This will be a live event. Should be fun and will raise money for brain tumor research!

The Japanese version of FDA granted approval of this new treatment for Glioblastomas!   The clinical trial had amazing success for recurrent Glioblastoma.  We are trying to get access here!

 I wrote this Op-Ed about the Promising Pathway Act.  It is worth a read. We will be launching a letter writing campaign as soon as the House version of the bill is ready. The senate version was introduced a few weeks ago.

Very interesting concept. They did a mathematical model of numerous biomarkers to try to figure out which is best for an individual patient with newly diagnosed, methylated MGMT Glioblastoma: Temozolomide, Lomustine or the combination of the 2.  In general the combination was best for most but there are cases where one or the other drug alone was better than the combination.  Theoretically, they could predict which path to take, improving the effects of chemotherapy, and in some cases, minimizing unneeded side effects. It has to be tested in patients to make sure it works, but this is the type of research we need. Figure out why and in which patients treatments work instead of blindly using the same treatments on everyone.

 They announced an increase of 3-4 months in median overall survival. That sounds like nothing but for DIPG it is significant. It is very rare for a DIPG trial to show any benefit.  This could be the start of major progress against DIPG as part of a combination of therapies.

 These 2 grants are exciting. 

We did a webinar about the Gallium Maltolate project. See https://virtualtrials.org/video2021.cfm?video=202105 This is a new approach to treating brain tumors. They are trying it on Glioblastomas but it may work for most cancer types - not just brain tumors.

To be honest, I never heard of uORFs before this grant application came in. I had to research what it is, and found that they regulate gene expression and thereby can influence how much of a protein is made by the cell.  Dysfunction of the uORFs may be the underlying cause of many cancers, and very little research has been done into the connection between uORFs and cancer. The researcher chose to work on Group 3 Medulloblastomas because it is known that these tumors rely heavily on translational regulation. If this project is successful it may lead to breakthroughs in all cancer types.

We have worked with this organization for a few years. They have a survey for Glioblastoma patients and / or the caretakers, where they will pay you to participate. They also donate to the Musella Foundation!  Please participate if you can!

 We had high expectations for this treatment, and were shocked when it failed the clinical trial.  this article may explain why and opens the door to try again using a way to get the treatment into the brain - perhaps with focused ultrasound, or other ways around the blood brain barrier.

 I will be speaking tonight about the Promising Pathway Act.  It is difficult to get across how important this bill is in just a short text blurb, so I am going to give it the treatment it needs. I will start with a little history of the Musella Foundation, and the reasoning for our various programs, and how it all ties into the Promising Pathway Act. This bill is a game changer. It will quickly impact the lives of brain tumor patients if passed.  I thought the same about the "Right To Try" bill, which passed but turned out not to make much of a difference (except to the few lucky enough to be able to use it!). We looked at the drawbacks of the Right To Try law,  accelerated approval, as well as the expanded access pathway and helped to create a new pathway that solves the problems and hopefully can allow us to make quick progress in the fight against brain tumors - while increasing the amount of research conducted, getting access to more treatments and holding costs down!

 This may be a breakthrough in the treatment of Glioblastomas.  Teserpaturev is the new name for the treatment that we used to call G47 Delta.   In the clinical trial for recurrent Glioblastoma, they had a 92.3% one year survival rate, compared to historical rates of about 15%!   It should be available in Japan soon and we will work on getting it here!

 Of course 1 case report doesn't prove a treatment works but it is fantastic to see a long term survival with just one injection of CAR T cells!

 I think this is the perfect combination.  The original phase 3 trial of tumor treating fields (TTF) for recurrent glioblastoma did not show a benefit because the patients were too far gone - TTFs are slow and gentle, and need a few months to turn the tide and shrink the tumor away. Most patients in the trial did not have the time for TTFs to work.    GammaTile is implanted at the time of surgery, and slowly releases radiation to the tumor bed.  It possibly could buy the time needed for the TTFs to kick in. I will keep an eye on this trial.

 Metastatic brain tumors are about 10 times more common than primary brain tumors, but there has been a severe lack of research in these tumors.  In the past, once you had a cancer that spread to the brain, it was considered over and impossible to really do anything for it.  Times have changed and now that we have better ways to control those primary tumors, we also have better treatments for the brain mets. Some of these also apply to primary brain tumors. Our special guest speaker today is  Dr Tawbi, who is the co-director of the brain metastasis clinic at MD Anderson Cancer center!

 Most of the funds used for these grants were raised by our National Walks To End Brain Tumors. We had an actual live 5k walk in Florida and a virtual walk in MN. We have 2 virtual walks coming up in NJ and another in UT. See https://walktoendbraintumors.org/ for details. A special thanks to those volunteers and participants who made this possible!

For the grant from our DIPG All-in-initiative fund, some of the funding came from our partners in the DIPG All-In-Initative:  The McKenna Claire Foundation and the Prayers From Maria Foundation. 

 Unfrotunately, this trial failed to show any improvement by adding a Parp inhibitor to the standard treatment for MGMT Unmethylated Glioblastoma. It highlights the need to develop new treatments, as survival was only 1 year in both the experimental group and the standard of care group.

 I think this is one of the most important bills that can speed up our access to brain tumor treatments.  I helped write some of the words!    This would work perfectly hand-in-hand with our patient navigation program. Right now, our patient navigation program has brain tumor experts thinking up the best combinations of treatments for each individual patient. We give the patient and their doctors a list of 5-10 plan options, which we feel would be the best for them. Invariably, the best plans are hard or impossible to get, because they involve experimental treatments. We managed to get some of them under expanded access or under the right to try pathway, but that it too much of a barrier for widespread use. We need direct, easy access to these treatments.

Do not do anything yet. We will soon announce how to help.  Hate to ask - but we could also use some donations to help us with this. Make a donation at https://virtualtrials.org/donate11.cfm and be sure to specify "unrestricted" so we could use it for things like paying for the program that allows our members to contact congress easily.

 The MRI lecture was a little too technical at some points - just gloss over that as it all came together by the end, and showed how important this imaging technique is. It should be available at the larger centers. You can ask your doctors about it. They can not go back and reconstruct it from old images - you need to request it before the scan.  It can tell much sooner if a treatment is working or not, and if there is a tumor recurrence or necrosis / pseudoprogression. 

The clinical trial lecture was fascinating. The speaker goes through the history of the drug, explaining his thought process on how he chose this drug to try in people. He shows results in animals which are very good.  I think this treatment (Gallium Matolate) has the potential to make a big impact.

 Paresis is a muscle weakness. In this trial, they treated patients who developed paresis after surgery for a brain tumor.  The treatment is transcranial magentic stimulation, which is non invasive.

They showed a large benefit in a small randomized trial. This therapy is available now, so if you develop weakness after a surgery, ask your doctors about it!

 This trial is for Glioblastomas, Ependymoma or Medulloblastomas that have leptomeningeal spread.  Leptomeningeal spread is when the tumor cells spread via the cerebral spinal fluid to the lining of the brain and spinal cord.  It is a very bad sign. Doing a google search for it shows only completely hopelessness.  Most trials exclude patients with leptomeningeal spread because the outlook is so grim and nothing has really been shown to provide lasting benefit.  This trial is targeting these people with the worst of the worst tumors.  It is worth a try as there really aren't many alternatives. [Disclaimer: Mustang Bio is a sponsor of the Musella Foundation]

 We have 2 related topics tonight. We start off with a talk on how perfusion MRIs can be used to better understand it a treatment is working or not, followed by an announcement and discussion of a new clinical trial which will test a new type of brain tumor drug and use perfusion MRI to evaluate it! Should be interesting.