Lentivirus‐mediated gene therapy for Fabry disease: 5‐year End‐of‐Study results from the Canadian FACTs trial

This was the first gene therapy clinical trial to be completed for Fabry disease. There were no adverse events of any grade attributable to the cellular gene therapy intervention or host conditioning throughout the follow-up interval of 5 years. After reduced-intensity melphalan treatment, all patients engrafted their autologous modified α-gal A expressing cells. All patients synthesized and secreted α-gal A throughout the course of the study. Expression of α-gal A resulted in a decrease in plasma lyso-Gb3 in four of five patients and stabilization of kidney symptoms in all patients.

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Publication Details

Journal
Clinical and Translational Medicine
Published
2025-01-01
DOI
https://doi.org/10.1002/ctm2.70073
Citations
11
Primary Topic
Lysosomal Storage Disorders Research
Type
article
Field-Weighted Citation Impact
12.81

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article

Lentivirus‐mediated gene therapy for Fabry disease: 5‐year End‐of‐Study results from the Canadian FACTs trial

Ronan Foley, William M. McKillop, Anzalee Khan, Dwayne L. Barber et al.
11 citations
Clinical and Translational Medicine
Lysosomal Storage Disorders Research
12.81
article

Lentivirus‐mediated gene therapy for Fabry disease: 5‐year End‐of‐Study results from the Canadian FACTs trial

Ronan Foley, William M. McKillop, Anzalee Khan, Dwayne L. Barber, Jeffrey A. Medin, Graeme Fraser, Armand Keating, Michael L. West, C. Anthony Rupar, Daniel H. Fowler, Christiane Auray‐Blais, Alexandra Berger
article en
11 citations

Abstract

This was the first gene therapy clinical trial to be completed for Fabry disease. There were no adverse events of any grade attributable to the cellular gene therapy intervention or host conditioning throughout the follow-up interval of 5 years. After reduced-intensity melphalan treatment, all patients engrafted their autologous modified α-gal A expressing cells. All patients synthesized and secreted α-gal A throughout the course of the study. Expression of α-gal A resulted in a decrease in plasma lyso-Gb3 in four of five patients and stabilization of kidney symptoms in all patients.

Clinical and Translational MedicineVol. 15(1)
Western University (CA), Dalhousie University (CA), University Health Network (CA), University of Toronto (CA), Medical College of Wisconsin (US), Juravinski Hospital (CA), Princess Margaret Cancer Centre (CA), Hôpital Fleurimont (CA), McMaster University (CA)
Midwest Athletes Against Childhood Cancer, Kidney Foundation of Canada, Canadian Institutes of Health Research
Openalex Percentile: Top 1%
Lysosomal Storage Disorders Research
12.81
DOIOpen Access PDFOpenAlex
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