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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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01/24/22 Mark Cuban Cost Plus Drug Company's Online Pharmacy Launches with Lowest Prices on 100 Lifesaving Prescriptions        

 Take a look at this website. Mark Cuban created the company to sell generic drugs at his cost.  A few of the drugs are used by brain tumor patients. For example, Generic Gleevec which usually cost $2,500 for 30 tablets is being sold for $17.10 (plus shipping).  Keppra XR which usually sells for $94.65 is available for $6.30.     Zofran which is usually $160 is now $6.30.   They have 100 different medications.  They do not take insurance but this is usually way less than you would pay for a copay. Check all of your drugs. I found a few that I use and will give it a try.

I already sent a request for them to add Temozolomide and Lomustine.  I did not hear back yet - but it couldn't hurt if a LOT of people requested those drugs! Go t their website and use the contact form!

01/22/22 Results of a post hoc analysis of the EF-14 study showed Optune + TMZ was associated with increased median OS vs TMZ alone in patients ineligible for surgical resection        

 They went back and analyzed the results of the big EF-14 clinical trial for Newly Diagnosed Glioblastoma patients of Optune plus Temozolomide vs. Temozolomide alone, broken down by extent of surgery. They divided the patients into 3 groups, those with biopsy only, those with a partial resection and those with a total resection of the tumor.   They found that in all 3 groups, there was a significant survival advantage to Optune.  The survival times I talk about are from the start of the trial, which was about 2-3 months after initial diagnosis. For the Biopsy only patients, average survival with Optune was 16.5 months compared to 11.5 months without Optune.  This is a 43% increase in survival time. This might not sound like much but now that we have so many other options to add, this 43% increase can buy the time needed to try other treatments which may synergize with Optune. 

For the patients with a partial resection, the outlook is somewhat better, 21.4 months with Optune vs. 15.1 months without - a 41% increase in survival. And finally, for those with a complete resection, the median survival with Optune was 22.6 months vs. 18.5 without Optune, for a 22% increase in survival.

01/20/22 Prolonged response of recurrent IDH-wild-type glioblastoma to laser interstitial thermal therapy with pembrolizumab        

 This is only a report on 3 patients and needs more research but looks promising. It makes a lot of sense to use this combination. LITT is an fda approved treatment for brain tumors that is sometimes an alternative to open surgery. It is a minimally invasive surgery where the surgeon makes a small drill hole in the skull and inserts a fiber optic cable that delivers laser energy to kill the tumor. They follow the progress inside an MRI machine. This causes the release of tumor specific antigens into the environment. Your body can then mount an immune response to the tumor and possibly stop the tumor from recurring.   

Pembrolizumab is an immune checkpoint inhibitor which takes the brakes off of the immune reponse.  By themselves, they do not do much with  glioblastoma.  However, there was an earlier report that if you start the Pembrolizumab before a surgery and continue after the surgery, it works much better than starting after surgery. The thinking is that the surgery exposes the tumor specific antigens and the Pembrolizumab revs up the immune system response to that. Without the priming of the immune system, the Pembrolizumab has nothing to work on.  This article talks about the same effect with LITT.  

In the future, we need more work on additional components of a cocktail - perhaps add Optune after the LITT which can continuously kill tumor cells slowly over time, which can prime the immune system, and add Pembrolizumab to enhance that effect.

01/20/22 Vigeo Therapeutics Advances VT1021 Into Phase 2-3 Registrational Study for Glioblastoma        

 VT1021 is an experimental treatment for brain tumors.  Follow the link below to see the mechanism of action and the impressive results of the initial trial. The GBM AGILE trial is the most advanced clinical trial system. It is a learning system where multiple treatments are evaluated against each other.

01/13/22 Systemic high-dose dexamethasone treatment may modulate the efficacy of intratumoral viral oncolytic immunotherapy in glioblastoma models        

 This study shows that (at least in the mouse model used) using high doses of steroids reduces the effectiveness of the immunotherapy.    We knew that already. This study confirms the magnitude of the effect and it is large.  This brings me back to why we need the Promising Pathway Act. I keep talking about this but can't get the help we need to get it passed.  In this case, there was a drug in trials many years ago called Xerecpt.    It was to treat swelling from brain tumors, without the side effects of steroids. Early testing looked very good but it failed it's pivotal study.  Only a few patients were able to come off steroids entirely, but all were able to greatly reduce the dosage of their steroids.  At the time, it did not seem worth pursuing it as an alternative to steroids if it still required use of a small amount of steroids. But now with this current study, we see a great value in being able to reduce the dosage of steroids. It will make the immunotherapies work better. And it will greatly reduce the side effects of steroids.  I hate to see pictures of little kids with brain tumor who get bloated from steroids - as well as problems sleeping, increased chance of infections, irritation to stomach, increase in blood sugar, muscle wasting and weakness, skin thinning, emotional problems and eye problems.  As if they did not have enough problems with the tumor. Under the Promising Pathway Act, we could get drugs like this (both Xerecept and the immunotherapy mentioned) approved and try them in combination with immunotherapies and get better results and less side effects. 

01/07/22 Extent of MGMT promoter methylation modifies the effect of temozolomide on overall survival in patients with glioblastoma: a regional cohort study        

 This is a complicated study to read.  The shocking data to me is in the supplemental materials.  These show how few patients - especially the elderly - get the standard Temodar regimen.   Over half of MGMT unmethylated patients do not even get Temodar at all. Only 26% of MGMT methylated patients complete the standard schedule of Temodar and 35% do not even get any Temodar.  They did note that if you do complete the entire course of Temodar (which they say is 6 weeks during radiation then 6 months after), you have a much higher overall survival - 24.7 months in unmethylated and 36.3 months for methylated patients.   I do not know if that means there is a benefit of Temodar to unmethylated patients, or just that patients in good enough shape 6 months after radiation to take Temodar will just do better in general. Bottom line - the old standard of Temodar and radiation (without Optune)  is not really good enough and we need to find better ways forward.

01/07/22 Adult H3K27M mutated thalamic glioma patients display a better prognosis than unmutated patients        

This is in adults only.  Unfortunately, the median overall survival is only 15.9 months - worse than a Glioblastoma.

01/06/22 DIPG All-In-Initiative awards $35,000 grant for pediatric brain tumor research!        

 Although this is for pediatric DIPG, if it works, it could be used for any type of brain tumors!

01/04/22 Musella Foundation Copay Assistance Program is now open and another drug added to coverage list!        

 We received generous donations and are able to reopen the program!  Do not feel bad about asking for help. That is what we are here for. We understand what you are going through and want to help. If you could use the help - apply!

01/04/22 Efficacy and safety of bevacizumab combined with other therapeutic regimens for treatment of recurrent glioblastoma: A network meta-analysis        

Bevacizumab is an FDA approved treatment for Glioblastoma, which inhibits VEGF (Vascular endothelial growth factor).  By itself, for glioblastomas,  it did not increase overall survival, but it did make people feel better for a longer time - with an increased progression free survival.  It is thought that combining it with other drugs is more rational than using it by itself if the purpose is to extend life.  (It can be used by itself as a super steroid to get rid of swelling). This study reviews the medical literature and reports on which combinations did the best.  Unfortunately, the best combination they found was Bevacizumab plus Rindopepimut. I say unfortunate because Rindopepimut is no longer available.   It is a therapeutic vaccine against EGFRvIII.  It did very well in early trials, but in a large randomized trial, it did not do better than the control group - which was an immune enhancer that is also part of Rindopepimut.  Both groups did better than expected by historical controls, but the way the  trial was designed  resulted in failure and they no longer make the drug.

IF the Promising Pathway Act ever passes, there is a possibility of going back and reviving some of these treatments that "failed" even though they had good results in some patients and did what they were supposed to do. In this case, Rindopepimut was supposed to inhibit EGFRvIII and it did. It helped a little but not enough to get approved by itself. It's real strength would have been as part of a cocktail approach as mentioned in this current study where it says adding Bevacizumab to Rindopepimut resulted in the best results.  I think we need 4 or 5 drug combinations to hit the home run, but to get that far we need access to the components!

01/04/22 Drug Resistance in Glioma Cells Induced by a Mesenchymal - Amoeboid Migratory Switch        

 We (The Musella Foundation with the help of our fantastic donors!) helped fund this research project.  They found that just adding a drug that inhibits the main way the Glioblastoma cells move did not really help much. In fact, they found that the cells easily and quickly used a different mechanism to move.  Combining inhibitors of both pathways did help. A lot, but not completely -  a 3rd drug might be needed to target the resistance pathway.  They did find a biomarker in the blood that may predict recurrence of Glioblastoma. More work needs to be done to validate the biomarker and to completely eliminate invasion! Good work!

01/03/22 Combination therapies for pediatric brain tumors        

 This is one of my favorite webinars. Dr Dunn explains his thoughts on how to find the best combinations of treatments for pediatric brain tumors. 

Well worth watching.  It is a little technical - it is like an entire high level college course compressed into an hour or so, so if there is anything you do not understand, we can discuss it in our facebook group (link in the article!)

01/01/22 Musella Foundation awards $25,000 brain tumor research grant!        

 This is an exciting project with potential for a breakthrough in the treatment of Glioblastomas.  This experiment is in mice and if successful will be quickly translated into a human trial.  Thanks to the donors who made it possible for us to fund this grant and our medical advisory board who worked over the holiday to get it approved quickly!

The drug is in clinical trials by itself now - delivered at the time of surgery for recurrent Glioblastoma.

12/30/21 A word about donations...        

 First - a thank you to the many people who have donated!    We had a bad year with fundraisers and would like to ask you to get your friends and families to make a donation!

12/30/21 Reminder - Brain Tumor Webinar tonight! 7pm Eastern        

 This is a very important webinar - not just for patients and families - but brain tumor doctors and researchers will be interested in this one.   It may change the way we approach the treatment of brain tumors!

12/30/21 Musella Foundation Copay Program now closed to new and renewal patients        

 We have run out of funding and the copay program will remain closed until we get enough donations to the program to reopen.    You can make a donation at and specify it is to be used for the copay program if you want to help us reopen the program!  Note that only donations that are specified for the Copay program can help us reopen the program. If no usage is specified, 100% of donations go to brain tumor research!

Note - the program actually has 3 spots open right now  - if you already started filling out the application, send it in today. We will process them in order until we run out of money.  They will be filled by tonight.

12/30/21 The Impact of Tumor Treating Fields on Glioblastoma Progression Patterns        

 This study shows that the tumor recurs further away from the primary tumor when using Optune than without Optune.  The significance of this is that the new tumor grows in areas that have a low dose of tumor treating fields. First - this proves that Optune has an effect - it prevents the regrowth in areas of high field strength.  However, it also points to the fact that other treatments are needed along with Optune to prevent recurrences - or the arrays have to be aimed differently to cover the areas more likely to get a recurrence.  

12/29/21 Important Brain Tumor Webinar Thursday Dec 30, 2021 7pm Eastern Time        

 This is one of our most important webinars!  Dr. Dun explains his ideas on how to put together a cocktail approach in the most scientific way. He focuses on a tumor called pediatric diffuse midline glioma, but the methodology should apply to any type of brain tumor (or any cancer for that matter).   I think that these methods used will lead us to the right cocktails quickly!   

12/29/21 Armodafinil Does Not Reduce Cancer-Related Fatigue in High-Grade Glioma        

They found that 96% of patients with high grade glioma sugger from the side effect of moderate to severe fatigue. Unfortunately, this randomized trial failed to find any benefit for the drug Armodafinil (Also known as Nuvigil). 

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