Laboratory work etc

Clinical Trials and Research

Research into GIST is moving very fast, and we now know very much more than we did ten years ago. However there are still many questions unanswered:

  • We DO know that most GIST tumours have mutations in the KIT or PDGFRA genes, and we know how to identify which exon these mutations are in.

  • We DON'T know why some GISTs don't have mutations at these sites, the wild type GISTs. (Presumably they have mutations in other locations.)

  • We DON'T know why pædiatric GIST behaves differently from adult GIST.

  • We DON'T know why GISTs with mutations in different exons react differently to Glivec and Sutent.

  • We DON'T know why some GISTs behave more aggressively than others, even if they have the same mutations.

  • We DON'T know whether all patients need the same dose of Glivec or Sutent in order to get the same concentration in the blood.

  • Etc.etc.!

Current research is trying to find answers to some of these questions, as well as continuing to search for new drugs or combinations of drugs which will offer patients total long-term remission or a cure.

There is a very good booklet called Understanding Clinical Trials produced by UK Cancer Research Collaboration UKCRC This explains the terms you may hear, like placebo, control arm etc. If you are invited to enroll in a trial, this is recommended background reading.

National GIST Guidelines...

UK clinical practice guidelines for the management of gastrointestinal stromal tumours (GIST) 2017

We are delighted to announce that the UK National clinical practice guidelines for GIST have been updated and published and can be accessed via this link... 


Trials relevant to GIST


The Intrigue Study

The INTRIGUE Phase 3 study of ripretinib is now open to enroll second-line GIST patients, regardless of their mutational status, who have progressed on, or are intolerant to front-line therapy with imatinib.

The following links provide more information: (this link shows the study locations) (this link provides more information about the trial)

Please contact Dr Robin Jones at the Royal Marsden in London or Dr Robin Young at the Sheffield Teaching Hospitals NHS Trust to register for this trial. 


The Invictus trial

Testing DCC-2618 in patients with advanced GIST who have been treated with other anticancer therapies – incl. imatinib, sunitinib, regorafenib – before.

Eligible patients are those Patients with confirmed advanced or metastatic GIST with all types of mutation in KIT or PDGFRa (excluded are patients without KIT or PDGFRa mutations) Patients must have progressed on imatinib, sunitinib, and regorafenib or do not tolerate any of these treatments.

This link provides more information.

Please contact Dr Robin Jones at the Royal Marsden Hospital in London to register for this trial.

The Voyager trial:

Avapritinib vs Regorafenib in unresectable or metastatic GIST.

A Phase 3 study in patients with locally advanced unresectable or metastatic GIST (advanced GIST) of avapritinib versus regorafenib in patients previously treated with imatinib and 1 or 2 other tyrosine kinase inhibitors (TKIs).

This link provides more information as do these

Please contact the following hospitals to register for this trial:

Glasgow - Beatson West of Scotland Cancer Centre

Sheffield - Sheffiled Teaching Hospitals NHS Foundation Trust - Dr Robin Young

London - Guys Hospital

London - The Royal Marsden Hospital - Dr Robin Jones

“Navigator” trial:

This Phase 1 clinical trial is now recruiting patients with second-line GIST. At the time of writing (October 2018) there are about 20 or so slots available in this second-line GIST patient cohort. Note this study has additional patient cohorts for PDGFRα D842V-driven GIST and third-line or later GIST, but these are now fully enrolled.

more information about the Navigator trial is available at or

This trial is open at the Royal Marsden Hospital, London - Dr Robin Jones

The Scandinavian 3 versus 5 years adjuvant imatinib study is:

Open at the following centres:

  • Addenbrookes Hospital, Cambridge (Please contact Dr Ramesh Bulusu)
  • The Royal Marsden, London (Please contact Dr Robin Jonnes)
  • University College Hospital, London (Please contact Dr Palma Dileo)
  • Beatson (West of Scotland Cancer centre), Glasgow (Please contact Dr Jeff White)
  • Weston Park Hospital, Sheffield (Please contact Dr Robin Young)
  • Queen Elizabeth Hospital, Birmingham (Please contact Dr Mariam Jafri)
  • Nottingham University Hospital (Please contact Dr Ivo Hennig)

The CABOGIST Trial: 

The EORTC CABOGIST study for cabozantinib for relapsed metastatic GIST.

This link takes you to a Liferaft group report about this drug and GIST. 

Please contact Dr Charlotte Benson at the Royal Marsden Hospital to register for this trial.


'BLU-285-1101' - A  Study of BLU-285 in Patients with Gastrointestinal Stromal Tumors (GIST) and other Relapsed and Refractory Solid Tumors

Dr Robin Jones has the BLU285 GIST study open at the Royal Marsden. To register for this trial pleas contact Dr Jones. 

This link  takes you to the news released late in 2016 about the Phase 1 trial results...

'ALT' GIST Trial now open! - December 2016

A new first line metastatic GIST trial called ‘Alt GIST’  is now open at the Royal Marsden Hospital in London.

This is a randomised Phase II trial being run through the EORTC (European Organisation for Research and Treatment of Cancer) and is comparing standard imatinib treatment with imatinib and regorafenib alternating monthly.

Eligible patients are those with metastatic GIST who have had no previous drug treatment or patients who have been started on imatinib for 3 weeks or less.

Centres in the UK that will be opening include:

  • The Royal Marsden - London (lead site)
  • UCLH - London
  • The Christie Manchester
  • Leeds
  • Birmingham
  • Clatterbridge
  • Glasgow
  • Norfolk.  

To register for this trial patients will need to be referred by their oncologist to closest centre listed above.


Masitinib Trial

The biotech company that is developing this drug have initiated both first and second line trials for GIST. This means that if you are about to start treatment with Imatinib or Sunitinib then you may be offered the opportunity to enter one of these trials. If you are being treated in a hospital that is not listed below and are interested in participating then your clinician can connect you to the relevant clinicians.Masitinib is a tyrosine kinase inhibitor similar to imatinib and sunitinib but with a slightly different profile. The 1st line trial is a direct head to head randomised trial of imatinib v masitinib. This means that patients who enrol will not be aware which drug they are receiving however should the trial centre feel that the patient is not responding then the patient will be unblended and have the option of conventional GIST treatment. Similarly the 2nd line trial is head to head of sunitinib v masitinib and the same criteria as above will enable the patient to receive conventional treatment should there be no benefit. There are a number of trial centres in the UK who have already signed up (with more to come). Centres who have signed up to-date for the 1st line trial (imatinib v masitinib) are:

  • Dr Marples in Leeds
  • Dr Leahy at the Christie
  • Dr Hayward in Edinburgh
  • Dr Sivamarlingam in Preston
  • Dr Morgan in Cardiff
  • Dr Ali in Clatterbridge

For the 2nd line trial the following centres have signed up so far:

  • Dr Morgan Cardiff
  • Dr Hickish Bournmouth
  • Dr Leahy Christie

We are trying to get updated information on the completed phase 2 trials for both of these trials and will hopefully be able to give an update at our 2016 patient meeting in Nottingham and also post information on Listserve.

Mitigate Poject

This is a pan-European project exploring new ways of treating metastatic GIST.  For more information go to 

Various Drug Trials

Imatinib (Glivec)

There are several trials trying to find out whether taking imatinib (Glivec) after complete removal of a GIST helps to prevent recurrence and long-term survival. This is called adjuvant treatment. (ie taking a drug when there is no obvious disease.)

One trial in the US has shown that taking imatinib for a year seems to dramatically increase the time before recurrence after surgery for Kit-positive high risk GIST. (Click here to download the full text of the NOVARTIS press release.) However it seems to make no difference to patients who had low risk GIST.

There are two other trials looking at adjuvant treatment. The pan-European adjuvant trial EORTC 62024 closed after recruiting 750 patients. Patients with a complete resection (no marginal disease or mets) were randomised to either 400mg imatinib for two years, or to no treatment (which is the standard care). The aim is to answer the question about whether imatinib has value in preventing recurrence and giving long-term survival. Unlike the US trial, the patients will be followed up for years, so we will have no results for a long time.

Another similar trial being conducted in Scandinavia is comparing the results of taking 400mgs per day of Glivec after surgery, for one year or three years.

These trials show that for high-risk patients taking imatinib for three years after surgery increases both time to progression and overall survival. Taking imatinib for one year was better than not taking it at all, but not as effective as taking it for three years. Many doctors now think that high-risk patients should take imatinib for ever, but the cost implications of this are significant (or at least until it goes off patent!). In the UK the adjuvant use of imatinib is not yet approved by NICE, but it is available in Scotland and also in parts of England and Wales through the Cancer Drugs Fund.


A trial of Regorafinib (made by Bayer) has shown very encouraging results. There are no trials in the Uk for this drug, but it has now become available via THE CANCER DRUGS FUND, after an appeal by GSUK.  The use of Regorafinib for GIST is subject to review by NICE

Updated 15/10/16

Masitinib and AB Science (to be updated)

We are able to update you on the above new drug. An application has been submitted to the EMA (European Medicines Agency) for a conditional licence for masitinib in 2nd line GIST. It is expected that a decision will be made before the year end and this means that clinicians will be able to choose between sunitinib and masitinib for patients who have progressed on imatinib 400mg or 800mg. Based on the phase 2 trial results, (masitinib v sunitinib) there appears to be a benefit for patients who received masitinib.

There is also an on going phase 3 trial of Masitinib currently open and several UK hospitals are participating in the study although full results will not be available until 2015. Be aware that this trial has now closed. Click on this Cancer Research UK link for more details and suitabililty.

AB Science have also initiated a compassionate use programme in the UK, offering compassionate access to Mastinib (third line) for patients who have progressed on current licensed treatments (imatinib & sunitinib). This appears to be not widely known by all health care providers. We can put you in contact with the right people if you think this could help.

If you require further information please contact:

David Falconer Special Adviser GSUK


Can you help Professor Eamonn Maher with his research Study?

We have received a request from Professor Eamonn Maher at the University of Cambridge

asking if we can help him make contact with anyone who has had:

  • two or more primary GISTs  or   a GIST and another tumour

for inclusion in the multiple primary tumour study he is working on.

Though this combination of tumours is an uncommon event it can have several causes.

Professor Maher and colleagues at the University of Cambridge are investigating how often this can be caused by inherited genetic changes.

They have requested that any GIST patients who fit the above criteria contact them directly for further information at:

or write to:

Prof. Eamonn Maher, Box 238, Addenbrooke’s Hospital, Cambridge Biomedical Campus, Cambridge CB2 0QQ.

Participants will need to provide a blood sample and clinical information but the study does not require a visit to Cambridge.

Background information on the study (for all tumours and not specifically for GIST) can be found at:

Many thanks for your help.

We hope that we will be able to help Professor Maher to help us!

Updated 1/08/13 & 13/08/15


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The next PAWS-GIST clinic is happening on Friday 7th December 2018. Registration is via PAWSGISTCLINIC.ORG.UK 

Our next Patient Meetings are in Edinburgh on Thursday 31st January 2019, Birmingham on Friday 8th March 2019 and Leeds on Friday 4th October 2019. If you would like to join us please email  to register. We look forward to seeing you...

Rare Cancer Patient Survey - collecting feedback on rare cancer patient experience with clinical trials...

On the 10th October the Scottish Sarcoma network are holding a day called RISE at the Western Park Hotel in Falkirk. The day is dedicated to people with a sarcoma diagnosis and their families to focus on "what matters to me". This poster contains the registration details if you would like to attend. GIST Support UK will be represented at this meeting and we look forward to seeing you. 

A big thank you to Scotmid Co-operative for choosing to support GIST Support UK and the development of our national tissue bank. Read more here


If you missed any of our previous meetings, you can view the speakers' presentations here

Clinical Trial - 3 v 5 years adjuvant imatinib - trial centre list update here

19th August 2017 - Page 19 of the Times GIST report - READ HERE

New UK clinical practice guidelines for the management of gastrointestinal stromal tumours (GIST) published by British Sarcoma Group - NEWS >>>

GSUK's Chair Nic Puntis discusses the BBC's report on the Cancer Drugs Fund on BBC Wiltshire Radio - NEWS>>>.  

Update on GIST clinical trials that are recruiting and in the pipeline....

We have launched our first GIST patient survey and hope to gather a better understanding of the landscape for GIST patient treatment in the UK. The survey closes on 10th September 2016 so if you would like to participate please complete the survey by clicking this link

'ALT' GIST Trial to open in the UK soon! We have been alerted to a new trial that will be opening in the UK in the near future for metastatic GIST patients. Read more on our Trials & Research page 

PAWS-GIST Clinic 7 - Newsletter June 2016

GSUK attends Cancer52 parliamentary reception to raise awareness of rare and less common cancers.

GSUK Research Update 2016 - Read all about it here

NHS England's Sarcoma Service Specification consultation is open until Weds 20th April 2016.       Please register your views by responding directly via this link.                             GSUK's response can be viewed here

Cycle for GSUK! We have four allocated charity places on the RideLondon event for this year and also for 2017 and 2018. Go to our News page to find out more!

The Masitinib Trial is now open.  For further details of this trial, and to find out which centres and clinicians are involved, please go to our Research & Trials page. 

Regorafenib has remained on the NHS England Cancer Drug Fund List for GIST patients! Read more!

Can you help Professor Eamonn Maher with his research Study? If you have had, or have, two or more primary GISTs, or a GIST and another tumour, find out more at the bottom of our research page

PRESS RELEASE - UKTV announces GIST Support UK as its Charity of the Year 2015

TUMOUR BANK UPDATE: "We have been granted approval from the NHS ethics committee for our National GIST Tissue Bank which will store these valuable GIST tumour samples for research. If you are about to have an operation and would like to donate your tissue for research please email  with your contact details. Visit our Retention of tissue page for regular updates.

Scottish Sarcoma Network: Interested in attending their Sarcoma education days or finding out about the services on offer ? Read our News page

Use of human tissue
Do go to our new "Retention of Tissue" page and please sign a consent form!

GSUK funds research!
GSUK provides £20,000 from donated funds to help start a research project at the Royal Marsden. Visit our News page for further information and picture.

Funds being raised for UK PAWS GIST clinic
See our News Page for more information.

NICE approves improved treatment for ascites
The PleurX system allows the patient to drain fluid from the abdominal cavity without having to pay repeated visits to hospital. More information on our News Page.

European Commission approves three years' adjuvant use of Glivec
Visit our News page for more details.

A press release in February claiming the effectiveness of this drug as a replacement for Sunitinib appeared to be premature at the time. However subsequent news may be more encouraging! See our News page for more details.