Thai researchers outline early-phase trial of a new GD2 CAR-T product

A published protocol describes a planned first-in-human trial of an investigational product: an autologous chimeric antigen receptor T-cell therapy designed to recognize GD2, a tumor-associated sugar molecule linked to several aggressive childhood cancers. The TARGETS study, led by researchers in Thailand, will enrol approximately 20 patients with relapsed or refractory GD2-positive solid tumours across multiple centres. It is a prospective, open-label, single-arm phase I/IIa basket trial, meaning that participants will receive the same investigational treatment while researchers first assess its safety and then explore signs of anti-tumour activity. The protocol was accepted by BMC Cancer and published online on 19 October 2026, while the trial is registered in the Thai Clinical Trials Registry as TCTR20251222009.

The trial focuses on solid tumours for which CAR T-cell treatment remains far less developed than it is for leukaemia, lymphoma and multiple myeloma. Among the representative cancers named in the protocol are neuroblastoma, glioma and osteosarcoma. These diseases can carry aggressive biological features, respond poorly to conventional treatment and frequently relapse despite intensive therapy. Patients whose disease returns or proves resistant to existing approaches have few effective options, particularly when standard agents have been exhausted. TARGETS therefore addresses both a scientific problem—the limited success of CAR T cells against solid tumours—and a clinical one—the shortage of promising treatments for patients with advanced refractory disease.

GD2 is a ganglioside, a class of molecules composed of sugars linked to a fatty component, and it is expressed by a variety of tumours. Its presence can make it useful as a target, but the effectiveness of an immune treatment depends on more than simply recognising the molecule. A CAR is a synthetic receptor that gives a T cell the ability to detect a specified surface antigen and trigger an immune attack when that antigen is encountered. The investigational product described in TARGETS uses a newly engineered second-generation CAR directed against GD2, combining the antigen-binding machinery of the receptor with intracellular signalling designed to activate the modified T cell.

All participants will receive autologous CAR T cells, meaning that the therapeutic cells are manufactured from each patient’s own immune cells rather than supplied as an off-the-shelf donor product. Before treatment, investigators will collect peripheral blood through leukapheresis, isolate the relevant cells and engineer them to express the anti-GD2 receptor. Manufacturing will follow Good Manufacturing Practice requirements, while broader trial procedures will adhere to Good Clinical Practice principles and the Declaration of Helsinki. The approach is personal to each participant but also complex: it requires coordinated blood collection, specialist cell processing, quality testing and transport before the finished product can be returned for administration.

Before the CAR T cells are infused, patients will receive lymphodepleting chemotherapy. This preparatory treatment reduces the number of lymphocytes in the blood and creates space for the engineered cells to expand, while modifying the immune environment to support their persistence and activity. The CAR T product is then administered intravenously. Because the trial involves a newly engineered construct and is the first clinical evaluation of this particular product, investigators will combine intensive inpatient observation after infusion with longer follow-up. The study plans to monitor participants for up to four years, allowing researchers to capture both immediate toxicities and potential longer-term immune effects.

Safety is the primary focus of the first part of TARGETS. The investigators will use a conventional 3+3 dose-escalation scheme across three planned dose levels. In this design, small groups of participants receive an initial dose, and additional patients are enrolled only when the observed toxicities permit escalation. The process helps identify a dose-limiting toxicity and establish the maximum tolerated dose while balancing treatment exposure against risk. A Data and Safety Monitoring Board will oversee the study, and the researchers will record adverse events, serious adverse events and other safety signals using recognised clinical-trial criteria.

A basket design can accelerate the evaluation of a targeted therapy across several tumour types because the defining feature is shared biology rather than a single organ. Here, eligibility centres on tumours that express GD2 and have relapsed or refractory disease. The approach allows the same CAR T-cell product to be tested in neuroblastoma, glioma, osteosarcoma and other qualifying solid tumours, while also revealing whether biological responses differ markedly between diseases. At the same time, the small planned cohort of roughly 20 participants means the trial is not designed to provide definitive comparative evidence. Its role is early-stage: establish tolerability, characterize dose-related effects and look for signals that justify larger, disease-focused studies.

The protocol has passed ethics review by the Human Research Ethics Committees of the Faculty of Medicine at Chiang Mai University, the Faculty of Medicine Ramathibodi Hospital at Mahidol University, and the Faculty of Medicine at Prince of Songkla University. Written informed consent will be obtained from participants or, where applicable, their parents or legally authorized representatives before enrollment, and assent will be sought from children in line with local regulations and institutional policies. Several investigators involved in the study also report a Thai petty patent covering a GD2-specific CAR T-cell receptor. The work was supported by Genomics Thailand and the Health Systems Research Institute, with partial support from Chiang Mai University.

CAR T cells are not a routine treatment for relapsed GD2-positive solid tumours, and the publication describes a study protocol rather than clinical results. No efficacy figures or conclusions about tumour shrinkage are therefore available from this research. The central scientific question is whether a second-generation anti-GD2 receptor can produce a tolerable and potentially useful immune response in a difficult group of cancers. TARGETS will provide early safety data, define a workable dose and offer a first look at whether engineered T cells can shrink established tumours. If those early signals prove encouraging, they could provide the foundation for larger trials designed around the tumour types most likely to benefit.

Editorial clarification: This article describes a published trial protocol, not treatment results or independent confirmation that recruitment has started. “First-in-human” refers to this particular investigational product.

Subject of Research: A phase I/IIa basket trial of autologous anti-GD2 CAR T cells for relapsed or refractory GD2-positive solid tumours

Article Title: A basket trial of autologous Anti-GD2 chimeric antigen receptor (CAR) T Cells in patients with relapsed or refractory GD2-positive solid tumors (TARGETS): study protocol for a phase I/IIa multicenter open-label trial

Article References: Koonrungsesomboon, N., Anurathapan, U., Chaiyawat, P., Dukaew, N., Kittilukkana, A., Orrapin, S., Choed-amphai, C., Sathitsamitphong, L., Charoentum, C., Hongeng, S., Sripornsawan, P., Sangkhathat, S., Preedagasamzin, S., Atjanasuppat, K., Jittorntrum, B., Suanpan, K., Suthapot, P., & Pruksakorn, D. (2026). A basket trial of autologous Anti-GD2 chimeric antigen receptor (CAR) T Cells in patients with relapsed or refractory GD2-positive solid tumors (TARGETS): study protocol for a phase I/IIa multicenter open-label trial. BMC Cancer. https://doi.org/10.1186/s12885-026-17149-x

Image Credits: AI Generated

DOI: 10.1186/s12885-026-17149-x

Keywords: CAR T cells, GD2, solid tumours, neuroblastoma, glioma, osteosarcoma, immunotherapy, dose escalation, clinical trial, Thailand, basket, trial