Clinic leads worldwide hunt for rare cancer treatment


A Cambridge University Hospitals NHS Foundation Trust clinic has teamed up with experts in America in a race against time to find a treatment for one of the world’s rarest children’s cancers.

The collaboration, the first of its kind by a European clinic, is so significant the news was announced by the former Vice President of the United States, Joe Biden, at a dedicated cancer summit in Washington DC last weekj (21 September).

Paediatric, adolescent, wild type, syndromic, gastrointestinal stromal tumours (PAWS-GIST) generally affect children and young adults and conventional treatments are less effective. The subset represents a tiny percentage of the 15 in one million mainly adult suffers of classic GIST.

GISTs are most common in the stomach or small bowel, but can occur anywhere along the digestive tract. They can spread are to the liver and peritoneum/omentum and less commonly to bones and lungs.

Cambridge University Hospitals PAWS-GIST clinic and the New Jersey-based Life Raft Group of America have collaborated to form the International Paediatric and SDH-Deficient GIST Consortium.

The consortium’s aim is to focus the best research brains in the world to find within three years at least one effective treatment for the rare children’s cancer subset.

The starting point will be to create a dedicated data sharing platform, a tissue bank in America to compliment an existing one in the UK, establish guidelines for clinical stakeholders, expand clinical trials, and speed up results. Strategies include educational programmes for healthcare professionals and patients and families worldwide.

The Cambridge team includes PAWS-GIST Clinic lead, Dr Ramesh Bulusu, consultant oncologist, Dr Ruth Casey, consultant endocrinologist, Dr Olivier Giger, consultant pathologist and GIST Support UK/PAWS-GIST patient advocates, Jayne Bressington and Victoria Bassett.

Dr Bulusu, who has been the consultant clinical lead for PAWS-GIST since its inception in 2014 said: “This is an extremely exciting moment for us and has the potential to improve understanding, research and outcomes for those who have PAWS-GIST.

“When we launched the clinic one of our most ambitious goals was to improve outcomes for these young patients and hopefully, find a cure. By collaborating with specialists in Europe, America and elsewhere we are jointly increasing our chances of doing that.”

Norman J Scherzer, Executive Director of the Life Raft Group, added:We have more than doubled progress for patients living with GIST with a major exception - paediatric and SDH-Deficient GIST. That is our unfinished business: Nobody wants to lose any more children.”

News of the initiative was outlined by Vice President Biden and Dr Jill Biden at the Biden Cancer Summit designed to bring together leading problem solvers.

He said: “The goal of the Biden Cancer Summit is to show progress from partners and to engage communities to tell us what issues most plague them, and to bring all resources possible to the fight to identify issues and solutions that matter locally and nationally.”

More information on PAW-GIST clinic can be found on the website at

Image:  Members of  the new International Paediatric and SDH-Deficient GIST Consortium


About Cambridge University Hospitals

Cambridge University Hospitals NHS Foundation Trust (CUH) is one of the largest and best known hospitals in the country, delivering high-quality patient care through Addenbrooke’s and the Rosie Hospitals. CUH is a leading national centre for specialist treatment for rare or complex conditions and a university teaching hospital with a worldwide reputation.

CUH is a key partner in Cambridge University Health Partners (CUHP), one of only six academic health science centres in the UK, and is at the heart of the development of the Cambridge Biomedical Campus (CBC), which brings together on one site world-class biomedical research, patient care and education.  As part of the Campus development, Papworth Hospital is creating a bespoke, purpose-built hospital, and AstraZeneca is building a new global R&D centre and corporate headquarters.  The Campus is one of the government’s  National Institute for Health Research (NIHR) comprehensive biomedical research centres.

To read more information, click here.

Cambridge University Hospitals is one of the largest and best known trusts in the country. As the local hospital for our community we deliver care through Addenbrooke’s and the Rosie hospitals.

Cambridge University Hospitals NHS Foundation Trust