This paper compares the incidence and survival times of brain tumor patients within the VA system compared to the entire USA.  For younger patients, aged 18-49, the survival rates were the same inside and outside the VA, but  those aged 50-69 did worse in the VA. For 70+, the survival was only 4 months for both VA patients as well as USA patients.   This shows that we really need better treatments.   The Musella Foundation was mentioned as one of the funders!

 This is exciting news - but still very early.  It has been reported to be used in 8 patients so far.  Five of these had an immune response, and one had tumor regression and remained tumor free for 31 months - which is amazing for a recurrent H3K27M mutant diffuse midline glioma patient.   There is a trial going on in Germany that should report more data in a year or two.

This article contains inaccuracies. Temodar was initially approved for recurrent anaplastic astrocytoma in 1999, not for glioblastoma as stated. The FDA declined the request to approve it for glioblastoma at that time. It wasn't until 2005 that the FDA extended its approval to include newly diagnosed glioblastoma. The most recent update added approval for newly diagnosed anaplastic astrocytoma.

 This is from our friends at the University of Florida.  A fun event raising money for patients being treated for brain tumors!

Researchers at Brigham and Women's Hospital have developed a tiny device to improve glioma treatment. Implanted during surgery, the device tests multiple drugs on the tumor in real-time, offering personalized treatment options. Initial trials show no adverse effects, making it a promising tool for tackling this hard-to-treat brain cancer.

Northwest Bio has announced plans to apply for approval of DCVax within the coming month. Following the submission, the approval process is expected to take approximately 150 business days. This represents a significant milestone for the company. DCVax is a dendritic cell-based vaccine that is personalized to an individual's tumor. To create the vaccine, a sample of the tumor is required. The results from a large Phase 3 trial have been highly promising, and preliminary data from ongoing trials involving combination therapies appear to be remarkable.

 This video explains how you can donate your child's brain tumor tissue. This can be from a surgery if there is extra tissue available, or from an autopsy.   The donation will help the researchers design better treatments. It is an interesting video and shows how the donation  will be used.

The Promising Pathway Act: A Game-Changer in Medical Treatment Access

I'm a strong advocate for focused ultrasound, a versatile tool for treating brain tumors. The article introduces an innovative application for this technology. The issue at hand is that the immunotherapy drug intended for use cannot penetrate the tumor in sufficient concentrations to be effective. To address this, researchers have developed a pro-drug—a modified version of the drug—that can infiltrate the tumor. This pro-drug is then converted into the active drug specifically within the tumor area using focused ultrasound. This approach has the potential to facilitate the delivery of various drugs to the tumor. The selected drug has shown promising results in mouse models, and hopefully, clinical trials in humans will commence soon.

 I started the Musella Foundation because a family member had a Glioblastoma!  I came across an article about High Dose Tamoxifen for Glioblastomas where a small number of patients did well with it, so we tried it and it worked for her.  She used high dose Tamoxifen for about 5 years, until her insurance hit it's lifetime maximum and stopped paying for any treatments.   (Luckily there is no more lifetime maximum on insurance now!).  She had a massive recurrence on her next scan, and then died. She lived for about 8.5 years from diagnosis.   Hundreds of patients then tried it based on her experiences, and it only helped a small % of them. For most patients it did nothing.  A few trials were done that showed only a small number of patients benefitted.  I am not thinking that since it had relatively little side effects, it may be worth trying it in combinations now.

 These Open Houses from Novocure are interesting and fun.  You will get to meet others who are using Optune and get a chance to ask questions directly to the Novocure people.  It is free, and it is for GBM patients and Caregivers!  They have limited space so if you are thinking of going, register now.

We have covered Onc201 extensively in past News Blasts. The Musella Foundation has been fighting for years to get patient access to this drug, as we know it can help patients with diffuse midline glioma (DMG) with the H3K27 mutation. We have funded $875,000 in grants to help develop this treatment (we were not mentioned in the article because we did not fund this specific project).  Now that results showing Onc201 nearly doubles median survival for DMG-H3K27 patients have been published in a peer-reviewed journal, there will be a wave of news coverage for this drug. But the important part of this story is that patients STILL cannot easily access this drug.  This is not right, and it is a clear example of why we need the Promising Pathway Act. If you haven't already, please click here to send a letter to your Congress reps in support of the Act.

 A recent study published in Immunity on August 11, 2023  found that an immunotherapy drug called anti-CTLA-4 prolonged survival in a mouse model of mesenchymal-like glioblastoma. Anti-CTLA-4 was one of the first drugs designed to stimulate the immune system to fight cancer, but it was quickly followed by another, anti-PD-1, that was less toxic and has become more widely used in clinical trials. Upon investigating why the anti-CTLA-4 therapy was more effective in this mouse study, researchers found that the drug prompted CD4+ T cells to secrete interferon gamma which activates microglia to 'eat' more tumor cells. Although promising, this is still early-stage research.

This is a new form of targeted radiation.   We did a webinar about it: https://virtualtrials.org/video2022.cfm?video=202205.   They reported on the early results of their phase 1 trial, which was a dose escalation trial. They reported an amazing median survival of 10 months.  They did not have an external control group but in general, people with leptomeningeal mets only live 2 weeks to 4 months.   As the find the correct dosages it should get better. And as I always say - combinations are the way to cure these things. In the past, the main problem was that nothing really had time to work.  Now, if you could buy 10 months - that gives you a lot of time for other treatments to work. Very exciting. 
Disclaimer: Plus Therapeutics is a sponsor of our organization. 

 As I mentioned many times recently, Sonodynamic therapy is one of my favorite experimental treatments.  It is too early to tell how effective this will be, but the concept is elegant and preclinical work showed very good results.  From the patient's point of view, it is relatively easy as it is non-invasive - no cutting through the skull!   A dye is injected into the arm veins, which is taken up by tumor cells and not normal brain. Focused ultrasound is applied  (similar to the way a sonogram is done on a fetus) which kills cells that have taken up the dye. Net effect is killing cancer cells while leaving normal cells alone.  

There are two competing systems that are in clinical trials now. Nobody knows which is best. The one mentioned in the article is from the company SonALAsense, Inc. This trial is for progressive or recurrent Glioblastoma and requires that only one tumor be present. They also have a trial of the same system for newly diagnosed Glioblastoma - but only in Italy right now (check clinicaltrials.gov for updates).  The other is from  Alpheus Medical, Inc. and they can accept multifocal tumors.