The survival curves for this clinical trial are very good, and this was for people with inoperable tumors which makes it much more impressive.    The concept is they give a dye - 5-Ala - to the patient. The dye gets taken up by tumor cells much more than normal cells.   This dye is FDA approved as Gliolan for use during surgery so the surgeon can tell tumor from non tumor.  However, they found that when the dye is excited by light (or by ultrasound when used with Sonodynamic therapy), it kills the cells that take up the dye and leave the normal cells mostly alone.  They use fiber optics to deliver the light through a small bur hole in the skull.    We do not yet have results for sonodynamic therapy but they have to be compared to this to see which way is better. Sonodynamic therapy is non invasive, and has the advantage of being able to treat  large areas of the brain, wheras photodynamic therapy requires direct access to the area.

This is an exciting development in the fight against brain tumors. While previous attempts at using Car-T cells against one target have shown some success, they fell short of the desired outcome. However, the new plan to target four different targets simultaneously holds great promise. In other types of cancers, Car-T cell therapy has led to cures, not just modest improvements in survival as seen in most brain tumor trials. Brain tumors pose a unique challenge due to their heterogeneity; some cells may have that one target, while others might not. This means that targeting only one antigen is not enough, as the remaining cells without the antigen will continue to grow the tumor. By targeting four different antigens, the hope is that all tumor cells will have at least one target, leaving no way for the tumor to grow back. For more information on how Car-T cells work, check out the video in the link below. The Musella Foundation, in collaboration with other pediatric brain tumor organizations, provided a grant to Dr. Vitanza at Seattle Children's to support early work on this project through our "DIPG All-In-Initiative."

 Of course it is only in mice so we can't get too excited about this just yet but it is an interesting new way to deliver treatments to tumors.  The two components they used sound logical and if it isn't as effective in people, they can try other combinations using the same delivery system.  

 The Musella Foundation gave the researchers a grant about 9 years ago for the early work on this new drug!  They have trials for newly diagnosed glioblastoma and recurrent high grade glioma, and this new study is trying an oral form of the drug (it is also being used IV).  There is also an expanded access program for H3K27 mutant diffuse midline glioma including DIPG.

 From our friends at UCLA!   This is always a great conference and there is live and virtual sessions available!

 I am participating in the Society of Neuro-oncology Community Neuro-Oncology Committee.  .   The majority of brain tumor patients are treated at community centers rather than major academic medical centers.  We want to make sure that these doctors have the support they need to offer best of care to their patients.  To start, we are conducting a survey to identify these doctors and create a directory.  The next step is facilitating networking opportunities with other community doctors as well as those from the major academic centers. The goal is to make sure all patients have access to excellent care.    So if you are a community doctor who treats brain tumor patients, please fill out the survey below.  If you are a brain tumor patient that is being treated at a community center, ask your doctor to fill out the survey!

   For those of you with Optune who travel, it may be worthwhile to bring a copy of this article in case the TSA gives you any trouble!

I love how responsive Novocure is to help in siutations like this.  

 This is a new modality of treatment - antisense oligonucleotides. It worked for spinal muscular atrophy - which proves it can get and stay in the brain and spinal cord and make a big difference.  They are now doing preclinical work on DIPG.   We (The Musella Foundation) gave him his first grant to get started with DIPG back in 2016! Hopefully this will not only make a difference in DIPG, but the technology could be altered to work on most mutations. 

 I know this sounds crazy but it is possible that it works.  The xCures team  (I am a consultant to them) are working on a similar project for Pancreatic cancer. They are running a clinical trial (https://clinicaltrials.gov/ct2/show/NCT05078775) of a specially designed food that removes a few amino acids.  Too early to tell how well it works but I hear interesting things.  https://faeththerapeutics.com/  created the program. 

 The Optune Open Houses were popular before the pandemic and they are now coming back, starting in Depew, NY.   It is a free dinner meeting where people who are using (or thinking of using) Optune can get together to share their experiances with each other and to learn about Optune.  

 We have run out of funding and the copay program will remain closed to new and renewal application until we get enough donations to the program to reopen.  Of course, we will continue to pay claims for those that have an active grant!

 Our Copay program  already gave out grants to 152 patients this year which puts us on pace for our best (or worst - as it is criminal that our program is even needed) year ever.  Our best year previously was 2021 in which we gave out 311 grants.   

Unfortunately we are running low on funds and will have to close to new and renewal applicants soon. We do not know when or if the program will reopen as we work on donations. This is a life saving program, as many of the recipients tell us they wouldn't take the treatments without our help.  If you would like to help, make a donation and select "copay fund" for where it is to be used!

We have our 5K fundraisers coming up in May - but those funds are dedicated to brain tumor research only. We will award brain tumor research grants in June.  (Researchers can apply now - contact me for details).

 We (the Musella Foundation) helped fund this first in kids project.    It was intended to see if it was safe enough to use this treatment on kids - and they found it is. 3 of the 8 patients who received the single CED infusion treatment had at least stable disease for a time. One patient was still alive at the end of the study at 22 months.  These were heavily pre-treated patients who had failed an average of 3.5 lines of therapy before trying this treatment.  Although this group of kids did much better measured from first recurrence to death than the average survival expected, it is hard to tell how much of that is due to this treatment as they had so many other treatments as well.  More research is needed.

 The experimental drugs Onc201 and Onc206 work on this pathway.   We need to get them approved so we could use them in combinations.

 This is in a small group of patients but very promising!

 This experimental drug is available via expanded access through xCures (I am a paid consultant for xCures) https://clinicaltrials.gov/ct2/show/NCT04566393

 This shows the importance of having genetic testing done on your tumor.  The BRAF V600E mutation is rare, but when found, this drug combination is useful.  This mutation is more commonly found in kids with low grade brain tumors, but also sometimes in adult glioblastoma. Although approved for kids with low grade tumor, it can be used off label for adults and kids with high grade tumors when they have this mutation.

 This drives me crazy. About 20% of the patients have a major response and at least 1/3 of the patients are helped somewhat with onc-201 alone. This is for dmg and dipg, where no drug has ever shown a response before. The logical next step is to try the combinations we think will make it work for most patients. There are small trials underway to slowly test a few combinations. However, instead of concentrating on making it work better, they decide to waste years, lots of money, and kill patients with a literal sugar pill placebo controlled trial. Shows a complete lack of understanding of what is needed. Drugs like this should be fda approved so every patient can get easy access and we can learn how best to use it.