I think this is one of the most important bills that can speed up our access to brain tumor treatments.  I helped write some of the words!    This would work perfectly hand-in-hand with our patient navigation program. Right now, our patient navigation program has brain tumor experts thinking up the best combinations of treatments for each individual patient. We give the patient and their doctors a list of 5-10 plan options, which we feel would be the best for them. Invariably, the best plans are hard or impossible to get, because they involve experimental treatments. We managed to get some of them under expanded access or under the right to try pathway, but that it too much of a barrier for widespread use. We need direct, easy access to these treatments.

Do not do anything yet. We will soon announce how to help.  Hate to ask - but we could also use some donations to help us with this. Make a donation at https://virtualtrials.org/donate11.cfm and be sure to specify "unrestricted" so we could use it for things like paying for the program that allows our members to contact congress easily.

 The MRI lecture was a little too technical at some points - just gloss over that as it all came together by the end, and showed how important this imaging technique is. It should be available at the larger centers. You can ask your doctors about it. They can not go back and reconstruct it from old images - you need to request it before the scan.  It can tell much sooner if a treatment is working or not, and if there is a tumor recurrence or necrosis / pseudoprogression. 

The clinical trial lecture was fascinating. The speaker goes through the history of the drug, explaining his thought process on how he chose this drug to try in people. He shows results in animals which are very good.  I think this treatment (Gallium Matolate) has the potential to make a big impact.

 Paresis is a muscle weakness. In this trial, they treated patients who developed paresis after surgery for a brain tumor.  The treatment is transcranial magentic stimulation, which is non invasive.

They showed a large benefit in a small randomized trial. This therapy is available now, so if you develop weakness after a surgery, ask your doctors about it!

 This trial is for Glioblastomas, Ependymoma or Medulloblastomas that have leptomeningeal spread.  Leptomeningeal spread is when the tumor cells spread via the cerebral spinal fluid to the lining of the brain and spinal cord.  It is a very bad sign. Doing a google search for it shows only completely hopelessness.  Most trials exclude patients with leptomeningeal spread because the outlook is so grim and nothing has really been shown to provide lasting benefit.  This trial is targeting these people with the worst of the worst tumors.  It is worth a try as there really aren't many alternatives. [Disclaimer: Mustang Bio is a sponsor of the Musella Foundation]

 We have 2 related topics tonight. We start off with a talk on how perfusion MRIs can be used to better understand it a treatment is working or not, followed by an announcement and discussion of a new clinical trial which will test a new type of brain tumor drug and use perfusion MRI to evaluate it! Should be interesting.

Two items of note:

1. The manufacturing  plant is ready! Just waiting for the final approval.

2. We are getting close to getting the long awaited results of the DCVAX phase 3 trial for glioblastoma. 

It is a great sign that they put the money into getting the plant ready - shows confidence that the vaccine will show success! 

DCVAX is a custom made vaccine that treats the tumor. This version is for Glioblastomas but the concept should be able to be applied to any type of cancer. I am happy they chose Glioblastoma as the first target - that is the hardest, and if they can make progress there, the other cancers should be easier!  I will let you know as soon as the data is released!

GammTile is an FDA approved treatment for brain tumors. It is a biodegradable wafer that is implanted at the time of surgery and it releases radiation to the tumor bed.  This trial will directly compare GammaTile to stereotactic radiosurgery for metastatic brain tumors.

  This will fund a phase 3 trial into superselective intra-arterial infusion of treatments. This is a way of delivering drugs by threading a catheter into the artery that supplies the tumor and injecting the drugs there. The result is a very high concentration of the drug where it is needed with minimal exposure to the rest of the body.  This should be tried with all of the old drugs that looked good in the test tube but failed in people because the drug doesn't get to the target. For this trial, they are using Avastin and Temodar. Early trials showed that it is relatively safe and had promising results. This will prove if it works or not.  This technique was mentioned on our webinar on drug delivery last week. Great to see it is going forward and it is worth considering this trial for newly diagnosed glioblastoma.

 I have been saying this for years. I am a big fan of Optune, but by itself (or even with Temodar), it is not enough. It will improve survival by a few months (longer than any other approved treatments), and a small % of patient will go on to become long term survivors, but we can do so much better. This article explains how Optune could be used in a cocktail approach to bring us closer to the cure.  I love to see so much research, but the pace is too slow  for me.   They talk about possible mechanisms of resistance to Optune and ways around it. This is the research we need. 

They talk about clinical trials. One of my favorite theoretic combinations is Optune + Choraquine.  Many years ago there was a presentation about resistance to Optune, and autophagy was mentioned as a main resistance pathway.  Chloraquine may inhibit autophagy, which may make Optune work better.  This was finally formalized into a clinical trial which was posted on Clinicaltrials.gov May 21, 2020.  Almost a year ago  - but it is still listed as "not yet recruiting".  The timeline of having an idea, creating a clinical trials, running the trial, then getting results published and made useful to people is about 5-10 years.  We do not have that much time, as we need many iterations of the process to get to the cure.  And here we are talking about approved treatments (and off label treatments) that are relatively easy to get access to.  Trying it with experimental treatments would be much harder and take much longer.

My solution to this is a program like our patient navigation program, where a team of experts (now including 3 neuro-oncologists, a few PhD researchers, nurse navigators and our artificial intelligence system) reviews patient records and comes up with what they feel is the best treatment plan options. The patient and their health care team can pick one of the options we present, or do whatever else they want. The key is that we then track their case as if in a clinical trial, and learn from every patient.  We can create virtual trials of combinations like that mentioned above.  Try them on a set number of people then analyze the outcomes.  Improve with each new iteration.  This is actually working now, and we have it listed on clinicaltrials.gov as A Patient-Centric Platform Trial for Precision Oncology.  We just need a lot more patients to participate to speed up the learning process! To join, go to https://virtualtrials.org/xcelsior.cfm

 This is another must see video.  I learned a lot from it that I did not know about focused ultrasound. I can see focused ultrasound becoming a very important part in the treatment of brain tumors!

 Happy Mother's Day!  What a nice way to celebrate - find out about this treatment that may be a breakthrough in the treatment of braintumors!

For those tied up tonight, we will record it and post it to the website soon, but participating in the webinar allows you to ask questions!

 This is our major source of funding for the brain tumor research grants we give out. It has been very hard to raise money for brain tumor research and we can not afford to take a year off.  Please participate in the virtual events!

 This is another small study (we wrote about a similar study last year with the same results) which shows that for MGMT methylated patients, taking Temodar in the morning works better than at night.  The difference is about a 6 month average survival increase if you take it in the morning compared to taking it in the evening.  The level of evidence needed to prove it is not yet there but with 2 studies showing the same result, I feel it is worth taking the Temodar in the morning.  Traditionally it is given in the evening so you sleep through the nausea it causes but now there are good anti nausea drugs that can avoid that problem. If you still get nausea, talk to your doctor and try other anti nausea drugs.

 Another exciting webinar!   This was a must see. It is a little technical. If there is anything you do not understand, feel free to ask us in our discussion forum

 They reported pretty good survivals for recurrent GBM. This experimental treatment is delivered via convection enhanced delivery for tumors that overexpress IL-4R, about 75% of recurrent glioblasomtas overexpress it. Unfortunately, I do not think there are any clinical trials open for it right now, but I will be keeping an eye on that.

 If your tumor has the BRAF V600E mutation it may be worth looking at this combination.   Although only a small % of patients responded, they had a few complete responses that are lasting a long time!

I love the way these doctors think..  they look at the best treatments, why they fail, and work on improving them.  With each iteration, we get closer to the cure. The link to the story below has an animation that shows how this new approach works. It is very elegant. It was able to cure brain tumors in rats where CAR-T cells couldn't.  Can't wait to see how it works in people.

Disclosure: Dr. Okada is a friend of mine and a member of the Musella Foundation Medical Adviosry Board.  

Focused ultrasound is a treatment modality that can work in a few different ways. It is noninvasive, using sound energy waves that can be precisely focused on targets within the brain. The main way it has been used is to open the blood brain barrier so other drugs could get into the tumor at the right concentration.  Many drugs look good in the lab but failed when used in people because they do not get to the right area in the right concentration. This can help.  A new way it is being used it to sensitize the tumor with a dye, then the focused ultrasound beam can be used to kill those cells that take up the dye.   Very exciting. 

 This is the problem with the current clinical trial system.  These vaccines seem to work in a small % of patients, perhaps 30%.  So when you have a trial report that the median overall survival and median progression free survival did not change, they wrongly conclude the trial was a failure. I say wrongly because the median does not move if you cure 30% of the patients, only if you help over 50%.  They should be looking at the 2,3,5 year survival rates. And they should be looking at the differences between the responders and non responders.

 We changed the title of tonight's webinar to reflect the importance of the delivery system! Convection enhanced delivery is a way to get the drug to the tumor in the right concentration. This has been a major stumbling block in the treatment of brain tumors, and a lot of progress has been made!