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Elivaldogene autotemcel (eli-cel) gene therapy for cerebral adrenoleukodystrophy has shown efficacy in clinical trials, but concerns about oncogenesis have emerged. A recent study examines the development of hematologic cancer in patients treated with eli-cel, providing critical insights into the risks and mechanisms associated with this innovative therapy.
Key Points:
- Hematologic cancer developed in 7 out of 67 patients (10%) treated with eli-cel across two clinical studies (ALD-102 and ALD-104).
- The incidence rate of hematologic cancer was 2.1 per 100 person-years (95% CI, 0.8 to 4.2) over 338 person-years of total follow-up.
- Six of the seven cases were associated with lentiviral vector insertions in known oncogenes, primarily MECOM (5 cases) and PRDM16 (1 case).
- Additional factors, including somatic mutations and chromosomal abnormalities, were observed in patients who developed hematologic cancer.
- The study suggests that vector design, conditioning regimens, and mobilization strategies may influence the risk of insertional oncogenesis.
- Despite the oncogenic risk, 81% of patients treated with eli-cel remained free of major functional disabilities at 4 years post-treatment.
- Ongoing monitoring and follow-up are crucial for patients treated with eli-cel to assess long-term safety and efficacy.
HCN Medical Memo
Although eli-cel gene therapy shows promise for treating cerebral adrenoleukodystrophy, the risk of hematologic cancer must be carefully considered. Physicians should weigh the potential benefits against the risks, considering patient-specific factors and alternative treatment options. Close monitoring and long-term follow-up are essential for patients receiving this innovative therapy.
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