Glioblastoma & Brain Cancer Treatment Options: What Patients Should Know

•There are many promising new therapies, but you can’t access a lot of them in a combination. You have to pick and choose which ones you're going to pursue.

•If a treatment doesn't work, you have to pivot really quickly to the next one because the disease is very aggressive.

•The current drug development process is too slow and costly for rare diseases like glioblastoma.

•Only a very select few, like 5% of the population, can access clinical trials. How can we overcome the regulatory hurdles to accessing treatments and accelerate treatment development? You should support “The Promising Pathway Act”, pending legislation which will create a conditional approval pathway for the FDA. After you see that a drug is pretty safe, and it has the effect you want it to have, it would get a conditional approval. Any doctor could prescribe it, and insurance would handle it like any other approved drug. Any patient who uses these drugs would be followed as if they're in a clinical trial. We would learn from every patient. We would continue the research. The only difference from the standard pathway is that it's more flexible, where you can do combinations if you like, and any patient could get access. The information and opinions expressed on this website or platform, or during discussions and presentations (both verbal and written) are not intended as health care recommendations or medical advice by Cancer Patient Lab, its principals, presenters, participants, or representatives for any medical treatment, product, or course of action. You should always consult a doctor about your specific situation before pursuing any health care program, treatment, product or other course of action that might affect your health. Meeting Notes SUMMARY KEYWORDS glioblastoma, patient, clinical trial, tumor, people, drug, treatment, cells, years, vaccine, good, radiation, talk, GBM, call, trial, pathway, question, working, vanessa SPEAKERS Al Musella (72%), Brad Power (12%), Vanessa Hugo (7%), Brian McCloskey (5%), Adrienne Nugent (2%), Ebrahim Nana (1%), Lisa Collman (1%), David Plunkett (1%) OUTLINE 1.Brain cancer treatment and patient advocacy. (0:03) 2.Developing a platform for glioblastoma treatment. (2:18) 3.Glioblastoma treatment options and research. (8:16) 4.Personalized cancer treatment options. (14:29) 5.New brain tumor treatments with promising results. (18:55) 6.Personal journey with glioblastoma and treatment options. (23:27) 7.Cancer treatment options and advocacy. (28:11) 8.Genetic testing for glioblastoma. (34:02) 9.Optune mechanism of action and potential for other cancers. (39:25) 10.Cancer treatment options and clinical trials. (43:56) 11.Brain cancer treatments and advocacy. (48:54) SUMMARY

•Co-founder of Cancer Patient Lab Brad Power introduces webinar on brain cancer, with guest speaker Al Musella sharing insights after decades of advocacy.

•Al Musella, president of the Musella Foundation, discusses the organization's vision for speeding up the search for a cure for glioblastoma, which involves collecting medical records, using AI to structure the data, and presenting treatment options to patients and doctors.

•The organization's virtual tumor boards bring together world experts to suggest treatment plans for patients, and the registry records the rationales for these suggestions to feed back into the AI loop.

•Al Musella proposes the "Promising Pathway Act" to provide easy access to experimental treatments for patients, including a conditional approval pathway for the FDA to approve drugs after they've shown safety and effectiveness in a smaller group of patients.

•He believes that this approach would allow for more flexible combinations of drugs to be tried, and would provide valuable learning opportunities for doctors and researchers.

•Al Musella prioritizes experienced surgeons for glioblastoma surgery and advanced genomic testing.

•He discusses the use of gamma tiles for glioblastoma treatment, including their effectiveness in recurrent cases and potential for combination with other therapies.

•He also mentions the importance of advanced imaging modalities, such as fractional tumor burden mapping, for identifying effective treatment targets and monitoring patient response.

•Vanessa Hugo discusses the challenges of accessing personalized cancer treatments, including the need for fresh dendritic cells for the DC-Vax vaccine and the requirement for the Compassionate Use program in the UK.

•Al Musella provides updates on the availability of the vaccine under the Special Access Program in the UK and the potential for importation from the UK once approved.

•Combining DC-Vax with Keytruda shows promising survival results for glioblastoma patients, with potential for further improvement with additional therapies.

•Al Musella presents research on Optune therapy for brain tumors, showing doubled 5-year survival in a randomized phase 3 trial.

•Combining Optune, with the new arrays and frequent use, and Keytruda, we may be able to get in the range of 50% 5-year survival, comparable to DCVax with a different mechanism.

•Al Musella discusses a new technology (sonodynamic therapy) using FDA- approved dye and ultrasound to kill cancer cells in the brain with minimal side effects.

•The technology involves repeating the treatment over time to target cells far away from the main tumor, with potential to treat glioblastoma.

•Al Musella shared his family’s experience with brain cancer, and the importance of serial MRI scans to monitor tumor growth or shrinkage.

•Al Musella's personal journey with brain tumors began when his sister-in-law was diagnosed in 1992, and he has since dedicated himself to advocating for patients and creating resources for them.

•He has learned that the key to success in advocacy is to be proactive, persistent, and adaptable, and to be willing to take risks and challenge the status quo.

•Al Musella discusses the challenges of clinical trials for rare diseases, includ including the risk of receiving a placebo instead of the actual drug.

•He discusses the need for legislation to help patients access treatments outside of clinical trials.

•Brian McCloskey discusses proton therapy for tricky places to treat, including glioblastoma, and the potential benefits and limitations of using radioligands for cancer treatment.

•Brian McCloskey shares his personal experience with glioblastoma running in his family and asks for insights on proton therapy and radioligands.

•Genetic testing for glioblastoma. 34:02

•Brian McCloskey asks if glioblastoma is a germline-based cancer, citing his aunt and son who also had the disease.

•Al Musella explains that sequencing is important for diagnosis and treatment, but there are different types of sequencing and only a small percentage of patients have actionable results.

•Vanessa Hugo mentions that xCures has a registry of 140,000 total patients, including over 1000 GBM patients.

•Brian McCloskey asks if liquid biopsies are relevant in the brain cancer space, and Vanessa Hugo replies that tissue-based biopsies are more common.

•Al Musella explains the mechanism of action for Optune, including how it disrupts cell division and exposes new antigens to the immune system.

•He highlights the importance of using immune checkpoint inhibitors in combination with Optune to enhance the immune response.

•Optune and sonodynamic therapy have potential to treat various cancers beyond GBM, including lung and pancreatic cancer.

•Lisa Collman asks about a new tumor treating field device called Voyager, which is in clinical trials but not yet available.

•Al Musella discusses a magnetic device in Texas that is more promising but still early in development.

•He expresses frustration with the clinical trial system and the lack of progress in developing a vaccine for cancer.

•Adrienne Nugent and Vanessa Hugo discuss the latest research on glioblastoma, including a Stanford study on familial GBM and a patient navigation program at Cancer Commons.

•Lisa Collman shares insights on hyper-mutated TMZ and glioblastoma, and the group discusses how to stay informed and learn from each other.

•Brad Power mentions the launch of an online discussion forum for a continuing conversation on brain cancer treatment ideas and questions.

•Al Musella suggests passing the Promising Pathway Act to get rid of problems and make treatment easier, with a follow-up session planned for when it's reintroduced in Congress.

•the data,

•the AI navigation program, and

•access to treatments. First, the data: This is the important part. It starts with a regulatory grade registry that xCures is running. All they need is your permission, and a few details about you, and they can collect all of your medical records from every doctor, every medical facility, and every MRI place. They collect thousands of pages of records on each patient, and the artificial intelligence percolates that data and structures it, theoretically. It's not perfect yet. That's why I say it's a vision. But we're getting close. It picks up the important pieces so you can, at a glance, see the entire history of the patient. You’ve got all the biomarkers, the treatments, and the outcomes. Then we have virtual tumor boards, where we present cases to some of the world's experts. Then we record the rationales for what they're suggesting to the patient. We record the experts’ suggestions. Some of them are here, right now. I'll explain that role.