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Wednesday, November 24, 2021
Issue 5847

The most important bill ever introduced into congress for brain tumor patients needs your support!
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Latest News

Discuss these articles in our forum!
  • Musella Foundation 2021 Highlights and End of Year Appeal!        

    This is our annual fundraising appeal!  We are trying to speed up the search for the cure, and frankly - lack of resources is slowing us down. This is the most exciting time ever in the brain tumor world. Everything is finally coming together - the basic science research is finally figuring out how things work.  There are promising treatments in the pipeline, and an abundance of drugs being approved for other cancers that we can use off label.  We created a learning network to take advantage of all of this. We are working on legislation that will drastically speed up the process. 

    But we need your help!  Please make a donation and get your extended families to donate!

  • XCELSIOR - A real time, real world learning platform for patients with advanced cancer        
    I am proud to announce that we presented a poster on our learning system at the recent Society for Neuro-oncology conference in Boston!  This was done in partnership with Cancer Commons, xCures and the Musella Foundation.  The poster explains the learning system and how it benefits everyone in the brain tumor community - starting of course with Patients - helping patients is the reason we created the system!  It also helps doctors, researchers, nonprofit patient organizations, pharma and  biotech companies.     I may be biased because I helped create the system and I am a consultant for it, and the Musella Foundation uses it for our Patient Navigation system - but I feel every brain tumor patient (or patients with other advanced cancers) - as well as the doctors who treat these patients - should participate in the system.  We learn from every patient and every patient gets access to xINFORM, which is our patient portal where you can get a customized, curated list of treatment options as well as see a concise summary of your treatment journey.
    Doctors get the xDECIDE portal, where they can register their patients for xINFORM, and see the treatment options report and ask for help with any of their cases. "Help" can be having specialized virtual tumor boards which the doctor can participate in and ask questions of other experts, or help with getting expanded access for a patient.  Doctors can suggest treatment options or combinations for "Virtual Trials" and track the outcomes.
    Nonprofits can use the system as part of their own patient navigation system - to get a scientific way of offering treatment options.
    Pharma and Biotech can use the system in many ways - as a synthetic control group for single arm trials, to run expanded access programs in a way which generates real world data,  to allow our learning system to figure out the best ways to use their drugs alone or in combination with off label treatments - perhaps find new indications for their drugs, and even to run prospective decentralized or observational trials.
    These services are currently free to all patients, doctors and nonprofits, but the Pharma and Biotech companies pay to support allowing us to offer the services to others for free!  Note that there is an ethical wall between the scientists who generate the treatment options and the business people, so we will suggest options only based on merit.

  • Using Virtual Trials to Screen for Potential Glioblastoma Therapies        

     These are my thoughts on the fastest way to screen new combination therapies for brain tumors!

  • Chimerix Announces Positive ONC201 Data in Recurrent H3 K27M-mutant Diffuse Midline Glioma to Be Presented at the Society for Neuro-Oncology Annual Meeting        

    For this type of tumor, recurrent H3K27M mutant  Diffuse Midline Glioma, there are no good historical controls - as it was only recently created as a new classification of tumor.  It is thought to be worse than a regular Glioblastoma. Historically, recurrent glioblastomas has an average survival of 6 months (which can be read as 50% survival at 6 months)  and we would expect recurrent H3K27M mutant  DMG to be less than that.  This report showed that with Onc201, 57% of patients were alive at 12 months and 35% alive at 2 years – more than doubling what I feel is the correct control.   Objective response rates are hard to determine in this area - but they reported 20% response rate including one complete response, using one grading system, and 26% using a different grading system.   Doesn't sound like much but there is nothing that has been working for this tumor type.. the jump from 0 to 20 or 30% is major, and I think should be good enough to get FDA approval. Once it gets approved, we can try combinations of treatment and find the best way to use it and significantly improve those numbers. The DMG-ACT trial recently opened which will try a few combinations.

    We funded and helped run the first expanded access program for Onc-201 along with Cancer Commons, xCures, The Cure Starts Now foundation, the Michael Mosier Defeat DIPG Foundation, Dragon Masters Foundation and the Finn Family Foundation.  Some of the patients from our program were including in this analysis.  They included the first 50 patients who met the inclusion criteria from all of the trials and the expanded access program! This is your donations at work!

  • Reproducibility of clinical trials using CMV-targeted dendritic cell vaccines in patients with glioblastoma        

     This is data from three vaccine trials for Glioblastoma, and they all show about the same effectiveness: 5 year survival rate of about 33-36%. Historically, the 5 year survival of glioblastoma is about 4%. This is a major improvement. As I mentioned in a few recent articles, I beleive the ultimate cure is going to be a combination of treatments. If we could get 35% survival at 5 years with this vaccine, then maybe add Optune, which alone -if used above 90% of the time - can get another 30%, perhaps these would be complimentary and add up to 65% 5 year survival.  Maybe add in a checkpoint inhibitor, oncolytic virus, Car-T cell, or a targeted therapy or 2, and we could get to a cure! Our patient navigation system is designed to test and track combinations like this - but it will only work once they are approved. Otherwise, it is too hard to do these combinations. Which is why I keep saying we need the promising pathway act! 

  • Safety and efficacy of PVSRIPO in recurrent glioblastoma: long-term follow-up and initial multicenter results        

     This is the "Duke Polio Vaccine".  For recurrent glioblastoma, they reported 1 year survival rates of 54% for 1 trial and 50% for the other trial.  This compares nicely with historical controls of 35%. Survivals were about 12 months, compared to historical control of 9 months. It is very hard to improve the outcome in recurrent glioblastoma. These results are meaningful.

  • DNAtrix Announces Oral Presentation of Positive Overall Survival Data with DNX-2401 in DIPG at the Society for Neuro-oncology (SNO) Annual Meeting        

     DNX-2401 is an experimental  oncolytic virus in trials for adult and pediatric brain tumors. They report promising results in a small DIPG trial:  25% response rate (using RAPNO) with tumor reductions in 75%.   Under RAPNO guidelines, the tumor has to shrink 25% in DIPG to be considered a "response".  (This is 50% in other types of brain tumors). So 1/2 of cases had some tumor shrinkage but did not hit the 25% reduction level. This is still very good. It is very hard to get a response in DIPG.  Median survival was 17.8 months with 3 (out of 12) patients still alive at the cutoff date. Historical survival rates are about 9-12 months for DIPG so this compares favorably.

    My thoughts: this will be very useful, but by itself is not enough. I feel the same way about Onc-201.  We need to get these treatments approved by the FDA so we can try combinations - that is how we will find the cure.

  • First experience with maximal safe resection and Gammatile brachytherapy as treatment for recurrent glioblastoma        

     Very impressive results with Gammatile for recurrent Glioblastoma. This is an FDA approved treatment that is widely (although not everywhere yet) available.  If you are going to have a brain tumor surgery, ask your neurosurgeon about it.  Surgery by itself doesn't really prolong survival much - adding Gammatiles at the time of surgery may significantly extend survival! We recently did a webinar about this: https://virtualtrials.org/video2021.cfm?video=202101

  • Plus Therapeutics Announces Positive Interim Data from ReSPECT-GBM Phase 1 Clinical Trial at the 2021 Society for Neuro-Oncology Annual Meeting        

    This is a new way of delivery radiation to the tumor using nanoliposomes and Rhenium-186. Visit their website https://www.plustherapeutics.com/ to see how it works.  This phase 1 trial had impressive results: It was a dose finding trial. Those at the highest dose did not have any dose limiting toxcicities and 7 of 13 recurrent glioblastoma patients are still alive at an average of 453 days. This compares very favorably to history which tells us these patients live about 6  months (183 days). The trial for recurrent glioblastoma is still open, and they will soon start trials for pediatric brain tumors and leptomeningeal metastases.

  • France allows experimental brain cancer treatment free of charge        

     This is the drug Onc-201 which we have been writing about a lot. It is for H3K27M mutant brain tumors, usually found in pediatric DIPG, DMG and some Glioblastomas (check your pathology report).  A recent presentation at SNO reveals that for recurrent DMG, it helped at least half of the patients, with 20-30% having shrinkage of the tumors. It doesn't help everyone but by itself I have seen it perform miracles on a few kids. 

    France now offers it for free to patients who need it.  There are clinical trials going on involving this drug, and there is an expanded access program for it in the USA.

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The article commentaries are the opinions of Al Musella, DPM and do not represent the official position of the Musella Foundation. Copyright 1992-2021 Musella Foundation - All rights reserved. No part of the Brain Tumor News Blast can be reproduced without the express written permission of the Musella Foundation.