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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02/27/24 Impact of Reirradiation Utilizing Fractionated Stereotactic Radiotherapy for Recurrent Glioblastoma        

 This doubled survival for recurrent glioblastoma and is easily available. Worth considering.



02/25/24 Promising Pathway Act Open House in Washington, DC This Tuesday!        

 We are planning to introduce version 2.0 of the Promising Pathway Act sometime next week. This open house is for patient advocates to hear about the act and to network with other groups. If you are in the area, we need people to show up to let them know we support this bill!  



02/25/24 Theranostic Uses of the Heme Pathway in Neuro-Oncology: Protoporphyrin IX (PpIX) and Its Journey from Photodynamic Therapy (PDT) through Photodynamic Diagnosis (PDD) to Sonodynamic Therapy (SDT)        

 Excellent paper on sonodynamic therapy - the history, mechonism of action, current trials  and future prospects. The authors discuss all of the available devices - not just their own.    I beleive this therapy is going to be  a major breakthrough in the treatment of brain tumors. It is very elegant, non-invasive, early indications are that it is safe and well toerated, and it can be repeated as often as needed.



02/24/24 Biodexa Reports Positive Phase 1 Trial Results For Diffuse Midline Glioma        

   This study showed that it is relatively safe and feasible to do repeated convection enhanced delivery of drugs to the brain - particularly the pons.  The drug they used, MTX110 is a nanoparticle formulation of Panobinostat.  This was a dose escalation study - so most patients did not get an effective dose - but it still showed an improvement in survival compared to what we know about the outcome of this disease.



02/23/24 New GT Medical CEO Per Langoe on driving adoption of brain tumor therapy        

 There is a problem with slow adoption of new treatments.  I was taught to never be the first one to prescribe a new treatment, or the last one to prescribe a bad treatment.    That is ingrained in most doctors, but it doesn't apply when dealing with a deadly disease.  You need to take the risk / benefit ratio into account.   For something like gamma tiles, which have shown very impressive results with glioblastomas as well as metastatic tumors, we do not have the time to wait. Patients who need brain tumor surgery need to ask their doctors if it GammaTile is appropriate for thier case.  Check the list of doctors who are trained in the use of GammaTiles at https://gtmedtech.com/gammatile-centers/   If your doctor is not on the list and says they are not appropriate, get a quick second opinion from one who is on the list.  It is worth checking. 



02/23/24 Tumor Treating Fields (TTFields) combined with the drug repurposing approach CUSP9v3 induce metabolic reprogramming and synergistic anti-glioblastoma activity in vitro        

I love this combination.  We usually recommend the CUSP0v3 protocol as an option to patients and usually suggest adding Optune to it.  This study confirms that the combination makes sense.  We do not have enough patients using this combination yet to tell how well it works but will be watching it! So if you are thinking of trying it, participate in our patient navigation program / registry so we can track it! Go to https://virtualtrials.org/xcelsior.cfm to get started!



02/21/24 Promising Pathway Act Open House in Washington, DC This Tuesday!        

 We are planning to introduce version 2.0 of the Promising Pathway Act sometime next week. This open house is for patient advocates to hear about the act and to network with other groups. If you are in the area, we need people to show up to let them know we support this bill!  Note: A previous version of the invitation had the wrong date. It is Feb 27, 2024.



02/21/24 Sonodynamic Therapy Webinar added to website!        

 Sonodynamic therapy is one of my favorite new treatments.  It is very elegant in how it works to noninvasively target cancer cells while minimizing damage to the normal cells. Early results look very good. Keep in mind the first few patients presented had a low dose and few treatments.  It will get better and better as they learn how best to use it!

We have the ability to speed up the process of making this available - it is a matter of money - but in this case a lot of money.   So as the speaker mentions in the talk, if you have any interest and ability to make either a large ($1-5 million) donation or investment in the company, contact me!



02/20/24 'Olive oil' drug shows promise in treating aggressive brain cancer at London hospital        

 Very small trial but interesting results.  



02/20/24 FDA and EMA Accept Vorasidenib Regulatory Submissions for the Treatment of IDH-mutant diffuse glioma        

 Vorasidenib did very good in trials for IDH mutant gliomas and have now applied for FDA approval. We should hear the decision by August!



02/20/24 Webinar on Sonodynamic Therapy For Brain Tumors this Wednesday 2/21/24!        

Sonodynamic therapy is an exciting, non-invasive experimental treatment for brain tumors. I think it will be shown to be a breakthrough and will significantly impact this disease. This webinar is at a different date and  time than usual - we wanted to get it to you as soon as possible!  



02/19/24 OurBrainBank Launches Nationwide Survey to Address Healthcare Disparities in Glioblastoma Care        

 From our good friends at Our Brain Bank.  



02/14/24 IN8bio Announces Publication on Novel Gamma-Delta T Cell Therapy for Glioblastoma in Frontiers in Immunology        

 Interesting new approach to treating glioblastoma. Very early results look good! Will keep an eye on this.



02/11/24 Webinar on Sonodynamic Therapy For Brain Tumors this Wednesday!        

Sonodynamic therapy is an exciting, non-invasive experimental treatment for brain tumors. I think it will be shown to be a breakthrough and will significantly impact this disease. This webinar is at a different date and  time than usual - we wanted to get it to you as soon as possible!  



02/09/24 Brain cancer a thing of the past?        
It's far too premature to determine the efficacy of this treatment, as it has not yet been tested in animals or humans. Experience has shown that many treatments effective in lab settings fail in human clinical trials. The purpose of sharing this article is to highlight the development process of a new lab-proven treatment. The next steps typically involve further lab research, animal testing, and the challenging task of securing funding for human trials. Recently, we were approached for a grant to support an early-stage brain tumor treatment, which required a staggering $1 million per patient. Unfortunately, we had to decline due to financial constraints. The plan  was to treat approximately 250 patients in total over the next decade before seeking FDA approval, a venture that no one is eager to invest $250 million in, given the risks and the potential to become obsolete by the time approval is granted.
 
I advocate for the Promising Pathway Act, which offers a more feasible approach. If the drug proves to be highly effective, it could undergo a small phase 1/2 trial with about 25 patients for an estimated $25 million, potentially receiving conditional FDA approval within three years. This would allow the drug to be accessible sooner, with continued research over eight years and costs covered by insurance or out-of-pocket payments. This method not only makes fundraising more manageable but also enables setting a reasonable price for the treatment, recovering the investment without the need for exorbitant pricing. This approach addresses the financial challenges of bringing new treatments to market.


02/08/24 Biodexa Reports 12 Month Survival in MAGIC-G1 Study of MTX110 in Recurrent Glioblastoma Patients        

 This looks promising. Still too early to tell, but early results look good.



02/07/24 Ivy Center Announces First Patient Treated in Targeted Phase 0/1 Clinical Trial for High-Grade Glioma        

 This is a new generation of EGFR inhibitor that gets through the blood brain barrier and targets multiple mutations in EGFR.    Worth looking into if your pathology report showed alterations in EGFR!



02/06/24 Northwest Biotherapeutics Moves From Optimization of Flaskworks Prototype to Fabrication of GMP-Compliant Units For Installation, Validation and Final Testing Prior to Regulatory Certification        
This announcement heralds significant progress in the realm of personalized medical treatments, a field historically challenged by the dual hurdles of scaling production and managing costs effectively. The bespoke nature of custom-made therapies, while groundbreaking, has led to exorbitant prices and a notable scarcity, compelling physicians to allocate these life-saving treatments to only the most critically ill patients. The traditional method of meticulously crafting each treatment individually is far from efficient.
 
However, Northwest Bio's recent breakthrough introduces a game-changing solution. They have developed a machine capable of automating this intricate process, enabling the simultaneous creation of vaccines for multiple patients. Remarkably, this innovation also eliminates the need for the sterile environments mandated by manual production methods. This advancement is poised to significantly streamline the production of DCVAX, when it receives approval, meeting any level of demand with newfound ease.


02/03/24 Copayment Assistance Program update!        

 We increased our income guidelines - see the website for details but for a family of 4, we now allow up to $156,000 in income!   We also take into account if you will make less money due to the tumor.



02/02/24 Australian government steps in to fund experimental brain cancer drug        
 
Stories like this ignite my frustration. The video highlights a young Australian girl battling DIPG (Diffuse Intrinsic Pontine Glioma) who has shown remarkable progress. Her treatment involves a combination of drugs, including ONC-201, for which her family has incurred expenses exceeding $100,000. Fortunately, the Australian government has decided to cover the costs moving forward. The demand for this drug is global, as it has demonstrated potential to significantly extend survival rates and, in some cases, produce miraculous outcomes in a condition where such results are exceedingly rare. The family is also experimenting with other drugs, although specifics are not disclosed.
 
In the United States, access to such treatment combinations outside of clinical trials, which include a placebo group, is virtually non-existent. The urgency for ONC-201's approval cannot be overstated. This underscores my dedication to advocating for the Promising Pathway Act. Passage of this act would not only facilitate access to drugs like ONC-201 but also establish a learning system to expedite the evaluation of drug combinations and optimize their application. Meanwhile, the grim reality is that DIPG claims the life of approximately one child almost  every day in the USA.


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