The shocking part of this article is that only 10.7% of glioblastoma patients get surgery at a high surgery volume medical center. It has been shown that patients who have surgery at these high volume centers do better than those that get surgery at low volume centers.   There is a high bias against the poor, minorities and those with bad or no insurance.

This shows why our patient navigation program is so important.  

For the vast majority of brain tumor patients who get treated at smaller medical centers, it is absolutely imperative to get other opinions - like our patient navigation program. There are so many treatment possibilities now that it is very difficult to stay up to date on them all. 53 new cancer drugs were approved in the last year alone.  Any one of them can be used off label if you have the right genetic mutations.  Our team of neuro-oncologists and PhD researchers track them all and can help match you to the best drug combinations.

 This is very good news!   The drug Prozac, which is an old drug approved for the treatment of depression, OCD, Bulimia and panic disorders, has been shown in mice to help brain tumors!  They did a unique type of research to show that it helps patients with glioblastomas:  they looked at medical records to see which glioblastoma patients also happened to take Prozac (or it's generic equivalent), and how they did.

There was an increase in median overall survival of glioblastoma patients who also took Prozac of 545 days with Prozac and 318 without Prozac, This is a 71% increase in survival just by adding a repurposed drug, which has the side effect of helping with depression and mood!

The study is too small and not the gold standard randomized phase 3 but the drug is relatively innocuous, cheap, easy to get. Might be worth asking your doctor about it.   

This trial is for diffuse midline glioma and DIPG in patients 2-29 years old. They can be newly diagnosed or recurrent. It tests the drug Onc-201 with either Paxalisib or Panobinostat.   Onc-201 by itself has the best effect on these tumor types so far, but not good enough. Hopefully adding another drug will make them work better, and this is a great way to test the combinations. Nobody gets placebos, they are testing what is thought to be the two best drug combinations against each other.

 This drug should help most other treatments work better. The Musella Foundation gave a research grant to get this drug started a few years ago!  Your donations at work!

 We will be following this closely at the meeting and I will let you know the details after the meeting!

Delytact is an oncolytic herpes simplex virus that in a small trial showed amazing results in recurrent glioblastoma.  Japan conditionally approved it and the treatment is now available only in Japan. We have been trying to get access here in the USA but it most likely won't happen for a while.  It is an embarrassment that Japan and France (See our recent article on Onc-201 being approved in France) are so far ahead of the USA in giving approvals to drugs for serious diseases. We in the USA can not get access to the most promising treatments.  We are trying to fix that. Go to https://virtualtrials.org/activism.cfm#/1/ for details on our proposed law to fix this!

This is groundbreaking.   The Jaime Leandro Foundation for Therapeutic Cancer Vaccines created a new Personalized neoantigen vaccines and got permission from the FDA to distribute it under expanded access.  They just announced that they treated their first brain tumor patient!  If you are interested in getting the vaccine, go to their website at https://personalizedvaccines.org/

 They released data from the recurrent H3 K27M mutated diffuse midline gliomas.  This is a really bad prognostic group - amoung the worst of the worst with no effective treatment. They reported a 20% response rate with a 11.2 month duration of response.   There was only one serious adverse effect that may possibly have been related to the drug out of the 50 patients.    20% may not sound like much but it is a big step forward. Much better than 0%. And this is only the beginning.  Hopefully this is good enough to get FDA approval, then we will be able to experiment with combinations and find the right way to use the drug.  Onc-201 will be at the heart of any combinations and we should quickly be able to find ways to make it work better!

 Onc-201 is an experimental therapy for cancer - especially brain tumors - in the USA and as far as I know, is not available for sale anywhere in the world yet.    This article says that Onc-201 is now available - as in today -  in France, but they are a little vague on the mechanism.  It may be under some type of expanded access program but I am not familiar with the regulations in France.  If anyone has any more details, please join the discussion in our forum at https://forum.virtualtrials.org/forum/main-forum/1307-onc-201-in-france

 The article talks about the use of external control groups specifically for rare diseases such as Glioblastoma. and shows that the more prognostic characteristics that are controlled for the better the results.   I recently wrote about xCures' new xUTILITY program - which creates external control groups with all known prognostic characteristics accounted for!    This can be used both for single arm trials as well as randomized trials as described in the current paper!

Disclaimer: I am a founder of xCures, own stock in xCures and am a paid consultant for xCures!

 This was a study in the test tube and in rats and it showed that tumor treating fields (Optune)  has a strong effect against tumor cells.

 xUtility is a set of tools and a regulatory grade dataset of de-identified cancer patient data.  This can easily be used to create a synthetic control group for a clinical trial. This means we do not really need placebos any more!  By being able to match patient characteristics along with the biomarkers, a valid control group can be created. We could also go back and reanalyze phase 2 trials with matched synthetic controls to better see how they are doing. A major problem in the past was phase 2 trials results appeared great when compared to the median survival of all patients, then when they get into a phase 3 trial they fail.  The main cause is those patients in the phase 2 trial were not typical and would have lived longer even without the experimental treatment.  Now we can go back and create matched controls to really tell how the phase 2 trial went. This could avoid phase 3 trials that are doomed to fail - saving lives!  This is a major advance.

Disclaimer: I am proud to say that I am a paid consultant for xCures and own stock in xCures!

 This is a new treatment approach, and it looks great in the lab. Will let you know when human trials start!

 This is a very important paper that shows a large benefit for Optune in the elderly (defined in this paper as 65 years old or older)..   The headline and the abstract missed the main point.  Read the entire paper. Bottom line is that if Optune (plus Temozolomide) is used correctly, that is over 75% of the time (over 90% was used in a different article with even better results but 75% might be an easier target for the elderly patients), there is a very large benefit over Temozolomide by itself or the combination at less than 75% usage.    Look at Figure 3 and table 4. They show that for those patients who used Optune over 75% of the time, the median survival was 21.7 months, compared to Optune at less than 75% with 12.5 months.   Looking at some other numbers, At the 3 year point, there were 20 patients still alive.  2 of them were on Temozolomide alone without Optune. 7 of them also used Optune but at less than 75% compliance, and 11 used Optune over 75%.    At 3.5 years, 9 patients were still alive.  None without Optune. 3 with Optune under 75% and 7 with Optune over 75%.  At 5 years, survival was 0% in the control group and 15% in the Optune group.      Look at https://virtualtrials.org/newsarticle.cfm?item=6607 for the paper looks at all of the patients - not just elderly. It shows a major benefit for compliance rate of 90%.

    Of course it is too early to tell how well this works but I love the concept. They are trying to bypass the blood brain barrier by grafting abdominal tissue into the cavity left when removing a brain tumor.  This should allow chemotherapies to reach the tumor better.

 This is a new approach to leptomeningeal disease - which is when the cancer spreads to the lining of the brain and /or spinal cord.  This is usually a very bad sign, with an average of about 2-3 months survival after diagnosis and no effective treatments approved yet. I wish them luck!

 This shows why brain tumor patients are more likely to develop blood clots than people without brain tumors.  They do not suggest anything to prevent it but it may be worth talking to your doctor about it.  All brain tumor patients should know about blood clots. Go to https://www.cdc.gov/ncbddd/dvt/facts.html for a nice overview with symptoms and how to prevent it!  This can be life threatening so you absolutely need to know the warning siges:  Swelling, pain, tenderness and /or redness of the skin can occur in any body part but most commonly the back of the leg. Left untreated, blood clots can travel to your lungs (pulmonary embolism) - the signs of which are difficulty breathing, faster or irregular heart beat, chest pain or discomfort which gets worse with taking a deep breath or coughing, coughing up blood and very low blood pressure, lightheadedness or fainting.

If you have any of these symptoms, it is an emergency and you need to get it checked quickly. They are very treatable if caught early but can cause huge problems or even death if ignored.

 ONC-206 is a new experimental therapy, related to Onc-201.  The first clinical trial of Onc-201 just opened at UCSF for patients with newly diagnosed diffuse midline gliomas or recurrent high grade gliomas.  

 This survey is from our friends at Pinpoint Patient Recruiting.  They are trying to learn what brain tumor patients need, so they can help you better.  This is similar to the request we made last month but they changed the requirements. Now they are looking for patients diagnosed with a Glioblastoma within the last 24 months, or their caregivers.   You get $75 for doing the survey!

 This is a different way to use Avastin: superselective intra-arterial cerebral infusion (SIACI) after Blood Brain Barrier Disruption.  The "standard" way involves IV infusion every other week until progression. This new way is delivering the Avastin only where it is needed by using a catheter inserted through the groin and threaded up to the area of the tumor. They can map out which arteries feed the tumor and inject the Avastin directly into those arteries in the brain, after blood brainso the drug is delivered in very high concentration exactly where it is needed, much higher dose to the tumor than using the IV methods. And the rest of the body doesn't get exposed, or gets a very insignificant dose, so it theoretically shouldn't have the systemic side effects of Avastin.  The procedure to perform this does add a risk, but it is only 3 injections, compared to many more the standard way.  

As to results, they look promising. The abstract did not include any controls so I looked up the original Avastin trial for newly diagnosed glioblastoma https://www.nejm.org/doi/full/10.1056/nejmoa1308573  using the standard IV method, and that did have a control group. This is just a rough comparison, as the results vary widely by many factors so the numbers aren't directly comparable.

Control group:  PFS: 7.3 months  Overall Survival:  16.1 months

IV Avastin : PFS: 10.7 months  Overall Survival:  15.7 months

SIACI Avastin:  PFS: 11.5 months  Overall Survival:  23.1 months

This is a proof of concept.  I would love to see this method of delivery used with a customized set of drugs based on the molecular pathology report from the surgery.