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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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09/18/23 Real-world validity of randomized controlled phase III trials in newly diagnosed glioblastoma: to whom do the results of the trials apply?        
The Limitations of Phase 3 Randomized Trials in Glioblastoma: A Call for a Comprehensive Learning System
 
Clinical trials are the gold standard for evaluating the efficacy of new treatments. However, when it comes to Glioblastoma, phase 3 randomized trials often fall short of providing a complete picture. The stringent eligibility criteria for these trials mean that the majority of patients are excluded, leading to results that may not be generalizable to the broader population.
 
This article indicates that patients ineligible for clinical trials have significantly poorer outcomes, living less than half as long as those who meet the criteria. This stark difference underscores the need for a more inclusive approach to understanding this complex disease.
 

The Need for a Comprehensive Learning System
To address these limitations, we propose the implementation of a comprehensive learning system that tracks all Glioblastoma patients, not just those who qualify for clinical trials. This system would collect real-world data on a variety of factors, including treatment responses, side effects, and overall survival rates.
 
 
Matched Contemporaneous Control Groups
In addition to tracking all patients, the use of matched contemporaneous control groups could offer more accurate insights into treatment efficacy. By comparing similar patient profiles, this approach would allow for a more nuanced understanding of how different treatments perform in real-world settings.
 
 
Conclusion
The current approach to Glioblastoma research, focused primarily on phase 3 randomized trials, is insufficient for capturing the complexities of this disease. By adopting a comprehensive learning system and utilizing matched contemporaneous control groups, we can develop more effective and personalized treatment strategies. This is not just a theoretical exercise; it's a necessary evolution in how we approach Glioblastoma research and treatment.
 


09/16/23 FDA expands approval of Temodar to treat additional form of brain cancer        

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.

 


09/14/23 xCures Platform advances progress on the White House Cancer Moonshot mission to end cancer as we know it.        
I'm incredibly proud to be a part of xCures. The White House recently highlighted our work in a fact sheet about the Cancer Moonshot initiative, specifically mentioning our contributions to Diffuse Midline Glioma (DMG) and DIPG research. xCures operates a regulatory-grade registry that meticulously tracks data on patients with DMG, DIPG, and various other diseases. We swiftly collect and organize all of the patient's medical records. This de-identified data is then made freely available to researchers, benefiting both the research community and the patients involved.
 
For instance, this data can serve as a contemporaneous matched control group, thereby eliminating the need for placebos in pediatric brain tumor trials. This is particularly useful in phase 2 trials, where it can act as a control group to provide a more accurate understanding of a treatment's impact on the disease. One of the most common reasons treatments excel in phase 2 but fail in phase 3 trials is the use of poorly matched historical controls in the former, leading to an inflated perception of efficacy. Our project aims to prevent such outcomes, potentially saving lives and reducing both time and financial costs for pharmaceutical companies.
 
Disclaimer: I am a paid consultant for xCures and also one of the company's founders.


09/14/23 Deep vein thrombosis as a side effect of cancer: 9 things to know??        
Patients with brain tumors and their caregivers should be aware of the symptoms of deep vein thrombosis (DVT). The initial symptoms of DVT in cancer patients include swelling, redness, and pain in one or both arms or legs; abdominal pain; or swelling or redness around the port site. The major risk is that the clot could dislodge and travel to the lungs, leading to a potentially fatal pulmonary embolism. Symptoms of a pulmonary embolism include shortness of breath, chest pain, lightheadedness, and difficulty breathing.
 
If you suspect you may have a blood clot or pulmonary embolism, seek immediate medical attention. Contact your doctor and inform them of your symptoms. If you're unable to reach your doctor, go to the emergency room and clearly state that you suspect you have a blood clot or pulmonary embolism. This is a medical emergency.


09/11/23 Cancer Chomp: A Brain Tumor Event in Gainesville, FL        

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



09/11/23 Study shows how microdevices can be used to treat brain cancer        

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.



08/29/23 Northwest Biotherapeutics Announces Completion of Prerequisites, and Plans for Submission of Marketing Authorization Application        

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.



08/29/23 Journey of pediatric brain tumor tissue donation        

 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.



08/26/23 Update on the Promising Pathway Act        

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

Every day, patients reach out for assistance in accessing treatments, and for many, the options are limited or non-existent. The Promising Pathway Act has the potential to quickly change this grim reality. 
 


08/26/23 Association of Tumor Treating Fields (TTFields) therapy with survival in newly diagnosed glioblastoma: a systematic review and meta-analysis        
This article doesn't reveal anything new, but it does underscore the significance of Optune. A glance at the survival curves in Figure 3 indicates that adding Optune boosts the 5-year survival rate to approximately 20%, compared to just 5% without it. This is a substantial improvement.
 
One issue I have with the article is its implication that Optune is not part of the standard care for treating Glioblastoma. Contrary to this, Optune is actually considered standard care. According to current NCCN guidelines, the preferred treatment sequence is to first consider a clinical trial. If that's not feasible, the next best options are surgery, radiation, Temodar, and Optune. Dr. Roger Stupp, the namesake of the Stupp protocol—which is the standard of care for GBM—has stated at a SNO meeting that Optune is now integrated into the Stupp protocol and is considered standard care.
 
Another point of contention I have with the article is its emphasis on a 75% compliance rate as the threshold for effectiveness. I believe this target is too low and should be raised to 90%.


08/26/23 Prodrug-loaded semiconducting polymer hydrogels for deep-tissue sono-immunotherapy of orthotopic glioblastoma        

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.



08/23/23 Estrogen-negative cancers respond to anti-estrogenic therapies        

 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.



08/19/23 Optune Open House in Tarrytown, NY Sept 12th, 2023.        

 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.



08/18/23 Scientists Find New Weapon Against Brain Tumor: 'Major Crack in the Armor'        

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.



08/18/23 Using the Body's 'Invisible Scalpel' to Remove Brain Cancer With Immunotherapy at Salk Institute        

 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.



08/12/23 She went to Germany for a new brain cancer treatment. She wonders why she had to learn about it online        
The current drug approval system is in dire need of reform. While this article focuses on a Canadian patient's experience, the issue is equally pressing in the USA, particularly concerning the drug Onc-201.
 
Onc-201 has demonstrated potential in treating diffuse midline gliomas with the H3K27M mutation. On its own, it provides some relief to about half of the patients and significantly helps about one-third. However, as the article rightly points out, the current approval process requires a drug to benefit the majority of patients.
 
The crux of the problem, which I have repeatedly emphasized, is that the ultimate cure for many conditions will likely involve a combination of treatments. Individual treatments may not benefit the majority, but when used in conjunction, they could prove highly effective. Onc-201 represents a significant advancement in this direction, especially for this specific tumor type. It's an orally administered drug with minimal side effects, and its efficacy should have warranted approval following the data presented to an FDA ODAC panel years ago.
 
Contrast this with Germany's approach, where patients have the right to access any experimental drug, albeit at their own expense. This policy ensures that potentially life-saving treatments are within reach for those who need them.
 
The USA could take a significant step forward with the passage of the Promising Pathway Act (details and an easy way to help are  available at Virtual Trials). This legislation would not only expedite access to treatments like Onc-201 but also encourage insurance coverage and, crucially, establish the infrastructure needed to test combination therapies.
 
In conclusion, the time for change is now. The current system's limitations hinder progress and deny patients access to promising treatments. By embracing a more flexible and patient-centered approach, we can foster innovation and offer hope to those battling serious illnesses. The Promising Pathway Act represents a vital step in this direction, and its passage would mark a significant victory for patients and medical practitioners alike.
 
Disclaimer: Chimerix is a sponsor of the Musella Foundation


08/12/23 Plus Therapeutics Reports ReSPECT-LM Phase 1 Trial Data at the 2023 SNO/ASCO CNS Cancer Conference        

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. 



08/10/23 Perlmutter Cancer Center Enrolls First Participant in Study of Noninvasive Treatment of Glioblastoma        

 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. 



08/07/23 GT Medical Technologies Marks a Clinical Milestone: 200th Patient Enrolled in the Multicenter Observational Registry of GammaTile┬« Therapy        

 GammaTile is an FDA approved treatment for newly diagnosed and recurrent primary and metastatic brain tumors.  It is implanted at the time of surgery and slowly releases radiation to the tumor bed. It may be able to replace standard radiation.  I love that they are collected the real world data in a registry so they learn from every patient. All brain tumor patients show be tracked in a registry so we can learn from each of them!



08/02/23 Phase 1 clinical trial underway to test City of Hope-developed cancer treatment        

 This is very early - just starting human trials.  However it is also a shot at a home run therapy not only for brain tumors but most solid tumors.  It is the holy grail of cancer treatments - oral, doesn't hurt normal cells, may work on all cancers by targeting a pathway that is central to all cancers.  Will be keeping an eye on this.   The trial is open in California now and will open in Arizona soon. https://classic.clinicaltrials.gov/ct2/show/NCT05227326



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