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.

Trent Bldg Autumn x440


'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.)

Mentimeter feasible delivery strategy Nov 2021 x465

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.


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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.


Nottingham 2021 workshop delegates x735


List of institutions represented at the workshop

Delegate institutions



Funding bodies

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

Semmelweis University

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 

WPD Pharmaceuticals

Research Design Services East Midlands 

Oncoheroes Biosciences











The Netherlands

















Abbie’s Army

Brain Tumour Research

Children with Cancer UK