Sponsored by
The Promising Pathway Act 2.0 was officially introduced to Congress last week! If you haven't already, clink this link to contact your Congressional representatives in support of the bill! It takes 2 minutes.
We're proud to announce the following research grants:
1. $48,836 grant to Dr. Ekokobe Fonkem at Medical College of Wisconsin to fund the project: "Advancing Immunotherapy for Glioblastoma: Pre-Clinical Development of a Novel PI3K Inhibitor"
2. $35,000 grant to Dr. Monika Haack at Beth Israel Deaconess Medical Center to fund the project: "Computer modeling for tumor treating fields"
3. $10,053 to Dr. Samuel Singer at The Feinstein Institutes for Medical Research to fund the project: "Assessment of sST2 Staining in Tumor Tissue from Glioblastoma, IDH-Mutant High Grade Glioma and High Grade Meningioma"
Niraparib is a PARP inhibitor approved for ovarian cancer. It disrupts cancer cells' ability to repair DNA damage. In the Phase 0 part of this study, presumed newly-diagnosed glioblastoma patients were given the drug for 4 days prior to resection, and resected tumor tissue was then tested to measure drug accumulation. All Phase 0 patients (n=46) met the accumulation threshold. Of the 27 patients with MGMT-unmethylated tumors, 19 were enrolled in the Phase 2 part of the study, where they received niraparib during radiation followed by a maintenance phase of niraparib alone. Median overall survival was 20.3 months, which is significantly better than the historic median of only 12.3 months for unmethylated GBM. While this study was small, a larger Phase 3 study is being planned which will compare niraparib with temozolomide for newly diagnosed MGMT-unmethylated GBM (more here).
Preclinical research has shown that endogenous hydrogen sulfide (H2S) can act as a tumor suppressor against glioblastoma. Directly giving patients H2S is risky, so researchers at the Cleveland Clinic are exploring whether the body's natural production of H2S can be increased by reducing thyroid hormone levels. In addition to continued preclinical work, Dr. Hine and Dr. Peereboom will run a small Phase 2 trial to evaluate the safety and efficacy of using oral methimazole (drug approved to treat hyperthyroidism) in patients with progressive GBM. They'll also look at other potential ways to enhance H2S signaling in patients, aside from using thyroid hormone inhibitors.
This is very exciting! This partnership to develop renewable energy facilities will direct 90% of profits towards cancer research and services, focusing specifically on glioblastoma and diffuse midline glioma. We are honored to be a part of this project, and we will invite organizations and researchers worldwide to collaborate in applying these funds towards the most promising research when the funds start flowing in!
As noted above, we have a webinar on this trial on Wednesday. IMVAX expects to be able to share top-line results sometime in 2025, but we'll cover the latest status and updates during our webinar.
The Promising Pathway Act 2.0 will be introduced into Congress this Thursday. Before it gets introduced, we need to contact all of our legislators and ask them to cosponsor it. Use our form for details and a quick way to send emails to your senators and representatives! Then ask all of your friends to do the same!
Please do this even if you've already contacted your representatives on behalf of the original bill. We're closer than ever before, and garnering additional cosponsors will help push this bill across the finish line!
'Empowering Brain Tumor Patients' webinar this Thursday, May 23, 2024 (6pm EST) with Dr. Isabelle Germano, Dr. Kevin Elmore, and Dr. Stanislav Lazarev. This webinar is hosted by the Icahn School of Medicine at Mount Sinai, in collaboration with the National Brain Tumor Society and the Musella Foundation. The webinar can be accessed by visiting https://bit.ly/3UuvmRK.
Video recordings for all of our Brain Tumor Awareness Month Webinars are available here. Our final event for the series will be Wednesday, May 29, 2024 (7pm EST) IMVAX Phase 2b trial, Dr. Brad Zacharia.
Our next webinar is today! Monday, May 20, 2024 (2pm EST) ReSPECT GBM Phase 2 Trial, Dr. Norman LaFrance & Dr. Marc Hendrick, Go to virtualtrials.org/webinar to participate!
Our next webinar is tonight! Thursday, May 16, 2024 (7pm EST) University of Michigan high-grade glioma trial, Dr. Maria Castro & Dr. Pedro Lowenstein. Go to virtualtrials.org/webinar to participate!
The Musella Foundation became an official 501(c)(3) non-profit on May 12, 1998. Read more about the organization, its history and accomplishments HERE!
Go to virtualtrials.org/webinar a few minutes before each event to participate!
Tuesday May 14, 2024 (7pm EST) Duke Clinical Trials, Dr. Annick Desjardins
Thursday May 16, 2024 (7pm EST) Results from University of Michigan high-grade glioma trial, Dr. Maria Castro and Dr. Pedro Lowenstein
Monday May 20, 2024 (2pm EST) ReSPECT GBM Phase 2 trial, Dr. Andrew Brenner and Dr. Marc Hendrick
Thursday May 23, 2024 (6pm EST) Empowering Brain Tumor Patients, Dr. Isabelle Germano, Dr. Kevin Elmore, and Dr. Stanislav Lazarev (NOTE: This webinar is hosted by Icahn School of Medicine at Mount Sinai and may be accessed by visiting https://bit.ly/3UuvmRK)
Wednesday May 29, 2024 (7pm EST) IMVAX Phase 2b trial, Dr. Brad Zacharia
This is a very important publication. It shows that combining a personalized dendritic cell vaccine with an immune-boosting adjuvant can improve treatment efficacy. This Phase 2 study specifically looked at two different immune modulators - poly-ICLC and resiquimod - and found that poly-ICLC triggered a stronger immune response when given in combination with the vaccine. Although the trial was not designed to make survival comparisons, the authors reported noticeable differences between the study groups. For the IDH mutant/grade 3 cohort, all 4 patients who received the vaccine + poly-ICLC were still alive at the time of data cutoff (3 patients >10 years and 1 patient >9 years). For the grade 4 (GBM) patients, there was a trend towards improved progression-free and overall survival with the vaccine adjuvant compared to the vaccine alone, but the differences didn't reach statistical significance (likely due to small number of patients).
As part of the National Walk to End Brain Tumors, the 15th annual Minnesota Brain Tumor 5K is coming up on May 19th. This is a great event for everyone in the Minnesota area. All funds raised go directly to the Musella Foundation and will be used exclusively for brain tumor research!
Very exciting news! DB107 is the new name for a previously tested 2-drug combination (Toca 511 + Toca FC). The Musella Foundation was an early supporter of the treatment via grants, back when the drugs were owned by a company called Tocagen. Early trials did well. The large Phase 3 trial failed, but there was a small subset of very successful responders. At first, nobody knew why only some patients benefited, but the company that now owns the drugs (Denovo) discovered a new biomarker they believe accounts for the favorable response. Based on this discovery, new trials will be launched to confirm the treatment helps patients who have the relevant 'DGM7' biomarker. This large grant will help fund the trials, and we hope to see enrollment opening soon!
To watch our GBM AGILE trial webinar from last week, click HERE.
Coming up next:
Tuesday May 14, 2024 (7pm EST) Duke Clinical Trials, Dr. Annick Desjardins
Thursday May 16, 2024 (7pm EST) Results from University of Michigan high-grade glioma trial, Dr. Maria Castro and Dr. Pedro Lowenstein
Monday May 20, 2024 (2pm EST) ReSPECT GBM Phase 2 trial, Dr. Andrew Brenner and Dr. Marc Hendrick
Thursday May 23, 2024 (6pm EST) Empowering Brain Tumor Patients, Dr. Isabelle Germano, Dr. Kevin Elmore and Dr. Stanislav Lazarev (Hosted by Icahn School of Medicine at Mount Sanai)
Wednesday May 29, 2024 (7pm EST) IMVAX Phase 2b trial, Dr. Brad Zacharia
Check out our upcoming webinars!
Wednesday May 1, 2024 (7pm EST) GBM AGILE (Adaptive Global Innovative Learning Environment) trial, Dr. Timothy Cloughesy
Tuesday May 14, 2024 (7pm EST) Duke Clinical Trials, Dr. Annick Desjardins
Thursday May 16, 2024 (7pm EST) Recent results from University of Michigan high-grade glioma trial, Dr. Maria Castro and Dr. Pedro Lowenstein
Monday May 20, 2024 (2pm EST) ReSPECT GBM Phase 2 trial, Dr. Andrew Brenner and Dr. Marc Hendrick
Wednesday May 29, 2024 (7pm EST) IMVAX Phase 2b trial, Dr. Brad Zacharia
We are also partnering with the Icahn School of Medicine at Mount Sinai for a webinar on Thursday May 23 - details coming soon!
From our friends at the Pediatric Brain Tumor Foundation. We need help encouraging NY state legislature members to support The Pediatric Cancer Neuropsychological Needs Assessment Act (S.8750). This bill will add pediatric cancer to the list of disabilities covered by insurance so that children with brain tumors will be able to qualify for Individualized Education Programs (IEPs) to help them thrive and succeed at school. For more information, please reach out to patientadvocacy@curethekids.org.
Experts at the University of Michigan are investigating a two-pronged attack for treating high grade glioma. Their Phase 1 first-in-human trial tested two adenoviral vectors (HSV1-TK and Flt3L) injected directly into the tumor bed after resection. Of the 18 patients enrolled, 14 had gbm, three had gliosarcoma, and one had anaplastic ependymoma. The treatment was well-tolerated, and median OS was 21.3 months. The 2-year survival rate was 40%, with seven patients living beyond two years, three patients beyond three years, and one still living 60 months after enrollment. Study investigators believe this treatment approach may be even more effective for IDH1-mutated patients. We'll be doing a webinar on this exciting new treatment on May 16 (see above)!
Last week, FDA granted accelerated approval for tovorafenib (brand name OjemdaTM). This is great news for pediatric low-grade glioma (pLGG) patients and families. FDA had approved another drug combo (dabrafenib with trametinib) for BRAF V600E-mutated pLGG last year, but OjemdaTM is the first approved drug for both BRAF mutations and fusions. Early research for this drug was supported by the Team Jack Foundation in Nebraska, and it was brought to market by Day One Biopharmaceuticals - congratulations to all who contributed!
