Two small British charities have made history by creating what they believes is the first ever international partnership to test a new vaccine for cancer. 

The aggressive childhood disease, neuroblastoma, affects two children a week in Britain and more than half of these children will suffer potentially fatal relapses.  

If successful, the new drug will provide the first demonstrably effective vaccine, meaning that in the future children may not have to endure ongoing chemotherapy and can have significantly more hope for survival.

But the trial couldn’t have happened without a US and European alliance to pool funds and expertise.

The partnership is called INBRACED (International Research and Collaboration for Effective Delivery).

London based NCCA UK (Neuroblastoma Children’s Cancer Alliance) has steered the partnership with UK partner J-A-C-K and US based Solving Kids’ Cancer.

Chief Executive Bettina Bungay-Balwah comments: “The three charities involved in this partnership share the view that just because neuroblastoma is rare doesn’t mean we can forget the pain and heartbreak it causes families worldwide. The success of this new partnership lies in the amazing determination of UK and US charities, parents, research institutions, doctors and pharmaceutical organisation to help more children survive.”

Scott Kennedy, the Executive Director of Solving Kids’ Cancer comments:- “This is the first time that paediatric cancer research charities have collaborated to launch an international clinical trial. Our hope is that through this multi-centre international phase I trial, we can quickly bring this cutting-edge treatment  to kids in urgent need of more options today.”

Stephen Lowis, Macmillan Consultant in Paediatric and Adolescent Oncology, Bristol Royal Hospital for Children, comments: “I am delighted to be involved with this international cooperative study, which will use a novel vaccine to stimulate an immune response to neuroblastoma cells in patients who have previously been treated, but relapsed. The project builds on a recent successful study of immunotherapy for similar patients, run at Memorial Sloan-Kettering Cancer Center (MSKCC). It is important for novel therapies for children with neuroblastoma to be available as widely as possible. Collaborative studies such as this will mean that children need not travel elsewhere, and can maintain contact with their families and supporting healthcare teams throughout their treatment, which must be a good thing.”

Richard Brown, Chair at J-A-C-K, said: "We are extremely excited by the chance to join with other charities in order to provide a funding framework for collaborations between doctors and institutions across continents. We truly hope this will speed the exploration and evolution of potential therapies for the children and their families blighted by neuroblastoma."

How would a vaccine be made available after the trial?

The company is responsible for all commercial clinical development of the neuroblastoma vaccine.

MabVax is finalizing the manufacture of the vaccine and is undertaking the regulatory work necessary to initiate the study in three countries.

David Hansen, CEO of MabVax stated: “The early trial conducted at MSKCC was very encouraging. The company has designed this trial to enable transition to a larger follow-on Phase II trial, which will be a potentially registration eligible trial”.

How does the treatment work?

The low-toxicity vaccine was developed by researchers at MSKCC in New York City, which is participating in the trial alongside Bristol Children’s Hospital.

It works with the child’s own immune system – helping the child’s body recognise and destroy the cancer cells that express the two antigens most commonly expressed on the surface of neuroblastoma cells—GD2 and GD3, potentially promoting long-term remissions in these children.

A Phase I trial conducted in 2009 at MSKCC with 15 patients has demonstrated very promising results.  

How is the trial funded?

This is the first grant awarded by INBRACED (International Neuroblastoma Research and Collaboration for Effective Delivery) which was established by NCCA UK, J-A-C-K and SKC last year to improve access to promising clinical trials for children with high-risk neuroblastoma in North America, the UK and in Europe.

The charities have raised $400,000 to pay for the trial.

What is neuroblastoma?

Neuroblastoma is a cancer of the sympathetic nervous system that most commonly occurs in infants and children under five.

The cancer is usually discovered after it has spread.

Half of all children diagnosed have high-risk neuroblastoma, which is one of the most aggressively treated of all cancers.

For many children, even after intense treatment, remission is short lived.

This is because strategies to prevent relapse have had very limited success.

Source: INBRACED