	Week 21 post-intervention	Number of participants who exhibited both an immune response as defined by Outcome 3 and a hematologic or cytogenetic response as defined by Outcomes 1 and 2, respectively.