Experimental DIPG Therapy

While the search for a DIPG cure continues, children with DIPG usually must endure one experimental trial after another, usually suffering awful side-effects. Research is showing that single agent trials will NOT be curative in DIPG...multi agent and multi modes of therapy will be needed.

There are now more trials that are testing efficacy in DIPG for combinations of drugs matched to tumour profiles from biopsy.

We are also beginning to see new developments and some combinational approaches using technologies (multi modal) that by-pass the blood brain barrier and deliver agents directly to the brain stem,  available on 'compassionate' grounds in the UK (See CED). Some are more well documented than others and there are fees for these treatments.

Unfortunately for families the costs can also be HUGE!...None of the experimental options are currently with any data driven 'approvals' that would allow their adoption within the NHS here in the UK, or make them available on insurance for overseas patients.

All consequently are currently found in private 'self-pay' clinic settings...these are the procedures that have the most interest and are more widely used by the DIPG community.

You will find many family accounts of all those trying these procedures if you follow any of the social media Facebook pages.

Convection Enhanced Delivery (CED)

Convection Enhanced Delivery - CED

The Harley Street Clinic, London (Re-located from Bristol Children's Hospital)

To call this .experimental' is rather a misnomer as it has been around for considerable time - CED trials are available worldwide using varying agents ( see Worldwide Trials Listing ) and UK trials are hopefully coming soon.

CED trials using soluble panobinostat (MTX110) sponsored by 'Midatech Pharma' are now running through the PNOC (The Pacific Pediatric Neuro-Oncology Consortium) in the US at UCSF ( San Francisco) and MSKCC ( New York) ...catheter designs however vary to the London/ Renishaw delivery system discussed below. Trial Ref NCT03566199 (PNOC015)

Abbie's Army has been involved with the lab team and Functional Neurosurgery Group in Bristol investigating agents used in CED in combination with carboplatin.

Children with brainstem tumours have a very poor prognosis with almost all patients succumbing within 18 months of diagnosis. One of the major obstacles to the effective treatment of these tumours is the blood-brain barrier (BBB) which prevents the free passage of chemotherapies from the bloodstream into the brain.

To date, no clinical trial of intravenous chemotherapy has shown a beneficial effect on the outcome for children with brainstem glioma. Children also suffer significant side-effects when chemotherapies are administered intravenously in high doses.
In Bristol (initially) they have been treating children with progressive brainstem tumours using a novel neurosurgical method of drug delivery to the brain called Convection-enhanced Delivery (CED).

The method involves the robotic implantation of very fine catheters into the brain and infusion of drugs directly into the tumour, thus bypassing the BBB . They have found this technique to be safe and to show some promising signs of efficacy. Importantly the risk of children suffering intolerable side-effects when chemotherapy is administered by CED is very low.

Figure 1. We have developed a unique implantable catheter system (a) which allows us to repeatedly administer drugs to the brain through a port mounted on the skull (b). Using custom made software we can plan the implantation of the catheters with submillimetre precision (c).
Figure 2. Treatment with CED of carboplatin has so far proven safe and well tolerated with some promising signs of efficacy.

Agents known to be currently in use - carboplatin and valproate (VPA) and panobinostat.

Since the closure of the 'compassionate program' in Bristol and its relocation to London in January 2017 , all CED enquiries and sending of scans should now be directed to The Harley Street Clinic  and addressed in the first instance to:


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Intra-Arterial Chemotherapy & Immunotherapy

IA Chemotherapy and (Dendritic Cell/ACT) Immunotherapy

Monterrey, Mexico

There are now more than 70 families who have traveled to Mexico to seek out this new treatment for DIPG in Monterrey, including some from the UK. Again you will find accounts of some of the families following these procedures on Facebook pages where several write a daily blog with any progress. 

A Note for DIPG Parents - With the view of making this treatment as effective as possible the Doctors in Mexico would prefer patients without prior radiation treatment. A thorough review with your treating oncologist and the clinic is essential.

The treatment consists of combined Intra arterial chemotherapies ( with infusion of unknown various drug combinations) all agents are FDA approved, and (Dendritic Cell) immunotherapy. In some cases it seems to be showing some clinical responses and suggestion of tumour stabilisation or shrinkage, and improved clinical condition from symptoms. It's worth noting however that any 'evidence'  of that is not widely corroborated by home treating oncologists.

Entry to the basilar artery which supplies the pons in the brain is accessed via the femoral artery (groin) to allow for infusion to the local area. Little is known about the exact combinations of drugs used because there is little information relayed by families or doctors. There is not strictly an NDA ( Non disclosure agreement) in place but an agreement between patients and doctors who obviously have no wish to jeopardize the procedure for their child.

There are around 11 different drugs being used.

Here is a film from Johns Hopkins that roughly explains the IA part of the procedure.

If you would like further information you can direct your enquiry to Stephanie Skinner msalaza1@hotmail.com  or Cesar Mendoza at:: gcesar.ml@gmail.com 

This website gives more information https://www.idoimexico.com/ 

Costs are very high approximately:- Intra-arterial £12,500 per treatment and Immunotherapy £12,500 per treatment. So combined treatments £25,000 each. Children cannot start any immunotherapy in conjunction with the IA until they are completely off steroid medication.

There are minimal side effects reported, however mild fever, headaches, vomiting, tiredness can be expected several days after. Patients are generally in hospital for one night only after procedure plus a couple of visits to the clinic between procedures.

The treatment Cycle:- minimum 17 days and maximum 35 days between treatment, however early days, they suggest 3 week cycles

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Immunotherapy - IOZK Clinic

IOZK Clinic

Cologne, Germany

This is a form of immunotherapy...actually virotherapy being used to promote an immune response in the patient against DIPG. A vaccine is made from each patient’s own white blood cells and tumour antigens, which are proteins that produce an immune response. The goal is to allow the immune system to recognise and attack the cancer cells.

Stefan Van Gool is the treating physician in Cologne, as of February 2017 and 29 children the results are said to be 'encouraging' but still too early to know if there is life extension.

Check out the website here for contact details and information : https://www.iozk.de/die-iozk-immuntherapie/iozk-immuntherapie/