Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy
Hematologic cancer developed in a subgroup of patients who were treated with eli-cel; the cases are associated with clonal vector insertions within oncogenes and clonal evolution with acquisition of somatic genetic defects. (Funded by Bluebird Bio; ALD-102, ALD-104, and LTF-304 ClinicalTrials.gov numbers, NCT01896102, NCT03852498, and NCT02698579, respectively.).
Authors
- Richard A. Colvin (ORCID: https://orcid.org/0000-0001-5851-7019)
- Francis John Pierciey
- Geoffrey Parsons (ORCID: https://orcid.org/0000-0003-1767-9489)
- Vinod K. Prasad (ORCID: https://orcid.org/0000-0001-8151-8212)
- Melissa Bonner (ORCID: https://orcid.org/0000-0001-7154-1777)
- Marianna Foos (ORCID: https://orcid.org/0000-0001-5275-4566)
- Himal L. Thakar
- Melissa A. Kinney (ORCID: https://orcid.org/0000-0003-1528-6163)
- Jörn‐Sven Kühl (ORCID: https://orcid.org/0000-0002-1739-1090)
- Florian Eichler (ORCID: https://orcid.org/0000-0001-6836-1089)
- Bartosz J. Grzywacz (ORCID: https://orcid.org/0000-0003-0075-688X)
- Marian H. Harris (ORCID: https://orcid.org/0000-0002-9956-664X)
- Paul J. Orchard (ORCID: https://orcid.org/0000-0001-9426-1292)
- David A. Williams (ORCID: https://orcid.org/0000-0001-7057-6863)
- Sarah Slauson (ORCID: https://orcid.org/0000-0002-5432-4241)
- Jacob R. Bledsoe (ORCID: https://orcid.org/0000-0002-2387-5462)
- Ashish O. Gupta
- Ankit Lodaya
- Amy Beckman (ORCID: https://orcid.org/0000-0002-1139-1321)
- Andrew C. Dietz (ORCID: https://orcid.org/0000-0003-2810-2466)
- Gerald Downey
- Nicole Floro
- Christine Duncan (ORCID: https://orcid.org/0000-0002-2090-4897)
Institutions
- Dana-Farber/Boston Children's Cancer and Blood Disorders Center (US)
Publication Details
- Journal
- New England Journal of Medicine
- Published
- 2024-10-09
- DOI
- https://doi.org/10.1056/nejmoa2405541
- Citations
- 98
- Primary Topic
- Virus-based gene therapy research
- Type
- article
- Field-Weighted Citation Impact
- 53.93
Funders
- University of Minnesota
- bluebird bio