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!
This article shows how the various accepted ways to assess response to treatment are completely inadequate. We need to use advanced imaging techniques and come up with an improved global assessment that works with all types of treatments. We had a few webinars on this topic and another one is scheduled for May 16.
Impressive results. 11 out of 1 kids had at least some repsponse to the treatment and they already doubled expected survival with patients still alive so that will get better. There are no approved treatment for this pediatric brain tumor.
Although this is for lung cancer, I included it with the brain tumor news blast to show Tumor Treating Fields have uses outside of the brain and it is becoming mainstream. The article talks about a large randomized phase 3 trial for lung cancer. The final results are not in yet but the independent data monitoring committee said that based on the interim analysis, it is unnecessary and possibly unethical to randomize patients into the control group. That is a great sign.
Exciting but very early results for this experimental treatment. Will be keeping an eye on it.
Exciting report. This combination of the drugs drugs atezolizumab and ipatasertib were tried on 10 glioblastoma patients. They report two patients did very well, and these patients had a PTEN mutation. Atezolizumab is a PDL1 inhibitor, and is easily available. Ipatasertib is an experimental small molecule inhibitor of AKT and hard to get. I would like to see this tried in a larger trial.
This is good news for the Gliovac vaccine: the FDA said the phase 2 data (for recurrent glioblastoma) looks so good that the FDA wants the company to stop the phase 2 trial early and get started on the phase 3 trial with the assumption that if the phase 3 trial turns out good, the FDA will approve Gliovac.
Howevr, I look at it differently. If the FDA says the phase 2 data is so good, and I know from previous reports that there were no significant side effects, why can't the FDA just stop the trial, and approve the vaccine so everyone can benefit. The vaccine has doubled the median survival which is unheard of in brain tumor trials. Most of the other immunotherapies that are promising help a small % of the patients, so the median survival doesn't move.
This vaccine is available under the right to try program now. For details go to http://erc-immunotherapy.com/ or contact me and we can help facilitate the process.
This type of dose intensification is the wave of the future. This study is too small and not controlled to prove it but the concept makes sense and more research needs to be done.
We are only able to approve 20 grants this time so it might go really fast and might close quickly. Check the website for the current status.
Although it is a small study, the difference in outcome is so large and there is no downside to changing from taking the drug in the morning vs. at bedtime that it might be worthwhile to talk to your doctor about it and consider changing to a morning dose!