Allogeneic Mesenchymal Cell Therapy in Anthracycline-Induced Cardiomyopathy Heart Failure Patients: The CCTRN SENECA Trial.

cardiac repair cardiooncology chemotherapy heart failure stem cells

Journal

JACC. CardioOncology
ISSN: 2666-0873
Titre abrégé: JACC CardioOncol
Pays: United States
ID NLM: 101761697

Informations de publication

Date de publication:
Nov 2020
Historique:
entrez: 6 1 2021
pubmed: 7 1 2021
medline: 7 1 2021
Statut: ppublish

Résumé

Anthracycline-induced cardiomyopathy (AIC) may be irreversible with a poor prognosis, disproportionately affecting women and young adults. Administration of allogeneic bone marrow-derived mesenchymal stromal cells (allo-MSCs) is a promising approach to heart failure (HF) treatment. SENECA (Stem Cell Injection in Cancer Survivors) was a phase 1 study of allo-MSCs in AIC. Cancer survivors with chronic AIC (mean age 56.6 years; 68% women; NT-proBNP 1,426 pg/ml; 6 enrolled in an open-label, lead-in phase and 31 subjects randomized 1:1) received 1 × 10 A total of 97% of subjects underwent successful study product injections; all allo-MSC-assigned subjects received the target dose of cells. Follow-up visits were well-attended (92%) with successful collection of endpoints in 94% at the 1-year visit. Although 58% of subjects had non-CMR compatible devices, CMR endpoints were successfully collected in 84% of subjects imaged at 1 year. No new tumors were reported. There were no significant differences between allo-MSC and vehicle groups with regard to clinical outcomes. Secondary measures included 6-min walk test (p = 0.056) and Minnesota Living with Heart Failure Questionnaire score (p = 0.048), which tended to favor the allo-MSC group. In this first-in-human study of cell therapy in patients with AIC, transendocardial administration of allo-MSCs appears safe and feasible, and CMR was successfully performed in the majority of the HF patients with devices. This study lays the groundwork for phase 2 trials aimed at assessing efficacy of cell therapy in patients with AIC.

Sections du résumé

BACKGROUND BACKGROUND
Anthracycline-induced cardiomyopathy (AIC) may be irreversible with a poor prognosis, disproportionately affecting women and young adults. Administration of allogeneic bone marrow-derived mesenchymal stromal cells (allo-MSCs) is a promising approach to heart failure (HF) treatment.
OBJECTIVES OBJECTIVE
SENECA (Stem Cell Injection in Cancer Survivors) was a phase 1 study of allo-MSCs in AIC.
METHODS METHODS
Cancer survivors with chronic AIC (mean age 56.6 years; 68% women; NT-proBNP 1,426 pg/ml; 6 enrolled in an open-label, lead-in phase and 31 subjects randomized 1:1) received 1 × 10
RESULTS RESULTS
A total of 97% of subjects underwent successful study product injections; all allo-MSC-assigned subjects received the target dose of cells. Follow-up visits were well-attended (92%) with successful collection of endpoints in 94% at the 1-year visit. Although 58% of subjects had non-CMR compatible devices, CMR endpoints were successfully collected in 84% of subjects imaged at 1 year. No new tumors were reported. There were no significant differences between allo-MSC and vehicle groups with regard to clinical outcomes. Secondary measures included 6-min walk test (p = 0.056) and Minnesota Living with Heart Failure Questionnaire score (p = 0.048), which tended to favor the allo-MSC group.
CONCLUSIONS CONCLUSIONS
In this first-in-human study of cell therapy in patients with AIC, transendocardial administration of allo-MSCs appears safe and feasible, and CMR was successfully performed in the majority of the HF patients with devices. This study lays the groundwork for phase 2 trials aimed at assessing efficacy of cell therapy in patients with AIC.

Identifiants

pubmed: 33403362
doi: 10.1016/j.jaccao.2020.09.001
pmc: PMC7781291
mid: NIHMS1654834
doi:

Types de publication

Journal Article

Langues

eng

Pagination

581-595

Subventions

Organisme : NHLBI NIH HHS
ID : F32 HL139046
Pays : United States
Organisme : NHLBI NIH HHS
ID : UM1 HL087318
Pays : United States

Commentaires et corrections

Type : CommentIn

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Auteurs

Roberto Bolli (R)

Department of Medicine, Division of Cardiovascular Medicine, University of Louisville, School of Medicine, Louisville, Kentucky, USA.

Emerson C Perin (EC)

Division of Cardiology Research, Texas Heart Institute, CHI St. Luke's Health Baylor College of Medicine Medical Center, Houston, Texas, USA.

James T Willerson (JT)

Division of Cardiology Research, Texas Heart Institute, CHI St. Luke's Health Baylor College of Medicine Medical Center, Houston, Texas, USA.

Phillip C Yang (PC)

Department of Medicine and Cardiovascular Institute, Division of Cardiovascular Medicine, Stanford University School of Medicine, Stanford, California, USA.

Jay H Traverse (JH)

Department of Medicine, Cardiovascular Division, Minneapolis Heart Institute Foundation at Abbott Northwestern Hospital, and University of Minnesota School of Medicine, Minneapolis, Minnesota, USA.

Timothy D Henry (TD)

The Carl and Edyth Lindner Center for Research and Education, The Christ Hospital, Cincinnati, Ohio, USA.

Carl J Pepine (CJ)

Department of Medicine, Division of Cardiovascular Medicine, University of Florida College of Medicine, Gainesville, Florida, USA.

Raul D Mitrani (RD)

Department of Medicine, Cardiovascular Division, University of Miami, Miller School of Medicine, Miami, Florida, USA.

Joshua M Hare (JM)

Department of Molecular and Cellular Pharmacology, Division of Cardiology, University of Miami, Miller School of Medicine, Miami, Florida, USA.

Michael P Murphy (MP)

Department of Surgery, Division of Vascular Surgery, Indiana University School of Medicine, Indianapolis, Indiana, USA.

Keith L March (KL)

Department of Medicine, Division of Cardiovascular Medicine, University of Florida College of Medicine, Gainesville, Florida, USA.

Sohail Ikram (S)

Department of Medicine, Division of Cardiovascular Medicine, University of Louisville, School of Medicine, Louisville, Kentucky, USA.

David P Lee (DP)

Department of Medicine and Cardiovascular Institute, Division of Cardiovascular Medicine, Stanford University School of Medicine, Stanford, California, USA.

Connor O'Brien (C)

Department of Medicine and Cardiovascular Institute, Division of Cardiovascular Medicine, Stanford University School of Medicine, Stanford, California, USA.

Jean-Bernard Durand (JB)

Department of Cardiology, Division of Internal Medicine, M.D. Anderson Cancer Center, Houston, Texas, USA.

Kathy Miller (K)

Department of Medicine, Division of Hematology/Oncology, Indiana University School of Medicine, Indianapolis, Indiana, USA.

Joao A Lima (JA)

Department of Medicine, Cardiology Division, Johns Hopkins University, Baltimore, Maryland, USA.

Mohammad R Ostovaneh (MR)

Department of Medicine, Cardiology Division, Johns Hopkins University, Baltimore, Maryland, USA.

Bharath Ambale-Venkatesh (B)

Department of Medicine, Cardiology Division, Johns Hopkins University, Baltimore, Maryland, USA.

Adrian P Gee (AP)

Department of Pediatrics, Division of Hematology and Oncology, Center for Cell and Gene Therapy, Baylor College of Medicine, Houston, Texas, USA.

Sara Richman (S)

Department of Pediatrics, Division of Hematology and Oncology, Center for Cell and Gene Therapy, Baylor College of Medicine, Houston, Texas, USA.

Doris A Taylor (DA)

Department of Regenerative Medicine Research, Texas Heart Institute, CHI St. Luke's Health Baylor College of Medicine Medical Center, Houston, Texas, USA.

Shelly L Sayre (SL)

Department of Biostatistics & Data Science, UTHealth University of Texas Health Science Center at Houston School of Public Health, Houston, Texas, USA.

Judy Bettencourt (J)

Department of Biostatistics & Data Science, UTHealth University of Texas Health Science Center at Houston School of Public Health, Houston, Texas, USA.

Rachel W Vojvodic (RW)

Department of Biostatistics & Data Science, UTHealth University of Texas Health Science Center at Houston School of Public Health, Houston, Texas, USA.

Michelle L Cohen (ML)

Department of Biostatistics & Data Science, UTHealth University of Texas Health Science Center at Houston School of Public Health, Houston, Texas, USA.

Lara M Simpson (LM)

Department of Biostatistics & Data Science, UTHealth University of Texas Health Science Center at Houston School of Public Health, Houston, Texas, USA.

Dejian Lai (D)

Department of Biostatistics & Data Science, UTHealth University of Texas Health Science Center at Houston School of Public Health, Houston, Texas, USA.

David Aguilar (D)

Department of Epidemiology, Human Genetics and Environmental Sciences, UTHealth University of Texas Health Science Center at Houston School of Public Health, Houston, Texas, USA.

Catalin Loghin (C)

Department of Medicine, Division of Cardiovascular Medicine, UTHealth University of Texas Health Science Center at Houston McGovern Medical School, Houston, Texas, USA.

Lem Moyé (L)

Department of Biostatistics & Data Science, UTHealth University of Texas Health Science Center at Houston School of Public Health, Houston, Texas, USA.

Ray F Ebert (RF)

Division of Cardiovascular Sciences, Basic & Early Translational Research Program, National Institutes of Health, National Heart, Lung, and Blood Institute, Washington, DC, USA.

Barry R Davis (BR)

Department of Biostatistics & Data Science, UTHealth University of Texas Health Science Center at Houston School of Public Health, Houston, Texas, USA.

Robert D Simari (RD)

Division of Cardiovascular Diseases, University of Kansas School of Medicine, Kansas City, Kansas, USA.

Classifications MeSH