Global trial to find a cure for rare childhood cancer opens in Europe

A global trial has opened two sites in mainland Europe for children who have not responded to treatment for B-cell Non-Hodgkin Lymphoma (B-NHL), or who have relapsed.

Glo-BNHL, a global clinical trial run by the University of Birmingham, is trialling multiple new treatments to offer a better chance of survival to hundreds of young people across the world. The trial is now open in three countries across Europe including the UK following the announcement today.

Clinical trials traditionally compare one new treatment to the standard treatment used by doctors today. In around 10% of cases of B-NHL that has not been cured (refractory) or has come back (relapsed), there are no adequate treatments anywhere in the world and most children will sadly die from the disease.

In the Glo-BNHL trial, several new drugs will be tested at the same time, meaning that there should be at least one arm of the trial containing a new drug suitable for a child with relapsed or refractory B-NHL. Advanced statistical methods will be used to decide if a treatment is working or not with a very small number of patients, very quickly.

A treatment that is not working will be identified early and removed, to be replaced with another treatment. For treatments that do work, researchers believe there will be enough evidence to show regulators that the treatment should be adopted as standard care in the NHS and beyond. In this way, the usual lengthy process of trial and regulatory approval will be shortened significantly.

Professor Amos Burke, Director of the Cancer Research UK Clinical Trials Unit at the University of Birmingham and Chief Investigator of the Glo-BNHL trial said:

“Opening sites for the Glo-BNHL in Europe is a hugely important moment taking this innovative new trial design to more countries around the world. This trial represents a step change for children with rare disease like relapsed/refractory B-NHL. This trial uses a new design, developed with ongoing input from our patient advocates. The design has been supported by regulators and pharma companies, and the result is a trial that offers the possibility of better chances for children with relapsed or refractory B-NHL.”

Hidde’s story: “It feels like you’re being haunted by a shadow”

24-year-old Dutch national Hidde Plieger was four when he was diagnosed with Burkitt’s Lymphoma.

Hidde’s mother, Willemijn recalls when they first realised that something was wrong: “We were on holiday on one of the Dutch islands and Hidde’s tummy became really swollen as the tumour is very fast growing. We had to first get transported to a hospital on the mainland on an emergency boat. After three days and a transfer to a specialist hospital in Nijmegen, the devastating diagnosis became really clear.”

Hidde was given a 75% chance of survival and immediately started an intense regime of treatment. Willemijn and her family were hopeful about the odds, although she knew a tough period lay ahead for them.

“Of course, there was the chance that it (Burkitt’s Lymphoma) would come back again. Hidde was really monitored carefully after treatment, which felt safe but still you cannot prevent yourself being quite worried for the disease to return.

“It feels like you’re being haunted by a shadow. We knew there would be treatment options [if Burkitt’s Lymphoma relapsed], but the survival chances would be significantly lower in that case. Because Hidde was so young at the time of diagnosis, he was not consciously aware of the risks both during and after treatment. We are grateful that he did not have to bear that burden.

Despite the fears over relapse, Hidde did respond positively to treatment and slowly, Willemijn and the family were able to increasingly adjust towards a more daily life of school and work.

Willemijn quickly found herself supporting research, using her experience as a parent to advocate for patients in research projects both within the Princess Máxima Center for pediatric oncology in Utrecht (the research hospital in the Netherlands for paediatric cancer treatment) and beyond, nationally and internationally. Through this involvement, Willemijn was invited, along with other advocates, to support the development of Glo-BNHL where her insights into Burkitt’s Lymphoma are invaluable.

What is B-cell Non-Hodgkin Lymphoma?

B-NHL is a type of cancer that develops inside or outside of lymph nodes (glands) and organs such as the liver or spleen. The medicines used now are very powerful with many side effects. Around nine in every ten children treated are cured however, those who have relapsed or refractory B-NHL have very little chance of survival.

Pamela Kearns, Emeritus Professor at the University of Birmingham and President of the Innovative Therapies for Children and Adolescents with Cancer (ITCC) consortium said:

“There are a lot of new drugs in clinical trials for this type of disease in adults, but in rare diseases like childhood lymphomas, there are not enough patients to test them using conventional approach to clinical trials. We need to select only the most promising drugs to test in children and then get the answers as fast as possible.

“Innovative trials like the Glo-BNHL trial are paramount to finding better treatments for paediatric cancers, many of which are rare and have little or no treatment options. This trial is hugely exciting as it has seen industry partners committing to a collaborative process that is allowing multiple drugs tested at the same time, and we are very grateful for those that have already come on board.”

Bringing all parties together

The idea for Glo-BNHL came from discussions between academics, regulators, patient advocates and drug companies at the 2^nd Paediatric Strategy forum of the ACCELERATE platform.

Glo-BNHL gratefully receives funding from Cancer Research UK and Fight Kids Cancer, and financially supported by drug companies working with Glo-BNHL.

Glo-BNHL is also supported by Birmingham’s Precision Health Technologies Accelerator (PHTA) Industry Trials Hub, driving the trial’s novel approach of an academic clinical trials unit delivering fit-for-filing trials. This will ensure that the trial is designed and delivered with market authorisation in mind, providing a more rapid pathway to clinical adoption at the end of the study.