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!
06/12/23
Dr Henry Friedman podcast This is from our friends at The Glioblastoma Research Organization: an interview of Dr Henry Friedman of the Preston Robert Tisch Brain Tumor Center at Duke University. Interesting stories, and he mentions me at the 31:40 mark!).
06/08/23
Senators Braun, Gillibrand, colleagues introduce bipartisan Promising Pathway Act to help patients with ALS and other life-threatening diseases get treatments In my opinion, this is one of the most crucial bills we have ever encountered. It builds upon existing frameworks such as the accelerated approval pathway, expanded access, and right to try laws, with the primary objective of ensuring convenient and widespread accessibility to experimental therapies. Furthermore, it places a strong emphasis on systematically gathering data from every patient who undergoes these treatments, allowing us to derive valuable insights from their experiences. This bill is precisely the transformative measure needed to make a significant impact in the ongoing battle against cancer. I am currently working on an effortless way for you to express your support for this bill to your representatives in Congress. I will notify you as soon as it is ready!
06/05/23
Use of proton pump inhibitors (PPI) in glioblastoma (GBM) and relationship to overall survival in a national real-world evidence (RWE) database. This is an impressive application of real-world evidence. Dr. Castro hypothesized that the use of proton pump inhibitors (PPIs) may have a negative impact on the survival of patients with Glioblastomas. Typically, testing such a hypothesis would be challenging and costly. However, he utilized a regulatory-grade registry that maintains a comprehensive record of the medications patients take. By analyzing this data, they were able to demonstrate a significant negative effect associated with PPI usage. This effect could potentially be as significant as the difference between methylated and unmethylated MGMT status.
While it is advisable to replicate these findings using other datasets for confirmation, until counter-evidence emerges, it would be worthwhile for individuals taking the drugs listed below to discuss the matter with their healthcare providers and consider alternative medications.
The proton pump inhibitors include:
- Omeprazole (Prilosec) - available over-the-counter without a prescription.
- Esomeprazole (Nexium) - available over-the-counter without a prescription.
- Lansoprazole (Prevacid) - available over-the-counter without a prescription.
- Rabeprazole (AcipHex)
- Pantoprazole (Protonix)
- Dexlansoprazole (Dexilant)
- Zegerid (Omeprazole with sodium bicarbonate) - available over-the-counter without a prescription.
Disclaimer: I am a founder and paid consultant to xCures and hold stock in the company.
06/05/23
Brain Tumor Biotech Summit 2023 - this Friday June 9, 2023 This conference holds a special place in my heart as it remains my favorite event of the year. It serves as a unique platform that brings together esteemed researchers, biotech leaders, and members of the investment community. Its primary purpose is to showcase the latest treatments in the pipeline and facilitate fundraising efforts to support their development. It is important to note that this conference is specifically tailored for doctors, researchers, biotech companies, and investment professionals, and unfortunately, it is not open to patients.
06/04/23
DCVax®-L: Mechanism of Action, Immunological Effects, and Clinical Trial External Controls Methodology
The presentation on the DCVax clinical trial for glioblastoma, which was showcased at the ASCO meeting yesterday, brought attention to the use of an external control group in the trial. This aspect of the trial has been the subject of much criticism, particularly from individuals on the internet. However, I am of the belief that external control groups, when utilized correctly, can potentially offer more beneficial insights than a randomized control.
There have been several instances recently where long-running trials did not stratify by biomarkers that were unknown at the time the trial was initiated. These biomarkers were later discovered to significantly influence outcomes, potentially affecting the overall results of the trial. The purpose of randomization is to theoretically distribute these different prognostic factors evenly between the treatment and control groups. However, in these instances, the randomization failed to achieve this balance.
By using external control groups, we can account for every known prognostic indicator. Furthermore, if a new prognostic factor is identified before the data is reported, we can recreate the external control group taking this new factor into account. This methodology can also be more ethical, as it prevents wasting a patient's life by administering a placebo, or a standard of care that does not cure the disease.
The presentation also detailed the mechanism of action of DCVax. In one case, they found that DCVax targeted over 11,000 targets, a remarkable finding considering that many other vaccines currently in trials target only one to six targets. This suggests that DCVax could potentially be more effective due to its wide range of targets.
06/04/23
Vorasidenib first 'game changer' in 20 years for patients with IDH-mutant grade 2 glioma
I am genuinely thrilled by these findings. The study shows that Vorasidenib has more than doubled the median progression-free survival time from 11 months to 27 months. Moreover, more than half of the patients are still alive, indicating that the median survival has not been reached yet. It's important to note that this study was focused on patients with grade 2 IDH mutant glioma.
Vorasidenib is a targeted drug, designed to work specifically on patients with a mutation in either IDH1 or IDH2. With the new nomenclature of brain tumors, it's crucial to understand that Glioblastomas cannot have this mutation. If they do, they are classified under a different name and can no longer be referred to as Glioblastoma. However, some older centers might still use the old naming system, so it's important to check your pathology report and look for IDH1 or IDH mutant.
If your report states "IDH wildtype," it means that you do not have the mutation. If you do have the mutation, I would strongly urge you to discuss this drug with your doctor. It's critical not to assume that your doctors will retroactively go through all their old pathology reports to identify patients who could benefit from this drug. Although most probably will, your life is at stake, so it's worth taking the initiative to check the report yourself. If your tumor was not tested for the IDH mutation, I recommend requesting that they perform this test. Your health and well-being are paramount, and this could be a significant step in your treatment journey.
05/30/23
GT Medical Technologies Marks a Clinical Milestone: 200th Patient Enrolled in the Multicenter Observational Registry of GammaTile® Therapy
GammaTile is an FDA-cleared treatment option for both newly diagnosed and recurrent malignant brain tumors, such as Glioblastomas and metastatic tumors. This innovative therapy involves the surgical insertion of radiation-emitting wafers at the time of surgery, precisely targeting the affected area. Unlike external beam radiation, GammaTile begins its therapeutic effects immediately, potentially eliminating the need for additional radiation treatments and significantly benefiting patients in terms of convenience and time savings. This groundbreaking treatment has been readily accessible for three years and is currently available in over 80 hospitals across the United States.
05/22/23
Updated NCCN Guidelines for Brain Tumors
The updated guidelines suggest more testing and get more into the genomic markers. For Glioblastoma, the big change is that Optune (listed as "Tumor Treating Fields (TTF)") is now included in the preferred regimen for patients with a karnofsky score of 60 or more. In the last version of the guidelines, it was just one of a few choices.
05/22/23
New legislation proposed to expedite FDA approval process for life-threatening disease treatments This is the Promising Pathway Act that I have been talking about for the past few years! It will soon be reintroduced to Congress. When it is, we must collaborate to exert pressure on Congress to pass it. The exciting update is that we now have Senator Kirsten Gillibrand on board! She is a Democrat representing my home state of New York! Previously, when we attempted to get this bill passed, we struggled to find Democrats who would support it. With bipartisan support now in place, our chances have significantly improved!
05/20/23
Congress should support a new strategy for approving lifesaving drugs Being one of the contributors to this bill, I believe it paves the fastest way to discovering cures for critical diseases. Its nature is disruptive, as it fundamentally reshapes the existing system by incorporating a novel conditional approval pathway for FDA drug approvals, which starts after a phase 2 trial.
Any medication administered through this pathway necessitates that patients be enlisted in a virtual trial registry. There, their treatment protocols and outcomes are diligently observed. This real-time registry is accessible to healthcare professionals, equipping them to discern which treatments are effective, what combinations are in use, and to assess preliminary results.
Traditional practitioners who rely on the rigorous data from Phase 3 trials may have reservations, but it's worth noting that this model provides similar comprehensive data, albeit in a more dynamic context.
The bill's effects might be less favorable for major pharmaceutical corporations, which currently generate substantial profits from treatments that are only marginally effective. As a range of innovative treatments receive approval, competition could intensify based on efficacy and pricing.
Consequently, the financial requirements to get a drug approved are anticipated to diminish significantly. This shift will likely make it more enticing to develop drugs targeting rare cancers, such as brain tumors, thereby bringing hope to many patients with these conditions.
05/18/23
Two Leading Nonprofits Join Forces to Fund Brain Tumor Research We are thrilled to share the exciting news of our partnership with StacheStrong to support groundbreaking research on brain tumors! Together, we have contributed $80,000 each, resulting in a total funding of $160,000 for five exceptional brain tumor research projects. This collaboration highlights the importance of working together to achieve remarkable outcomes. In addition to StacheStrong, we are proud to collaborate with esteemed organizations such as Cancer Commons, The DIPG/DMG Collaborative, The DIPG/DMG Research Funding Alliance, The Children's Brain Tumor Network, and the DIPG All-In-Initiative. Collectively, these groups encompass more than 100 brain tumor nonprofits, demonstrating the collective effort towards addressing this critical cause.
05/18/23
5K for brain tumor research (MN May 21, 2023) Mark your calendars for our upcoming MN 5k fundraiser taking place on May 21, 2023! This year, we have high hopes of surpassing the remarkable milestone of $1 million in total donations generated through this event. Thanks to the generous support from our this as well as our other events, we have been able to fund five promising brain tumor researchprojects today - in partnership with StacheStrong!
To join us in this meaningful endeavor, you can participate either in person or by making a donation through our dedicated website at
WalkToEndBrainTumors.org.
We are excited to announce three additional 5k events in different locations: Salt Lake City on May 29, 2023, Clark, NJ on June 10, 2023, and Street, MD on September 10, 2023. Your involvement and support are invaluable as we strive to make a difference in the fight against brain tumors.
05/11/23
The Musella Foundation Celebrates 25 Years of Advancing Brain Tumor Research and Supporting Patients
Reflecting on 25 Years of Impact: A Personal Journey with Brain Tumors
When I reflect on the past 25 years, it's remarkable to think about how my personal experiences with brain tumors led to the creation of The Musella Foundation For Brain Tumor Research and Information, Inc. It all started when my sister-in-law was diagnosed with a Glioblastoma in 1992, followed by my dad's diagnosis in 1999. These events propelled me into action, and I became determined to make a difference in the lives of those affected by these devastating tumors.
In the early days, I recognized the need for support and information for brain tumor patients and their families. That's when I took the initiative to start the first online support group dedicated to brain tumors. Little did I know at the time, this small step would snowball into something much greater.
Today, as we celebrate our 25th anniversary as a 501(c)(3) nonprofit organization, I am filled with immense pride and gratitude for the incredible journey we have undertaken. The Musella Foundation has grown into a leading force in advancing brain tumor research and providing vital information to patients and caregivers.
Our dedication and relentless efforts have led to significant achievements, including funding groundbreaking research projects, empowering patients through comprehensive resources, advocating for improved treatment options, and building a strong community of support. Together, we have made a lasting impact in the lives of brain tumor patients and their families.
I invite you to read more about my personal story and the origins of The Musella Foundation at
virtualtrials.org/musella.cfm. It is a testament to the passion, resilience, and unwavering commitment of our community.
As we look to the future, our commitment remains steadfast. We will continue to drive progress in brain tumor research, provide support and resources to those in need, and advocate for improved treatment options. Our journey is far from over, and with the continued support of our community, we are confident that we can make even greater strides in the years to come.
Thank you for being a part of this incredible journey and for your ongoing support.
Warm regards,
Al Musella, DPM
President, The Musella Foundation For Brain Tumor Research and Information, Inc.
05/08/23
Join Us for Brain Tumor Awareness Month: Free Webinar Series on Innovative Treatments and Clinical Trials Tonight Monday May 8 at 7PM Eastern!
Last night's webinar was very informative. We posted the recording to our video library.
Tonigth we have Dr Mark Malkin talking about the Clinical Trials for Brain Tumors that are being done at the Cleveland Clinic!
We host these webinars in our Zoom room, which offers the best platform for interactive participation and asking questions. However, we also attempt to simulcast the sessions on Facebook, as our Zoom room has a capacity limit of 500 attendees, which we've exceeded on a few occasions.
Last night we did not get to most of the questions asked on Facebook. If you would like to ask questions, join in our zoom room. It is really easy. Starting about 15 minutes before each event, our virtualtrials.org/webinar page will have a link to the room. Just click it and you are in!
