Collection of Samples From Patients With MDS

Study Overview
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Brief Summary
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The purpose of this study is to collect information and bone marrow, blood, saliva, cheek cells and skin to be used in the laboratory to assist the sponsor in identifying a new way of treating MDS.

Detailed Description
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Goals of the study: The purpose of this study is to collect the blood and marrow samples, and non-involved fibroblasts, that are required to identify the unique, personalized array of mutation-driven neoantigens that are expressed by the subject's MDS cells and to assess the feasibility of immunizing and expanding one or more of the patient's T cells ex vivo for investigation of their use as adoptive cellular immunotherapy.

Official Title
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Collection of Bone Marrow, Peripheral Blood (PB), Epithelial Tissue, and Saliva Samples From Patients With Myelodysplastic Syndromes (MDS) to Identify MDS-Specific Antigens (MSA) for Use in Cellular Immunotherapy.

Conditions
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Myelodysplastic Syndromes(MDS)

Participation Criteria
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Eligibility Criteria
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Patients must meet the following initial inclusion criteria: Diagnosis or suspected diagnosis of MDS or CCUS Age 18 or older Patient exclusion criteria: Currently receiving or within 3 months has received hypomethylating agent(s), lenalidomide, cytotoxic agents, or within the previous 4 weeks corticosteroids > 5 mg prednisone daily or any other immunosuppressants Previous allogenic transplant Inability to provide consent Prisoners

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Genomics of patients with MDS | To sequence the exome and transcriptome obtained from MDS hematopoietic cells and the exome from non-hematopoietic cells (e.g. fibroblasts). | 2 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Identification of patients' MDS-specific variant | To select variants by comparing MDS versus non-MDS cell exome sequences. MDS-specific variant sequences are defined as those that differ between the two and are not common polymorphisms. We will also compare myeloid and lymphoid hematopoietic cells and assess the number of myeloid-specific vs myeloid and lymphoid MDS-related variants | 2 years | 
| Immunogenic mutant neoantigen peptide selection | To select putative mutation-driven neoantigen-related peptides, which represent the sequences obtained from Aim 2, according to their ability to bind to the patient's MHC using PersImmune's licensed and proprietary algorithms. | 2 years | 
| Peptide Immunogenicity confirmation and donor T cell stimulation | To test the neoantigen peptides for their in vitro immunogenicity for autologous T lymphocytes. | 2 years | 
| Peptide immunogenicity confirmation and donor T cell stimulation | To test the potency and specificity of neoantigen peptide-stimulated T cells for the patient's MDS cells that express the defined neoantigens. | 2 years | 
| Data analysis and interpretation | To create a database summarizing the data obtained. | 2 years |