2024 Beverly Rollnick Memorial Lecture: Ordering "The Cure" with a Side of Trepidation: The Challenges of Gene Therapy for Sickle Cell Disease

On December 8, 2023, the U.S. Food and Drug Administration approved two cell-based gene therapies for sickle cell disease (SCD), including one that utilizes CRISPR technology - the first of its kind to be approved. Despite the intention of this demonstrating a commitment from the medical community to cure this condition, there is mixed reaction among those in the SCD community, given the history of inequities in research, and disparities, mistreatment, and disproportionate availability of adequate medical care for many individuals living with SCD. Through a facilitated discussion with individuals with SCD from a variety of perspectives, we will highlight their experiences and consider the factors, both positive and negative, that influence the decision to undergo a transformative therapy, and more broadly contemplate the issues that need to be addressed to ensure equitable opportunities to utilize them.

Presented by:
1. Barbara W. Harrison, MS, CGC
2. Aarin Ables Williams, MS, CGC
3. Rebekah Hutchins, MS, CGC, LCGC
4. Terry Jackson, PhD
5. Lakiea Bailey, PhD
6. Victoria Gray
7. Taylor John  

Learning Objectives:
1. Describe the curative therapeutic options available for sickle cell disease, focusing on gene therapy approaches, including the use of CRISPR.
2. Identify the risks, benefits and potential unknown clinical effects of gene therapy in sickle cell disease.
3. Summarize the patient experience with curative therapies, both successful and not successful, to inform genetic counseling practice.
4. Analyze accessibility strategies for gene therapy and other transformative treatments both within the United States, and globally, where most people with sickle cell disease reside.
5. Examine the impact of the approval of gene therapy, int he midst of social and other factors that impact the sickle cell disease community, including mistreatment, disparities in appropriate care and medical mistrust.
6. Identify personal decision-making factors for patients and families regarding the pursuit of gene therapy for sickle cell disease.
7. Explain the role of genetic counselors play in patient awareness and decision-making amongst curative gene therapy options in sickle cell disease. 

Continuing Education Unit Approval:
The National Society of Genetic Counselors (NSGC) has authorized National Society of Genetic Counselors (NSGC) to offer up to 1.25 Category 1 contact hours for 2024 Beverly Rollnick Memorial Lecture: Ordering “The Cure” with a Side of Trepidation: The Challenges of Gene Therapy for Sickle Cell Disease. The American Board of Genetic Counseling (ABGC) will accept CEUs earned at this program for the purposes of genetic counselor certification and recertification. 

Successful Completion:
1. View recording
2. Pass quiz
3. Complete evaluation