They reported median survival of 20  months for recurrent glioblastoma.  84% alive 1 year after starting treatment.   This is unheard of and is a breakthrough in brain tumor treatments.  It was a single arm trial so no randomized controls, but we would expect 8-9 months.    It is approved in Japan, but we can not get it in the USA. I tried asking the company to open a trial or expanded access here and they do not want to yet, because of our regulatory system.  We need to change this now.   

 This is a potential game changer.   These authors looked for biomarkers that can predict success for Optune users, and found that a score composed of 3 biomarkers could differentiate between patients who do exceedingly well and those that do not. The difference is tremendous:   If your score was high, overall survival with Optune was about 82% at 45 months, compared to about 16% without Optune. 

Of course I would love for this to be validated in another group of patients  but the difference is so large we should consider it now when making decisions.   

If you have a high score, it makes sense to use Optune instead of a clinical trial that doesn't allow Optune use,  No clinical trial has ever shown the results this study has.

If you have a low score, either enter a clinical trial, or use Optune but add treatments that counteract the biomarkers..

These results were for Optune patients with poor compliance and were stll impressive. I would love to see the results with high compliance.

 We helped fund this project  (through the DIPG Collaborative).  It shows that a liquid biopsy of CSF or even Blood can tell if the patient is responding or not to Onc-201.  With DIPG it is hard to tell how the tumor is doing with just MRIs.  It also shows impressive results for Onc-201!

 Although median survival wasn't helped much, 10% were alive at 4 years and one was progression free at 5 years.

 Cancer Commons has been a partner of ours for our Patient Navigation Program for the last few years.  We are constantly improving the system and xCures has just completed the next genertion of artificial intelligence tools for us to use... they are in testing now but we will use them for this project which we hope can prove that our learning system can directly increase patient survival.   

We are looking for partners to help fund and run this exciting program. If interested, contact me (Al Musella, DPM at  musella@virtualtrials.org)

 We gave out 2 more research grants!    We already surpassed last years giving -  we gave out $430,000 in brain tumor research grants so far this year, and  $427,000 in 2021! Still not back up to pre-Covid levels - as we gave over $600,000 in each of 2018 and 2019, but we are building back up! Thank you for your help!

This is the publication of one of the projects we (the Musella Foundation)  helped fund.  The concept is perfect and elegant - select the best treatment plan for a child with DIPG based on the biological makeup of the tumor.  Each patient was reviewed by a board of experts and they decided on up to 4 approved drugs to use in combination to treat the kids.   This is similar to how our patient navigation program works except we do not limit ourselves to approved treatments.

 There was no improvement in survival for these kids comparing those that followed the recommendations and those that did not follow the recommendations.  . Overall survival was 13.1 months - which is somewhat  better than the accepted median survival for these tumors.  They did not compare to external controls, but both groups did better than expected. Perhaps the group that did not follow the recommendations were accepted into a clinical trial that is helping. 

They did learn a few things as noted in the article, which lays the groundwork for the next iteration of this project  Unfortunately they can not yet use treatments  like Onc-201 or DCVAX because they are not approved yet.  Hopefully that will change soon.

 Caruso Physical Therapy and Nutrition has taken over as director of this walk from Bruce Blount. Bruce did a terrific job for 11 years and helped us raise a lot of money which was used for brain tumor research!   This is one of my favorite events and I plan on being there!  The location is the Laurita Winery - a perfect setting for the event!  You will get to meet other families going through the same thing. If you can't make the event, you can still donate to it!

 This shows how stupid our current system is and the need for change.   The video in the link below is about families with kids fighting brain tumor who want to get an experimental drug that I believe will be the basis for a cocktail approach that will lead to a major breakthrough as soon as we are allowed to try combinations with it.    I get calls and emails from around the world requesting help getting access to it and most patients can not get it. There is really no approved alternatives.  Imagine being a parent of a child with an incurable disease and you know there is a treatment available that may help but you can not get it for your child.   That is my worst nightmare and keeps me up at night,

At an FDA meeting over 3 years ago, all of the data was presented about this drug - it helps by itself and had no side effect.  I asked (at about the one hour mark in the video) the FDA to approve it immediately. They did not - they wanted more trials. Meanwhile, kids who could benefit from this drug are dying.  The mission of the USA FDA is "The Food and Drug Administration is responsible for protecting the public health by ensuring the safety, efficacy, and security of human and veterinary drugs".  They are too conservative with their approach to terminal diseases.  Their policies are destroying families and they are not protecting our kids. The system serves only to prop up the huge expense of drugs and create monoplies by making it so expensive and time consuming to get a drug approved that many good drug candidates never make it into humans and only large companies can even try to get a drug approved.   Even worse - drugs that may be part of the ultimate cocktail approach may not get approved because they do not help enough on their own.  There are many examples.

 It is time for a change. I believe  the Promising Pathway Act is our best chance now. It is a proposed law in congress right now.  It will create a new pathway to FDA approval for drugs for incurable diseases where the approval is conditional - the drug company has 6 years to prove the drug works to get full approval or loses the conditional  approval. The approval will happen as soon as the drug is shown to be relatively safe compared to the currently used treatments, and has a biologic effect such as increasing survival, shrinking tumors on scan, or improving biomarkers.  This may mean conditional approval after a small phase 1/2 trial instead of a prolonged phase 3 trial.  After conditional approval, the drug can be sold and any doctor can prescribe it.  However, every patient who uses such drugs has to be followed in a virtual trial so we can see how the drug does and learn from every patients' experiences.   So this law would basically move most of the research from before approval to after approval, Doctors will have access to the ongoing results so they see how the drug does and which combinations are being tried and the results - which would let us home in on the ultimate cocktail quickly.    The key is knowledge  and research - this plan requires much more research and openness  than the current pathways. Under the current pathway to approval, the drug is only tested on highly selected patients (very few minorities, poor people, rural people, people with comorbidities and people who are in bad shape) before approval, then after approval there is no more research.  Nobody can get full access to the research. Parts are usually reported but not individual patient details.

 The Promising Pathway Act will hold down the cost of drugs for a few reasons:  the cost and time of getting FDA approval  is reduced by about 90%. There will be more competition as the barriers to approval are reduced, allowing us to try the best new ideas without having to raise a bullion dollars for each one we try.  

We need help getting this bill passed.  Right now it is stalled in Congress as other more "important" issues take precedence.  We tried writing letters.  It helps a little and we still encourage it at https://virtualtrials.org/activism.cfm#/1/. It may be time to call and visit your representatives - especially those running for office this year.   We have the support of many other foundations - mostly pediatric brain tumor foundations as well as pancreatic, ovarian and ALS foundations but need all brain tumor foundations to work together on this (or I am open if they have other ideas to get access to these drugs and increase research as Promising Pathway Act does.).

 Our copay program is open again.  If you think you may need help - apply. Do not feel bad about taking advantage of this program. Going through the brain tumor journey is stressful enough. Taking a little off of the expenses may help reduce stress and you will do better! 

 Roon is a new social media site dedicated to patients and families  dealing with high grade brain tumors.     We are considering partnering with them to run our online support groups.. so join it, test it out and let me know what you think of it!

 The GBM Agile trial is a new style clinical trial that screens multiple treatments against each other at the same time, minimizing the number of patients needed for a control group as each of the arms acts like a control to the other arms.    It does personalize by one biomarker - MGMT methylation status.  Currently, some arms only accept unmethylated patients, but most accept any methylation status.  It uses an algorithm to assign a patient randomly to any arm that patient is qualified for, but it is weighted to assign more patients to the trials doing the best and less to the trials not doing as well.   It quickly can tell if a treatment is worthy of graduating to the next level or not. If not, it is dropped from the rotation and another treatment added.    

Unfortunately for Kazia Therapeutics, it's drug, Paxalisib, which was tested by itself did not do well enough to graduate on, and was dropped from the trial.   This does not mean the end of the drug - it just did not perform well enough by itself in the specified circumstance of newly diagnosed Glioblastoma with unmethylated MGMT and recurrent GBM.  

As I have always been saying, I doubt if the cure is going to be a single magic bullet where a drug will work good enough by itself to cure brain cancer.  Instead, I think a rational combination of drugs will be needed and we need access to these drugs which is impossible (or difficult) under the current regulatory system.  This drug, Paxalisib,  is a PI3K inhibitor.  The PI3K pathway is one of the most important regulators of most cancers.  Unfortunately, targeting this pathway by itself is futile. The cancers that are driven by this pathway just mutate around it and use a different pathway when this is blocked.  However the cancers that were driven by this pathway are weakened when they can't use this pathway, giving other treatments a better chance of working.  There were two presentations about this drug being used in pediatric brain cancers, in combinations.     This was mostly in animal models but they presented two DIPG patients who used the combination of Onc-201 and Paxalisib under compassionate use and both demonstrated dramatic reductions in tumor volume (which is very hard to do with DIPG) and complete resolution of symptoms, with extended overall survival.   The combination is now being tested in a clinical trial.

 I love the concept of the GBM Agile trial, but I do not think we are at the stage where we have effective enough treatments to test.  I think what is needed is our "A Patient-Centric Platform Trial for Precision Oncology" to think up and try combinations that are personalized to each patient.  Our team of experts figures out a list of the best possible treatment options. The patient and their doctor decides which one to try. We try to help get access to it. Then we follow up on each patient to see how it works.  It is like a screening trial of combinations.  When we find a combination that works for a specific scenario, that combination should then be validated ina trial like GBM Agile - they would work together symbiotically.

 Two articles came out about this viral therapy that is approved in Japan for Glioblastoma.

The first is a phase 2 study reporting very promising results:   the 1 year survival rate for recurrent Glioblastoma was 84.2%. 16 of 10 patients were alive at the one year mark. This treatment is injected directly into the tumor up to 6 times.   As a result of this study, the treatment was approved in Japan with the name: DELYTACT .   I have been trying to get it for patients here in the USA with no luck but will keep trying!

The second article is the phase 1/2 dose finding study which had less impressive results: the 1 year survival rate was 38% but they did have 2 long term survivors.

 This press release talks about an article in the journal Science, which is behind a paywall so I can't get the details.   The press release says a team of researchers improved upon Temozolomide in such a way that the usual resistance mechanism won't work.   It sounds interesting.

  It appears that they did not take into account the reason why some people continued taking Temozolomide past 12 months - could it be they were in better shape so they continued? Or the opposite - Temozolomide stopped working so they stopped taking it.  This question can only be answered by a randomized blinded trial - I doubt that will ever happen because Temozolomide is generic and such a trial is expensive - and I doubt anyone would volunteer for such a trial.

 This study showed moderate activity in pretreated high grade gliomas.  May be worth considering regorafenib - especially if combined with something.  This drug is approved for other types of cancer but can be used off label for brain - although cost may be an issue.   No treatments really "work" well in this scenario, so it is impressive that this drug showed the results it did. Of course it is not good enough - which is why I say it needs to be combined with something.

 I recently wrote about the exciting results of the expanded access program for this therapeutic vaccine (Sitoiganap - which used to be known as Gliovac or ERC1671). Now the USA FDA granted them Fast Track Designation, which speeds up the USA process for approval!

 This video talks about a new way to reformat the DICOM images from an MRI scan.  The new views show the difference between treatment effects (Pseudo-progression) and tumor growth much better than standard MRIs. They also can show the best spot to do a biopsy, and tell much earlier if a treatment is working or not.   Sometimes these can be applied to the scans you already did (if they happened to do the correct techniques which they commonly do!). 

This is an update from a webinar we had on this topic last year! Well worth watching!

I love research like this. First they figure out how cytotoxic T lymphocytes kill tumor cells. Then they figured out how the tumor cells defended themselves against the T cells.  Then they figured out a way to suppress the defense mechanism, and made the T cells work dramatically better.   The problem is the defense mechanism is also used by normal dividing cells so in this case it might be difficult to create a drug to this vulnerability.  But they are working on it and think they can ultimately solve the problem! I wish them luck.