Dataset Information

Fostering open collaboration in drug development for paediatric brain tumours.

ABSTRACT: Brain tumours have become the leading cause of child mortality from cancer. Indeed, aggressive brainstem tumours, such as diffuse intrinsic pontine glioma (DIPG), are nearly uniformly fatal. These tumours display a unique set of driver mutations that distinguish them from adult gliomas and define new opportunity for the development of precision medicines. The specific association of ACVR1 mutations with DIPG tumours suggests a direct link to neurodevelopment and highlights the encoded bone morphogenetic protein receptor kinase ALK2 as a promising drug target. Beneficial effects of ALK2 inhibition have now been observed in two different in vivo models of DIPG. Nonetheless, such tumours present a huge challenge for traditional economic models of drug development due to their small market size, high failure rate, tumour location and paediatric population. Moreover, a toolkit of different investigational drugs may be needed to fully address the heterogeneity of these tumours in clinical trials. One new business model is suggested by M4K Pharma, a recent virtual start up that aims to align diffuse academic and industry research into a collaborative open science drug discovery programme. Fostering scientific collaboration may offer hope in rare conditions of dire unmet clinical need and provide an alternative route to affordable medicines.

SUBMITTER: Wong JF

PROVIDER: S-EPMC6824674 | biostudies-literature | 2019 Oct

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Fostering open collaboration in drug development for paediatric brain tumours.

Wong Jong Fu JF Brown Elizabeth J EJ Williams Eleanor E Bullock Alex N AN

Biochemical Society transactions 20191001 5

Brain tumours have become the leading cause of child mortality from cancer. Indeed, aggressive brainstem tumours, such as diffuse intrinsic pontine glioma (DIPG), are nearly uniformly fatal. These tumours display a unique set of driver mutations that distinguish them from adult gliomas and define new opportunity for the development of precision medicines. The specific association of ACVR1 mutations with DIPG tumours suggests a direct link to neurodevelopment and highlights the encoded bone morph ...[more]

PMID: 31551357

Similar Datasets

Project description:BackgroundInterprofessional education has emerged as a key concept in education of health professionals over the last 20 years. Positive effects of interprofessional education have been shown, but it has proved to be more time-consuming than traditional teaching methods. We therefore developed a 30-minute interprofessional learning activity, using peer-teaching methods. We were interested in effects on and ways of interprofessional learning, including conditions and resources that make it successful despite limited time.MethodsSpeed InterprofESsional Peer Teaching PaediAtric (SIESTA) was developed in the context of an interprofessional training ward. 20 paediatric nursing trainees and 20 medical students were enrolled in the study. Two students from each profession participated in a total of four SIESTA sessions each, supervised by registered paediatric nurses and paediatricians. We used a mixed-methods approach of quantitative and qualitative data (questionnaires, semi-guided focus group interviews) to evaluate self-perceived interprofessional competencies, interprofessional learning gains and ways of interprofessional learning.ResultsQuestionnaires were obtained from all participants (n = 40) and n = 26 took part in the group interviews. Participants from both professions reported an increase in self-perceived understanding of interprofessional roles and tasks. Communication and cooperation emerged as important aspects. The workplace-based nature of SIESTA promoted interprofessional learning, while peer teaching fostered a safe learning environment. Regarding time constraints participants suggested thorough preparation and structuring by facilitators as a solution.ConclusionsOur short interprofessional peer teaching activity showed promising results. Participants reported enhanced interprofessional competencies and provided suggestions for successful learning in limited time. Further studies should include an objective assessment of the interprofessional learning progress. The SIESTA concept can be easily adapted to other medical fields, providing interprofessional learning opportunities for many more health care professionals to come.

Dataset Information

Fostering open collaboration in drug development for paediatric brain tumours.

Publications

Fostering open collaboration in drug development for paediatric brain tumours.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets