Brexit Blocks UK Children from Accessing Life-Extending Drug Trial for Aggressive Brain Cancer
核心洞察
The Biomede trial, testing a US-approved drug combined with radiotherapy for DIPG, has been delayed in the UK due to post-Brexit regulatory divergence from the EU framework established in 2022.
Approximately 30 UK children are diagnosed annually with DIPG, an incurable brainstem tumour with a survival rate of "essentially zero," where most children die within two to three years of diagnosis.
Cancer Research UK reports that nine in 10 children's cancer doctors rely on international funding for trials, and the lack of UK-EU harmonisation has driven families to seek treatment overseas.
UK children diagnosed with diffuse intrinsic pontine glioma (搜索) (DIPG), a rare and uniformly fatal brain tumour, are being denied access to a potentially life-extending international drug trial because of regulatory barriers created by Brexit. The Biomede trial, which is testing a US-approved drug in combination with radiotherapy, has been approved across Europe but remains stalled in the UK, where it is being treated as an entirely new trial requiring fresh regulatory approval.
A disease with no treatment and no cure
DIPG is an incurable and inoperable tumour that develops in the brainstem, the region controlling vital functions such as breathing, swallowing, and facial expressions. Approximately 30 children in Britain receive this diagnosis each year. Professor Chris Jones, head of the Division of Cancer Biology at The Institute of Cancer Research, described the stark prognosis: "So currently, the survival rate is essentially zero. Almost all children will pass within two or three years of diagnosis, often much, much quicker. And so really, we're looking for just anything which might impact that survival rate and show a difference."
Because of the unique biological makeup of DIPG tumours, treatments developed for adult cancers cannot be repurposed, leaving clinicians and families with virtually no therapeutic options.
The Biomede trial and the Brexit barrier
The international Biomede trial launched in 2017, recruiting patients from France, the UK, Denmark, Sweden, Australia, Spain, and the Netherlands. Its second phase, now approved by European regulators, is evaluating two drugs already authorised for market — one approved in the United States for use at relapse, and another used in other cancer indications.
However, the UK has not approved the second phase because, under the post-Brexit regulatory framework, it is being classified as a new trial requiring separate authorisation. Britain's withdrawal from the European Union means it no longer participates in the European regulatory framework introduced in 2022 that monitors clinical trials across the continent.
Professor Amos Burke, director of the Cancer Research UK Clinical Trials Unit, explained: "The lack of harmonisation between the UK and the EU on clinical trials since Brexit" has created a situation where, since the first part of the trial concluded in the UK, "the options for children with this rare cancer are nonexistent." He stressed that regulatory alignment between the UK and the EU is vital to prevent treatment gaps, adding: "There is an appeal being launched at the moment, but this sort of asynchrony between the UK and the EU is problematic."
Families forced to seek treatment abroad
Professor Jones noted that the absence of UK access "is very problematic, and over the last few years, it's driven many families to go overseas to get access to this drug."
Debi Oliver, whose daughter Emily died from DIPG in 2019 at age 21, described her reaction to the delays: "I'm angry and quite upset that, considering what we went through and it's still very raw, even six, seven years later… I'm very upset to hear that things have not progressed more, especially as the trial is there." Emily's family had travelled monthly to Germany for a bespoke vaccine trial run by Tübingen University after being told she had three months to live.
While two other trials for DIPG patients are operating in the UK through Great Ormond Street Hospital (搜索) using personalised immunotherapy, experts emphasise that any additional barrier to research in this area is deeply concerning.
Broader impact on paediatric oncology research
Cancer Research UK has documented that Brexit has led to delays in launching clinical trials and increased costs for multiple drugs. Research by the charity revealed that dozens of researchers reported greater difficulty initiating new trials with EU-based scientists and recruiting researchers to UK trials since Brexit.
Laura Williams, Europe and global affairs manager at Cancer Research UK, highlighted that nine in 10 children's cancer doctors surveyed last year relied on international funding to conduct clinical trials. She warned: "For some people, it may be that that trial is their only treatment option."
Dr Karen Noble, Director of Research, Policy and Innovation at Brain Tumour Research (搜索), called on "government bodies to engage with industry to build a more attractive financial and regulatory environment that encourages investment in brain tumour drug development, and facilitates UK involvement in pan-European trials."
Regulatory response
A Department of Health and Social Care spokesperson stated: "This government has been clear about our ambition to turbo-charge medical research and introduced rules to speed up access to new treatments and cement the UK as a world-class destination for clinical trials. Our clinical trials regulations remain in line with the EU, where appropriate, to avoid unnecessary issues that cause delays."
The Medicines and Healthcare Products Regulatory Agency (搜索) (MHRA) said it could not comment on specific clinical trials but indicated that new regulations introduced from April 2026 will make it easier and faster to run trials in the UK, with changes including "stronger alignment with international standards."