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Musella Foundation Logo and Name of Email Blast
Friday, July 30, 2021
Issue 5834
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The most important bill ever introduced into congress for brain tumor patients needs your support!
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  • Help support the Promising Pathway Act        
    We have not had much progress in the treatments of Glioblastomas. Ever.  Of every phase 3 clinical trial that was ever done for Glioblastomas, only a handful were considered a success, and the best of them, the Optune EF-14 trial,  only increased median survival by 5 months.    We tried this methodology for about 70 years and it did not work. Time to try something new.
     
    The current system is too rigid and slow. A trial is designed, then has to run out for 3-15 years to get a result. Then they run another trial based on those results. The cost and time are so significant that most of the promising treatments in the lab never get into human trials.
     
    This proposal will turn the system upside down. It would minimize phase 3 trials.  Once a treatment is tested in a small group of patients and considered safe (compared to other approved treatments) and has a biologic effect, it can get a conditional approval.  All patients who use the drugs approved under this pathway have to go through an informed consent process as if they are going into a clinical trial and be made aware of the risks of using a drug not fully approved. They all have to be tracked in a virtual trial so we can learn from every patient's experience. The net results is way more research than the traditional pathway - as only about 10% of less of adult Glioblastoma participate in clinical trials.  Instead of each iteration of a new combinations taking 3-15 years and millions of dollars to test, multiple Virtual Trials of combinations can be run  at the same time, and can take a year or less and virtually no cost to the researchers and clinicians who create these cocktails.
     
    These researchers and clinicians will have access to the live data so they can see which drugs and combinations have been used, how safe they were, how effective they were, and what combinations were used and how they worked out. They can propose a virtual trial of any number of combinations, and limit those to patients with specific markers.  Bad drugs will be weeded out quickly instead of lingering for decades as they do now.
     
    And finally, prices should be kept under control. The payment part of the bill is still being hammered out but as it stands now, insurance should pay for the drugs.  At first that may increase costs as a lot of new drugs come out and they are used in combinations, but over time, the cost of developing drugs will plummet as will the time needed to do so, which should drop prices. Then the free market system should prevail, with many treatments with similiar results competing based on prices.  Furthermore, nonprofits like the Musella Foundation will create drugs ourselves, as nonprofits, at a reasonable price with proceeds going to the next generation of drug.  We know of many candidate drugs that did great in the lab but never made it to human trials. We could create an assembly line of pre-clinical testing and phase 1 trials, and take the best of the phase 1 results and move those onto phase 2 trials and try to get those approved.  We will see progress continuously instead of now where there is a small bump up every few years.
     
    Please help.  Go to https://virtualtrials.org/activism.cfm#/1/ to send letters.   Or call your legislator.  Spread the word. Everyone in your family and circle of friends can send their own letters.
    Thanks
     
     



  • Radiation therapy combined with intracerebral convection-enhanced delivery of cisplatin or carboplatin for treatment of the F98 rat glioma         This is the publication of a brain tumor research project that we (the Musella Foundation) funded.  It has the best results ever reported for the treatment of this rat model.    This is old (published a year ago), but I am including it now because the clinical trial that resulted from it was recently published. See next article!


  • Phase I trial of intracerebral convection-enhanced delivery of carboplatin for treatment of recurrent high-grade gliomas         This is the results of the clinical trial of CED delivery of carboplatin for the treatment of recurrent high grade gliomas.  It is based on data from a grant we gave for a rat trial (see previous article). This was funded by our friends at Voices Against Brain Cancer.  Overall the results are not impressive. The dose was way too low but the FDA required they start so low and slowly increase it. They started at 1 ug, then upped it to 2ug, then ended with 4ug, and did not get significant side effects. In the rat study, they found the best dosage for rats was 72ug and rats are way smaller than people.  So I would like to see this repeated but with reasonable doses.


  • Brain tumour patients and COVID-19 vaccines: IBTA international survey         From our good friends at the IBTA!  This is for adult (18+) brain tumor patients only!



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The article commentaries are the opinions of Al Musella, DPM and do not represent the official position of the Musella Foundation. Copyright 1992-2021 Musella Foundation - All rights reserved. No part of the Brain Tumor News Blast can be reproduced without the express written permission of the Musella Foundation.