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01/15/17 Immunotherapies for GBM: Tumor Vaccines by Linda Liau, M.D., Ph.D., M.B.A.        

 This is a video about the DCVax trial, given by one of my all time favorite brain tumor doctors, Dr. Liau.  This looks very promising. We are hoping to hear the results of the big phase 3 trial soon.

 In the earlier trials and in the early progressors which were excluded from this trial but were followed as they took the vaccine,  about 25% of the patients went on to be long term survivors, with many alive and progression free 5-10 years later.  That is unheard of with the standard treatments. Some other immunotherapies have also reported similiar results (although not as long follow up).    We are hoping this large phase 3 trial shows something close to that so it can be approved quickly and all patients can benefit from it.  Best of all it had no serious side effects.

Dr. Liau is on the Musella Foundation Medical Advisory Board.  The Musella Foundation has supported Dr Liau's immunotherapy research with $375,000 in grants and we have never received any financial  support from the company developing this treatment, Northwest Biotherapeutics.

01/14/17 National Walk To End Brain Tumors – Volunteers needed        

 We desperately need to fund more brain tumor research. We have many research proposals on our desk that are just waiting to be funded. Any of them might lead to advances.  If you have the energy - please volunteer. (And when the time comes - donate!)

01/13/17 Salmonella Has Been Genetically Engineered to Consume Brain Tumors        

 This is way too early to tell if it will work on people but I like the thinking behind it. Good luck to the researchers.

01/03/17 Musella Foundation Copay Assistance Program reopens!        

 This program will probably close quickly so if you think you may need it, apply as soon as possible!

12/31/16 Top Brain Tumor News Stories for 2016        

 2016 was an exciting year - but I think 2017 will be the year we see big steps forward!

Happy New Year!

BTW: The copay program will open again very soon - maybe Monday or Tuesday. Will announce it as soon as it opens!


12/31/16 Florescent light helps surgeons target brain tumors        

 This is approved and a standard treatment in Europe. It is not yet approved in the USA so it is only available in clinical trials. Hopefully the new administration will relax some FDA rules and allow potentially life saving treatments to be approved sooner rather than later.

12/31/16 Pathway linked to slower aging also fuels brain cancer        

 They need to find a way to target only the tumor cells and not have the side effects on the normal cells.  There is a lot of research going on to accomplish this with other treatments, such as antibody drug conjugates, convection enhanced delivery, super selective intraarterial intracranial infusion, gene therapies such as used by Tocagen and a few others.

12/31/16 Prognostic parameters and outcome after re-irradiation for progressive glioblastoma.        

 This suggests that stereotactc radiosurgery may be worth consdering for recurrent gbm. This is another controversial issue in the field.  Some doctors I talk with never use it, and others use it a lot.  This is why it is so important to get second opinions.

12/31/16 A Rational Approach to Target the Epidermal Growth Factor Receptor in Glioblastomas.        

 We (the Musella Foundation) are partially funding this work!  Good luck to them.

12/31/16 Absence of cytomegalovirus in glioblastoma and other high-grade gliomas by real-time PCR, immunohistochemistry and in situ hybridization.        

 This study shows that CMV is NOT associated with GBMs. Other studies show 100% of GBMs have it.  It is very important to get the 2 sides together and work out why one group finds it and the other group doesn't. Perhaps have both labs test the same samples, and also have them compare methodology.

12/29/16 Novel Type of Immunotherapy Helps Man Fight Brain Cancer        

 Of course it is too early to tell how it will work but great sign that they were able to get rid of all of the tumors for a while, and that they have shown it is safe to use multiple times.

12/25/16 First patient enrolled in brain tumor trial combining 9 repurposed drugs - CUSP9v3 project        

 This is the 3rd version of CUSP9... it appears that they are only taking patients from Germany at this time, however, since all of the treatments are already approved and readily available, it can be done outside of that trial. If you try it, please register for our virtual trial project first at That way we can track results. 

12/25/16 ONC201 Glioblastoma Trial to Expand Based on Promising Initial Results        

 It's a great sign that they expanded the trial to include more patients and has shown signs of helping patients as well as of not hurting them.

12/25/16 Some glioblastoma patients benefit from 'ineffective' treatment, Stanford researchers say        

 This is the problem with clinical trials.. if the average patients doesn't benefit, they call it a failure.  They do not identify the patients who do benefit. In this case, they identified a group of patients who would benefit from Avastin, even though an average patient doesn't. I have seen many patients with a huge benefit from it, which makes no sense when you look at the trial results. This may explain why. They should go back to the original Avastion trials,  and see if this group of patients did better,

12/19/16 Use of an anti-viral drug, Ribavirin, as an anti-glioblastoma therapeutic.        

 Aside from the mechanism of action these authors proposed, perhaps the theories about cytomegalovirus playing a role in GBMs are valid, and this drug also fights the CMV, just like Valcyte does.

12/13/16 Medicenna Announces Initiation of Phase 2 Clinical Trial of MDNA55 for the Treatment of Recurrent Glioblastoma        

 (Disclosure: Medicenna is a new sponsor of the Musella Foundation).   This immunotherapy had some very impressive results in a small early trial. Of course we have to be careful when there are small numbers involved - sometimes that doesn't pan out when the large trials are done, but it is a good sign.  In a group of patient with recurrent gbm who did not have a resection at the time of recurrence, just one infusion of this new treatment, they had 20% complete response rate.  That means the tumor disappeared after just one infusion.  The response rate was 56% and if you include stable disease, the benefit rate (Stable + partial response + complete response) was 68%.   They were no serious safety problems.

This treatment is a fusion protein which targets Interleukin-4 which is overexpressed on 3/4 of GBMs, and 100% of brainstem gliomas. It carries along a toxin that can kill the cells which overexpress IL-4. Normal cells have a low level of IL-4. Delivery is via convection enhanced delivery.  Prior trials involving CED have failed, mainly because the early versions of CED were not accurate - they couldn't get the drug to the target.  The Tocagen trials proved that CED is now at a point where they can routinely get the treatment 

12/13/16 Tocagen Expands Toca 5, Phase 2/3 Clinical Trial for Recurrent Brain Cancer, to Israel        

 We have a lot of members from Israel asking about coming to the US for clinical trials - now they have one of my favorite trials available there!

12/09/16 NW Bio Provides Update About Phase 3 Trial Of DCVax®-L for Newly Diagnosed Glioblastoma Multiforme Brain Cancer        

Very exciting news.  Nobody knows why the trial was placed on hold. Usually that is a bad sign but there are many reasons a trial can be placed on hold that have nothing to do with how good a treatment is.  See .  I doubt it is for safety reasons, as there were no safety issues at all in the first few trials and in similar trials. That is the one we would worry about if it were true.  There are many other reasons. One possibility is that the FDA thinks that the trial will not prove anything.  There has been arguments back and forth about appropriate endpoints. At one time the FDA allowed progression free survival as an endpoint, but then the Avastin trial showed excellent progression free survival but no increase in overall survival, so the FDA changed its stance and said it wanted overall survival as an endpoint and that progression free survival is not enough. This happened after the trial was designed. The primary endpoint is progression free survival.  This trial allows patients in the control group to cross over and get the vaccine when they have a recurrence.  This messes up the endpoint of overall survival because you will be comparing people who get the vaccine to people who get the vaccine.  The FDA may think it is futile to try with the current design and may want the trial redesigned and done over again, which would probably be too expensive and lead to abandoning of the treatment.  I would protest that it is unethical to not allow cross over for the control group. (Let alone that it is unethical to have a control group in the first place).



Anyway - this press release says they have stopped enrollment in the trial and will just analyze the results they have.  They already enrolled 331 patients, which should be enough to tell how it looks. IF it shows a progression free survival benefit, we will be fighting to get it approved based on this trial and hopefully not have to go through another phase 3 trial.

12/07/16 Take a survey - get $75!        

 We have worked with these people before.. make sure to tell them you were refered by the Musella Foundation and they will also make a donation to us!


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