A unique consortium of patients, carers, and experts united in the fight against a little-known cancer affecting children and young people will have multiple reasons to celebrate Rare Diseases Day on Friday (February 28).
Not only does 2025 mark 14 years since the launch of the Paediatric, Adolescent, Wild-type Syndromic Gastrointestinal Stromal Tumour (PAWS-GIST) Alliance, but its successes include the UK’s first clinic at Addenbrooke’s – and only the second worldwide.
Gastrointestinal Stromal Tumours (or GISTs) are most common in the stomach or small bowel, but can occur anywhere along the digestive tract. They can spread to the liver and peritoneum/omentum and less commonly to bones and lungs.
There are some sub-variants of GIST which are extremely rare, and which are grouped together as "PAWS-GIST" - Paediatric/Adolescent, Wild-Type and Syndromic (Carney's Triad and Carney-Stratakis Syndrome).
The fight for UK patients began in began in 2010 when 15-year-old patient Eve Bressington and mum Jayne – frustrated that there was “no cure, no treatment and nobody doing any research in the UK” – decided to fly to the USA.
They attended the Paediatric, Wild-type GIST Clinic in Washington DC after which Jayne was so impressed she felt determined that UK patients should have a similar service.
They held a crucial meeting with Addenbrooke’s consultant oncologist Dr Ramesh Bulusu and, following a joint plea for help from other UK specialists, set up a group focussed on paediatric and wild-type GIST.
Since then, the alliance has achieved goals including establishing in 2014 the world’s second PAWS-GIST clinic at Addenbrooke’s Hospital and creating a national GIST register and biobank.
The clinics are held three times year, each one bringing together up to eight UK patients who meet with multi-disciplined specialists to review medical history, treatment, response, scans, tumour histopathology, and genetic/molecular analyses. Patients may undergo further tests and receive advice tailored to individual needs.
The specialists include national experts in oncology, genetics, histopathology, endocrinology, GIST, soft tissue cancers and more. Remaining at the helm as clinical lead is Dr Ramesh Bulusu while Jayne, whose daughter died aged 23, is patient director.
The team has grown wild-type cells to aid research, secured funding for new research projects, and collaborated with specialists in Europe, America and elsewhere in pursuit of their ultimate dream – a cure.
Jayne said:
Since 2010 PAWS-GIST supporters have raised funds for research and to help patients and their families attend the PAWS-GIST clinics. These donations have funded seven key PAWS-GIST research projects.
Attending the clinic allows a patient to meet others living with this rare cancer which in itself is an invaluable experience and collectively generates a wealth of information that is assisting the PAWS-GIST multi-disciplinary team to discover the underlying mechanisms behind paediatric, adolescent, wild-type and syndromic GIST.
Jayne Bressington
Dr Bulusu added:
2025 marks the 11th anniversary of PAWS-GIST clinics at Addenbrooke’s and we are very excited to be embarking on clinical trials specifically for PAWS-GIST patients, which we hope will lead to improved treatments.
Dr Ramesh Bulusu
To make a donation to PAWS-GIST, follow this link (opens in a new tab).