Children who develop neuroblastomas, a rare form of cancer which develops in nerve cells, may benefit from receiving certain anti-tumour drugs as well as chemotherapy, a new trial has found.

The results of the BEACON trial conducted by the Cancer Research UK Clinical Trials Unit (CRCTU) at BHP founder-member the University of Birmingham found that combining anti-angiogenic drugs, which block tumours from forming blood vessels, alongside various chemotherapy drugs led to more young people seeing their tumours shrinking, from 18% in the control group to 26% among those on Bevacizumab.

The findings have been published in the Journal of Clinical Oncology. The trial saw 160 young people aged 1-21, from 43 hospitals in 11 European countries, randomised with half receiving the anti-angiogenic drug called Bevacizumab on top of conventional therapy. The group who received Bevacizumab had an increase in the likelihood of responding to treatment, from 18% among those who only had the established therapy to 26% for those with the additional drug. Patients who received Bevacizumab additionally had better one year progression-free survival rates.

The trial constituted one of many collaborations between the University of Birmingham and European expert groups SIOPEN (International Society of Paediatric Oncology European Neuroblastoma) and ITCC (Innovative therapies for children with cancer).

Simon Gates, Professor of Biostatistics and Clinical Trials at the University of Birmingham and senior lead author of the paper said: “These are very exciting results that hopefully get us closer to finding treatments for children who develop neuroblastomas. Currently, the outcomes are really poor for children who get this horrible cancer and so even seemingly small increases in the chance that a patient is going to be able to shrink their tumours is significant.

“We are delighted that the BEACON trial has helped to shape treatment for children with relapsed and refractory neuroblastoma going forward.”

Dr Lucas Moreno, Head of Paediatric Haematology and Oncology at Vall d’Hebron University Hospital, Barcelona, Spain and Chief Investigator for the study said: “BEACON was a hypothesis-generating trial that has served to identify active regimens that are now being further investigated. We are delighted that the data generated has been incorporated into the current UK Clinical Practice Guidelines and Bevacizumab is incorporated into standard treatment for relapsed neuroblastoma.”

A major UK clinical trial of an oral spray containing cannabinoids to treat recurrent glioblastoma has opened in the UK. Funded by The Brain Tumour Charity and coordinated by the Cancer Research UK Clinical Trials Unit at BHP founder-member the University of Birmingham, the three-year phase II trial  will investigate whether combining nabiximols and chemotherapy can help extend the lives of people diagnosed with recurrent glioblastoma.

Anyone interested in this study, which is called ARISTOCRAT, should speak to their medical team first to ensure they are eligible to participate.

It will recruit more than 230 glioblastoma patients at 14 NHS hospitals across England, Scotland and Wales in 2023 including Birmingham, Bristol, Cambridge, Cardiff, Edinburgh, Glasgow, London, Liverpool (Wirral), Manchester, Nottingham, Oxford and Southampton.

Professor Pamela Kearns, Director of the Cancer Research UK Clinical Trials Unit (CRCTU) at the University of Birmingham, which is co-ordinating the trial, said:

“ARISTOCRAT represents a significant step in our journey towards finding safe and effective treatments for the most aggressive brain tumours. By testing innovative combinations of drugs we hope to improve the outcome for this challenging disease.

“We’re immensely proud to be able to bring this trial to patients with the support of the Brain Tumour Charity and thanks to the generosity of all those who gave to the crowdfunding campaign.”

Glioblastoma is the most aggressive form of brain cancer with an average survival of less than 10 months after recurrence.

In 2021, a phase I clinical trial in 27 patients found that nabiximols could be tolerated by patients in combination with chemotherapy, and has the potential to extend the lives of those with recurrent glioblastoma.

Should the trial prove successful, experts hope that nabiximols could represent a new, promising addition to NHS treatment for glioblastoma patients since temozolomide chemotherapy in 2007.

In August 2021, a fundraising appeal by The Brain Tumour Charity, backed by Olympic champion Tom Daley, raised the £450,000 needed for this phase II trial in just three months, and Jazz Pharmaceuticals has generously agreed to provide nabiximols and matched placebo free-of-charge to patients on the ARISTOCRAT trial.

Participants will self-administer nabiximols or a placebo spray and will undergo regular follow-ups with the clinical trial team, including blood tests and MRI scans. This will also be one of the first trials to integrate with The Brain Tumour Charity’s app BRIAN.

Principal Investigator, Professor Susan Short, Professor of Clinical Oncology and Neuro-Oncology at the University of Leeds, said:

“We are very excited to open this trial here in Leeds and very much look forward to running the study which will tell us whether cannabinoid- based drugs could help treat the most aggressive form of brain tumour.

“The treatment of glioblastomas is extremely challenging. Even with surgery, radiotherapy and chemotherapy, nearly all of these brain tumours re-grow within a year, and unfortunately there are very few options for patients once this occurs.

“Cannabinoid-based drugs have well-described effects in the brain and there has been a lot of interest in their use across different cancers for a long time now. Glioblastomas have receptors to cannabinoids on their cell surface, and laboratory studies on glioblastoma cells have shown these drugs may slow tumour growth and work particularly well when used with temozolomide.

“We now have the opportunity to take these laboratory results, and those from the phase I trial and investigate whether this drug could help glioblastoma patients live longer in this first-of-a-kind randomised clinical trial.”

How can I take part in the trial?

Your treating oncologist will be aware of the study if it is open in your hospital or can refer you to a treating centre if necessary. Please speak to your treatment team about eligibility for the trial.

For more information visit the ARISTOCRAT web page on the Cancer Research UK Clinical Trials Unit website. 

BHP founder-members University Hospitals Birmingham NHS Foundation Trust (UHB) has become the UK’s first site to launch the BioNTech Messenger RNA (mRNA) cancer vaccines trial which aims to recruit 10,000 people across the UK.

Launching within the NIHR Clinical Research Facility (CRF) at Queen Elizabeth Hospital Birmingham, mRNA vaccines are one of the most exciting experimental developments to emerge from the COVID-19 pandemic – with strong indications that they could become powerful anti-cancer treatments.

Traditionally, vaccines use dead or weakened viruses to stimulate the immune system into recognising or creating harmless antibodies, so when exposed to the real virus, the body is better placed to fend off an overwhelming infection. mRNA is a genetic material that copies instructions found in DNA, using them to make proteins that carry out functions in the body.

Efficiency and speed are part of the appeal of mRNA vaccines. The manufacture of traditional inactivated virus vaccines takes months as scientists are required to grow these on a huge scale, inactivate the virus, and then formulate it to administer in the general population. mRNA vaccine manufacture only requires the right sequence of genetic instructions.

At UHB, this mRNA trial aims to recruit patients with high-risk stage II and stage III colorectal cancers where there is no standard of care treatment to offer the patient following surgery. Each mRNA vaccine delivered will be personalised to the individual patient.

Around 42,900 people are diagnosed with colorectal cancers in the UK each year. It is the 4th most common cancer in the UK. In Birmingham and Solihull alone, almost 700 people are diagnosed with a colorectal cancer each year.

Dr Victoria Kunene, Consultant Oncologist and Principal Investigator for the trial at UHB, said: “I am really very excited that we have been able to lead the way in setting up this arm of the trial, and am looking forward to being part of the wider vaccine program at UHB.

“We are proud to have an impressive team aptly capable of safely delivering these studies here in the West Midlands, and it is a real pleasure to be part of this transformational trial.”

Prof Simon Ball, Chief Medical Officer, said: “A diagnosis of cancer is devastating for patients and their families; this trial represents a monumental step forward in providing not just hope, but a real promise of delivering better outcomes for patients with colorectal cancer, for whom there is not always a standard of care treatment available following surgery.

“Our research teams, supported by the NIHR, have a proven, distinguished track record in delivering vital trials that make significant contributions to medical and scientific discovery with the patient at the very heart; we’re immensely proud to be able to play a strong part in this here in the West Midlands.”

Participants randomised to receive the study treatment will receive 15 treatments of over one year, followed up for at least 36 months. The treatment is, in essence, a personalised medicine for post-operative patients with high-risk stage II/III colorectal cancer, for whom there is no standard of care treatment and involves the development and testing of an individualised cancer treatment called RO7198457.

UHB is the first site open for this trial – a multi-site, open-label, Phase II, randomized, controlled trial to compare the efficacy of RO7198457, versus watchful waiting in resected, Stage II (high risk) and Stage III colorectal cancer patients who are ctDNA positive following resection.

‘Individualised’ means that the treatment is made individually for each participant according to their unique cancer. This is then tested for mutations which create a unique fingerprint. The goal of an individualised cancer treatment approach is to help train the immune system to recognise and attack cancer cells.

Participants who are randomised to the observation group will be followed up for at least 48 months and visit the research site every three months. Care is provided to ensure safety during trial participation, including an informed consenting process, regular follow up where biomarkers and all reported outcomes are collected and analysed.