Method of determining responsiveness to cell therapy in dilated cardiomyopathy
Inventors
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Assignees
MemberUniversity of MiamiUniversity of MiamiThe University of Miami, established in 1925 and based in Coral Gables, Florida, is a private research university recognized for its comprehensive academic offerings, robust research infrastructure, and strong interdisciplinary focus. Home to more than 19,000 students and with more than 400 acres of campuses across the Miami region, its mission encompasses education, research, innovation, and community service. The institution supports clinical, biomedical, marine, and atmospheric research initiatives, delivers diverse undergraduate and graduate programs, and maintains numerous research centers and institutes dedicated to scientific, medical, and societal advancements.
The University of Miami, established in 1925 and based in Coral Gables, Florida, is a private research university recognized for its comprehensive academic offerings, robust research infrastructure, and strong interdisciplinary focus. Home to more than 19,000 students and with more than 400 acres of campuses across the Miami region, its mission encompasses education, research, innovation, and community service. The institution supports clinical, biomedical, marine, and atmospheric research initiatives, delivers diverse undergraduate and graduate programs, and maintains numerous research centers and institutes dedicated to scientific, medical, and societal advancements.
Publication Number
US-12540357-B2
Publication Date
2026-02-03
Expiration Date
Abstract
The present disclosure is directed to methods for determining responsiveness to cell therapy in a subject suffering from a cardiovascular disorder (e.g., cardiomyopathy) and methods of treating subjects suffering from a cardiovascular disorder.
Core Innovation
The patent describes genetically guided selection of patients for mesenchymal stem cell therapy for cardiomyopathy. A subject is identified as a hyper-responder to mesenchymal stem cell therapy by determining the absence of a pathologic variant relevant for cardiomyopathy in a specified gene set. The described approach links genotype status to response to mesenchymal stem cells by defining hyper-responder status as PV− (negative for relevant pathologic variants).
In the described context, the method is applied notably to non-ischemic dilated cardiomyopathy (NIDCM) and compares hyper-responder groups against groups defined by other genetic classifications. Hyper-responder (PV−) subjects show greater improvement in cardiac function and functional capacity after MSC therapy than subjects in VUS and PV+ groups. The improvements described include ejection fraction and functional measures such as 6-minute walk and MLHFQ/NYHA.
The patent further associates the hyper-responder genotype selection with vascular and clinical outcomes. PV− hyper-responder subjects show improved endothelial progenitor colony (EPC) and flow-mediated vasodilation (FMD), and better survival and MACE outcomes compared with VUS and PV+ groups. The described patient grouping and genetic testing within the POSEIDON-DCM study context are used to relate the absence of pathologic variants in the specified genes to therapeutic response and adverse outcomes.
Claims Coverage
The partial content identifies one independent claim and describes multiple dependent refinements. The core claim involves patient selection by a defined gene set for hyper-responder status and administering MSCs effective to treat the cardiomyopathy.
Hyper-responder identification by absence of pathologic variants in a cardiomyopathy gene set
A method of treating cardiomyopathy in a subject includes identifying the subject as a hyper-responder to mesenchymal stem cell therapy by determining the absence of a pathologic variant relevant for cardiomyopathy in a listed set of genes.
MSC administration effective to treat cardiomyopathy in the identified hyper-responder
The method further comprises administering to the identified hyper-responder an amount of mesenchymal stem cells effective to treat the cardiomyopathy in the subject.
Overall, the claimed coverage centers on determining hyper-responder status through absence of pathologic variants relevant for cardiomyopathy across a specified gene list, followed by administering mesenchymal stem cells effective to treat the cardiomyopathy.
Stated Advantages
Greater improvement in cardiac function and functional capacity (ejection fraction and 6-minute walk, including MLHFQ/NYHA) in PV− hyper-responder subjects versus VUS and PV+ groups.
Improved endothelial progenitor colony (EPC) and flow-mediated vasodilation (FMD) in PV− hyper-responder subjects versus VUS and PV+ groups.
Better survival and MACE outcomes in PV− hyper-responder subjects versus VUS and PV+ groups.
Documented Applications
Genetically guided selection of cardiomyopathy patients (including non-ischemic dilated cardiomyopathy) as hyper-responders to mesenchymal stem cell therapy in the POSEIDON-DCM study context.
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