Latest News

• Musella Foundation Awards Research Grant        

  We're proud to announce that we've funded a $50,000 grant to Katherine Onk and David Needham at DNKO LLC to fund the project: Effective Delivery of Doxorubicin to Invasive Margins in GBM: Establishing Optimal Warming Criteria for a Thermal Sensitive Liposome with Laser Interstitial Thermal Therapy (LITT) in a Porcine Brain Model. We only partially funded the project total amount of $322,000 and are saving up for the rest. 

  
  
• FDA Expands Orphan Drug Designation for SurVaxM, Immunotherapy Developed at Roswell Park        

  SurVaxM is an off-the-shelf vaccine that stimulates an immune response against a tumor cell protein called survivin. The Phase 2b trial for newly diagnosed adult glioblastoma is ongoing (no longer recruiting), and there's also a Phase 1 trial currently recruiting for children with relapsed medulloblastoma, high grade glioma, ependymoma and newly diagnosed DIPG. SurVaxM received orphan drug status for treatment of adult GBM back in 2017, but the status has now been expanded in scope to include any malignant glioma in children or adults. This designation, given to drugs aimed at treating rare diseases, helps encourage drug development through incentives like tax credits and extended market exclusivity, but it does not speed up drug approval. We'd love to see an expanded access program offered for SurVaxM, but the cost would be significant. If any organizations or individuals are interested in partnering to help fund such a project, please let us know!

  
  
• No benefit from TMZ treatment in GB with truly unmethylated MGMT promotor: Reanalysis of the CE.6 and the pooled Nordic/NOA-08 trials in elderly GB        

  MGMT promotor methylation status in glioblastoma is usually determined by molecular testing of tumor tissue samples, but testing methods and cutoff values vary across institutions. This study re-examined data from previous clinical trials of elderly glioblastoma patients using a strict definition of "unmethylated" (added safety margin below a main cutoff point to define "truly unmethylated" MGMT promotor) to determine the effectiveness of temozolomide (TMZ). Unsurprisingly, results showed that patients with truly unmethylated MGMT promotor status did not benefit from TMZ. This study concludes that validated, assay-specific methylation cutoffs should be applied in clinical trials and that better stratification of MGMT methylation status in clinical management will reduce toxicity without compromising outcomes for patients.

  
  
• Application of Delta T1 maps for quantitative and objective assessment of extent of resection and survival prediction in glioblastoma        

  This study evaluated a new MRI technique called delta T1 (difference between pre- and post-contrast T1-weighted images) to measure extent of resection and residual tumor volume. The technique provides an objective way to routinely assess surgical outcomes for glioblastoma, and delta T1 residual tumor volume of greater or less than 5 cm3 could be a potential metric in predicting patient survival.

  
  
• Exhaustive in vitro evaluation of the 9-drug cocktail CUSP9 for treatment of glioblastoma        

  The CUSP9 protocol is an experimental treatment approach for recurrent glioblastoma that combines 9 repurposed drugs with low-dose continuous temozolomide (TMZ). While early studies showed the protocol can be safely administered under careful monitoring, researchers recently tested all subset combinations of the 9 drugs, in combination with TMZ, on two clonal cultures of glioma-initiating cells from patient samples. Results showed that several subset combinations produced equivalent effects to the full protocol. This study highlights the importance and feasibility of personalized treatment approaches based on functional testing. We are following research in this area closely!