This is an exciting new treatment. The Musella Foundation helped fund part of it. OS2966 is a brand new class of treatment, which targets CD29/B1 integrins which are highly upregulated in Glioblastomas and have been shown to drive tumor progression and invasion as well as participate in the resistance pathways.  

This is a new treatment approach. They have shown they can get this new treatment into the brain and that it kills tumor cells, but all of the patients died. They do not say how long the patients lived or if it helped them at all.  

 Very exciting news. This is a new approach which targets CD29 which controls a tumors' growth, invasiveness and resistance.  I wish this patient well (as well as future patients in the trial!). The Musella Foundation played a small part in the development of this drug by giving research grants to them!

 We are so proud to have been a part of this.  It is so unfair that families have to go through this nonsense when dealing with such a horrible disease. We are working on ways to fix that!

 I have been involved with brain tumors for almost 30 years. For most of that time I believed that a phase 3 randomized controlled trial was the best way to tell if a drug is working.  I saw too many cases where a phase 1 or 2 trial had spectacular results and then failed in phase 3.   I have always known that phase 3 clinical trials results (even those in a "standard of care" arm) were better than how patients outside a trial did, and I used to think that meant that any trial is better than no trial and perhaps the extra monitoring of patients in a clinical trial helped.

This article explains why trial patients do better and it has nothing to do with being in a trial. It is the entry criteria.  The vast majority of Glioblastoma patients are not eligible for clinical trials, and those people do much worse than the patients who are eligible.  This also means that the results of a phase 3 trial do not represent how the treatment would work in the general Glioblastoma patient population.

We need to rethink how we test drugs. We absolutely need to track all brain tumor patients as if they are in a trial to see how treatments work on everyone, not just the selected few.

From our friends at UCLA.   This is an excellent conference and now we can all participate online!

 Many of these patients told us they wouldn't have been able to get their medicines if not for our grant. We were the only program open for brain tumor patients on Medicare. Now there are none.  You can try goodrx.com for coupons which may help.  Will let you know when we reopen!

 It is a small study so needs to be repeated but the results are very impressive.  Adding Chloroquine and Avastin to re-radiation for recurrent Glioblastoma more than doubled the survival in the control group.  This is an easily available drug and more research needs to be done on this combination.

 Although the results were only good but not remarkable, it is a simple and well tolerated. Perhaps it could be added to other treatments as part of a cocktail approach.

 Patient reported outcomes should be a part of every clinical trial. Makes sense to have standards so they can be compared across trials and accepted by the FDA as endpoints for approving new drugs.  A good example of the need is a recent trial where the number of Glioblastoma patients working their normal job was way higher than expected, but the median survival did not change so the trial was deemed a failure.  The ability to be able to work at my normal job may be more important than just survival, especially if "surviving" means being bedridden.  Quality of life is of utmost importance and was never really taken seriously until recently because until recently nothing preserved quality of life for these patients.

 This is a market research study to look at the decision making involved when considering Optune. They are looking for patients who were prescried Optune but did not yet decide if they want to try it.  There is a payment for your time, and the Musella Foundation will get a small donation.  Let me know if you participate in the phone interview!

 Exciting research done in a zebrafish.  This model can mimick the way Glioblastoma cells spread in the brain of humans, and they are working on ways to stop the spread of the tumor. 

 I love the thought process involved. They looked at how the tumor becomes resistant to treatment, and then found a pathway the tumor uses to get around attempts to stop the resistence.  Hopefully this will be tried in humans soon.

 This article discusses the effects of releasing ongoing data from clinical trials on the trials themselves.   Obviously it helps the trial if the data is good and hurts the trial if the data is bad. What the article doesn't discuss is the effect on the patients.   If patients knew which trials were better than others, they would only go to the better trials. Maybe the worse trials should shut down and reformulate in a better way.   Our patient navigation program is one way to find the better trials. We have over 700 patients in our registry and we see what treatments they are doing (including trials) and the outcomes.  We help guide the patients to the better treatments.

 The Musella Foundation helped fund this project through the DIPG Collaborative.  Sounds exciting but it is early and more research needs to be done. Will be keeping an eye on this!

 This is only in mice, but it is very interesting.   Until recently, very little work has been done with dopamine receptor antagonists and brain tumors.  They used the drug Quetiapine, which is approved to treat schizophrenia, bipolar disorder, and depression, along with Lipitor, which is used to treat high cholesterol.  The great thing about this research is if they find it does work in people, it is easy to get access to these drugs and it can make a difference immediately.  Of course, human testing is needed first.

On a related idea - the drug Onc-201 is a powerful dopamine receptor antagonist and is known to penetrate the blood brain barrier. There is a trial of using it at the same time as radiation in kids with DIPG. Wonder if adding Lipitor might be worth it. 

 They present very impressive results -  anyone who is about to have a surgery for a brain tumor should ask their neurosurgeon about this. It is not yet available everywhere so you might have to ask your neurosurgeon if he has access to this treatment. The webinar shows where it is available at the time of the webinar, but check the https://www.gammatile.com/ website for a current list. You can also request that your surgeon make this available for you.

For those participating in this fundraiser for us using the Amazon mobile app, you need to turn it on again.  Every 6 months Amazon shuts off the donation program and you have to turn it on again. It is easy and free - see instructions below.

If you participate via the website, no further action is required - just continue to use smile.amazon.com!

And if you do not participate but use Amazon a lot, please sign up for the program. It doesn't cost anything to you and it helps us raise badly needed funds! Just go to smile.amazon.com and select "Musella Foundation" as your favorite charity!

Thanks!

 These 2 projects are exciting. The first is looking to see if a new drug crosses the blood brain barrier and works in tumor models. If it does, they will follow up with a human trial.  The second helps fund the Children's Brain Tumor Network.  We are a foundational partner of the CBTN now.  This collaboration will speed up the pace of research, making it possible to do things we never imagined! I think this will speed up the search for the cure. 

 This combination makes sense and it is worth looking at this trial: https://clinicaltrials.gov/ct2/show/NCT04479241  

It is for patients with recurrent Glioblastoma.