2021 Pediatric Grand Rounds | Cellular Therapy for Pediatric Cerebral Adrenoleukodystrophy (ALD)
Cellular therapy for genetic conditions began more broadly with hematopoietic stem cell transplantation, however, advances with gene therapy have allowed much more targeted interventions to address rare disease. Initial impressive gene therapy results in severe combined immunodeficiency syndrome have engendered an interest in pursuing gene therapy across a range of diseases, including sickle cell disease, which we offer at City of Hope, and adrenoleukodystrophy, as this presentation will describe. The gap in practice exists as this is a novel therapeutic application for the relatively new and exciting platform of gene therapy. Gene therapy is newly being utilized to treat adrenoleukodystrophy (ALD). This lecture is intended to bring awareness of the novel application of gene therapy for ALD, for which there is no known cure.
Pediatric Hematology/Oncology physicians, advance practice practitioners, staff and research team.
- Outline the indications for cellular therapy (allogeneic stem cell transplantation and autologous gene therapy) in children diagnosed with cerebral adrenoleukodystrophy (ALD).
- Describe the differences in treatment strategy and potential benefits between allogeneic stem cell transplantation and autologous gene therapy for children diagnosed with cerebral ALD.
- Contrast the risks associated with stem cell transplantation with autologous gene therapy.
- Discuss currently available outcome data from the ALD gene therapy studies.
Virtual Meeting- Zoom
Christine N. Duncan, MD Medical Director of Clinical Research and Clinical Development, Gene Therapy Program; Boston Children's/Dana-Farber Blood Disorders Center
Dr. Duncan has indicated that she does not have anything to disclose.
Dr. Duncan will be discussing the off-label or investigational use of Lenti-D Drug Product.
ACCREDITATION STATEMENT: City of Hope is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.
CREDIT DESIGNATION: City of Hope designates this live webinar for a maximum of 1.0 AMA PRA Category 1 Credit™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
The following credit type(s) are being offered for this course:
• AMA PRA Category 1™ 1.0
The following may apply AMA PRA Category 1 Credit™ for license renewal:
Registered Nurses: Nurses may report up to 1.0 credit hours toward the continuing education requirements for license renewal by their state Board of Registered Nurses (BRN). AMA PRA Category 1 Credit™ may be noted on the license renewal application in lieu of a BRN provider number.
Physician Assistants: The National Commission on Certification of Physicians Assistants states that AMA PRA Category 1 Credit™ accredited courses are acceptable for CME requirements for recertification.
- 1.00 AMA PRA Category 1 Credit™City of Hope is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.
City of Hope designates this 2021 Pediatric Grand Rounds | Cellular Therapy for Pediatric Cerebral Adrenoleukodystrophy (ALD) for a maximum of 1.00 AMA PRA Category 1 Credit™ requirements. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
- 1.00 Attendance