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The Multitude Of DIPG "Treatment-trials" Choices & The Lack Of Data For All Of Them

Updated: Sep 27, 2023


Sept 27h, 2023: Methionine Diet update

June 21th, 2023: ONC201 twice per week dosing; ONC206; low-methianne died; Metformin; Low-methionine diet; Avapritinib (Ayvakit)

Hello, we are just starting and I will be posting all of our findings and thoughts and expand into deep-dive posts for each. But here is the main areas and treatment options/ideas outside of the radiation (standard of care). Some of them are already showing a glimmer of hope and statically outliers/ patients.

  1. Main Treatment/Clinical-Trials Categories

  2. Longer tail of earlier/start-up ideas

  3. Add-on treatments (not the first line of defense, but maybe some booster)

My quick summary of the top types of DIPG treatments being tested, trialed or considered as ideas. I will later share my non-profesional understanding of plus and minuses of each. There are close to 100 clinical trials ongoing (maybe the best list of most/all trials) and it is hard to keep track which one are actually recruiting/accepting new patients let alone clearly understanding all the inclusion and exclusion criteria, and how such trials effect your chances of getting into other trials. And there are also many side and add-on treatments and ideas the community is talking about. For new patients I would recommend for experts guiding you to the best trials, this is mostly for the US, feel free to reach out to us as we are pushing to have a Europe-wide (and hopefully global) equivalent.

1. Main Treatments

  • Car-T cells, a type of immunotherapy: multiple locations have trials. Different types of surface proteins being targeted (but only one at a time, so far). Pricy but promising. Main Cons so far is that immune response causes inflammation in the pons and this often sends kids in the ER for a tough recovery. As of 2022 most trials have moved from IV delivery of the Car-T to intercranial (direct delivery to the brain) to minimize the systemic toxicity in other organs. Leading centers are Seattle, Stanford and Texas, in 2023 a trial targeting 4 different proteins started, we are eagerly awaiting the initial safety and dosing results.

  • "Chemo": various drugs (or combinations) given through an IV, or orally - there are many old and ongoing trials in the range of hundreds (one location that tried to summarize them) we are tracking our own list of drugs and are supporting (clinical and pre-clinical) work to test some of the more promising ones for DIPG in the DIPG/DMG Center Zurich. Some exciting drugs and combinations are being tested after good pre-clinical (in test tubes) trials as well as in-vivo (in mice most often) results.

    • There are many subtypes here as various drugs target various things -> your biopsy and DNA/RNA sequencing could provide a lead what could be targeted.

    • !!! most drugs are pure repurposing, new drug development (is pricy) for DIPG remains a far hope.

    • My personal most promising drugs:

      • ONC201 and Paxalisib: "ONC201 + Paxalisib has shown emerging clinical efficacy for patients diagnosed with DMG" (PNOC022, NCT05009992)

        • As of Q2 2023: "Twice per week dosing on two consecutive days expected to increase duration of therapeutic exposure... can increase glioma sensitivity to ONC201 in vitro" Source: Chimerix May 04, 2023 ppt

        • Metformin (used for diabetes) has also shown synergetic data with Onc201 (shared in the community around end of Q1 2023) and lowers sugar in the blood.

        • Onc206 is very similar to Onc201, binds in a different way, and is up to 10x more potnet in pre-clinical tests. Phase 1 trial for MTA (max toleratable dose is ongoign now). Evitenly also avaialble as German Sourced compasionate care prescription.

  • Oral AMXT 1501 in Combination With IV DFMO (I have concerns with DFMO passing the BBB, but in-mice trials have shown tremendous results (maybe with CED and/or BBB opening combination?), but some positive patient reports do exists.

  • Avapritinb or Ayvakyt repurposed drug is going to be tested in Clinical Trial NCT04773782 targeting PDGFR (maybe more) as a single agent, but I am more excited of its work as a combo. Risk of bleeding has been reported as well as possible other toxicity so this is a bit risky. Also it costs Euro 20K/month (even more in the US) if you get a prescription. As of this writing (June 2023) all sellers form India are offering fakes (PM me for details as I've investigates a lot)

  • Selinexor

  • Drug repurposing is ongoing (100s of drugs & combinations) are tested and some of them have shown promises in the lab but yet to be proven in the clinic. Novel targeted drug development remains too expensive.

  • OncoVirus - special viruses (ex. DNX-2401 Oncolytic Adenovirus single site Phase 1 trial results) that infect only cancer cells with direct to tumor delivery: no open trials as of today. (2023 might be positive to have at least one trial, but there are challenges). There is a discussion if stem-cells loaded onco-virus (ex. Calidi Biotherapeutics) is better vs. the naked virus DNX-2401. For DIPG we seem to only have naked-virus information, GBM has wider options.

  • CED (Convection Enhanced Delivery) - directly infusing (with catheter(s)) some drug/chemo into the tumor. Very similar procedure as the brain tumor biopsy (often done/advisable to be done at the same time). Benefit: bypass the BBB (Blood Brain Barrier) and remove most of the systemic toxicity. Trials with CED delivery have to be done per drug thus the technique, although very promising, remains to be approved for general use. Best leads:

    • Mark M. Souweidane, M.D the leader in the space for years with good success and most data.

    • neuroinfuse™ drug delivery system great tech allows multiple infusions with up to 4 catheters (better coverage of the tumor), but there has not been a push/support to get a real clinical trials going. 7+ Compassionate Care cases in the UK have been rumored/reported.

    • Drugs considered/tested MTX110 (Panobinostat) NCT03566199, NCT04264143

    • As of March 2023 NCT05063357 is enrolling patients for CED of the drug 131I-Omburtamab

  • Focused Ultrasound FUS (see full blog) - new but promising (various use cases) some clinical trials are ongoing.

    • 1. microbubbles to temporarily open a selected section of the BBB and allow drugs to enter the brain as a start or in larger quantities (for the BBB penetrating drugs).

2. Longer tail of earlier/start-up ideas

The longer tail of new devices and ideas that could be SciFi amazing in start-ups of pre-clinical research.

  • Hype fraction Radiation: take smaller doses of radiation: for example 1 week at a time, but repeat it as needed. An ongoing trial(s) exist , but no previous supporting data that it is better than the current standard of care. March 2023 recruiting trial: NCT05077735

  • RADIO FREQUENCY ENERGY (ulRFE) SPECTRUM: Nativis Voyager device (maybe this is the old name) tries to "directly influence metabolic pathways" by measuring the specific electromagnetic fingerprints of a molecule and record their molecular “song.” Which then get replayed by a coil worn by the patient. Ref: EMulate Tx . In short, they record the ghost of the drug and then replay this without having to actually take the drug: this has a much wider application than DIPG, and we at Swiss to Cure DIPG have funded some pre-clinical work in UCSF to get an idea on the efficacy in mice bearing DIPG. It should work with any drug that work by non-covelantly binding (just by exchange electro-magnetic signals) with patient cells: this should represent over 50%-66% of all currently used drugs.

3. Add-on ideas (nor primary treatment so far)

Add-on ideas that do not have any solid clinical data proving efficacy, dosage and/or safety for DIPG/DMG but are commonly used and discussed But dosage varies widely and efficacy is based in self-reported cases or they have very limited pre-clinical only tests (eg. CBD). For all of those always be aware of liver problems like elevated enzymes ALV (this could be very serious and could exclude you from clinical trials until the liver recovers): some of them like CBD might add extra load on the patient liver other could help (like green juices, but check which green, lemon juice...)

  • CBD &/or THC/CBG (see fully blog post here) - pre-clinical trials show CBD killing cancer/DIPG cells (dedicated blog for CBD/THC/CBG linked above). THC remains questionable as the high-effect as well as not clinical proves of efficacy. One claim (no data/confirmation could be found) that there is no cabinoid system in the pons and thus for DIPG it might not work as treatment but just for symptoms.

  • Turmeric and Curcumin as a food supplement, but also as special set of molecules in FDA registered drug(s) currently being trialed for GBM and other head and neck tumors like's APG-157 drug (we are starting dome pre-clinical testing and combination testing on DIPG cell-lines )


  • Ketogenic Diet, Vegan/no animal(Methionine Restrictive Diet) products diet or ... (I am still looking into that, but for kids it could be a stretch and risky based on some papers that I've read and expert opinions) We've tried Keto for a 5yo and it was no problem at all, as of June 2023 we started low-methionine. On the other side I've seen long(er)-term servitors beating the odds using Keto ...

    • Keto Diet (really a modified Atkins Diet, as real Keto is not feasible for kids and required at least 1 week in-patient set-up) has been recommended as an add-on to the Onc201+Pax combination. Note the high fats & salts (if not the good fats like avocado, olive oil...) could be taxing on the liver as well.

      • Metformin (used for diabetes) has also shown synergetic data with Onc201 (shared in the community around end of Q1 2023) and lowers sugar in the blood. Also a good option if not doing the KETO diet.

  • No-animal products (Methionine Restrictive Diet: link for a dedicated article) was mentioned as best option for patients Onc201 only. Example (1 min video) results of recently funded research by Dr. Cinthia Hawkins in mice. This link gives a rough idea what the diet looks lie (for another amino acid but the principle is the same) In May 2023 there was data shared form Canadian researchers that have shown great results in mice with DIPG. In general low-methionine diet has lots of history, publications and other types of cancer patients living by it: Example source: PM me for more details as I am actively deep diving into that, finding, products, recipoies, brands, reserach as well oral enzymes that could help. Note the Oral-rMethioninase (O-rMETase) enzyme that helps removing some of the methioinine consumed through food: growing body of research (example) shows that seems to be a good combination with the diet. !!!! Note this diet requires checking for adequate amino-acids in the blood ot make sure proper body-growth, not loosing weight... (kids under 2 years should take even further caution as this amino-acid is key in growth and protein formaiton). Publication on the: "reliance on the AA methionine and its effects on the epigenome of DIPG [in mice]"

Antineoplastons by Dr. Salislaw Burzynski are highly controversial so I suggest you read this Fact Check side by Reuters on it. "VERDICT False. Antineoplastons therapy is not FDA-approved, and is not recommended by medical institutions or experts because of a lack of evidence from randomized controlled trials to support its safety or effectiveness in treating cancer."

If you have other ideas please do share and I will add more links and details for each (maybe in separate posts)

Other variations when the treatment is administrated

  1. Before/after/during radiation

  2. Before/after/during biopsy (with or without biopsy)

  3. After relapse/recurrence/progression of the tumor: something was done, maybe there was stable period, but the tumor worsens or spreads.

Good source for list of clinical trials:

We as Swiss to Cure DIPG remain confident that some combinations of some of the areas above (or new ones) will bring a light at the end of the tunnel and continue supporting targeted research, trials and connecting promising ideas to DIPG experts.

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3 comentarios

18 sept 2023

Oxygenated water - what kind of water is this? how to make it?

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20 jun 2023

One thing we have been doing as we are on ONC201 and Pax but do not have access to metformin is the Keto diet combined with an intermittent fast (6PM to 8AM) and giving ONC and Pax 1/2 way through the fast (1AM) to allow the drugs to activate when glucose levels are at there low point.

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Armelle de Madre
Armelle de Madre
12 feb 2023

thanks Bobi this (and your other blogs) are really good and clear summaries. any thoughts/paper on Avastin ? thanks Armelle

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