We are delighted that Dr. Daniel Morgenstern is once again hosting the NPGS live event, which he will open for the third year running. Dr. Morgenstern will welcome you to the event and help you to engage with the event platform and find resources before delivering the Symposium's first session on treating high-risk neuroblastoma.

This session is intended as a useful introduction for all those interested in high-risk neuroblastoma, including parents, clinicians, academics and health professionals. Dr. Morgenstern will give a basic overview of the current approach to treating high-risk neuroblastoma across the globe. This snapshot of where we are in 2022 will provide context to other sessions throughout the day, and we hope will be a good starting point for those who are new to the neuroblastoma landscape. 

Since his initial medical training in the UK, Dr. Morgenstern has focused his career on the treatment of neuroblastoma and the development of novel therapeutic approaches for hard-to-treat childhood cancers through early phase clinical trials. He is now the Director of the New Agent and Innovative Therapy and Therapeutic MIBG Programs at Sick Kids in Toronto. 

Challenges exist for all families that are faced with a neuroblastoma diagnosis, but there are unique challenges that come with the high-risk form of the disease. This session will look at some of these challenges that make high-risk neuroblastoma a complex disease to treat, and how the research community is looking to address them. It will be a useful overview for families who are interested in learning about the latest areas of research to improve treatments for neuroblastoma. 

Finding the right treatment, for the right patient with Dr. Jan Molenaar

Professor Jan Molenaar and his research group at the Princess Máxima Center in the Netherlands focus on research into precision medicine in paediatric cancer. This talk will look at the evolution of molecular research in neuroblastoma and how this might bring a more personalised approach to treating children with the disease. 

Chemoimmunotherapy, a major breakthrough? with Dr. Rajen Mody

Combining low doses of chemotherapy with immunotherapy is a new area of focus in treating high-risk neuroblastoma, particularly for refractory disease. Dr. Rajen Mody and his group at Michigan Medicine are leaders in this field, being one of the first to trial combinations of chemotherapy and the immunotherapy agent anti-GD2 antibody dinutuximab. In this talk he will focus on the significance of chemoimmunotherapy in frontline and relapse treatments, as well as current research on optimizing combination therapies. 

Is an extended induction right for some patients? with Dr. Sara Federico

One of the challenges in treating high-risk neuroblastoma is that the disease varies greatly from case to case, so different patients might need very different approaches to therapy. Dr. Sara Federico at St. Jude Children’s Research Hospital is currently exploring the effectiveness of extended induction chemotherapy. In this talk she will share details of this late preclinical work, what it might mean for the clinic and how clinicians might identify which patients would benefit.

High-risk neuroblastoma: toward more cures and better outcomes, 2022 ASCO publication, with Dr. Steven DuBois

Dr. Steven Dubois is an Associate Professor in Paediatrics at Harvard Medical School, with an active clinical and translational research program focused on patients with neuroblastoma. In this talk he will present a summary of his recent publication, which evaluates the current efforts to improve outcomes for children with neuroblastoma, both in frontline and relapsed/ refractory cases. 

Read the publication here: https://ascopubs.org/doi/full/10.1200/EDBK_349783 

Followed by a 45 minute Q&A with Professor Jan Molenaar, Dr. Rajen Mody, Dr. Sara Federico, Dr. Steven DuBois 

Moderated by Donna Ludwinski.

This 45-minute discussion panel will give parents the opportunity to bring questions to these leading health and research professionals in the neuroblastoma field. As well as answering questions on their presentation areas, the panel will also address other queries that parents may have about diagnosis or treatment. The discussion panel is an opportunity to ask questions and shape the discussion - all panellists have a wealth of knowledge and experience in dealing with neuroblastoma so can answer questions on a range of subjects in addition to their presentation topic.

CAR-T cell therapy is an emerging area of research for treating neuroblastoma, mostly in cases of relapse. This therapy type is now more common in other types of cancer including leukemia and lymphoma, but the science behind using CAR-T cells for neuroblastoma is still developing. This focused session has been included to give in-depth information on this area of cutting-edge research, based on feedback from our previous events.

Optimising CAR-T cells against neuroblastoma with Dr. John Anderson

Professor John Anderson will start this session with an overview of the basics on CAR-T cell therapy- what is it and where are we at now? He will also draw on the preclinical work of him and his group at UCL GOSH Child Institute for Health, on designing and developing CAR-T cells that may be effective in treating neuroblastoma. 

GD2 specific CAR and interleukin-15 expressing autologous NKT cells with Dr. Andras Heczey

Dr. Andras Heczey, based at Texas Children’s Hospital specialises in immunotherapy research for solid tumours. In this talk he will present his recent work on the GD2 NKT CAR (Natural Killer T cells are genetically modified with a GD2-CAR and IL15), which is now being investigated in a Phase I clinical trial in the United States. 

PHOX2B peptide-specific CAR-T cells with Dr. Mark Yarmarkovich

PHOX2B is a gene that plays an important role in the development of nerve cells. Previous research has shown that mutations in this gene can be associated with the development of neuroblastoma, which arises from early nerve cells. Dr. Mark Yarmarkovich at Children’s Hospital of Philadelphia is researching CAR-T cell therapy that is engineered to target PHOX2B proteins, and will present his near-clinical work during this talk. 

GPC2 CAR for neuroblastoma with Dr. Rosa Nguyen

Dr. Rosa Nguyen specialises in the discovery and preclinical development of immunotherapies for neuroblastoma. Her recent work focuses on identifying effective targets in neuroblastoma that can be used to develop CAR-T cell therapies. In this talk she will present her preclinical work on GPC2 targeting CAR, which will soon move to a first-in-human clinical trial in the United States. 

Followed by a 45 minute Q&A with Professor John Anderson, Dr. Andras Heczey, Dr. Mark Yarmarkovich, Dr. Rosa Nguyen, Dr. John Maris 

Moderated by Antonia Palmer

This 45-minute discussion panel will give parents the opportunity to bring questions to leading research experts in the neuroblastoma field. As well as answering questions on their presentations, the panel will also address more general queries that parents may have about diagnosis or treatment. The discussion panel is an opportunity to ask questions and shape the discussion - all panellists have a wealth of knowledge and experience in dealing with neuroblastoma so can answer questions on a range of subjects in addition to their presentation topic.  

This special interest talk will focus on the well-known type of immunotherapy using anti-GD2 antibodies. This is used as standard frontline therapy for high-risk neuroblastoma in a number of countries across the globe, and is also being developed in other areas through clinical research. It will be led by anti-GD2 experts Dr. Robbie Majzner and Dr. Juliet Gray. 

Overview and application

Dr. Robbie Majzner is based at Stanford University School of Medicine and specialises in bringing novel immunotherapies to clinical trials for patients with neuroblastoma. In this session he will give an overview of current exploratory work surrounding anti-GD2 therapies, including where they can be optimized and for whom. 

Clinical trials of anti-GD2 antibodies

Dr. Juliet Gray is a paediatric oncologist at Southampton Hospital and at University of Southampton’s Centre for Cancer Immunology. In this session she will provide an overview of the clinical landscape of anti-GD2 treatment - what is currently being progressed through trials and what direction is the research landscape heading in.

This session will also include a 20-minute discussion where you will have the opportunity to put your questions to the experts.

Moderated by Gregory Sizikov

“My child has finished treatment – It’s OK not to feel OK” 

Hayley Blackwell 

In this special interest talk, neuroblastoma parent, Hayley Blackwell, will share her honest experience of life after treatment and the mixed emotions it can bring. She will talk about ‘Scanxiety’ and relapse fears, guilt, adapting to a ‘new normal’ and processing the trauma of diagnosis and treatment whilst navigating late effects.  

This session is a safe space for parents with a child post treatment, to breathe a sigh of relief that it’s OK to feel emotions other than happiness after treatment.  

Discussion Panel

Anita Keresztes, Antonia Palmer, Jude Sibley

This session will include a 25-minute discussion where parents can share their experiences and ask questions to our parent panel, who will in turn, share their experiences and insights.

Dr. Daniel Morgenstern provides a summary of the day's presentations and the event closes with a moving tribute to neuroblastoma children from around the world.