FAQ's

Things should begin to happen very quickly, and it will feel very surreal. Suddenly normal 'life' is turned upside down and becomes filled with appointments and introductions to many professionals previously unheard of. New terminology will become your vocabulary and at first the 'fear' is overwhelming....

The first steps will usually include appointments with your appointed oncologist and radiologist. These are very difficult meetings to have as you will already know, if you find yourself on this page!

For radiotherapy to commence planning usually takes place in a CT scanner. A 'mask' must be moulded to hold your child's head in position for treatment, most young children will have a 'port' fitted and complete their moulding, planning procedures and radiotherapy under a general anaesthetic. This can be very worrying initially, if you are lucky, it will become part of routine over the next weeks ...most centres should have 'play specialists' to help explain what is going to happen.

YOU know your child the best, don't be afraid to ask for additional support to get them through certain difficult procedures.

Steroids which need to be taken in conjunction with radiation to reduce inflammation, can greatly change the mood of your child. They can become uncharacteristically aggressive and difficult to manage at times it can be almost impossible to recognise them, or to know if it is disease, medication or fear...others can sail through much more easily but be prepared for big changes in physical appearance and an extremely emotional journey.

Unfortunately, at the moment no one knows why, or entirely how DIPG occurs. It rarely affects adults and is thought to be a process within the developing brain. Hence the peak age of incidence with onset in children between 5 - 9 years of age.

Research in this field is very active, like most cancers DIPG occurs when something goes wrong with the process of cell reproduction, and a 'mutation' occurs. Scientists have homed in on the cells of origin in DIPG and the exact processes that are interrupting the normal maturation and correct lineage that give rise to tumour formation.

Scientists are now learning much more about the genetic mutations that occur or are present in a majority of cases of DIPG. This has been found due to the selfless donations of tissue from families either at autopsy or during biopsy (not always standard procedure in the UK). We need to do much more to understand how these mutations connect fully with the micro-environment in the brain, as they are very complex and to aid knowledge of the available drugs already approved which may be 're-purposed' to treat DIPG effectively.

There is absolutely no evidence that DIPG is a resultant of exposure to any environmental or lifestyle factors or inherited and hereditary factors.

DIPG almost exclusively affects children aged 4 to 11. Approximately 40 children per year develop DIPG in the UK, representing 10-15% of all childhood brain tumours. It is not hereditary and occurs equally among boys and girls.

Unfortunately, DIPG is highly malignant, and very aggressive. DIPG can present in a very short space of time which is why it is important to try and gain control of the local area as quickly as possible...either through radiotherapy or other means.

You will already know the devastating symptoms being exhibited by your child, because of its precarious location in the brain stem, DIPGs cause pressure on the cranial nerves that originate in the pons as they grow. As the tumour grows the symptoms get worse, they are extremely debilitating and this can happen very quickly.

If you would like to talk (it's not for everyone) to others including those parents that may have already lost a child, then there are numerous groups on social media that may support. The groups are not for the faint hearted. You’ll find numerous other families online and will have lots of parents willing to help at various stages of their experience with DIPG disease. These can be helpful to decide on treatments options or gain advice on worrying symptoms. There are many parents led charities you will discover who are often the 'driving forces' in the DIPG community. We are always available to speak personally with anyone should they wish or connect them to other parents we have come to know who are happy to help.

Absolutely not. There are numerous cases of DIPG affecting twins and even triplets where only one child is affected. The risk for the others is as rare as for any individual child to manifest DIPG. There is also no evidence that specific inherited genetic variations contribute to DIPG either... quite simply because sadly most patients will succumb to their disease, it cannot be 'passed on'.

Standard NHS treatment consists of 'palliative' radiotherapy (usually 54 Gray) which may be administered over differing lengths of time. It is important to begin this therapy as soon as possible, around 70% of patients are known to get symptom relief and tumour shrinkage BUT most importantly you will need to consider this in the context of prospective trials ... prior radiation may render a patient 'ineligible' and unable to take part in some studies.

Some trials for newly diagnosed patients, may have drugs administered adjuvantly with radiotherapy as it is thought the effects of the agents are increased. If there is a particular trial, you are interested in (elsewhere in Europe or the US for example) please check the enrolment information very carefully so as not to be excluded.

Most diagnoses of DIPG can be, and are made from MRI imaging and their anatomical location.

Because of the risks of the procedure, surgical biopsy has typically only been performed when it has not been possible to confirm a diagnosis based on imaging (atypical). In the UK and worldwide however more doctors that specialise in DIPG more generally are recommending biopsy (The UK does not currently have a biopsy led clinical trial, but 'targeted' agents may be offered).

It is in itself a dilemma - and important if an available option for parents to weigh up and question any risks. Some parents feel that biopsy can cause disease to spread if only one cell is dropped along the tract of the tissue sampling channel...however DIPG disseminates throughout the brain and research in any case considers it 'full brain disease' it is also becoming crucial for more individualised treatment options to know which alterations are 'present targets'.

Neurological surgical techniques are now greatly improved and recent research has shown that advanced stereotactic methods greatly reduce the risks of surgical biopsy, which can sometimes yield useful diagnostic information and influence treatment decisions.

Occasionally when biopsy for a presumed DIPG is performed, an alternative histology may be discovered, which can impact your child’s treatment plan and change a prognosis very significantly. Of course this should only be performed by the relevant specialists with experience of the procedure.

There are increasing numbers of clinical trials which do require biopsy and specifically the H3K27M diagnostic marker for entry - check if it's important to you, that if you pursue this route that results will be made available and select based on who is looking for the most relevant mutations through the sequencing panel, and how quickly they'll process the biopsy. There may be associated 'modelling' of the cell line sample and drug screens if part of a trial. Biopsy options will only be available in larger institutions with neurosurgeons who have repeatedly completed DIPG biopsies.

FULL characterisation of these tumours is extremely important. It is absolutely pointless treating a child with a drug when the mutational target is not present in their tumour. Only full sequencing can provide a broad analysis and individual profile. It may also be that we currently do not have drugs available that will 'target' every present mutation, but studies are beginning to show the most promising prevalent targets and pathways for inhibition.

Scarcity of tumour tissue itself has hampered treatment development for children, in the past DIPG was treated with agents that have showed some efficacy in adult disease. However, children are not 'little adults' and require specific drugs for the specific biology of their disease.

DIPGs are relatively rare, and the diagnosis and treatment of DIPG is complex and involves multiple specialists. At the UK specialist centres you should have a dedicated paediatric neuro-oncologist, neurologist, neuroradiologist, and possibly even a neurosurgeon.

You can ask to be put in touch with any eminent researchers or clinicians worldwide. Do make sure if considering an approach under a certain doctor that you contact them directly, most will be very helpful. Discuss options fully and get your referral should you choose another pathway.

Check the full listing of clinical trials in the relevant section on the site tab 'Worldwide Clinical Trials'.

Research over the last ten years has helped improve treatment for DIPG patients, but life expectancy has not been greatly extended, the prognosis is still not good–with the median survival range being from 8-11 months

Statistics overall with DIPG have also evolved, culminating in the release of updated data from the International DIPG Registry in 2018, identifying changes from previously assumed statistics, including:

• 2-year survival is approximately 10%

• 5-year survival is approximately 2% (up from 1% previously reported)

Unfortunately, DIPG is always considered a terminal disease, with patients receiving only 'palliative' radiation as standard of care worldwide.

Yes...If you wish to donate tissue in the event of biopsy (or autopsy) there are various registries, speak with your oncologist about supporting one of these.

Some trials depend on biopsy for entry to assess certain markers, these samples are hugely valuable and if cell lines can be reproduced are used widely in other research studies. Most are held in the paediatric tissue bank in Newcastle where other researchers can request them for study.

DIPG experts participating in the International and European DIPG Registry are available to patients, families and their doctors for second opinions also and may be able to recommend other clinical trials for which patients may be eligible.

The UK representative for the DIPG Registry is:

Simon Bailey, MD

simon.bailey@newcastle.ac.uk

You can find full details on all clinical trials here

Parents facing diagnosis of DIPG can only be provided with the overview of their own NHS (in the UK) clinical care team administering the treatment available to them in that location. They may not have the full guidance as to the available treatments being advocated and pioneered elsewhere throughout the world.

You may also not always have a clinician that is highly encouraging in pursuing other options. They may be highly sceptical due to historical reasons, prognosis and the lack of documented 'peer reviewed' process.

There are however many people, parents and some clinicians included who understand that conventional approaches to treatment are just not creating the clinical outcomes that we all wish to see. It is now well established within research that a multi agent and multi-pronged 'attack', will be required to fully manage or eliminate DIPG.

This may include surgery for direct drug delivery, a priming of the tumour microenvironment and immunotherapy approaches. If you are prepared to pay for private consultations, you may want to engage outside help or seek out other experimental therapies in 'self-pay' settings.

Unfortunately, any new approaches in other locations really have not been studied in sufficient depth individually and in combination to make them available to every patient and currently will likely come at a huge cost expense to families. If you will be undertaking any of these options that may include overseas travel do make sure that on your return that you have a consultant fully prepared to continue treating your child or provide any maintenance therapy and tests.

It can become blurred whose care they are actually under. In instances where full cooperation is not received and information is not shared, it must be appreciated that it can become difficult for UK doctors to know how to proceed in treating a patient on their return.

Available private and 'compassionate care' treatments and procedures are not clinical trials. Some processes may be well documented in comparison to others. Ideally Abbie's Army would wish to see sufficient research funding investment to generate all the pre-clinical testing necessary for robust data, ensuring the crucial protection of children enrolling in more experimental studies. Responses must come from a strong scientific rationale, above all be 'safe' for children, and have any research data 'outputs' freely shared.

With regulatory approvals and a clinical trial framework in place any therapy can be developed further with any successful outcomes adopted for the benefit of all patients, not only those that can fund raise and pay.

The average survival time from diagnosis for a child remains at just 9-11 months.

Although 75-85% of patients experience some symptomatic improvement after radiation therapy, DIPGs almost invariably recur or progress within 6 months from therapy according to published data. Once the tumour begins growing again, it typically does so rapidly, affecting critical brain regions. Any further treatment is generally supportive and aimed at relieving symptoms. Sadly, following tumour progression, the median survival time is alarmingly short, ranging from just 1 to 4.5 months based on reported clinical outcomes.

If you have relatively stable disease sometimes a second round of radiation may be offered known as re-radiation, this will depend on previous responses. Progression can also occur more than once in the disease course.

While the average survival statistics are dire, it's important to remember that every child's journey is different. If your child has been diagnosed with a DIPG, focusing on their wellbeing and quality of life is by far the most important thing.

Although there are currently no proven effective treatments or a cure available at present, there is now much more research into DIPG and discoveries are constantly being published. We remain hopeful this will lead to treatments to manage the progression of the disease in future cases.

The understanding of DIPG biology has improved greatly, along with a better known tumour micro-environment and the mechanism of action of certain drug classes (i.e HDACi's) on known mutations (H3 K27M) and activated pathways in the disease. There are also immunotherapy target 'antigens' presented on the outside of DIPG cells (such as GD2 and IL13-RA2) for which powerful CAR-T cell therapies are now being developed. This means we are in a better position to assess the efficacy of potential approaches and drugs in combination within the developing sub-set patient cohorts in relation to tumour biology in highly organised and structured clinical trials.

Scientists are also homing in on the origins of DIPG and its micro-environmental dependencies, it's generally understood that combinations and whole treatments approaches in the correct order and timing will be helpful. There is every reason to be more hopeful.

Towards the end of life, you may want to consider giving your consent to donating your child's whole brain (and sometimes spine) which can give researchers a full picture of disease and be a comfort to know they 'go on' to advance the research field, for children diagnosed in the future.

In the UK this is generally supported and coordinated through the CCLG tissue bank (Children's Cancer and Leukaemia Group) although there are other UK labs such as the Jones Lab that specialises in paediatric high grade glioma research, at the Institute of Cancer Research that regularly process samples. The family is fully entitled to choose any lab where they wish the sample to go.

Tissue donation does need to happen fairly quickly, but a family’s needs must come first, you will be able to change your decision at any time.

It may be important to consider if once consent is given, that you might want to discuss any findings afterwards with your consultant (and possibly a researcher) and whether that will be an option that is available to you. Your oncologist should advise you accordingly.

Information on the CCLG Tissue Bank is on this link https://www.cclg.org.uk/tissue-bank

Families have experienced all kinds of ways to preserve precious memories in the more difficult later stages of disease. Some meaningful ideas are :

To travel, and take trips to your child's favourite places any time that they feel they can.

To consider celebrating any birthdays, family occasions or public holidays early.

To collect imprints or moulds from which sculpture and jewellery can be made.

Cut a lock of hair to keep.

To take as many photos and videos of your child hopefully in happier days doing things they love.

As symptoms begin to worsen , they may feel too overwhelming to control yourself at home, if this is the case you may want to consider the use of a children's hospice. their facilities and expertise. They may have other different therapies to offer such as music, art or have a pool to help your child feel more stimulated as mobility decreases.

When facing the end of life, hospice focuses on providing peace and dignity during the dying process. The care is family cantered and can include siblings and other family members. Hospice teams will work with your doctor to ensure coordination of psychological, spiritual, pain management, and physical care.

The needs of the family will also not end with the child’s death. Paediatric hospices continue to help the family even after the child dies by connecting families for bereavement support.