Mutant CALR-peptide Based Vaccine in Patients With Mutated CALR Myeloproliferative Neoplasm
Key claim: A mutant CALR peptide vaccine is being tested for safety and tolerability in a small early trial of patients with CALR-mutated myeloproliferative neoplasms.
Abstract
The primary objective of this study is to assess the safety and tolerability of administrating mutated-CALR peptide Vaccine to patients with MPN. The researchers plan to enroll 10 patients over a 12 month period. Maximum length of participation in 80 weeks. Patients will be asked to complete questionnaires, bone marrow biopsies, research lab collection, and standard of care lab draw. This research will be taking place only at The Mount Sinai Hospital, specifically at the Ruttenberg Treatment Center.
Why this matters
Nearly all MPN therapeutics to date — JAK inhibitors, interferons, HMAs, BCL-2/XPO1 combinations — target downstream pathways or general cytoreduction rather than the mutant driver itself. A CALR-neoantigen vaccine is mechanistically unique because mutant CALR generates a shared C-terminal frameshift peptide across CALR-mutated ET and PMF, making it a tractable immunotherapy target that is genotype-defined rather than subtype-defined. Even as an early safety trial, it anchors a new category in the cross-MPN therapeutic landscape.