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!
10/18/23
Our next webinar will be on GammaTile, on Monday Oct 30, 2023
Our next webinar will be on GammaTile, on Monday Oct 30, 2023 at 7pm eastern Eastern.
GammaTile is an FDA approved treatment for brain tumors. It is implanted at the time of a surgery and releases radiation to the tumor bed. The speaker will tell us the most current data on outcomes.
10/13/23
Invitation to the Promising Pathway Act Press Conference in Washington, DC, Oct. 26, 2023.
There will be a press conference in Washington, D.C., on October 26, 2023, at 9 am. You are cordially invited to attend and join us in our efforts to support the passage of the Promising Pathway Act!
A limited number of individuals will have the opportunity to speak at the conference. If you are interested in being one of our speakers, please notify us via email at musella@virtualtrials.org.
Moreover, we extend this invitation to everyone, whether or not you can attend in person. We encourage you to create a brief video, with a maximum duration of 2 minutes, explaining why you believe the Promising Pathway Act is crucial. Share your video on social media platforms and include the hashtag #PPA. Don't forget to send us a link to your video. We will compile and submit these videos to the Senator's office, and a select few will be featured during the live event!
Your participation and support are invaluable in our endeavor to promote the Promising Pathway Act. Together, we can make a difference!
10/13/23
Optune Overview This is an overview of the most important studies on Optune for Glioblastoma and other cancers, and includes the latest research on mechanism of action! Worth reading!
09/24/23
Brain Tumors in United States Military Veterans
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!
09/24/23
Peptide vaccine targets H3K27M mutation in midline gliomas, shows promise in early trials
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.
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
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.
