In a special co-branded episode between Oncology On theGo hosted by CancerNetwork® and the American Society for Transplantation and Cellular Therapy (ASTCT)’s program ASTCT Talks, Nora M. Gibson, MD, MSCE, and Taha Al-Juhaishi, MD, spoke about real-world applications of betibeglogene autotemcel (beti-cel; Zynteglo) as a treatment for patients with beta (β)-thalassemia.They spoke in the context of a study that Gibson presented at the 2025 Tandem Meetings, which evaluated patients who received commercial beti-cel in a single-center cohort following the agent’s FDA approval in August 2022. ¹ ²

Nora is a fourth-year fellow in bone marrow transplant andcellular therapy at the Children's Hospital of Philadelphia (CHOP), with a background in clinical research and epidemiology. Al-Juhaishi is the Associate Director of the Hematopoietic Stem Cell Transplantation and Cell TherapyProgram at Oklahoma University Health Stephenson Cancer Center and an assistant professor of Medicine at the University of Oklahoma College of Medicine.

Findings from Gibson’s study revealed that among 10 patients who underwent stem cell collection at CHOP from 2022 to 2024, beti-cel yielded consistent red blood cell transfusion independence, with investigators notingprolonged platelet engraftment time and high platelet transfusion requirements. Beyond these findings, the conversation focused on how beti-cel compares withother currently available gene therapies for patients with hemoglobin disorders as well as non-curative therapies like allogeneic stem cell transplantation. Gibson and Al-Juhaishi also discussed strategies for mitigating occlusive disease and other potential toxicities associated with beti-cel.

“It's a really exciting time to be working in this fieldwhere we finally have really good options for these patients. From our experience and from clinical trials, beti-cel and likely exagamglogene autotemcel [Casgevy]...are very effective, curative therapies for thalassemia in the real-world setting, and we've seen very similar results in sickle celldisease,” said Gibson. “These therapies have been really life-changing for our patients, and they've had a huge reduction in their symptoms and a huge reduction in their burden of health care that's required.”

References

1.     Gibson NM, Friedman DF, Elgarten CW, et al. Post-approval, real-world experience with betibeglogene autotemcel for transfusion-dependent betathalassemia. Transplantation and Cellular Therapy. 2025;31(2):S254. doi:10.1016/j.jtct.2025.01.386.

2.     FDA approves first cell-based gene therapy totreat adult and pediatric patients with beta-thalassemia who require regular blood transfusions. News Release. FDA. August 17, 2022. Accessed April 21, 2025.https://tinyurl.com/3vrkk8kz

CancerNetwork®, in collaboration with the American Society for Transplantation and Cellular Therapy (ASTCT)®, hosted a Twitter/X Space during the 2025 Tandem Meetings. The live conversation featured Rahul Banerjee, MD, FACP, of Fred Hutchinson Cancer Center, and Shernan Holtan, MD, of Roswell Park Comprehensive Cancer Center. Together, they shared expert commentary on new data in CAR T-cell therapy and hematopoietic cell transplantation.

With insights drawn from posters, oral abstracts, and late-breaking presentations, the session unpacked both clinical takeaways and implications for patient care.

Highlights included:

• LBA1 – Ide-cel + lenalidomide maintenance post-transplant in MM patients
  “This merges two worlds,” said Banerjee. “Not a tandem in the classic sense, but a transplant followed by CAR T.”
  [Garfall et al., Abstract LBA-1]

• Abstract 50 – Real-world CAR T use in early relapsed/refractory LBCL
  Holtan noted, “In this cohort, no statistical survival difference was seen between second- and third-line therapy, though the curves suggest a trend.”
  [Rojek et al., Abstract 50]

• Poster 340 – CAR T targeting CD83 in breast cancer
  Banerjee speculated on future innovations: “If we could deliver these locally, without systemic effects, that would be a game changer.”
  [Betts et al., Poster 340]

• Poster 317 – IEC-colitis in cilta-cel-treated patients
  Banerjee reflected, “We assumed steroids would help, like with GVHD. But outcomes didn’t align.”
  [Chang Lim et al., Poster 317]

• Poster 572 – Driving restrictions post-CAR T
  Banerjee advocated for updated policy: “Patients often can’t regain full independence until driving is restored—this restriction lacks strong evidence.”
  [Banerjee et al., Poster 572]

• Presentation 58 – Physical function tests to predict ICANS and mortality
  “These simple tools helped flag patients unlikely to benefit from CAR T,” said Holtan. “This could refine patient selection.”
  [Herr et al., Presentation 58]

• Poster 618 – HCT outcomes for MDS patients by age group
  Holtan expressed optimism: “We need conditioning regimens that are effective yet gentler—especially for older patients.”
  [Duarte et al., Poster 618]

• Presentation 39 – Immune suppression trends post-HCT (18,000+ patients)
  Looking ahead, Holtan predicted a shift in GVHD care: “In 10 years, steroids may no longer be our go-to.”
  [Pidala et al., Presentation 39]

• Poster 516 – Patient experiences with chronic GVHD via social media listening
  Holtan emphasized the human element: “We must prioritize mental health, community support, and advocacy alongside clinical treatment.”
  [Cowden et al., Poster 516]

The discussion emphasized not only the clinical relevance of each study but also broader themes—improving quality of life, adjusting standards of care based on real-world findings, and advancing innovation through thoughtful, patient-centered research.

References:

1. Garfall AL, et al. Abstract LBA-1. Tandem 2025.

2. Rojek AE, et al. Abstract 50. Tandem 2025.

3. Betts BC, et al. Poster 340. Tandem 2025.

4. Chang Lim KJ, et al. Poster 317. Tandem 2025.

5. Banerjee R, et al. Poster 572. Tandem 2025.

6. Herr M, et al. Presentation 58. Tandem 2025.

7. Duarte FB, et al. Poster 618. Tandem 2025.

8. Pidala J, et al. Presentation 39. Tandem 2025.

9. Cowden M, et al. Poster 516. Tandem 2025.

Welcome to the fourth episode of ASTCT Talks’ exclusive 8-part series, supported by an educational grant from Sanofi US. In this episode, former ASTCT President Dr. Corey Cutler sits down with Dr. Connie R. Shi from the Cutaneous Oncology Program at Dana-Farber Cancer Institute. They discuss Dr. Shi's recent article, Cutaneous Chronic Graft-Versus-Host Disease: Clinical Manifestations, Diagnosis, Management, and Supportive Care.

Tune in as they explore the complexities of cutaneous GVHD, including acute and chronic presentations, diagnostic challenges and skin-directed treatment strategies such as topical steroids and phototherapy. They also cover key considerations for recognizing and diagnosing cutaneous GVHD in patients of all skin tones and managing long-term complications like skin cancer risk.

Welcome to the third episode of ASTCT Talks’ exclusive 8-part series, supported by an educational grant from Sanofi US. In this series, ASTCT President Dr. Corey Cutler sits down with authors from the latest Graft-versus-Host Disease and Late Effects Following Allogeneic Transplantation journal collection to discuss their research and its implications for patient care.

In this episode, Dr. Cutler is joined by Dr. Steven Z. Pavletic, the GVHD and Late Effects Section and Myeloid Malignancies Program Director at the NIH in Bethesda, Maryland. Widely recognized as a titan in chronic GVHD research, Dr. Pavletic explores an emerging yet often overlooked area in transplantation medicine—lifestyle medicine.

Together, they discuss the role of lifestyle interventions, including nutrition, exercise, sleep, and stress management, in improving long-term outcomes for patients with chronic GVHD. Dr. Pavletic highlights the growing body of evidence supporting these approaches and the urgent need for more research and clinical guidelines to help providers counsel patients effectively.

Tune in for an eye-opening conversation on how small, practical changes can make a significant impact on quality of life and post-transplant recovery. To dive deeper into this topic, read Dr. Pavletic’s full article here.

In this episode of ASTCT Talks, Dr. Taha Al-Juhaishi welcomes Dr. Lori Muffly, Associate Professor in the Division of Blood and Marrow Transplantation-Cellular Therapies at Stanford University, to discuss groundbreaking advancements in graft selection and manipulation in hematopoietic cell transplantation. They explore the evolution of graft engineering strategies aimed at improving patient outcomes, with a focus on Orca-T, an innovative cell therapy designed to reduce graft-versus-host disease while maintaining graft-versus-leukemia effects. Dr. Muffly provides insights into the clinical development of this promising therapy, its impact on patient recovery, and the future of transplantation. Tune in to learn about the latest research and what it means for the future of cellular therapy.

Welcome to the second installment of an exclusive 8-part ASTCT Talks series, supported by an educational grant from Sanofi US. In this series, ASTCT President Dr. Corey Cutler speaks with authors from each article in the latest

In this episode, Dr. Cutler is joined by Dr. Nathaniel S. Treister, a recognized expert in oral medicine, to discuss the intricacies of oral chronic GVHD and its impact on oral health following allogeneic hematopoietic cell transplantation. They explore the diagnostic criteria, effective management strategies, and long-term care considerations for patients, highlighting the importance of both supportive care and

For further insights, read Dr. Treister’s article, “Oral Chronic

In the next episode of ASTCT's "Titans of Transplant," Dr. Shernan Holtan hosts Dr. Renier Brentjens, a pioneer in CAR T-cell therapy and a transformative leader in transplantation and cellular therapy. Dr. Brentjens discusses the challenges and breakthroughs behind developing the first chimeric antigen receptors (CARs), the pivotal discovery of CD19-targeting hybridomas, and the determination needed to turn bold ideas into clinical reality. Tune in for an insightful look at how early experiments paved the way for today’s CAR T-cell advancements, capturing the passion, persistence, and pivotal moments that shaped a new era in cancer treatment.

Welcome to the first installment of an exclusive 8-part ASTCT Talks series, supported by an educational grant from Sanofi US. In this series, ASTCT President Dr. Corey Cutler speaks with authors from each article in the latest ASTCT Journal of Transplantation and Cellular Therapy GVHD supplement.

In this opening episode, Dr. Cutler is joined by Dr. Katie Luo, a leading ophthalmologist, to discuss the complexities of ocular GVHD, why it goes beyond “just dry eye,” and how an integrated, multidisciplinary approach can offer better patient outcomes.

For further insights, read Dr. Luo’s full article, “Understanding Ocular Graft-versus-Host Disease to Facilitate an Integrated Multidisciplinary Approach”, and stay tuned as Dr. Cutler delves into each article with its author, providing a comprehensive guide on GVHD management through this entire supplement series.

In a special co-branded episode between Oncology On the Go hosted by CancerNetwork® and the American Society for Transplantation and Cellular Therapy (ASTCT)’s program ASTCT Talks, Alexis K. Kuhn, PharmD, BCOP, spoke with Katie Bruce, PharmD, BCPPS, and Susie Long, PharmD, about the use of approved cell-based gene therapies for patients with sickle cell disease, beta thalassemia, adrenoleukodystrophy (ALD), and metachromatic leukodystrophy (MLD). These panelists shared the pharmacist’s perspective on ensuring quality care with these ex vivo gene therapies across all treatment phases, including mobilization, conditioning, and infection prophylaxis.

Kuhn is an ambulatory Pediatric Hematology/Oncology/BMT Pharmacist at the Mayo Clinic in Rochester, Minnesota, and an assistant professor of Pharmacy at the Mayo Clinic College of Medicine. Bruce is a pediatric clinical pharmacy specialist at the Sarah Cannon Pediatric Hematology/Oncology & Cellular Therapy program of Tristar Centennial Medical Center in Nashville, Tennessee. Long is a pediatric clinical pharmacist in the Blood and Marrow Team at the University of Minnesota Masonic Children's Hospital.

Specifically, the panelists spoke about the use of agents like elivaldogene autotemcel (Skysona) and atidarsagene autotemcel (Lenmeldy), which are FDA-approved for ALD and MLD, respectively. They also discussed the use of exagamglogene autotemcel (Casgevy) and lovotibeglogene autotemcel (Lyfgenia), which the FDA approved for treating patients 12 years and older with sickle cell disease in December 2023. The conversation broke down each stage of treatment, detailing optimal strategies for the cell manufacturing and storing processes as well as the management of toxicities like cytopenias. They also reviewed key considerations during the post-infusion period that may help maximize the quality of life for patients after they complete their therapy.

“It has been so amazing to be able to be a part of gene therapy and gene editing,” Bruce stated regarding the potential long-term impacts of these treatments. “We have patients who are able to hold full-time jobs they never were able to have before. We have patients who are climbing mountains and backpacking through Europe, which would have never been an option before because their sickle cell disease would have prevented them from [doing] that…. It’s not an easy process, and it has a lot of steps for the patient to go through, but the reward at the end of it all is worth it.”

References

1. bluebird bio receives FDA accelerated approval for SKYSONA® gene therapy for early, active cerebral adrenoleukodystrophy (CALD). News release. bluebird bio, Inc. September 16, 2022. Accessed October 7, 2024.https://tinyurl.com/mp8crxes

2. FDA approves first gene therapy for children with metachomatic leukodystrophy. New release. FDA. March 18, 2024. Accessed October 7, 2024. https://tinyurl.com/mrh659yk

3. FDA approves first gene therapies to treat patients with sickle cell disease. News release. FDA. December 8, 2023. Accessed October 7, 2024. https://tinyurl.com/3zbdnf4c

This episode of ASTCT Talks dives into outpatient CAR T therapy, exploring logistics, challenges, and success strategies. Host Dr. Zahra Mahmoudjafari leads a panel of experts to share insights from their innovative programs.

The panel features Robb Richards, Administrative Director of Cell Therapy and Transplant at Penn Medicine; Dr. Katie Gatwood, Clinical Pharmacy Specialist at Vanderbilt University Medical Center; and Dr. Taha Al-Juhaishi, Associate Director at the University of Oklahoma’s Transplant and Cell Therapy Program. Topics include outpatient program structures, toxicity management, and the evolving role of cell therapies beyond hematologic malignancies.

About the Host:
Dr. Zahra Mahmoudjafari is a board-certified oncology pharmacist and Clinical Pharmacy Manager in Hematologic Malignancies at the University of Kansas Cancer Center. She earned her PharmD and MBA from UMKC and focuses on clinical and operational management of cell and gene therapies. Dr. Mahmoudjafari is active in HOPA, ATOPP, and ASTCT and was honored with ASTCT's Pharmacy SIG Lifetime Achievement Award and ASCO’s 40 Under 40 in Cancer Award.

Meet the Panel:

Listeners will gain valuable insights into the complexities of managing outpatient CAR T therapy and strategies to enhance patient care.