Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy.

ABSTRACT:

Background

Gene therapy with elivaldogene autotemcel (eli-cel) consisting of autologous CD34+ cells transduced with lentiviral vector containing ABCD1 complementary DNA (Lenti-D) has shown efficacy in clinical studies for the treatment of cerebral adrenoleukodystrophy. However, the risk of oncogenesis with eli-cel is unclear.

Methods

We performed integration-site analysis, genetic studies, flow cytometry, and morphologic studies in peripheral-blood and bone marrow samples from patients who received eli-cel therapy in two completed phase 2-3 studies (ALD-102 and ALD-104) and an ongoing follow-up study (LTF-304) involving the patients in both ALD-102 and ALD-104.

Results

Hematologic cancer developed in 7 of 67 patients after the receipt of eli-cel (1 of 32 patien

SUBMITTER: Duncan CN 

PROVIDER: S-EPMC11846662 | biostudies-literature | 2024 Oct

REPOSITORIES: biostudies-literature