From our good friends at the ABTA. These events are better live in person as you can meet and interact with other families going through this as well as the doctors presenting - however, if you can't make it, the event will be streamed live!
This is one of my favorite options for patients with glioblastomas who have unmethylated MGMT. Will be watching for the results!
This provides a great overview of the treatments being used for diffuse midline glioma and DIPG. It also talks about the use of a liquid biopsy to monitor tumor response. Regular MRI scans are not conclusive when it comes to these tumors - parts of these tumors do not enhance and the edges are diffuse so it is hard to quantify how much tumor is there and if it is getting better or worse. A liquid biopsy might help!
This study shows that it is safe to keep the Optune arrays on the scalp during radiation. The amount of radiation blocked is negligible, opening the door for using Optune during radiation. Of course we need to see the results of the current trial but the combination of Optune and radiation may make a lot of sense. See https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6290966/ for why!
PNOC is a group of 22 hospitals that share clinical trials and research. Going to any of these 22 hospitals lets you get access to the clinical trials of the PNOC organization.
The doctor mentioned in the article, Dr Carl Koschmann, is one of my favorite pediatric brain tumor doctors. Patients rave about him.
This reports a pretty big improvement in overall survival when using Avastin with Temodar as first line therapy. There are other reports that there is no survival benefit. It is hard to prove one way or the other if Avastin (or any new treatment really) has a survival benefit. The trials that said there was no (or small) benefit to overall survival did show a benefit in progression free survival and they were larger and randomized - which is usually more trusted, but all of these studies had other problems such as most patients had other treatments that could confound the results. In the randomized trial, many patients in the control group crossed over to using Avastin after progression, or had more surgery or more radiation. Too many variables. The only way to really tell is to follow all patients in a registry so we have large numbers of patients and analyze the effects of each subsequent or concurrent therapy!
It is obvious, but nice to see proof that people who have a complete resection of the tumor do better than those that do not. It makes a big difference.
I read stories like this all of the time where patients have to raise a lot of money to travel outside of their country to get treatments. It is a sign of failure in our systems - both the USA and UK. There is a bill in congress called the Conditional Approval Act which may fix this problem in the USA.
In the mean time - wouldn't it make a lot more sense for these families to pool their resources to bring the treatment to their country instead of spending a fortune to go outside their country? For what 4 or 5 of these families raise to get access to a treatment, they could bring the treatment home for maybe 50-100 kids. Our organization, the Musella Foundation did just that with one pediatric cancer drug - for patients in the USA... no reason the foreign charities can't do the same.
I never understood the concept of how an insurance company could tell a patient "you can not use the treatment your doctor prescribed which will give you the best chance of beating your cancer - but instead you have to try a cheaper drug first and fail on it - then you can try the one that might help". What they do not understand is that the first try is your best try. When you have a recurrence, the tumor is usually worse - you may be left with permanent neurological problems and even if cured at that point, your lifelong medical costs are going to be higher.
This is great news for Pennsylvania patients. We need this country wide!
The program opens and closes as we have funding.
This shows the importance of testing every tumor sample. They usually do not find anything that will make a difference, but sometimes they do!
There will be a lot of FDA people and congressmen (assuming the impeachment trial is over) This is a great chance to educate them on how bad brain tumors are. Most people do not understand how bad these tumors are until they know someone with one. If they can understand, then perhaps they will speed up the search for the cure!
There is still time to register and go. There is a briefing at 11am on 2/13/2020 with a great lineup of speakers, then the congressional briefing at 2pm. We need people to show up so we have a stronger voice! You have to register (free!) at HERE
Then there is an optional informal pizza party for dinner!
This is an interesting concept. Traditional photodynamic therapy uses direct light on the tumor after the tumor is sensitized to the light with a drug. Obviously impossible for a brainstem tumor. Instead, they propose using radiation therapy as the light souce, sensitizing the tumor with the drug 5-ALA which is already approved and in common use for brain tumors as Gleolan. (It is used at the time of surgery because it makes the tumor glow when a special light is shined upon it so the surgeon can see where the tumor is). This has not been done it trials yet - so it is only a theory - I would love to see it tested.
This study says that of GBM patients using concurrent radiation and Temodar, 3% developed serious thrombocyopenia. In the Temodar package insert, it was reported that about 4% for adults, and 58% for kids have problems with platelets. This abstract did not mention the ages of the patients but the article said they were all adults. 19.6% that used the generic had thrombocytopenia. Big difference.
From this it is hard to tell which is better. I doubt there is a differencem but if there is and the generic causes more thrombocytopenia, the next question is which one worked better. More side effects might mean more potency but also might mean worse quality control.
I would like to take a quick survey of the readers. If you have ever used Temodar or the generic, please fill out this quick 3 question survey:
This is just theoretic at this point but they present a new way to orient the Optune arrays so that it can treat tumors in the cerebellum. Until now, those tumors were not able to be treated, Might be worth considering it if you have a gbm that extends into the cerebellum. Ask your doctor.
This is just a proposal but seems like a very good idea. To limit the toxcicity of CAR-T cells, they propose to create a new type of CAR-T cell that has to bind to 2 targets before it can kill the cell. That has been tried before, but the new idea is the logic part: there has to be EGFRvIII AND (Il-13Ra2 OR CD133). This may be a big improvement, however, as I understand it, only about 1/3 of GBMs have EGFRvIII and it is possible that in the natural course of the disease, even those that are EGFvIII positive can turn EGFRvIII negative. CD133 is a marker found on all of the GBM stem cells, which are the most important cells to target, but most non stem cells in the tumor won't have it. IL13Ra2 is overexpressed in a little over 1/2 of the GBMs but not distributed uniformly through the tumor. So the OR part should target most of the tumor cells, but the AND part might not be enough. Perhaps find a way to require (EGFRvIII or ???) AND (Il-13Ra2 OR CD133) where we have to figure out what the best target for ??? is! This is a complex math problem that can be solved and variations can be tested and perhaps even personalized to the patient.
This is great news. I will let you know when the trials begin. Diffuse midline glioma is a new category of brain tumor, for those with the H3K27M mutation. It is one of the worst types because it is more aggressive and is in the midline structures which control important brain functions.
Disclaimer: I am on an advisory board for the Brain Tumor Center at Duke and have sponsored research on this treatment - but have never received any compensation from it!
This is another option for those with recurrent Anaplastic Astrocytomas.
[ Disclaimer: Orbus Therapeutics is a sponsor of the Musella Foundation]
We have not heard any advances with meningioma treatments in a while. There was no comparison group so it is hard to tell how good it did, but both drugs are already readily available off label so this is already an option for people with aggressive and recurrent meningiomas.
Correction - I sent this out in the last news blast but my comments were incorrect. I said it adds 10 months to the progression free survival time, which is the number for all types of brain tumors, not just GBM. For GBM it is 4 months, which is still very good. For metastatic tumors and meningiomas it is a lot more!