Global trial finding a cure for rare childhood cancer opens

A global trial has opened 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 now open in the UK, trialling multiple new treatments to offer a better chance of survival to hundreds of young people across the world over the next few years. The first sites have opened in the UK, and Glo-BNHL will open at 45 sites across 19 countries in Europe, Asia, North America and Australia.

Clinical trials traditionally compare one new treatment to the standard treatment used by doctors today. In the case 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 always be at least one new drug for any 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:

“Launching the first sites for Glo-BNHL is a hugely important moment and 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.”

Alex’s story

Alex Hulme was born in Trafford on July 29th 1999 and led a full and fruitful life with his fun, easy going and mature approach to life.

In March 2011 Alex was diagnosed with B-cell Non-Hodgkin Lymphoma and his treatment worked well in the early stages.

Alex’s dad Dave reflects on the early treatment, saying “we kept hearing the phrase textbook in those early stages, which is what you want to hear in that situation. Alex even managed to get back to school for short periods of time.”

“However, despite having a clear scan, a few weeks later Alex started to deteriorate, and it turned out the lumps came back while he was on treatment.

“We now know that relapsing, particularly on treatment is a really bad issue. They gave him the next level of treatment and again he improved, but as soon as the treatment stopped, he deteriorated again.”

Alex’s cancer had returned in late June of the same year and although he initially responded well again, he sadly lost his six-month battle and died on Monday September 5th 2011.

Alex’s parents Dave and Nicola formed a charitable foundation in the October of the same year to begin to raise money for children’s Non-Hodgkin Lymphoma – to honour their son, and to help other children who get NHL.

After more than 12 years of fundraising for research into better understanding and treatments, Dave and Nicola are now part of the steering committee for the Glo-BNHL clinical trial, helping children like their son Alex who have relapsed or refractory B-NHL.

Dave said: “You know, if we were where we are now, then things may have been different for Alex, we would have added a lot more choice but we can't change that. But we have changed it to give somebody else that opportunity now and I think that's all we wanted to do when we set up the Alex Hulme Foundation.”

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, Professor of Clinical Paediatric Oncology and Director of the Institute of Cancer and Genomic Sciences at the University of Birmingham and co-investigator of the Glo-BNHL trial 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.

Nicole Scobie is President of children’s cancer charity Zoe4Life and is also a patient advocate involved in ACCELERATE and now also Glo-BNHL. Nicole set up Zoe4Life with Natalie Guignard-Nardin, mum of 4-year old Zoe who died after her cancer relapsed a year after receiving initial treatment. Zoe4Life supports children with cancer, their families and research into childhood cancer.

Nicole said:

“I am delighted to have been able to support the development of the Glo-BNHL trial. Myself and other patient groups and members of the public very much hope that this trial and others that follow in its footsteps can address these significant unmet needs, particularly across paediatric cancer.

“We are all incredibly grateful to take part in the ACCELERATE meeting that led to the Glo-BNHL trial being established and that regulators and industry are acting on the experience and recommendations being made.”

Glo-BNHL gratefully receives funding from Cancer Research UK and Fight Kids Cancer. The drug companies working with Glo-BNHL have committed over £20 million of funding so far.

Glo-BNHL is also supported by Birmingham’s 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.