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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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04/20/19 Cancer and the dopamine D2 receptor: a pharmacological perspective.        

 Interesting article.  They are correct that the D2R receptor family (also known as  DRD2) is very important in many types of cancers, including brain tumors – especially in tumors of the midline and brainstem.

However, they are incorrect when they say there is no useful way to target this. The drug Onc201 is a very selective antagonist to DRD2 and is now in clinical trials for brain tumors.    Although it is too early to tell how well it works, I have seen a few remarkable responses to it already. This article talks about trying to repurpose older drugs that are NOT selective.


Disclaimer:  The Musella Foundation was an early supporter of Onc201 and we are involved in funding the compassionate use program for this treatment.

04/18/19 Liquid Biopsy for Detection of Histone H3 Mutations in Pediatric Diffuse Midline Gliomas        

 This will be very useful as we can now detect this mutation without doing a biopsy, so it can be used not only to direct the original treatment but to monitor how well the treatment is working.

04/15/19 Successful treatment of brain radiation necrosis resulting from triple-negative breast cancer with Endostar and short-term hyperbaric oxygen therapy: a case report        

 This is just one case report so of course you can not say how it well it works but it opens the door for another treatment possibility for those who have symptomatic radiation necrosis that did not respond to current treatment options.

04/15/19 Use of metformin and outcome of patients with newly diagnosed glioblastoma - pooled analysis.        

 This study did not show a significant imporvement in outcomes when using Metformin.  Metformin is a drug used to control high blood glucose in diabetics but has been used off label to try to treat cancers including brain tumors.  More research needs to be done before we write this off.

04/11/19 'I Love What I Do' - Internationally Renowned Brain Surgeon Dr. Linda Liau        

 This is one of my all time favorite brain tumor doctors and proud to say she is a friend. Also an advisor to the Musella Foundation.  She just spent a lot of time helping us update our Guide for the Newly Diagnosed - which should come out next month!

04/09/19 Case Report in the Journal of Neurosurgery Highlights Potential of ONC201 in H3 K27M-mutant DIPG        

Of course a single case report is not proof that a treatment works, but it is great to hear of individual miracles like this.  I know of a few more that are just as miraculous with this drug.   The journal it is published in is one of the most prestigious in the field -  which means by publishing it they recognize how  important an achievement this is.

As noted in the press release, our organization, the Musella Foundation (along with The Cure Starts Now Foundation, The Michael Mosier Defeat DIPG Foundation, Cancer Commons and xCures) has been a supporter of this treatment. We identified this as one of the highest priorities for us to support and we need help raising funds to help speed up this project (and others) so we can help other families have miracles too!

To recap: This experimental treatment, Onc-201, is for tumors that have the H3K27M mutation, which is also sometimes reported as a H3F3A mutation. It is mostly found in younger brain tumor patients, especially with tumors in the midline of the brain, brainstem, thalamus and DIPG.  There is a clinical trial going on now for this treatment but if you do not qualify for the treatment, there is a compassionate use program that might be able to help.  Contact us for details if you have this mutation!




04/09/19 Optune Open Houses        

This is a chance to learn about Optune - or for those of you using it - to meet others using it and swap tips and ask the experts any questions you have!  Most are live but the last one is a webinar.

04/09/19 National Walk To End Brain Tumors        

  We are having a series of 5k fundraisers - visit the website to see Where and When.  If you can't participate in the event, you can still make a donation!


04/03/19 High frequency of H3K27M immunopositivity in adult thalamic glioblastoma.        

We already knew that there is a high frequency of this mutation in the pediatric thalamic tumors but this study shows that 60% of adult thalamic tumors have this mutation.    Glioblastoma in this area have the worst prognosis. This article shows why - all are MGMT unmethylated, and most have the H3K27M mutation.    The good news about all of this is that now we have options that we did not have before:

 For the H3K27M mutation, the experimental drug Onc-201 can be tried. There are trials available for it and if you do not fit the trial, we are involved in a compassionate use program for it - call us.  

 For the unmethylated MGMT, there is a different experimental drug - Val-083 which is in trial that is similar to Temodar but works in a different area of the DNA so it is unaffected by the MGMT status.


04/03/19 Phase 2 study of Val-083 with radiation therapy for newly diagnosed        

Val-083 is an experimental chemotherapy drug that is similar to Temodar but works in a slightly different way so that it is not affected by the MGMT repair enzyme, which means it should theoretically work on a Glioblastoma patient with unmethylated MGMT as well as Temodar works on a methylated MGMT glioblastoma.   This shows early results which show a hint that it does work this way.

04/03/19 New Glioblastoma Vaccine Shows Promising Results in Phase 1b Clinical Trial        

 This is an interesting new type of vaccine.  These are early results but looks good.

03/31/19 Could £400-per-year drug cocktail be the key to beating one of the most-deadly cancers?        

That is $521 US Dollars a year at current exchange rates.   This is a fairly large trial of 100 GBM patients and it shows pretty good results - excellent if you take cost into consideration.  There are a few projects looking at repurposing older drugs or using special diets.  This is an excellent avenue of research but the only way we will home in on the best combinations is a registry that tracks ALL brain tumor patients so we can not only see the effects of combinations, but also see how the standard treatments are doing. The standard treatments have improved a lot recently.   We are launching a new version of our brain tumor virtual trial soon.  Watch for details!

03/31/19 Phase 1 Study Begins Dosing High-Grade Glioma Patients with GMCI Combo Therapy        

 This is a new genet therapy trial for newly diagnosed gbm

03/28/19 New Strategies Take On the Worst Cancer—Glioblastoma        

 This opens another option for people about to have a brain tumor surgery.  This article describes how they give a cocktail of drugs  a few days before the surgery then when they do the surgery they can measure if the drugs got into the tumor and if they have shown efficacy.  If yes, they continue with that cocktail, if not, they switch to something else.    This prevents you from wasting time with an ineffective cocktail.  This team is also open to experimenting with different cocktails based on prior lab results. The article says that this team treats more glioblastomas than any other center in the country!

03/20/19 Pilot Trial of Adoptive Transfer of Chimeric Antigen Receptor-transduced T Cells Targeting EGFRvIII in Patients With Glioblastoma.        

 This is one of the more exciting treatments in the pipeline, but unfortunately this trial did not show good results for most of the patients.  2 patients out of 18 did well.   There are many variations of CAR-T cells, using many different targets. This doesn't mean all Car-t cell trials won't work.

03/15/19 First-Of-Its-Kind Treatment Beats Back Notorious Brain Tumor        

 I love articles like this.  Diffuse midlines gliomas are among the worst of the worst brain tumors.     Of course you can not make decisions based on a handful of case reports but they do give some hope where there really wasn't much before. Your donations have helped fund this - we gave 3 grants to help get this developed and it is in clinical trials, and there is a compassionate use program open.  Ask your doctors about this if you have the  H3K27M mutation (sometimes reported as H3F3A), or a brainstem tumor.  It is most common in younger people with tumors near the midline of the brain.    We will be dedicating part of the proceeds from our National Walk To End Brain Tumors 5k events to this project!

03/14/19 Optune Open Houses        
These are informative and fun meetings. Some of them are webinars available online for those not close to a live meeting.  All are free.  They are sponsored by Novocure and the Musella Foundation is not involved - I am just letting you know about them because they are important!  

03/13/19 Newly Diagnosed Glioblastoma: A Review on Clinical Management        

 Excellent review of glioblastomas and how to treat them.

03/12/19 xCures to Implement an Intermediate Size Expanded Access Protocol for ONC201 in H3 K27M-mutant Gliomas        
This is an exciting experimental treatment for brain tumors with the H3K27M mutation.  This is usually found in children with DIPG, and people with midline gliomas - especially of the thalamus.  There are clinical trials open for it, but if you do not qualify for the trials, you may be able to get it outside of the trial.   There is no cost for the expanded access program because we - the Musella Foundation - along with the other organizations mentioned in the press release - funded it with a $1 million  grant!   Early results presented at the last Society of Neuro-oncology meeting looked good with a few impressive responses, which is rarely, if ever seen in tumors with this mutation.
Disclosure:  I have a small ownership interest in xCures.


03/12/19 Northwell neurosurgeons remove “glowing” brain tumor using breakthrough technology in Long Island first        

 Gleolan is FDA approved - it helps the neurosurgeon know what is tumor and what is not while operating.   Good work to my friend Dr Schulder!

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