Myelodysplastic Syndromes Clinical Trial
Official title:
A Phase 1 Clinical Trial of Personalized, Adoptive Cellular Immunotherapy Targeting Patient-specific Neoplastic Stem Cell Neoantigens (PACTN) in Patients With Myelodysplastic Syndromes (MDS)
This study will evaluate the safety of autologous T cells that have been immunized ex vivo with patient-specific MDS stem cell neoantigens in patients with MDS.
PACTN is manufactured by a novel method to employ cancer-specific somatic variants
(mutations) as a means to immunize autologous T lymphocytes to specifically kill cancer cells
bearing the protein products of the mutations.
The PACTN method is based on the premise that somatic DNA mutations that cause cancer often
give rise to proteins with an altered amino acid sequence. Peptides derived from these
proteins, if expressed in the context of MHC Class I or II may be perceived as "non-self" by
the immune system; that is, they may be perceived as neoantigens (aka, neoepitopes). Such
neoantigens could therefore serve as immunogenic targets for the development of
patient-specific, personalized T cell mediated immunotherapy.
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