Al's Comment:

 I am excited over the progress made in 2023 and I think 2024 is going to be even better! Everything is coming together and we are on the verge of a breakthrough - which I believe is going to be a combination of the treatments I mention in the blog post!
Happy New Year!

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Posted on: 12/31/2023

2023 Year In Review: Major Progress in Brain Tumor Research and Treatment

Advancements in Low Grade Glioma Treatments

This year, we witnessed groundbreaking advancements for Low Grade Gliomas, particularly grade 2 IDH mutant gliomas. Vorasidenib, a new drug, demonstrated remarkable efficacy, extending progression-free survival from 11 to 27 months. With more than half the patients surviving beyond the study period, median survival remains undetermined. We are hopeful for FDA approval of Vorasidenib, potentially benefiting all patients with IDH mutant brain tumors. Read more about Vorasidenib's impact.

Diffuse Midline Glioma / DIPG Innovations

For Diffuse Midline Glioma / DIPG, Onc-201 continues to show promise, potentially doubling survival rates. Early results indicate that combinations with other treatments may yield even better outcomes. Discover more about Onc-201.

Glioblastoma Breakthroughs

In the realm of glioblastoma treatment, several advances have emerged:

• DC-Vax: Poised for approval, DC-Vax's large phase 3 study showed modest improvements in median survival and significant enhancements in long-term survival. A small study combining DC-Vax with immune enhancers exhibited exceptional results. Approval in the UK seems imminent, with the USA hopefully following. Explore DC-Vax developments.
• Vaccine and Immunotherapy Trials: Trials with the Survaxm Vaccine, MDNA55, and others reported excellent outcomes, heralding a new era in glioblastoma treatment. Learn about Survaxm Vaccine and MDNA55.

GammaTile Therapy: A Milestone Achievement

GammaTile therapy, approved for recurrent and malignant brain tumors, reached a significant milestone with 1,000 cases treated. This year's results solidify its potential for both newly diagnosed and recurrent glioblastomas and metastatic tumors. Read about GammaTile's achievement.

Optune: Revolutionizing Glioblastoma Treatment

Optune, an innovative treatment approved by the FDA and recognized by the National Comprehensive Cancer Network (NCCN), has continued to make significant strides in glioblastoma therapy. This non-invasive device uses Tumor Treating Fields (TTFs), a unique method employing low-intensity, alternating electric fields to disrupt cancer cell division and inhibit tumor growth.

In 2023, extensive research around Optune has been summarized in a comprehensive report, highlighting its multifaceted impact on glioblastoma treatment. Key findings include:

• Combination Therapy: Recent studies suggest that combining Optune with immunotherapy agents like Keytruda (pembrolizumab) may significantly enhance patient outcomes. This combination has shown potential to extend median survival by up to 9 months compared to Optune alone, a substantial improvement over current standards.
• Compliance Correlation: The effectiveness of Optune appears to be strongly linked to patient compliance. Use of the device with low compliance (less than 75% of the time) resulted in an added 5 months to median survival compared to Temodar (Temozolomide) alone. However, when used with high compliance (over 75% of the time), the extension in survival increased to about 13 months beyond Temodar treatment, underscoring the importance of consistent usage.
• Technological Advancements: A new generation of Optune arrays is in development. These arrays are designed to be thinner, more flexible, and more comfortable for patients, potentially increasing compliance. They are also expected to deliver a higher dosage of TTFs directly to the tumor, enhancing the treatment's efficacy.
• Extensive Research Base: The body of research supporting Optune is vast and continuously growing. This includes numerous clinical trials, patient experiences, and ongoing studies exploring its synergistic effects with other treatments.

For an in-depth understanding of Optune's clinical evidence and ongoing research, interested readers can refer to the NVCR Clinical Evidence Flipbook.

Focused Ultrasound: A Pioneering Approach in Brain Tumor Therapy

Focused Ultrasound (FUS) is a groundbreaking, non-invasive therapeutic technology that has shown significant promise in the treatment of brain tumors. As of 2023, while still considered experimental for brain tumors, FUS has garnered considerable attention for its potential applications and early successes.

• Sonodynamic Therapy: This novel approach involves the use of FUS in conjunction with sonosensitizing agents. These agents are preferentially absorbed by tumor cells and, when activated by ultrasound, can selectively destroy tumor tissues. The precision of FUS allows for targeted activation, minimizing damage to surrounding healthy brain tissue.
• Blood-Brain Barrier (BBB) Disruption: One of the most promising applications of FUS is its ability to temporarily and safely open the BBB. This transient opening enables enhanced delivery of therapeutic agents, such as chemotherapy drugs, directly to the brain tumor, which is otherwise a significant challenge due to the protective nature of the BBB.
• Hyperthermia Treatment: FUS can be used to heat targeted areas of the brain, creating a hyperthermia effect that can weaken or kill tumor cells. This technique is particularly valuable as it affects tumor cells more than normal brain tissue and can be finely controlled to avoid collateral damage.
• Ablation Therapy: In cases where tumor removal is necessary, FUS can be used for thermal ablation, destroying tumor cells with focused heat without the need for invasive surgery. This method is especially beneficial for patients who are not candidates for traditional surgery.
• Early Clinical Trials and Research: The ongoing research and early-stage clinical trials of FUS are showing promising results. Initial studies demonstrate its potential efficacy and safety in treating various types of brain tumors.
• Expanding Applications: Beyond direct tumor treatment, FUS is being explored for its utility in enhancing drug delivery, gene therapy, and immunotherapy effectiveness by altering tumor microenvironments or releasing tumor antigens.

As FUS technology continues to advance, it holds the potential to transform the landscape of brain tumor treatment. It offers a combination of precision, minimal invasiveness, and the ability to reach previously inoperable tumors, making it a highly anticipated development in neuro-oncology. While more research is needed to fully understand its capabilities and refine its applications, the early results from 2023 indicate a bright future for this innovative treatment modality.

Cost-Effective Medication: Cost Plus Drugs

Costplusdrugs.com, Mark Cuban's venture, made a significant impact by offering generic Temozolomide at about 90% off traditional pharmacy prices. This initiative is a game-changer in making treatments more accessible. Check out their offerings.

Advocacy: The Promising Pathway Act: Accelerating Access to Innovative Treatments

The Promising Pathway Act, gaining momentum in 2023, represents a transformative shift in the way new treatments are brought to patients, particularly those battling serious conditions like brain tumors. This legislation is designed to streamline the approval process for new drugs and therapies, providing quicker access to potentially life-saving treatments.

• Expedited Approval Process: The Act proposes a conditional approval pathway for drugs treating serious or life-threatening conditions. This pathway allows for drugs to be conditionally approved based on initial clinical data showing safety and effectiveness, rather than waiting for the completion of lengthy and comprehensive Phase III trials. The research is continued after conditional approval until enough evidence is generated to promote the drugs to full approval.
• Learning Health System: A unique aspect of the Act is its focus on creating a learning health system. This system would continuously collect and analyze data from patients using these conditionally approved drugs, thus providing real-time information on their effectiveness and safety. This approach ensures that treatments can be optimized and adapted based on patient outcomes. Low performing drugs will be quickly weeded out and high performing drugs will be quickly identified.
• Bipartisan Support: The Act has garnered bipartisan support, with a balanced number of co-sponsors from both major parties in the Senate and the House. This broad support underscores the recognized need for faster access to innovative treatments across the political spectrum.
• Impact on Brain Tumor Research and Treatment: For the field of brain tumor research, the Promising Pathway Act could be a game-changer. It would allow patients earlier access to cutting-edge treatments, such as those mentioned, which might otherwise take years to become widely available.
• Addressing Critiques and Rewriting: Efforts are underway to address critiques of the original bill and to rewrite it from the ground up. These revisions aim to ensure that while the approval process is expedited, patient safety and treatment efficacy remain paramount.
• Future Outlook: With its potential re-introduction, the Act will require renewed efforts to garner support in Congress. The advocacy community is poised to mobilize once the bill is ready, emphasizing its potential to revolutionize the treatment landscape for patients with brain tumors and other serious conditions. I will let you know when you need to take action again!

In summary, the Promising Pathway Act is a significant legislative initiative aimed at reshaping how new treatments are approved and delivered to patients. Its impact could extend far beyond brain tumors, offering hope and new possibilities to patients with various serious conditions. The Act's progress in 2023 marks a critical step towards a more responsive and patient-centric healthcare system.

Artificial Intelligence in Cancer Care: A Game-Changing Innovation

Mainstream Adoption and Collaboration in AI Technology: 2023 witnessed a groundbreaking shift with the mainstream emergence of Artificial Intelligence (AI) tools like ChatGPT, revolutionizing various sectors, including healthcare. Our commitment over the past five years has been to harness AI's potential in cancer care, working in close collaboration with Cancer Commons, xCures, and several other pioneering organizations.

Publication of Research and System Evolution: A milestone in our journey was the publication of a peer-reviewed article that details our approach to integrating AI in cancer treatment. This article, accessible here, highlights the strides we've made in developing an AI system that is continually learning and evolving.

Creating a Comprehensive AI System: Our mission harnesses Artificial Intelligence (AI) to develop innovative, AI-driven treatment options for cancer patients. We integrate advanced AI algorithms with a comprehensive patient registry, the latest research findings as well as expert opinions to create personalized, state-of-the-art treatment plans. This integrative approach is transforming the personalized treatment of cancer, ensuring patients receive highly effective and customized care. This tool is designed to guide patients through their treatment options, providing customized recommendations based on their unique medical profiles. Patients and caregivers can access this cutting-edge resource here to explore potential treatment paths and make informed decisions about their care.

 

 

 

Happy New Year!

The above comments are the opinions of Al Musella, DPM. As a disclosure, Novocure, GT Medical Technologies, and Chimerix are sponsors of the Musella Foundation. I am a founder of, own stock in, and am a consultant to xCures.

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