5-ALA is FDA approved in the USA as an optical imaging agent to be used during surgery for high grade gliomas.  It allows the surgeon to better tell the difference between tumor and normal brain..  This study shows that it is safe and useful even in the elderly and for both attempts at complete resection as well as for biopsies. It has been used as standard of care in Europe for a decade before it was approved here in the USA a few years ago!

 Pretty good results. I think this type of treatment is going to be part of the ultimate cocktail "cure".

   This is a new way to tell from an MRI scan (before biopsy) if a meningioma is high or low grade.  Most meningiomas are low grade but it is important not to miss the high grade meningiomas.  This can lead to more confident decisions on waiting and watching, or using radiation witout a biopsy.  Excellent work!

 This is results of a small trial of an experimental vaccine therapy for recurrent Glioblastoma:  Seven out of 10 (70%) of  patients were alive at 12 months, compared to historical controls of  30%. This is a major improvement. They will start a randomized trial soon.

 Fascinating statistics.  They looked at all Glioblastoma patients that were entered into the SEER system.  Over 38,000 patients, to see if survival improved over the years.   They found a small increase in survival from 2000 to 2011. but no increase since then.   The eye opening statistic is in the most recent years studied, 2015-2017, the average survival was only 11 months, with the 12 month survival of 45.7% and 2 year survival of 19%.

   This is in sharp contrast to all of the recent clinical trials results, where even the control groups have been reporting better results.  In the Optune EF-14 trial,(the best randomized  phase 3 trial results ever reported for  Glioblastomas),  the control group survived 16 months on average, and the Optune group survived almost 21 months.  To me, this means that most clinical trials do not reflect the average patients.  Only a small % of patients enter clinical trials, and they tend to be the patients with the best prognostic indicators.    The average patient does not enter trials, and we can't even tell if the clinical trials can be generalized to the average patient.  Others may say just entering a clinical trial improves your survival, which may be true, but I think it is more likely that hospitals that run clinical trials tend to have better results even outside of the trials.  So just by selecting a major medical center to treat you  improves your chances of survival.  

This points to the need to track all patients and see how treatments work in the majority of patients, not the special few that participate in trials.  The "Promising Pathway Act" bill will accomplish that.

 Correction added 12/3/21:   the statistics for the EF-14 Optune trial are incorrect. That is the time from randomization to the time of death, not time from diagnosis.  There was an average of 3 months from diagnosis to randomization, so the actual numbers I should have used were control group is 19 monhs survival and Optune group 24 months!

This is our annual fundraising appeal!  We are trying to speed up the search for the cure, and frankly - lack of resources is slowing us down. This is the most exciting time ever in the brain tumor world. Everything is finally coming together - the basic science research is finally figuring out how things work.  There are promising treatments in the pipeline, and an abundance of drugs being approved for other cancers that we can use off label.  We created a learning network to take advantage of all of this. We are working on legislation that will drastically speed up the process. 

But we need your help!  Please make a donation and get your extended families to donate!

 These are my thoughts on the fastest way to screen new combination therapies for brain tumors!

 This is the drug Onc-201 which we have been writing about a lot. It is for H3K27M mutant brain tumors, usually found in pediatric DIPG, DMG and some Glioblastomas (check your pathology report).  A recent presentation at SNO reveals that for recurrent DMG, it helped at least half of the patients, with 20-30% having shrinkage of the tumors. It doesn't help everyone but by itself I have seen it perform miracles on a few kids. 

France now offers it for free to patients who need it.  There are clinical trials going on involving this drug, and there is an expanded access program for it in the USA.

For this type of tumor, recurrent H3K27M mutant  Diffuse Midline Glioma, there are no good historical controls - as it was only recently created as a new classification of tumor.  It is thought to be worse than a regular Glioblastoma. Historically, recurrent glioblastomas has an average survival of 6 months (which can be read as 50% survival at 6 months)  and we would expect recurrent H3K27M mutant  DMG to be less than that.  This report showed that with Onc201, 57% of patients were alive at 12 months and 35% alive at 2 years – more than doubling what I feel is the correct control.   Objective response rates are hard to determine in this area - but they reported 20% response rate including one complete response, using one grading system, and 26% using a different grading system.   Doesn't sound like much but there is nothing that has been working for this tumor type.. the jump from 0 to 20 or 30% is major, and I think should be good enough to get FDA approval. Once it gets approved, we can try combinations of treatment and find the best way to use it and significantly improve those numbers. The DMG-ACT trial recently opened which will try a few combinations.

We funded and helped run the first expanded access program for Onc-201 along with Cancer Commons, xCures, The Cure Starts Now foundation, the Michael Mosier Defeat DIPG Foundation, Dragon Masters Foundation and the Finn Family Foundation.  Some of the patients from our program were including in this analysis.  They included the first 50 patients who met the inclusion criteria from all of the trials and the expanded access program! This is your donations at work!

 This is data from three vaccine trials for Glioblastoma, and they all show about the same effectiveness: 5 year survival rate of about 33-36%. Historically, the 5 year survival of glioblastoma is about 4%. This is a major improvement. As I mentioned in a few recent articles, I beleive the ultimate cure is going to be a combination of treatments. If we could get 35% survival at 5 years with this vaccine, then maybe add Optune, which alone -if used above 90% of the time - can get another 30%, perhaps these would be complimentary and add up to 65% 5 year survival.  Maybe add in a checkpoint inhibitor, oncolytic virus, Car-T cell, or a targeted therapy or 2, and we could get to a cure! Our patient navigation system is designed to test and track combinations like this - but it will only work once they are approved. Otherwise, it is too hard to do these combinations. Which is why I keep saying we need the promising pathway act! 

 This is the "Duke Polio Vaccine".  For recurrent glioblastoma, they reported 1 year survival rates of 54% for 1 trial and 50% for the other trial.  This compares nicely with historical controls of 35%. Survivals were about 12 months, compared to historical control of 9 months. It is very hard to improve the outcome in recurrent glioblastoma. These results are meaningful.

 Very impressive results with Gammatile for recurrent Glioblastoma. This is an FDA approved treatment that is widely (although not everywhere yet) available.  If you are going to have a brain tumor surgery, ask your neurosurgeon about it.  Surgery by itself doesn't really prolong survival much - adding Gammatiles at the time of surgery may significantly extend survival! We recently did a webinar about this: https://virtualtrials.org/video2021.cfm?video=202101

 DNX-2401 is an experimental  oncolytic virus in trials for adult and pediatric brain tumors. They report promising results in a small DIPG trial:  25% response rate (using RAPNO) with tumor reductions in 75%.   Under RAPNO guidelines, the tumor has to shrink 25% in DIPG to be considered a "response".  (This is 50% in other types of brain tumors). So 1/2 of cases had some tumor shrinkage but did not hit the 25% reduction level. This is still very good. It is very hard to get a response in DIPG.  Median survival was 17.8 months with 3 (out of 12) patients still alive at the cutoff date. Historical survival rates are about 9-12 months for DIPG so this compares favorably.

My thoughts: this will be very useful, but by itself is not enough. I feel the same way about Onc-201.  We need to get these treatments approved by the FDA so we can try combinations - that is how we will find the cure.

This is a new way of delivery radiation to the tumor using nanoliposomes and Rhenium-186. Visit their website https://www.plustherapeutics.com/ to see how it works.  This phase 1 trial had impressive results: It was a dose finding trial. Those at the highest dose did not have any dose limiting toxcicities and 7 of 13 recurrent glioblastoma patients are still alive at an average of 453 days. This compares very favorably to history which tells us these patients live about 6  months (183 days). The trial for recurrent glioblastoma is still open, and they will soon start trials for pediatric brain tumors and leptomeningeal metastases.

 This is the future of medicine.  All patients should be followed as if they are in a clinical trial. This would allow researchers to figure out the best treatment options and find what works, and for whom. It would allow for the creation of synthetic control groups that are better than randomization for a trial, since ALL of the prognostic factors can be accounted for.   The free service will help practicing brain tumor doctors figure out which mutations are the most important and which drug combinations or clinical trials might be best for your patient.  

 Very good results in a new Optune plus Pembrolizumab (Keytruda) trial for newly diagnosed glioblastoma.  They reported median progression free of 11.2 months compared to the original Optune trial without Pembrolizumab which had 6.7 months median progression free survival. The trial for Pembrolizumab alone for recurrent Glioblastoma only had a 2.8 month progression free survival.    This shows that Optune has an immunogenic effect, which the Pembrolizumab magnified.    Hopefully we will see an even better result when they start adding a vaccine to the mix!

 SNO is the big annual brain tumor meeting which is next week.   Novocure will have 32 presentations which is amazing. They are split between clinical and preclinical and we learn better how to use Optune with each presentation.  I will report after the conference on which have the biggest impact!

The presentation title is: XCELSIOR: A real-time, real-world learning platform for patients with advanced cancer.  This is part of our patient navigation program. The idea it to create a learning system where a team of neuro-oncologists, PhD research scientists and nurse navigators - aided by an artificial intelligence engine - can learn from the experiences of every patient.  The registry tracks the outcomes of every patient - so we know what is being used and how it is working out. The system can easily track trials, standard treatments, off label, expanded access, and right to try treatments as well as the new combinations being used by doctors around the country and identify which are doing the best. I feel this is the fastest way to home in on the cures! You can participate by going to our patient navigation program

Disclaimer: I am proud to say I am a founder of xCures, own stock and am a paid consultant to their braintumor program!