Juliane Gust, MD, PhD, on Evaluating Longterm Neurological Outcomes for CAR-T in Pediatric Patients


The assistant professor of neurology at University of Washington, Seattle Children's discussed efforts to address a gap in knowledge in the long-term effects of CAR-T, if any, on children’s neurological development.

“We really currently have no evidence that CAR T-cells are harmful for the brain in the long term. There may be more subtle things that happen—we really have not done the work yet. But my hope is that in the end, we will find that CAR T-cells are more gentle for the brain and its development than our standard therapies that we're currently using.”

One of the most well-known common adverse events associated with chimeric antigen receptor T-cell (CAR-T) therapies is immune effector cell-associated neurotoxicity syndrome (ICANS). Although, ICANS is an acute adverse event typically manifesting shortly after administration of the treatment and ideally subsiding soon after its appearance. Furthermore, despite the fact that pediatric patients have been receiving CAR-T therapies for over 10 years now, CAR-T is still a relatively new therapeutic modality for children and adults alike. As such, data on long-term neurological effects on patients receiving CAR-T therapy, if any, are limited. In children especially, further study is needed to determine whether CAR-T may have a negative effect on neurological development.

Juliane Gust, MD, PhD, an assistant professor of neurology at University of Washington, Seattle Children's, is giving a talk entitled “Delayed Neurotoxicities Following CAR T-Cell Therapy” at the 2024 Tandem Meetings | Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR, held in San Antonio, Texas, February 21-24, 2024. In an interview with CGTLive® held shortly before her talk, Gust discussed the rationale behind her presentation and the key points that she will be speaking on. She noted that standard of care therapies for children with cancer such as chemotherapy, radiation, and transplant have well-documented long-term effects, but that long-terms effects for CAR-T in children are still largely unknown. Gust also highlighted a study that is currently underway via the National Cancer Institute’s CARnation Consortium that will seek to address this gap in knowledge through the use of remote follow-up assessments.

1. Gust J. Delayed neurotoxicities following CAR T-cell therapy. Presented at: 2024 Tandem Meetings, February 21-24, San Antonio, Texas.
Related Videos
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
PJ Brooks, PhD
© 2024 MJH Life Sciences

All rights reserved.