I (along with my team:) published an article in the journal: "Artificial Intelligence in Medicine".  It is about our brain tumor virtual trial. We compared our data to the data from 7 randomized trials around the same time and found that we could have predicted the outcomes in 6 of the trials by using our data. For the 7th trial that was close but not perfect, we went back to look why we missed - we had different patient populations - they had recurrent GBM patients who never had chemotherapy before which is really strange. All of our patients failed Temodar up front, so when we looked at recurrent patients, we got a different readout.   Bottom line, what we did for basically free probably could have replaced trials costing millions of dollars.

 xCures is running an expanded access program of this drug for any type of cancer that has  MAPK pathway-altered solid tumor(s), including but not limited to KRAS, NRAS, HRAS, BRAF, MEK, and ERK mutations who have incomplete response to or have exhausted available therapies.    Low grade gliomas have a high chance of having this mutation. Glioblastomas, Pancreatic Cancer, Colon cancer, Ovarian and more types of cancers also sometimes get this mutation. Check your pathology report for it.

 At this time of year we like to reflect back on the year so far and share the highlights with you!  I also need to ask for donations.   The highlights letter linked below is part of a package we send out once a year by mail  to all of the people who have donated to us in the past - where we also ask for more donations!

I hate to ask our members for donations - I understand most families dealing with brain tumors have enough problems, but if you are in a position where you can make a donation - please do!  Ask your friends to donate in your honor!  If you can't - do not worry about it - I understand.

Have a Happy Thanksgiving!

 This is completely unacceptable to the brain tumor community. The FDA is taking it upon itself to use it's regulatory flexibility to change the way accelerated approval works. The article hints that this will add more than 3 1/2 years to the process of getting approval.   The cause of this change seems to be a mistake they made with approving a drug for Alzheimer’s disease.   There are a few drugs that should have already received accelerated approval for brain tumors and I am being told the FDA told these companies they couldn't even apply yet, and that they want a final phase 3 placebo controlled trial fully enrolled before the FDA would accept an application for accelerated approval. This will kill thousands of patients needlessly and reduce the number of treatments available to us.  This is the same FDA that told me they do not have the regulatory flexibility to grant accelerated approval to a treatment that has been shown to work well in phase 3 trial, but only in certain subgroups that made sense.  They wanted a repeat of the phase 3 randomized trial in those  subgroups, which makes perfect sense from a scientific viewpoint but wound up making the company go out of business, forcing us to lose the treatment forever, and probably killing 1,000- 2,000 brain tumor patients a year needlessly.   

I am working on a fix. I invite other foundations who are also interested in this to contact me.  My original plan was the promising pathway act, which would create a new conditional approval pathway to allow treatments to get approved after a phase 2 trial, but require all patients who use the drugs approved under this pathway to participate in a virtual trial until efficacy was proven, or failure was shown.   That bill is still in Congress - so it has a chance - but I have thought of another possible way forward. Change accelerated approval to require all patients who use drugs approved by the pathway to participate in a virtual trial, instead of requiring the confirmatory trial. This would go on until we either proved the drug works or not.  This would give us more knowledge as we learn from every patient.  

 The Children's Brain Tumor Network (CBTN) is a multi-institutional international clinical research consortium created to advance therapeutic development through the collection and rapid distribution of biospecimens and data via open-science research platforms for real-time access and use by the global research community.  We are proud to be a foundational partner!

Impressive results for newly diagnosed GBM with a "stock" vaccine - this is not custom made for each patient but instead targets Survivin, which is overexpressed in over 95% of GBM patients.  This is now in clinical trials for newly diagnosed GBM as well as children with progressive or relapsed brain tumors including GBM, AA, medulloblastoma,  Ependymoma and others. For DIPG, the trial accepts non relapsed DIPG (After radiation).  

 This is a must see by anyone involved with brain tumors. DCVAX is an experimental vaccine therapy. The results of the phase 3 trial were published recently Excellent results in newly diagnosed as well as recurrent GBM. Dr Liau is the co-inventor of the vaccine and ran the clinical trial.  As always, our webinars are free, however donations are appreciated. If you make a donation (by clicking the DONATE button on the webinar page), mention in the comment you want the donation to be used for vaccines only and we will use 100% of your donation to help speed up the approval of vaccine therapies.

This is the results of our Onc-201 expanded access program for DIPG and DMG.  I am one of the authors! The results are outstanding. We compared patients in our registry with DIPG and DMG who took Onc-201 compared to those that did not take Onc-201 but instead did other treatments including clinical trials.  There was a significant increase in median survival, but more importantly there is a long tail of about 25% of the patients went on to do well, whereas those without Onc201 all died.  And this is only the start.  Once it gets FDA approved, we will be experimenting to find the best combinations and hopefully move that tail up to 100%!  There were a lot of interesting reports of treatments at SNO this week that might be amenable to combining with Onc-201, but the key is getting the approval from the FDA so we can try these combinations!   

The poster also shows that immune checkpoint inhibitors do well if used as part of the treatment plan. Trial after trial of them alone showed no benefit, but in our registry we looked at patients who used immune checkpoint inhibitors vs. those that did not, and those who used them had a much higher tail!  We just need to learn how best to use them, and our (Musella Foundation, Cancer Commons and xCures) registry  / patient navigation program is going to be the fastest way to figure it out!

 I am very excited about this treatment.   This is the first trial to ever report extended survival in both newly diagnosed and recurrent Glioblastoma.  For newly diagnosed Glioblastoma, 5 year survival more than doubled  from 5.7% to 13%.  And for recurrent Glioblastoma, survival at 20 months and 30 months also doubled, from 9.6% to 15.7% at 24 months and from 5.1% to 11.1% at 30 months.  This is all with a few simple injections which had no side effects. 

 Early results but look pretty good. We did a webinar about this trial recently, it is worth watching. This is a new way to deliver radiation directly to where it is needed.

 This is a new approach that targets the tumor microenvironment.  See their website for details!  This trial is early, but has very impressive results in a bad patient population - those who have MGMT unmethylated.  90% of patients had tumors shrink, and 40% had a partial response. The median survival has not been reached yet as the patients are still alive after average follow up of 7.9 months.  There is a link to the poster presentation as well as a webinar they will be having about it soon, in the press release linked below!

 This article talks about the results of the DCVAX trial in plain english!  

 Optune is the most effective treatment approved for Glioblastoma. By far.  Yet it is still underused, mostly due (I think) to patients making the decision at a time when it has not yet sunk in how bad this disease is.They do not want to shave their heads and wear a device constantly. When they finaly realize, it is too late.  Another reason is lack of familiarity of the doctors with tumor treating fields.  It is a new concept to them, and the older doctors remember a fake electric device that was popular in the 1900s which did not work and they associated Optune with that.

There is a tremendous amount of research on tumor treating fields.  There are 25 research projects on Optune being presented at the SNO meeting which is going on now.  All of them reinforce the importance of Optune in the treatment plan for Glioblastoma and show how to make it even more effective.  One of the keys is using Optune the right way - which is with at least 90% or more complaince. It only works while it is on, so you give the tumor a chance to recover when you turn it off too long.

I think the ultimate cocktail that has a chance to be a cure is going to involve Optune, a vaccine, a checkpoint inhibitor and one or two other therapies.  There are many other treatments being presented at SNO this week that might be able to fill in the gaps and make it so most Glioblastoma patients become long term survivors, and not only survivors - but survivors in great shape. This is within reach now for the rich and hopefully for everyone next year..  We are trying to speed that up!

 This is the peer reviewed publication of the DCVAX trial that we have been waiting for!   Same excellent results that we reported in May but they go into details such as how the external control arm was constructed to account for all of the variables including the fact that the DCVAX trial rejected patients who progressed quickly during radiation.  I feel the article responds very well  to every critisism I heard from the May report..  And it was published in a high impact Journal - JAMA Oncology which has an impact factor of 33, where anything over a 10 is considered "excellent"!

 One of these posters will present the results of our expanded access program for Onc-201 in DMG/DIPG.    I am an author.  If you are at SNO this week, look for the poster:

BIOS 03 Real world clinical outcomes of patients with diffuse midline glioma in a longitudinal outcomes registry.  If not going to SNO, I will post the poster in the next news blast as it is embargoed until Friday!

The other poster is EPCO 10: Systems biology based therapeutic predictions with gbmSYGNAL and clinical correlates in the real world longitudinal outcomes registry XCELSIOR which is about our registry and some of the neat things you can do by just analyzing the data it contains!

 This is the abstract for Dr. Linda Liau's presentation of the DCVAX trial results at the SNO meeting this weekend!  The trial was a major success for both newly diagnosed and recurrent glioblastoma! I will have more details after she presents it!

I hate asking our members for donation - I understand most families dealing with a brain tumor have enough financial stress that I don't have to add to it.  However, this is a simple and free way to help us raise a few thousand dollars!  Just vote for the Musella Foundation on Facebook, Instagram and Twitter in this contest!

 Of course, if you aren't having financial stress - donations are appreciated!  We are held back from doing even more by lack of money.  

And don't forget to use smile.amazon.com when you buy from Amazon this holiday season.   We get a small % of your purchase amount and it doesn't cost anything!  Just select "Musella Foundation" as your charity.  We also participate with Walmart's Round Up program. When you buy something at Walmart.com, it will ask if you want to Round Up for charity.  It will cost you less than a dollar, but they add up quickly since so many people buy from Walmart.com.  Just make sure to select "Musella Foundation".

Having said that - we do send out one mailing a year to all of our donors, telling you about our accomplishments for the year and asking for another donation.  Of course - if you are stressed,  ignore it! We serve everyone the same regardless of if they donate or not.   I will send the list of accomplishments out next week. We have a huge one coming up this Friday!

 The Musella Foundation has been a foundational partner of the DIPG/ DMG Collaborative for the last 6 years and we have helped them fund over $15 million dollars of research grants. All of them are special and important but this one is extra special to me.  Dr. Marcus has been working on Sonodynamic therapy for 20 years and has been able to cure mice with it.  He finally got to try it - with success - on adults with Glioblastoma recently and tried it on one child with a DIPG. He came to the Collaborative to ask for funding to launch a clinical trial in kids with DIPG and DMG.  In this video, he doesn't know that his request was approved - we spring it on him!  This is just 8 minutes long - worth watching!

The guest speaker on this episode of xCures' "Target Cancer Podcast" series is Dr. Siddhartha Mukherjee.  He is a Pulitzer prize winning author (and my favorite author), researcher and Harvard trained cancer physician at Columbia Univerisity Medical Center in NY.  He has interesting new ideas about how diet (precision nutrition) can be used to help treat cancer.   Forget trying to reduce sugar. His approach is to figure out which essential ammino acids the tumor needs more than the normal cells, and then provide a diet that minimizes those amino acids.He co-founded Faeth Therapeutics to test his ideas. He is using xCures to help run a clinical trial of precision nutrition for the treatment of metastatic pancreatic cancer.    

This is not directly related to brain tumors but if it works, we can apply the same concept to all cancers.

Disclaimer: I am a co-founder of xCures and am a paid consultant.

 MTX110 is a reformulation of Panobinostat, which is an oral medication approved to treat multiple myeloma.  In the test tube, it worked great against brain tumors (actually tested as the most promising anticancer agent of all 84 drugs tested) but unfortunately it doesn't cross the blood brain barrier well.   Midatech Pharma reformulated it so that it could be injected directly into the tumor using CED (Convection Enhanced Delivery). There are ongoing trials using it for DIPG and Meduloblastoma.  This is the first time it is being used for recurrent Glioblastoma.