Alexis A. Thompson, MD, MPH, Considers Future Assessments of Beti-Cel in β-Thalassemia


Alexis A. Thompson, MD, MPH, reviewed the unmet needs of patients with β-thalassemia who will receive betibeglogene autotemcel.

Alexis A. Thompson, MD, MPH, chief of the Division of Hematology and Elias Schwartz, MD, Endowed Chair in Hematology at Children’s Hospital of Philadelphia, and a member of the American Society of Hematology (ASH) CONSA Steering Committee and former ASH president, spoke with CancerNetwork® about future analyses of the gene therapy betibeglogene autotemcel (beti-cel; Zynteglo) that was recently approved to treat adult and pediatric patients with transfusion-dependent β-thalassemia. She believes this treatment has the possibility to change the treatment paradigm for both adult and pediatric patients alike.


There are still more things to be learned. We know that by and large, the patient experience that’s been reported has been in children and young adults. We have experienced that down to the age of about 4 or 5 and up to the age of 35. There do not seem to be complications with beti-cel that are age dependent. They are dependent upon the general health of the individual. We see the possibility that patients who are considerably younger than 4 or older than age 35 who have β-thalassemia will be considered for this now that it’s in a commercial setting. In my opinion, this can be safely achieved as long as these patients are properly counseled about potential risks. Being able to potentially offer this to more patients is certainly something that I’m looking forward to.

In general, I think that this is an exciting time for the field. I certainly have spent my career taking care of patients with β-thalassemia, but I also take care of patients with sickle cell disease and other nonmalignant conditions. Gene therapy has always been envisioned for conditions that have a genetic basis that involves a single gene. Beti-cel as the very first application of this kind of technology to a relatively common disease, even though in the United States we think of β-thalassemia as being fairly rare. We are well beyond the proof of principle for using gene therapy to treat human diseases. For that, I’m quite indebted to my thalassemia patients who are willing to be part of the phase 1/2 studies, and even the more recent phase 3 trials to show the way and to demonstrate that we could use ex vivo transfection of autologous stem cells to look at long-term disease control and potential cures.


FDA Approves First Cell-Based Gene Therapy to Treat Adult and Pediatric Patients with Beta-thalassemia Who Require Regular Blood Transfusions. FDA. August 17, 2022. Accessed August 17, 2022.

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