Background: Paediatric Strategy Forums have been created to evaluate the current state of the science, facilitate dialogue, and provide an opportunity for constructive discussions between relevant stakeholders (patient advocates, clinicians, academics, biotechnology/pharmaceutical companies, and regulators) on specific topics in an open forum to assure development of medicines in the best interests of children and adolescents with cancer. The goal of these meetings is to share information, to facilitate the development of innovative medicines and ultimately their introduction into the standard-of-care of children with malignancies.

The Fourteenth Multi-Stakeholder Paediatric Strategy Forum organised by ACCELERATE in collaboration with the European Medicines Agency (EMA) and with the participation of the U.S. Food and Drug Administration (FDA) will focus on novel ways to target and enhance efficiency of anti-GD2 therapies.

The disialoganglioside GD2 is expressed on several paediatric malignancies: neuroblastoma, diffuse midline glioma, osteosarcoma, Ewings sarcoma and soft tissue sarcoma. GD2 has been very effectively targeted in neuroblastoma with monoclonal antibodies and this approach is now a component of standard of care both during residual disease therapy and, with chemotherapy in relapse. In addition to monoclonal antibodies, other tactics are now being utilised to target GD2, including CAR T-cells, antibody–drug conjugates, radiolabeled antibodies, T-cell engaging bispecific antibodies and vaccines. Furthermore, combinations are being explored to increase the expression of GD2 and thereby activity. GD2 CAR T-cells have been demonstrated to be active in neuroblastoma and, outside neuroblastoma, they have shown some early very promising results.

There is a need to develop a strategy for the development of new therapies targeting GD2 and to learn from past experience, understand the mechanisms by which these approaches target GD2, identify in which clinical setting they are most effective and prioritise products. 

This Paediatric Strategy Forum will discuss the mechanisms of action of different approaches to target GD2 and their clinical applicability as well as tools to detect GD2 and mechanisms to increase and modulate its expression. Lessons learnt from the development of anti-GD2 monoclonal antibodies in neuroblastoma will be highlighted, including predictors of response, the importance of diagnostics for HACA and HAHA and combination approaches. The clinical benefits and indications of other ways to target GD2 (e.g., CAR T-cells, antibody–drug conjugates, radiolabeled antibodies, and T-cell engaging bispecific antibodies) will be reviewed as well disease specific approaches for diffuse midline glioma, osteosarcoma and other malignancies.

The goals of the Forum will be to:

•Identify a rational strategy to selecting specific approaches to target GD2;

•Define a strategy for the further development of monoclonal antibodies;

•Define a strategy to develop and evaluate new products;

•Identify and prioritise most promising  new medicinal products;

•Highlight the importance of diagnostics for GD2 expression and HACA and HAHA.

Format: This forum will be held in person and all participants are strongly encouraged to attend it. For attendees unable to travel or if travel restrictions prevent an in-person event, a virtual option will be available.   

Forum Output: Publication of a summary and manuscript from all participants addressing the challenges and documenting the conclusions of the meeting. Potentially further specific meetings relating to areas which require more in-depth discussion will be arranged.

Participation: Possible only by invitation or by submitting an Expression of Interest (see below) before the 30 June 2024. 

Submit your Expression of Interest: The call for expressions of interest is open now – please use the box available below. Invitations will be sent out by the end of July 2024.