Clinical Trial Readiness for CNS Drug Delivery: 8-9th November 2021
Our workshop on 8-9th November 2021 was organised in response to a meeting of our Clinical Trials Working Group for CNS Drug Delivery in March 2021.
Run in collaboration with Mr Kristian Aquilina (Consultant Paediatric Neurosurgeon at Great Ormond Street Hospital, UK), this March meeting brought together 38 leading brain tumour research scientists, clinicians and regulatory experts, from the UK, EU and US, to discuss how we can accelerate paediatric clinical trials of CNS drug delivery devices.
During the meeting, these experts identified what they saw to be the main challenges in preclinical to clinical translation.
'A really enlightening event'
Sponsored by Children with Cancer UK, this was our third drug delivery workshop, this time with a focus on advancing 'clinical trial readiness' for CNS drug delivery.
It brought together around 50 delegates (clinicians, researchers, regulatory experts, policy makers, and representatives from funding bodies, brain tumour charities and industry) to discuss the challenges identified by the Clinical Trials Working Group. These included the need for accurate pre-clinical models, available funding options for preclinical to clinical translation, navigation of the regulatory landscape, and the need for multicentric working.
'Has helped focus my research efforts on the barriers to progress including improved biomarkers and preclinical models'
Over the course of the two days, delegates participated in sessions led by international research leaders, enabling them to explore opportunities to develop transformative research plans that will lead to new, innovative ways of overcoming the significant challenges presented by childhood brain tumour drug delivery:
- Dr Marcel Kool (KiTZ, Germany) - Accurate pre-clinical models for paediatric brain cancer drug delivery
- Dr Roy Harris (Research Design Service East Midlands, UK) - Funding options for preclinical to clinical translation
- Prof Anthony Chalmers (University of Glasgow, UK) - Roadmap for preclinical to clinical translation: the backbones
- Dr Joshua Savage (Birmingham CRUK Clinical Trials Unit, UK) - Navigating the regulatory landscape
- Dr Madhumita Dandapani (The University of Nottingham, UK) - Multicentric working
- Prof Kathy Warren (Harvard Medical School, Dana Farber Cancer Institute/Boston Children’s Hospital, US) - Taking your research into clinical trials: a Q&A clinic.
‘I will be able to enter new collaborations and research endeavors more thoughtfully with an eye to the ultimate clinical ‘translatability’ of my work'
We also took the opportunity to ask our delegates their opinion on which delivery strategy is most feasible to pursue as a priority right now (results shown in bar chart.)
The workshop also enabled us to start pulling together a ‘Roadmap’ document for preclinical to clinical translation. Once finalised, this interactive document will be freely available to the neuro-oncology research community via our website.
We rounded up the workshop by launching a pump prime funding opportunity for projects that will move trial proposals into an advanced state of readiness. We were delighted to be approached by the charity Abbie’s Army, who wanted to partner with us on this funding call, and agreed to fund one successful project specific to DIPG. Following independent peer review, three projects were chosen for funding:
- Dr Antonios Pouliopoulos, King’s College London: Focused ultrasound for targeted carboplatin delivery in Diffuse Intrinsic Pontine Glioma
- Dr Wenbo Zhan, University of Aberdeen: Optimisation of infusion catheter posture for improving convection-enhanced drug delivery to brain cancer
- The third project is confidential at present, whilst contracts are drawn up and agreed.
You can read more about the pump prime research projects here.
List of institutions represented at the workshop
CRUK Clinical Trials Unit
Dana-Farber Cancer Institute
DIPG Centre of Excellence
Great Ormond Street Hospital
Imperial College Healthcare Trust
Instituto de Salud Carlos III
King’s College London
National Institute of Health Research
Nottingham University Hospitals NHS Trust
Princess Máxima Center
Royal Manchester Children’s Hospital
Royal Marsden NHS Foundation Trust
University College London
University Hospital of Essen
University Hospitals of Bristol
University of Arizona / Phoenix Children’s Hospital
University of Birmingham
University of Bristol
University of Glasgow
University of Leeds
University of Maryland
University of Nottingham
University of Queensland
Weill Cornell Medicine
Pharma / Biotech
Renishaw Neuro Solutions
Research Design Services East Midlands
Brain Tumour Research
Children with Cancer UK