Drug Development

We live exciting times in the SIOPEN Drug Development Group. Our two cooperative randomised clinical trials for relapsed and refractory patients, RIST and BEACON, are recruiting well and it is expected that they will complete recruitment for the current randomisations soon. We continue to encourage recruitment to ongoing trials in order to speed up the development of new drugs. We are working to incorporate the evaluation of a chemoimmunotherapy strategy with anti-GD2 targeted therapy added to conventional chemotherapy in a future amendment of the BEACON trial.

During this year, we also have started to plan future trials for relapsed & refractory neuroblastoma, once BEACON and RIST close.


A number of early clinical trials are coming to reality thanks to our collaboration with ITCC, including the ESMART and INFORM2 personalised therapy trials opening in an increasing number of European countries.


An algorithm for the treatment of patients with relapsed neuroblastoma is under development and will soon be disseminated to all SIOPEN members.


Lastly, a molecular tumour board with specific neuroblastoma expertise to discuss options for targeted therapies matched to the patient’s genomic profile has been developed and will start on November 8th. More detailed information and a patient registration form are attached.

This virtual meeting will be open to all SIOPEN members which will be able to present their complex cases and have obtained molecular data of the respective patients. After discussion in the board, a treatment recommendation including potential targeted therapy clinical trials available across ITCC/Europe will be given. A recent call for people wishing to join the group has been sent by SIOPEN, but is still time to join, if people is interested in joining our group. There is plenty of work to do and we welcome new members.



Recent publications

Risk stratification of high-risk metastatic neuroblastoma: A report from the HR-NBL-1/SIOPEN study.

Morgenstern DA, Pötschger U, Moreno L, Papadakis V, Owens C, Ash S, Pasqualini C, Luksch R, Garaventa A, Canete A, Elliot M, Wieczorek A, Laureys G, Kogner P, Malis J, Ruud E, Beck-Popovic M, Schleiermacher G, Valteau-Couanet D, Ladenstein R.
Pediatr Blood Cancer. 2018 Nov;65(11):e27363. doi: 10.1002/pbc.27363. Epub 2018 Jul 17.


Phase I results of a phase I/II study of weekly nab-paclitaxel in paediatric patients with recurrent/refractory solid tumours: A collaboration with innovative therapies for children with cancer.
Moreno L, Casanova M, Chisholm JC, Berlanga P, Chastagner PB, Baruchel S, Amoroso L, Gallego Melcón S, Gerber NU, Bisogno G, Fagioli F, Geoerger B, Glade Bender JL, Aerts I, Bergeron C, Hingorani P, Elias I, Simcock M, Ferrara S, Le Bruchec Y, Slepetis R, Chen N, Vassal G.
Eur J Cancer. 2018 Sep;100:27-34. doi: 10.1016/j.ejca.2018.05.002. Epub 2018 Jun 21.


Accelerating drug development for neuroblastoma - New Drug Development Strategy: an Innovative Therapies for Children with Cancer, European Network for Cancer Research in Children and Adolescents and International Society of Paediatric Oncology Europe Neuroblastoma project.

Moreno L, Caron H, Geoerger B, Eggert A, Schleiermacher G, Brock P, Valteau-Couanet D, Chesler L, Schulte JH, De Preter K, Molenaar J, Schramm A, Eilers M, Van Maerken T, Johnsen JI, Garrett M, George SL, Tweddle DA, Kogner P, Berthold F, Koster J, Barone G, Tucker ER, Marshall L, Herold R, Sterba J, Norga K, Vassal G, Pearson AD.
Expert Opin Drug Discov. 2017 Aug;12(8):801-811. doi: 10.1080/17460441.2017.1340269. Epub 2017 Jun 26. Review.