Hematologic Cancer Develops in Some Patients Receiving Eli-Cel
By Elana Gotkine HealthDay Reporter
THURSDAY, Oct. 10, 2024 -- Hematologic cancer develops in some patients receiving lentiviral elivaldogene autotemcel (eli-cel) gene therapy for cerebral adrenoleukodystrophy, according to a study published in the Oct. 10 issue of the New England Journal of Medicine.
Christine N. Duncan, M.D., from Harvard Medical School in Boston, and colleagues analyzed peripheral-blood and bone marrow samples from patients with cerebral adrenoleukodystrophy who received eli-cel therapy in two completed phase 2 to 3 studies (ALD-102 [32 patients] and ALD-104 [35 patients]) and the associated ongoing follow-up study (LTF-304) to examine the risk for oncogenesis with eli-cel.
The researchers found that seven of 67 patients developed hematologic cancer after receipt of eli-cel: Myelodysplastic syndrome (MDS) with unilineage dysplasia developed in two patients at 14 and 26 months; MDS with excess blasts developed in three patients at 28, 42, and 92 months; MDS developed in one patient at 36 months; and acute myeloid leukemia developed in one patient at 57 months. Predominant clones contained lentiviral vector insertions at multiple loci in the six patients with available data. Cytopenias developed in several patients; most had vector insertions in multiple genes within the same clone. Of the seven patients, six also had somatic mutations and one had monosomy 7. Four of the five patients with MDS with excess blasts or MDS with unilineage dysplasia who underwent allogeneic hematopoietic stem cell transplantation (HSCT) remain free of MDS without recurrence of cerebral adrenoleukodystrophy symptoms.
"The risk of oncogenesis with eli-cel must be weighed against the severity and natural history of cerebral adrenoleukodystrophy as well as the availability of other treatments and their risks, including allogeneic HSCT," the authors write.
The study was funded by Bluebird Bio, the manufacturer of eli-cel.
Abstract/Full Text (subscription or payment may be required)
Editorial (subscription or payment may be required)
Disclaimer: Statistical data in medical articles provide general trends and do not pertain to individuals. Individual factors can vary greatly. Always seek personalized medical advice for individual healthcare decisions.
© 2025 HealthDay. All rights reserved.
Posted October 2024
Read this next
Higher Cardiac Marker Levels Tied to Long-Term Cancer Risk
TUESDAY, July 1, 2025 -- Slightly higher levels of cardiac biomarkers may predict an increased risk for incident cancer, according to a study published online in the July issue of...
Capsule Sponge + Biomarkers Can Stratify Risk in Barrett Esophagus
MONDAY, June 30, 2025 -- In adult patients with nondysplastic Barrett esophagus at last endoscopy, a subsequent capsule-sponge test can assist in identifying those at highest risk...
Adding Pembrolizumab to Standard Care Ups Event-Free Survival in Head, Neck Cancer
FRIDAY, June 27, 2025 -- For patients with locally advanced head and neck squamous cell carcinoma (HNSCC), the addition of neoadjuvant and adjuvant pembrolizumab to standard care...
More news resources
- FDA Medwatch Drug Alerts
- Daily MedNews
- News for Health Professionals
- New Drug Approvals
- New Drug Applications
- Drug Shortages
- Clinical Trial Results
- Generic Drug Approvals
Subscribe to our newsletter
Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.