Biological Medicine for Diffuse Intrinsic Pontine Glioma (DIPG) Eradication (BIOMEDE)
The BIOMEDE Trial is the only open UK trial, available for DIPG patients (Newly Diagnosed).
The principle investigator is Dr Darren Hargrave.
Dr Hargrave is a Consultant Paediatric Oncologist at Great Ormond Street Hospital. He is involved in many groups, both in the UK and internationally, that are driving forward clinical trials in children so that he, and his colleagues, are able to offer more effective and less harmful treatments to children with brain tumours.
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Thanks to all our amazing supporters, Alfie Bear's Journey Fund, funded £90,000 towards the BIOMEDE Trial.
What Is BIOMEDE?
BIOMEDE is the acronym given to the clinical trial Biological Medicine for Diffuse Intrinsic Pontine Glioma (DIPG) Eradication.
The aim of the trial is to change the standard treatment given to children with a DIPG to treatment/s that work better at slowing or killing the tumour and are kinder to the children.
DIPG tumours can be split into different groups depending on the changes within the tumour. It is thought that these different changes are what cause the tumour to start growing in the first place, and that by using drugs to target these specific changes the growth of the tumour can be slowed, or the tumour can be killed completely.
One of the reasons that there has been little research on DIPG tumours is that surgeons have been hesitant to undertake biopsy, when the child's tumour is in such an important area of the brain.
One of the innovative aspects of the BIOMEDE trial is that biopsy is compulsory. This has meant that surgeons in participation hospitals have been trained in how to do a good, safe biopsy and that there will be tumour tissue available to analyse for the specific changes they are looking for.
Where there is a choice of drug for the group the child's treatment will be assigned at random. This is because the researchers think that these are the best drugs for this type of tumour, but need to test them together to see which is the best drug.
One of the other innovative things about this trial is that it has been specifically designed to be adaptive. If a new possibly better, drug becomes available the researchers can add another group to the trial.
Likewise, if a particular drug is not working they can remove it without having to start the rest of the trial again.
This is particularly key because it takes YEARS to set up a trial and make sure that all the ethical and governance approvals are in place.
Therapeutic Targeting of ALK2 for the Treatment of DIPG
Alfie Bear's Journey Fund are proud to be fundraising for the DIPG research within The Structural Genomics Consortium (SGC) through The Brain Tumour Charity's new partnership. 2017/18
Who are The Structural Genomics Consortium?
The Structural Genomics Consortium (SGC) are an international group of researchers using new technologies to accelerate the discovery of potential treatments for incurable diseases.
The Brain Tumour Charity are one of just few charities who have entered this type of agreement, driving the next generation of discoveries forward, focusing specifically on the proteins and biology that play a part in brain tumour development.
General Aims of the Project
Diffuse Intrinsic Pontine Glioma (DIPG) is the second most common type of primary high-grade brain tumour in children. There is currently a lack of treatment options for this tumour type and prognosis for patients is poor. The SGC have had a long-standing interest in the gene ACVR1, which is found to be mutated in around 25% of cases of DIPG.
When ACVR1 is mutated, it causes over-activation of a protein known as ALK2 which in turn leads to excessive cell proliferation and hence tumour growth.
The SGC's previous research has enabled them to target ALK2 as a therapeutic option for another disease. With our support, they now propose to design and synthesise new drugs to block the actions of ALK2, with the ultimate goal of delivering a new clinical trial option for the treatment of DIPG.
Specific Aims of the Project
Optimisation of The SGC's current ALK2 inhibitors with proven activity against DIPG
Exploration of new ALK2 inhibitors using publicly available compound libraries from industry
To use a method known as Fragment Screening to identify new target sites on ALK2 for drug development
Formal Title: ALK2/ACVR1 Kinase Inhibitors for DIPG therapy
Principle Investigator: Dr Alex Bullock
Tumour Type: Diffuse Intrinsic Pontine Glioma (DIPG)
Research Type: Drug Discovery, Cell Discovery
Please visit The Brain Tumour Charity's website for more Information: https://www.thebraintumourcharity.org/our-research/our-funding/what-were-funding/sgc/