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Brain Tumor News!

Note: The comments under each article title are the opinion of our president, Al Musella, DPM,
and do not reflect official policy of the Musella Foundation!
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11/25/21 Targeted Drug Combo Shows Unprecedented Activity in Some Aggressive Brain Tumors        

 This is significanct because it is the first time a clinical trial ever reported success for Glioblastomas using targeted therapies.  This is only for patients with a rare mutation known as v600E in the BRAF gene.   The drugs  are approved for other diseases so can be used off label right now:dabrafenib and trametinib.  So check your pathology report - no matter what tumor type you have - for this mutation.

We have been using this combination for patients in our patient navigation program and have seen some good results!

11/24/21 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!

11/24/21 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.

11/24/21 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!

11/24/21 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.

11/22/21 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!

11/22/21 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! 

11/22/21 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.

11/22/21 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:

11/22/21 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.

11/22/21 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 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.

11/18/21 Presentation of XCELSIOR Observational Protocol for Real World Data Research        

 This is the future of medicine.  All patients should be followed as if they are in a clinical trial. This would allow researchers to figure out the best treatment options and find what works, and for whom. It would allow for the creation of synthetic control groups that are better than randomization for a trial, since ALL of the prognostic factors can be accounted for.   The free service will help practicing brain tumor doctors figure out which mutations are the most important and which drug combinations or clinical trials might be best for your patient.  

11/15/21 2-THE-TOP Phase 2 Trial Reports Positive Top-Line Results in Newly Diagnosed Glioblastoma        

 Very good results in a new Optune plus Pembrolizumab (Keytruda) trial for newly diagnosed glioblastoma.  They reported median progression free of 11.2 months compared to the original Optune trial without Pembrolizumab which had 6.7 months median progression free survival. The trial for Pembrolizumab alone for recurrent Glioblastoma only had a 2.8 month progression free survival.    This shows that Optune has an immunogenic effect, which the Pembrolizumab magnified.    Hopefully we will see an even better result when they start adding a vaccine to the mix!

11/15/21 Novocure Announces 31 Presentations on Tumor Treating Fields at Society for Neuro-Oncology 2021 Annual Meeting        

 SNO is the big annual brain tumor meeting which is next week.   Novocure will have 32 presentations which is amazing. They are split between clinical and preclinical and we learn better how to use Optune with each presentation.  I will report after the conference on which have the biggest impact!

11/13/21 xCures presents the results of their oncology platform at the 2021 SNO Annual Meeting        

The presentation title is: XCELSIOR: A real-time, real-world learning platform for patients with advanced cancer.  This is part of our patient navigation program. The idea it to create a learning system where a team of neuro-oncologists, PhD research scientists and nurse navigators - aided by an artificial intelligence engine - can learn from the experiences of every patient.  The registry tracks the outcomes of every patient - so we know what is being used and how it is working out. The system can easily track trials, standard treatments, off label, expanded access, and right to try treatments as well as the new combinations being used by doctors around the country and identify which are doing the best. I feel this is the fastest way to home in on the cures! You can participate by going to our patient navigation program

Disclaimer: I am proud to say I am a founder of xCures, own stock and am a paid consultant to their braintumor program!

11/13/21 Disparities in access to surgery for glioblastoma multiforme at high-volume Commission on Cancer-accredited hospitals in the United States        

 The shocking part of this article is that only 10.7% of glioblastoma patients get surgery at a high surgery volume medical center. It has been shown that patients who have surgery at these high volume centers do better than those that get surgery at low volume centers.   There is a high bias against the poor, minorities and those with bad or no insurance.

This shows why our patient navigation program is so important.  

For the vast majority of brain tumor patients who get treated at smaller medical centers, it is absolutely imperative to get other opinions - like our patient navigation program. There are so many treatment possibilities now that it is very difficult to stay up to date on them all. 53 new cancer drugs were approved in the last year alone.  Any one of them can be used off label if you have the right genetic mutations.  Our team of neuro-oncologists and PhD researchers track them all and can help match you to the best drug combinations.

11/13/21 Targeting glioblastoma signaling and metabolism with a re-purposed brain-penetrant drug        

 This is very good news!   The drug Prozac, which is an old drug approved for the treatment of depression, OCD, Bulimia and panic disorders, has been shown in mice to help brain tumors!  They did a unique type of research to show that it helps patients with glioblastomas:  they looked at medical records to see which glioblastoma patients also happened to take Prozac (or it's generic equivalent), and how they did.

There was an increase in median overall survival of glioblastoma patients who also took Prozac of 545 days with Prozac and 318 without Prozac, This is a 71% increase in survival just by adding a repurposed drug, which has the side effect of helping with depression and mood!

The study is too small and not the gold standard randomized phase 3 but the drug is relatively innocuous, cheap, easy to get. Might be worth asking your doctor about it.   

11/11/21 PNOC Study in Childhood Brain Cancer Enrolls First Patient        

This trial is for diffuse midline glioma and DIPG in patients 2-29 years old. They can be newly diagnosed or recurrent. It tests the drug Onc-201 with either Paxalisib or Panobinostat.   Onc-201 by itself has the best effect on these tumor types so far, but not good enough. Hopefully adding another drug will make them work better, and this is a great way to test the combinations. Nobody gets placebos, they are testing what is thought to be the two best drug combinations against each other.

11/11/21 Chimerix Announces Upcoming Presentations at the Society for Neuro-Oncology Annual Meeting 2021        

 We will be following this closely at the meeting and I will let you know the details after the meeting!

11/11/21 Temporary opening of the blood-brain barrier with the nitrone compound OKN-007        

 This drug should help most other treatments work better. The Musella Foundation gave a research grant to get this drug started a few years ago!  Your donations at work!

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