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!

 There is also a huge disparity based on ability to pay.  When diagnosed with a malignant brain tumor, even a middle class family can easily be thrown into financial difficulty due to the cost of treatments and  inability for the patient or caregiver to work.  We (the Musella Foundation) gets calls every day from people who can not afford their treatments. Even my own dad refused to buy his Temodar when he realized he would never work again and he might die anyway so he didn't want to use up his savings and leave my mom broke.   

That is why we set up the brain tumor copayment assistance program. To help remove the inequities in treatments due to ability to pay. So far we were able to help over 1,600 patients pay for their medicine. In many of these cases they would not have been able to get their medications without our help.  Unfortunately the program is out of funding right now and closed to new patients. We still get a lot of calls for help but can not help them right now.

 This article reports on very impressive results for an experimental vaccine therapy for high grade gliomas with the IDH1 mutation.  It is a small phase 1 trial (and we have seen great results in small phase 1 trials not pan out in larger phase 3 trials) but I am optimistic on this one. 

 Convection enhanced delivery is a way of giving drug directly to the tumor bed, bypassing the blood brain barrier.   This article reviews all of the CED trials to date, and some of them had impressive results. They discuss the use of an implantable pump which allows continuous ongoing delivery of the drug. Bottom line is this method of delivery is relatively safe, and makes the treatments more effective. On the downside, it is invasive which carries some risks.    I think we will soon see major breakthroughs using this methodology.

 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.