ASTCT Talks

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.

In this episode of ASTCT Talks, host Dr. Andrés Gómez De León is joined by Dr. Nandita Khera and Dr. Alexandra Gomez Arteaga to explore the critical issue of disparities and barriers to care in graft-versus-host disease (GVHD). The discussion delves into the factors contributing to unequal access to hematopoietic cell transplantation, such as socioeconomic status, race, and ethnicity, both in the U.S. and globally. The guests highlight initiatives like the ACCESS Initiative by ASTCT and the National Marrow Donor Program, aimed at addressing these disparities through advocacy, awareness, and training for junior faculty. They also discuss the importance of caregiver support, clinical trial accessibility, and the broader impacts of GVHD on patients’ lives. Tune in to gain valuable insights into ongoing efforts to promote equitable access to life-saving treatments.

About Dr. Nandita Khera

Nandita Khera is a Professor of Medicine in the Mayo Clinic College of Medicine and a Consultant in the Division of Hematology/ Oncology at Mayo Clinic Arizona. She treats patients with hematological malignancies and some solid tumors, especially those needing blood and marrow transplant/cell therapy (BMT/CT). Her research focuses on improving the delivery of care to patients with cancer including those undergoing BMT/CT to help them be better prepared for the psychosocial and financial consequences of the treatment. She has published several papers in outcomes, late effects, and quality of care in cancer patients and provides mentorship to trainees interested in projects in these areas. She has been a member and has held leadership positions at Mayo Clinic and in the various committees in organizations/ societies in hematology and BMT/CT.. She was the working committee co-chair for the Health Services and International Issues Working committee of CIBMTR from 2015 to 2020 and a member of BMT-CTN SOSS Late Effects committee in 2020. As the co-chair of Dissemination and Implementation committee at BMT CTN currently, she leads efforts in improving translation of evidence into practice in the field of BMT. She is the Director of Community or Clinical Practice at ASTCT.

About Dr. Alexandra Gomez Arteaga

Dr. Alexandra Gomez Arteaga is an Assistant Professor at Weill Cornell Medicine/NewYork-Presbyterian Hospital and directs the Allogeneic Bone Marrow Transplant Service and the Advanced Fellowship in Bone Marrow Transplantation. She earned her MD from Los Andes University, completed her residency at the University of Miami, her Hematology/Oncology fellowship at Weill Cornell, and her BMT advanced fellowship at Memorial Sloan Kettering Cancer Center. Dr. Gomez’s research focuses on improving outcomes in allogeneic stem cell transplantation for leukemia and myeloid malignancies, with an emphasis on young adults and alternative donors. Her work also addresses the critical need to decrease disparities in access to transplantation for minority populations, a cause she champions across her clinical practice, research, and advocacy. She currently serves as the Co-Chair for the Junior Faculty Initiative within the ASTCT ACCESS Initiative.

About Dr. Andrés Gómez De León

Dr. Andrés Gómez De León (@GomezDLeonMD) is an Associate Professor at Universidad Autonoma de Nuevo Leon in Monterrey Mexico and an ASTCT Content Committee member with an interest in acute leukemias and transplant and cell therapies in low and middle income countries.

In a special co-branded episode between Oncology On the Go and the American Society for Transplantation and Cellular Therapy (ASTCT)’s program ASTCT Talks, Rahul Banerjee, MD, FACP, and Noopur Raje, MD, discussed the risk of secondary malignancies in patients with multiple myeloma who receive CAR T-cell therapy.

Banerjee is an assistant professor in the Clinical Research Division of Fred Hutchinson Cancer Center and an assistant professor in the Division of Hematology and Oncology at the University of Washington. Raje is the director of the Center for Multiple Myeloma at Massachusetts General Hospital Cancer Center and a professor of medicine at Harvard Medical School.

Banerjee and Raje spoke in the context of prior advisories from the FDA on the potential development of secondary T-cell malignancies in patients who receive CAR T-cell therapy for hematologic cancers. Specifically, the agency required a boxed warning for secondary T-cell malignancy risks for BCMA- or CD19-targeting therapies in April 2024.1 The conversation also touched upon reports of secondary malignancies in cases and trials such as CARTITUDE-1 (NCT04181827), in which second primary cancers were highlighted in 9 patients who received treatment with ciltacabtagene autoleucel (Carvykti).2

Considering these reports and warnings, Banerjee and Raje emphasized shared treatment decision-making with patients after assessing the risks and benefits of CAR T-cell therapy compared with other agents like bispecific antibodies. They also reviewed optimal strategies for monitoring and referring patients based on the incidence of certain toxicities.

“[Treatment with] CAR T cells requires planning, and we need to have good control of the disease. We need to have 4 to 6 weeks of a lead time to get these effective treatments to our patients, so early referral is a good idea,” Raje said. “[For example], if you see chronic diarrhea in someone that is way out of the window of what you would expect, referring back to the CAR T-cell center is important so that we don’t miss some of these toxicities.”

In this episode of ASTCT Talks, Dr. Muhammad Salman Faisal is joined by Dr. Jens Hillengass. Dr. Hillengass discusses the latest advancements in CAR T cell therapy, focusing on its application in early-line settings following the results of the CAR-TITUDE 4 and KarMMa-3 trials. Tune in as he shares insights on the efficacy, patient selection and future directions of CAR-T cell therapy in multiple myeloma. This episode is supported by Johnson & Johnson and Legend Biotech. Don’t miss out on this informative discussion with one of the leading experts in the field.

About Dr. Jens Hillengass

Jens Hillengass, MD, PhD, is Professor of Oncology and Chief of the Myeloma and Amyloidosis Service at Roswell Park Cancer Institute, Buffalo, NY. Previously, he was the head of the autologous stem cell transplant program and the deputy chief of the multiple myeloma section at the University Hospital of Heidelberg, Germany. He earned his medical degree, residency, and fellowship at Heidelberg University, and completed a research fellowship at the National Cancer Institute. Dr. Hillengass co-chairs the bone and imaging group of the International Myeloma Working Group and serves on the Multiple Myeloma guideline committee at the National Comprehensive Cancer Network.

About Dr. Muhammad Salman Faisal

Dr. Muhammad Salman Faisal is currently a fellow at Roswell Park Comprehensive Cancer Center, in his third year of training, with keen interest in myeloma, CAR-T and access to care.

In this episode of "ASTCT Talks," Dr. Shernan Holtan sits down with Dr. Samer Al-Homsi, as he shares his insights on the future of graft versus host disease (GVHD) prophylaxis, particularly in haploidentical transplantation.

Dr. Al-Homsi delves into the innovative CAST regimen, which combines post-transplant cyclophosphamide, abatacept and short-duration tacrolimus. Learn about the challenges, breakthroughs and promising results that could revolutionize how we approach GVHD prevention and treatment, making a future without GVHD a reality.

Listen in as we explore the potential for reducing transplant toxicities and the impact on patient care.

About Dr. Samer Al-Homsi

A. Samer Al-Homsi, MD, MBA, is the System Chief of Blood and Marrow Transplant and Cellular Therapy at Northwell Health Cancer Institute and serves as Director of Faculty and Academic Affairs in Medical Oncology. He is a Professor of Medicine at the Zucker School of Medicine and of Cancer Research at Feinstein Institutes of Medical Research. Previously, he was the Executive Director of Blood and Marrow Transplantation at NYU Grossman School of Medicine.

Dr. Al-Homsi graduated from Damascus Medical School and trained in Hematology and Medical Oncology in France. He completed his residency at Advocate Health Care and fellowship at the University of Massachusetts. He has led programs in Malignant Hematology and Blood and Marrow Transplantation at several institutions, including NYU Langone Health.

His research focuses on preventing graft-versus-host disease (GvHD), developing innovative approaches such as the CyBor and CAST regimens, particularly in haploidentical transplants. Dr. Al-Homsi aims to address healthcare disparities due to donor shortages among minority groups.

Dr. Al-Homsi is also the President of the American Arab Assembly of Cellular Therapy and Transplantation (AAACTT), promoting collaboration among its members.

About Dr. Shernan Holtan

Dr. Holtan is a clinical/translational investigator. Her work focuses on increasing resilience, both at the tissue level to prevent and treat GVHD, and at the whole person level, designing exercise programs to mitigate cancer therapy-associated aging. In GVHD, her early work focused on epidermal growth factor and pregnancy hormones to facilitate tissue repair in GVHD, which led to a successful phase II study for the treatment of high-risk acute GVHD and discovery of the GVHD biomarker amphiregulin. More recently, she has led and published two prospective clinical trials regarding the use of PTCy as GVHD prophylaxis, the results of which are changing practice around the globe.

Dr. Holtan has also been a competitive powerlifter, setting a national record in 2019. She has leveraged her knowledge regarding strength training into translational studies and clinical trials that are helping to reverse some of the damage done by high-dose chemotherapy and radiation. Through her multidimensional accomplishments, Dr. Holtan has demonstrated a unique and powerful intersection of medical research and physical resilience, redefining standards of care in HCT, and innovatively combating the deleterious effects of cancer treatment.

In this episode "ASTCT Talks,” hosts Rebecca Epperly, MD, and Aimee Talleur, MD, from St. Jude Children's Research Hospital, dive into the pioneering realm of CAR T-cell therapy and its late effects on pediatric patients. The discussion unfolds at the intersection of innovation and caution, highlighting the recent sessions from the 2024 Tandem meetings. They explore the emerging challenges and lack of data concerning long-term impacts of this revolutionary treatment, particularly in pediatric, adolescent, and young adult populations. This episode sheds light on both the immense potential and the imperative need for comprehensive studies to better understand and mitigate the long-term consequences of cellular therapies in treating high-risk malignancies.

About Dr. Rebecca Epperly, MD

Dr. Rebecca Epperly is an Instructor in the Department of Bone Marrow Transplantation and Cellular Therapy (BMTCT) at St. Jude Children’s Research Hospital. After gaining undergraduate degrees in biochemistry (BS) and music performance (BA), she received an MD from the University of Iowa Carver College of Medicine. She then completed pediatrics residency at the UPMC Children’s Hospital of Pittsburgh and fellowships in pediatric hematology/oncology and BMTCT at St. Jude. As a clinician scientist, she is now working to improve outcomes for pediatric patients with high-risk malignancies using cellular based immunotherapy, with a focus on developing early-phase CAR T cell studies and evaluating the delayed effects of novel therapies.

About Dr. Aimee Talleur, MD

Dr. Aimee Talleur is an Assistant Member in the Department of Bone Marrow Transplantation and Cellular Therapy (BMTCT) at St. Jude Children’s Research Hospital, specializing in the clinical investigation of novel immunotherapies for the treatment of high-risk malignant disorders. She completed her BA at Union College, MD at SUNY Upstate Medical University, pediatric residency at Children’s National Medical Center, and fellowships in pediatric hematology/oncology and BMTCT at St. Jude. As a clinician scientist, Dr. Talleur focuses on the advancement of novel cellular therapies through early-phase clinical trials, including CAR T cell therapy. Additionally, her work includes the evaluation of acute and long-term toxicities of this immunotherapy approaches, seeking to better define such toxicities to inform upon predictive and intervention strategies.

In this latest episode of ASTCT Talks, Rachel Schollmeier and NMDP’s Samantha Watters host author Mike Niles, delving into the captivating history of the Anthony Nolan bone marrow transplant register. Explore the evolution of transplantation, from no donor registries to international initiatives, and gain a deeper understanding of the challenges faced by bone marrow registries today. Discover the inspiring narratives behind bone marrow donation and the transformative advancements that continue to shape the future of transplantation. Plus, don't miss the chance to connect with Samantha's podcast, NMDP ‘Explore Cell Therapy,' for even more insights into the world of cellular treatments.

In the next installment of ASTCT’s Titans of Transplant series, Dr. Stephanie Lee is interviewed by Dr. Jennifer Saultz.

Dr. Lee shares her journey from Seattle to becoming a professor at Fred Hutch Cancer Center. The conversation highlights early motivations, challenges faced during fellowship, and the influence of her father's advice to always strive for the best. They also discuss Dr. Lee's groundbreaking work in chronic graft-versus-host-disease research, collaborative efforts, FDA-approved treatments, and the evolving landscape in the field.

The Titans of Transplant series seeks to recognize, celebrate and chronicle the physicians, researchers, pharmacists, nurses, social workers and more who were on the frontlines of the early days of transplant.

About Stephanie Lee, MD, MPH

Dr. Stephanie Lee (@StephanieLeeMD) is a hematologist and physician scientist who is Professor and Associate Director at the Fred Hutchinson Cancer Research Center. Dr. Lee works to improve the lives of blood stem cell transplant and bone marrow patients by better understanding the chronic form of graft-versus-host disease. Dr. Lee is the former President of the American Society of Hematology.

About Jennifer Saultz, D.O.

Dr. Jennifer Saultz (@jennifernsaultz) is an Assistant Professor of Medicine and physician scientist in the Adult Transplant and cellular therapy program at Oregon Health & Science University. Her research focuses on augmenting NK cellular therapy in myeloid malignancies. She is also a member of the ASTCT Content Committee.