Haydar Frangoul, MD, on Weighing Gene Therapy Against Haploidentical Transplant for Sickle Cell Disease


The medical director of Pediatric Hematology/Oncology at Sarah Cannon Research Institute discussed the pros and cons of potentially curative treatment options for SCD.

“The main message I have is for hematologists caring for individuals with sickle cell disease. I think they need to be aware of the potential curative therapies available today for them and encourage their patients to seek consultation and understand these therapies and make an informed decision on what potentially should be done to cure their disease.”

Just 10 years ago, the only potentially curative treatment option for patients with sickle cell disease (SCD) was matched sibling stem cell transplant. Times have changed however, and as of 2024 potentially curative FDA-approved options for SCD can include haploidentical bone marrow transplant (haplo) and 2 different gene therapies: Vertex Pharmaceuticals' and CRISPR Therapeutics’ exagamglogene autotemcel (exa-cel), a gene-editing therapy marketed under the name Casgevy, and bluebird bio’s lovotibeglogene autotemcel (lovo-cel), a gene-addition therapy marketed as Lyfgenia. As such, there is now discussion in the clinical community about which option is best for patients, considering the unique advantages and drawbacks of each. This topic was the focus of a session titled “Sickle cell disease: haplo versus gene therapy debate” at the 2024 Tandem Meetings |Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR, held in San Antonio, Texas, February 21-24, 2024.

Shortly before the session was held on February 22, CGTLive® sat down with Haydar Frangoul, MD, the medical director of pediatric hematology/oncology at Sarah Cannon Research Institute and Pediatric Transplant and Cellular Therapy Program at TriStar Centennial, who spoke during the session. Frangoul gave an overview of the clinical trial data regarding the gene therapy products, emphasizing their strong efficacy and safety profiles. He also pointed out drawbacks of both the haplo and gene therapy modalities, noting that haplo carries the risk of graft versus host disease and infections and that the gene therapies have a less extensive history of testing and require a busulfan conditioning regimen that poses a fertility risk. He concluded by expressing optimism that ongoing long-term follow-up studies will reveal more about the gene therapies’ long-term effects and persistence over time.

Click here for more coverage of Tandem 2024.

1. Frangoul H. Sickle cell disease: haplo versus gene therapy debate. Presented at: 2024 Tandem Meetings, February 21-24, San Antonio, Texas.

Related Videos
M. Peter Marinkovich, MD, on Bringing RDEB Treatment to the Local Level
Caspian Oliai, MD, MS, the medical director of the UCLA Bone Marrow Transplantation Stem Cell Processing Center
Frederick “Eric” Arnold, PhD
Genovefa (Zenia) Papanicolaou, MD, an infectious diseases specialist at Memorial Sloan Kettering Cancer Center
Jeffrey Chamberlain, PhD, on Exciting New Research at MDA 2024
Alan Beggs, PhD, on Challenges in Therapeutic Development for Rare Diseases
Akshay Sharma, MBBS, a bone marrow transplant physician at St. Jude Children’s Research Hospital
© 2024 MJH Life Sciences

All rights reserved.