VJHemOnc Podcast Gene therapy in sickle cell disease: patient selection, potential complications, & more
May 23, 2025
In this insightful discussion, Dr. Akshay Sharma, a hematologist at St. Jude Children’s Research Hospital, explores the cutting-edge field of gene therapy for sickle cell disease. He details key considerations for selecting patients, emphasizing organ function and pre-existing health conditions. The conversation highlights the complex benefits and challenges of gene therapy, including the risk of leukemia linked to lentiviral treatments. Additionally, Dr. Sharma addresses the evolving role of palliative care in enhancing patient comfort during such innovative therapies.
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Gene Therapy Advances and Uncertainties
- Gene therapy for sickle cell leverages advances in genomics and gene editing like CRISPR-Cas9 for potentially curative treatment.
- Approved therapies show up to 90% reduction in painful crises but long-term effects beyond 3-5 years remain unknown.
Patient Selection Considerations
- Thorough organ evaluation is crucial before gene therapy to assess cardiac, pulmonary, renal function, and stroke history.
- Avoid gene therapy in patients with severe pulmonary fibrosis or unstable conditions preventing stem cell collection or safe transfusion discontinuation.
Immune Recovery Differences
- Gene therapy patients retain near-normal immunity immediately post-treatment, unlike allogeneic transplant recipients.
- They have lower risk of infections like CMV or EBV and may not require routine revaccination after therapy.