Webinar: Engineering a Cure: Updates in CAR-T and Gene Therapy-Webinar -Engineering a Cure: Updates in CAR-T and Gene Therapy

Video Transcription

Video Summary

The webinar reviewed key differences between CAR-T cell therapy and gene therapy. CAR-T therapy modifies mature T cells ex vivo to recognize cancer antigens and is primarily used for malignancies, while gene therapy alters genes or stem cells to address a broader range of conditions, including sickle cell disease and beta thalassemia.<br /><br />The presentation covered three CAR-T-related inflammatory toxicities: cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), and immune effector cell-associated hemophagocytic lymphohistiocytosis-like syndrome (IEC-HS). It discussed their symptoms, grading, and management, including tocilizumab for severe CRS and anakinra and corticosteroids as initial IEC-HS treatments. IEC-HS is a rare but potentially life-threatening complication.<br /><br />The final section focused on busulfan conditioning for gene therapy. Because busulfan has a narrow therapeutic range, pharmacokinetic monitoring is important to balance efficacy and toxicity. Overexposure can cause sinusoidal obstruction syndrome (SOS), so clinicians should monitor for signs such as abdominal pain, weight gain, and platelet-transfusion refractoriness. Patients with sickle cell disease may be especially vulnerable because prior disease-related liver injury and transfusions can increase hepatic risk.

Keywords

CAR-T cell therapy

gene therapy

cytokine release syndrome

ICANS

IEC-HS

tocilizumab

anakinra

busulfan conditioning

sinusoidal obstruction syndrome