Within one year of MIHRA’s incorporation as a 501c3 non-profit charitable research organization, the MIHRA Clinical Trial Sites Network (CTSN) Advisory Team announced the 1st CTSN Community Forum to occur on 14 December 2024. This was in response to pressing communications from predominantly earlier investigators regarding differentiating benefits between CAR-T / CAR-NK trial selection, logistics of trial initiation and patient selection. Thus, demonstrating the need for and the remarkable utility of a program like the MIHRA CTSN to galvanize the community. The recording of the entire forum can be found Here or in video segments below:
The forum was devised to elicit and delineate the spectrum of community perspectives and experiences on Overcoming Challenges of CAR Therapy Trials. In the weeks preceding the forum, the CTSN Advisory initiated a qualitative study to elicit and characterize patient and investigator perspectives, concerns and challenges perceived by the stakeholder community. The study will remain open and continue to collect the perspectives via the survey for people living with myositis and survey for investigators until 15 January 2025.
The public forum was chaired and moderated by scientific and patient experts in myositis and was attended by participants representing wide-ranging career levels, multiple specialties, disciplines, stakeholder types.
The program focused specifically on CAR therapies over other cellular therapies for two reasons:
- to ensure focused, clear discussions with distinct, interpretable content
- CAR therapies being very complex in multi-faceted ways is anticipated to provide a framework for future discussions and deliverables on other cellular therapies
Upon achieving its primary goals to the satisfaction of the attendees, the forum ultimately evolved into a summit. A preliminary road map prioritized the most pressing actionable concerns of the community identifying long and short-term deliverables to improve patient and investigator experience as well as the manageability of the clinical trial landscape
The forum began with video perspectives submitted by investigator and patient stakeholders that can be viewed here:
This was followed by the interim analyses of the ongoing qualitative investigations to characterize the climate of concerns and challenges perceived by the stakeholder community. This can be viewed here:
The forum then proceeded in three major discussion areas, preceded presentations to stimulate discussion:
i. Overview of CAR Therapy Trials and potential relevance in adult and pediatric autoimmune diseases with by presentations from Pedro Machado (adult rheumatology), Susan Shenoi (pediatric rheumatology) and Victoria Werth (dermatology):
ii. Logistics of CAR Therapy Trial initiation, budgeting, inter-reliance and division of oversight with oncology colleagues with presentations by Mazen Dimachkie (adult neurology) and Gary Gilkeson (immunology).
iii. Patient Safety, Outcomes, Selection, Vulnerability Perspective, Cost-Viability & Respectful Education presented by Marianne de Visser (adult neurology), Lesley Ann Saketkoo (adult/pediatric rheumatology), Karen Cheng (patient partner) and Ingrid de Groot (patient partner).
Patient advisors and investigators characterized urgent concerns related to lack of appropriate education materials for both patients and investigators that clearly outline procedures as well as efficacy and safety outcomes and appropriate patient selection and education as relates to patients with connective tissue diseases and myositis-related disorders. The recording can be viewed here:.
MIHRA CTSN Faculty:
Antonia Valenzuela, Lesley Ann Saketkoo (MIHRA CTSN Program Leads)
Helene Alexanderson, Karen Cheng, Lisa Christopher-Stine, Ingrid de Groot, Marianne de Visser, Mazen Dimachkie, Kristin Highland, Pedro Machado, Chester Oddis, Julie Paik, Vicky Werth (MIHRA CTSN Advisory)
Latika Gupta, Felix Muhlensiepen, Vincenzo Venerito (MIHRA CAR-T Project Data Stewards)
Barbara Shafranski, Elie Naddaf, Floranne Ernste, Helene Alexanderson, Julie Paik, Lesley Ann Saketkoo, Mazen Dimachkie, Pedro Machado, (MIHRA Board of Directors)