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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02/21/21 The addition of chloroquine and bevacizumab to standard radiochemotherapy for recurrent glioblastoma multiforme        

 It is a small study so needs to be repeated but the results are very impressive.  Adding Chloroquine and Avastin to re-radiation for recurrent Glioblastoma more than doubled the survival in the control group.  This is an easily available drug and more research needs to be done on this combination.

02/20/21 Phase I trial of intranasal NEO100, highly purified perillyl alcohol, in adult patients with recurrent glioblastoma        

 Although the results were only good but not remarkable, it is a simple and well tolerated. Perhaps it could be added to other treatments as part of a cocktail approach.

02/19/21 Setting international standards in analyzing patient-reported outcomes and QOL endpoints        

 Patient reported outcomes should be a part of every clinical trial. Makes sense to have standards so they can be compared across trials and accepted by the FDA as endpoints for approving new drugs.  A good example of the need is a recent trial where the number of Glioblastoma patients working their normal job was way higher than expected, but the median survival did not change so the trial was deemed a failure.  The ability to be able to work at my normal job may be more important than just survival, especially if "surviving" means being bedridden.  Quality of life is of utmost importance and was never really taken seriously until recently because until recently nothing preserved quality of life for these patients.

02/16/21 Are you Considering Optune Therapy for GBM?        

 This is a market research study to look at the decision making involved when considering Optune. They are looking for patients who were prescried Optune but did not yet decide if they want to try it.  There is a payment for your time, and the Musella Foundation will get a small donation.  Let me know if you participate in the phone interview!

02/16/21 New zebrafish model could help advance glioblastoma multiforme research        

 Exciting research done in a zebrafish.  This model can mimick the way Glioblastoma cells spread in the brain of humans, and they are working on ways to stop the spread of the tumor. 

02/16/21 Brain tumor study reveals surprising gene deletion and method to overcome drug resistance        

 I love the thought process involved. They looked at how the tumor becomes resistant to treatment, and then found a pathway the tumor uses to get around attempts to stop the resistence.  Hopefully this will be tried in humans soon.

02/15/21 The effects of releasing early results from ongoing clinical trials        

 This article discusses the effects of releasing ongoing data from clinical trials on the trials themselves.   Obviously it helps the trial if the data is good and hurts the trial if the data is bad. What the article doesn't discuss is the effect on the patients.   If patients knew which trials were better than others, they would only go to the better trials. Maybe the worse trials should shut down and reformulate in a better way.   Our patient navigation program is one way to find the better trials. We have over 700 patients in our registry and we see what treatments they are doing (including trials) and the outcomes.  We help guide the patients to the better treatments.

02/12/21 Sydney researchers identify/advance potential revolutionary new drug treatment for fatal childhood cancer        

 The Musella Foundation helped fund this project through the DIPG Collaborative.  Sounds exciting but it is early and more research needs to be done. Will be keeping an eye on this!

02/09/21 New combination treatment approach improves overall survival in mice with glioblastoma        

 This is only in mice, but it is very interesting.   Until recently, very little work has been done with dopamine receptor antagonists and brain tumors.  They used the drug Quetiapine, which is approved to treat schizophrenia, bipolar disorder, and depression, along with Lipitor, which is used to treat high cholesterol.  The great thing about this research is if they find it does work in people, it is easy to get access to these drugs and it can make a difference immediately.  Of course, human testing is needed first.

On a related idea - the drug Onc-201 is a powerful dopamine receptor antagonist and is known to penetrate the blood brain barrier. There is a trial of using it at the same time as radiation in kids with DIPG. Wonder if adding Lipitor might be worth it. 

02/06/21 GammaTile webinar posted to our video library!        

 They present very impressive results -  anyone who is about to have a surgery for a brain tumor should ask their neurosurgeon about this. It is not yet available everywhere so you might have to ask your neurosurgeon if he has access to this treatment. The webinar shows where it is available at the time of the webinar, but check the website for a current list. You can also request that your surgeon make this available for you.

02/06/21 Amazon Smile Fundraiser! Action Required!        

For those participating in this fundraiser for us using the Amazon mobile app, you need to turn it on again.  Every 6 months Amazon shuts off the donation program and you have to turn it on again. It is easy and free - see instructions below.

If you participate via the website, no further action is required - just continue to use!

And if you do not participate but use Amazon a lot, please sign up for the program. It doesn't cost anything to you and it helps us raise badly needed funds! Just go to and select "Musella Foundation" as your favorite charity!


02/04/21 Musella Foundation awards two brain tumor research grants!        

 These 2 projects are exciting. The first is looking to see if a new drug crosses the blood brain barrier and works in tumor models. If it does, they will follow up with a human trial.  The second helps fund the Children's Brain Tumor Network.  We are a foundational partner of the CBTN now.  This collaboration will speed up the pace of research, making it possible to do things we never imagined! I think this will speed up the search for the cure. 

02/02/21 First Glioblastoma Patient Dosed in Phase 2 Trial Testing PVSRIPO Plus Keytruda        

 This combination makes sense and it is worth looking at this trial:  

It is for patients with recurrent Glioblastoma.

02/01/21 Very Low Mutation Burden Is a Feature of Inflamed Recurrent Glioblastomas Responsive to Cancer Immunotherapy        

This reports the opposite of what we thought regarding tumor mutational burden.  A high tumor mutational burden in other cancers would make the tumor more likely to respond to immunotherapy but that did not work out with Glioblastomas.  This study says that a very low tumor mutational burden for glioblastomas results in a better response with immunotherapies. They also release some new data from the PVSRIPO trial showing impressive results for recurrent Glioblastoma patients with less than the median tumor mutational burden.  Of 21 patients in the trial, 10 in the high TMB group and 11 in the Low TMB group, at the 3 years point after treatment 3 of the patients in the low TMB group were alive, while none in the High TMB group were alive.  These 3 patients were still alive at the end of the trial, one at 36 months, one at 60 months and one at 96 months!  It is a small number of patients, but I would consider this trial if I had a low TMB.  TMB is usually reported on your pathology report.

01/29/21 Mebendazole and temozolomide in patients with newly diagnosed high-grade gliomas: results of a phase 1 clinical trial        

 It is difficult to interpret the results because there was a mix of anaplastic astrocytoma and glioblastoma, and they did not disclose the MGMT methylation status, the IDH status ir if they patients used Optune or any other treatment. However, they have shown that adding Mebendazole is safe, and it appears that the outcome is a little better than Temozolomide alone.  This is a pretty popular drug for Glioblastoma patients to take. It is part of the Care Oncology Protocol, which also adds 3 other repurposed approved drugs. The Care Oncology protocol has some imnpressive data behind it.

01/27/21 Imvax Announces Publication of Phase 1b Clinical Trial of IGV-001 for Patients with Newly Diagnosed Glioblastoma in Clinical Cancer Research        

 Impressive results for a phase 1 trial.  The link to the journal article is in the press release.

01/27/21 Learn More About GammaTile® Therapy, Surgically Targeted Radiation Therapy For Brain Tumors, At Patient Education Event On Thursday, February 4        

 GammaTile  is an FDA approved treatment for brain tumors that is implanted at the time of surgery (both newly diagnosed or recurrent) and can double the time before the tumor grows, which buys time for other treatments to work.

It is not available everywhere just yet, visit their website to see which centers are using it now and ask your neurosurgeon about it.

This free webinar will go into detail about it.

01/23/21 Targepeutics Receives Orphan Drug Designation for Lead Drug Targeting Malignant Glioma        

 This is a very interesting drug. It is a smart bomb. It seeks out IL13Ra2 which is a cell surface protein present on many types of cancer cells (including Glioblastomas, and DIPG), and rarely found on normal cells. It carries along a pseudomonas exotoxin that kills the cells that the drug attaches to. The net effect is that it kills tumor cells without harming the normal cells.  The Musella Foundation has given a few grants to support the creation of this drug over the last 11 years and I am so happy to see it has received orphan designation. That will make it easier and cheaper to  bring it to patients.

01/23/21 Outcomes and Patterns of Care in Elderly Glioblastoma Multiforme Patients: A Population-Based Study of the National Cancer Database        

Outcomes and Patterns of Care in Elderly Glioblastoma Multiforme Patients: A Population-Based Study of the National Cancer Database         One of the key findings is that only 42% of the elderly patients in the USA with Glioblastoma used any chemotherapy compared to 63% of younger people. Same problem with radiation and surgery, all 3 of which were shown to increase survival time if used.   They found the average survival time was only 9.1 months. We need to figure out why these people did not receive treatment.  Cost may be a factor.  The first Temodar prescription usually costs in the neighborhood of $8,000 with a Medicare copayment of about $1,000. Many people refuse to buy the Temodar due to the cost. The manufacturer of Temodar closed their assistance program a long time ago and right now we - the Musella Foundation - are the only copayment assistance program open that can help Medicare patients with their copayment for Temodar (and Optune and Avastin). (Others open and close but none are open now).  We helped over 1,500 patients pay for the medications they need. I am sure this has helped these people live longer. Having said that, we need to change the laws so people who have health insurance do not have to go without medicines due to their cost.

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