The role of the ‘innovative therapies for children with cancer’ (ITCC) European consortium

Zwaan, C. Michel; Kearns, Pamela; Caron, Huib N.; Verschuur, Arnauld; Riccardi, Riccardo; Boos, Joachim; Doz, François; Geoerger, Birgit; Morland, Bruce; Vassal, Gilles

doi:10.1016/j.ctrv.2010.02.008

Cited by 61 publications

(27 citation statements)

References 40 publications

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“…Several international cooperative groups dedicated to early drug development, such as the Innovative Therapies for Children with Cancer European network, run a biology-driven new drug development strategy for children with cancer (18). This strategy is based on identification and validation of relevant targets in pediatric malignancies to choose and prioritize drugs to be developed in children through innovative designs using biomarkers.…”

Section: Discussionmentioning

confidence: 99%

Is the European Pediatric Medicine Regulation Working for Children and Adolescents with Cancer?

Vassal

Geoerger

Morland

2013

Clinical Cancer Research

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The European Pediatric Medicine Regulation was launched in 2007 to provide better medicines for children. Five years later, the number of new anticancer drugs in early development in the pediatric population remains low, and most children with cancer are still largely denied access to innovative drugs in Europe, as compared with the United States. We analyzed individual pediatric investigation plan (PIP) and waiver decisions for oncology drugs and all oncology drugs that have been approved for marketing authorization since 2007 in Europe. Among the 45 approved PIPs, 33% concern leukemias and lymphomas, 29% solid tumors, 13% brain tumors, and 20% a drug for supportive care. No specific PIP exists for lifethreatening diseases such as high-risk neuroblastoma, whereas there are several PIPs in extremely rare malignancies in children and adolescents such as gastrointestinal stromal tumor, melanoma, thyroid cancer, and chronic myeloid leukemia. This paradoxical situation is due to approval of a PIP being driven by the adult indication. Twenty-six of 28 authorized new oncology drugs have a potentially relevant mechanism of action for pediatric malignancies, but 50% have been waived because the adult condition does not occur in children. The most striking example is crizotinib. Implementation of the pediatric regulation should no longer be driven by the adult indication but should be guided instead by the biology of pediatric tumors and the mechanism of action of a drug. This change will be achievable through voluntary PIPs submitted by Pharma or revocation of the oncology class waiver list.

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Section: Discussionmentioning

confidence: 99%

Is the European Pediatric Medicine Regulation Working for Children and Adolescents with Cancer?

Vassal

Geoerger

Morland

2013

Clinical Cancer Research

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“…C'est pour cela qu'il y a dix ans, a été créé le réseau européen innovative therapies for children with cancer (ITCC) (www.itcc-consortium.org) pour booster le développement précoce des médicaments et en prioriser les choix, en étroite collaboration avec les groupes européens au sein de la SIOPE qui conduisent la recherche sur les cancers de l'enfant ainsi qu'avec les industriels, les associations de parents et d'enfants et les agences réglementaires [22].…”

Section: Resultsunclassified

Nouveaux médicaments anticancéreux pour les enfants et les adolescents : où en sommes-nous en Europe ?

Vassal

2013

Revue d'Oncologie Hématologie Pédiatrique

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“…For this purpose, we modified liposomes by adding cyclic RGD-peptides to their surface, forming integrin-targeting liposomes. Because neuroblastoma and rhabdomyosarcoma are both known to be highly vascularized, 13,14 we predicted an additional benefit from this antiangiogenic therapeutic approach in addition to the antitumor effect.…”

Section: Discussionmentioning

confidence: 99%

“…11,12 Neuroblastoma and rhabdomyosarcoma are among the most frequent and aggressive tumors of childhood, and still have unsatisfactory outcomes, especially in the advanced stages. 13,14 New molecules and therapeutic approaches are therefore urgently needed. Both entities show particular characteristics in their molecular biology, and angiogenesis has been identified to be a potential new target in neuroblastoma as well as in rhabdomyosarcoma.…”

Section: Introductionmentioning

confidence: 99%

Treatment of neuroblastoma and rhabdomyosarcoma using RGD-modified liposomal formulations of patupilone (EPO906)

Scherzinger-Laude¹,

Schönherr²,

Lewrick³

et al. 2013

IJN

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Background: Patupilone (EPO906) is a microtubule stabilizer with a potent antitumor effect. Integrin αVβ3-binding (RGD) liposomes were loaded with EPO906, and their antitumor efficacy was evaluated in two pediatric tumor models, ie, neuroblastoma and rhabdomyosarcoma. Methods: Integrin αVβ3 gene expression, RGD-liposome cellular association, and the effect of EPO906 and liposomal formulations of EPO906 on cell viability were assessed in vitro in human umbilical vein endothelial cells (HUVEC), in the RH-30 rhabdomyosarcoma cell line, and in the Kelly neuroblastoma cell line. In vivo, mice bearing neuroblastoma or rhabdomyosarcoma tumors were treated with EPO906, EPO906-liposomes, or EPO906-RGD-liposomes. Tumor growth, cumulative survival, and toxicity were monitored. Results: Integrin αVβ3 was highly expressed in HUVEC and RH-30, but not in Kelly cells. Accordingly, RGD-liposomes were highly associated with HUVEC and RH-30 cells in vitro, but not with the Kelly cells. EPO906 and its liposomal formulations inhibited HUVEC, RH-30, and Kelly cell viability to the same extent. In vivo, EPO906 1.5 mg/kg and liposomal EPO906 potently inhibited tumor growth in both xenograft models without triggering major toxicity. At this dose, liposomal EPO906 did not enhance the antitumor effect of EPO906 in neuroblastoma, but tended to have an increased antitumor effect in rhabdomyosarcoma. Using a lower dose of EPO906-RGD-liposomes significantly enhanced cumulative survival in rhabdomyosarcoma compared with EPO906 alone. Conclusion: EPO906 shows a strong antitumor effect in neuroblastoma and rhabdomyosarcoma, without triggering major side effects. Its liposomal encapsulation does not alter its activity, and enhances cumulative survival when EPO906-RGD-liposomes are used at low dose in rhabdomyosarcoma.

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The role of the ‘innovative therapies for children with cancer’ (ITCC) European consortium

Cited by 61 publications

References 40 publications

Is the European Pediatric Medicine Regulation Working for Children and Adolescents with Cancer?

Is the European Pediatric Medicine Regulation Working for Children and Adolescents with Cancer?

Nouveaux médicaments anticancéreux pour les enfants et les adolescents : où en sommes-nous en Europe ?

Treatment of neuroblastoma and rhabdomyosarcoma using RGD-modified liposomal formulations of patupilone (EPO906)

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