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!
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
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.
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 feee 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!
These webinars are very informative and give you the chance to learn new things about brain tumors. They are all scientific. ! Well worth going to the live events! We will try to record them and post them on the website, but by going to the live event you can ask questions of the experts!
xCures is the company we use to run our registry and helps with our patient navigation program and they ran our Onc-201 expanded access program. (As a disclaimer, I am a consultant to xCures and own some xCures stock). They are now running a few other expanded access programs, including one for brain tumors with KRAS, NRAS, HRAS, BRAF, MEK, and ERK mutations. This announcement is about a new portal for doctors only where they can easily register patients for these expanded access programs. We have found that many doctors do not have the time to help a patient access a treatment via expanded access. This portal will make it a lot easier for them to do it, and make it much faster for them and you. They can also submit the required follow up information quickly in minutes. This is a big advance for doctors and removes a barrier to expanded access.
Sometime we forget that brain tumor patients do get unrelated problems. It is not always due to the tumor! Sometimes the positioning of your head during the surgery can trigger Paroxysmal Positional Vertigo and there is a really simple fix for most cases. You need to see an ENT doctor who can do a simple manuveur which cures it in some cases. Obviously talk to the neurosurgeon first to see if they think you are healed enough from the surgery to have the manuveur!
We are going to have a live 5k fundraising event on May 1st! First time in over a year!
Will let you know when (if) the program opens again.
This is an exciting new treatment. Animal studies looked really good. It is a new experimental oral treatment for brain tumors that dramatically inhibit tumor growth. The link to the full text has nice pictures of results in one animal. Will be keeping an eye on this!
This is an experimental gene therapy which I like a lot. Preclinical testing looked very good, and they just completed the phase 1 trial of patients with newly diagnosed high grade glioma. Results will be released next year.
There are a lot of variables to test such as the timing of the components, and perhaps even replacing or eliminating the temozolomide with a different drug for those that won't benefit from it. The temozolomide is a double edged sword. It will kill tumor cells, exposing the insides of the cell to the environment to allow a bigger immune response, but on the other hand temozolomide also kills the immune cells. It is a delicate balance that needs to be tested.
Still very early to say how effective it is but this is a new option for DIPG and DMG with H3K27m mutation. They identified a new type of side effect but also were able to treat it. They say one of the patients had a > 90% reduction in tumor size!