This is the type of research that will lead to the cure.    We need to know WHY things work and do not work in order to put together an intelligent combination of treatments.  

This was written in Japanese and the link takes you to a google translation of it so it is a little hard to understand but it looks like they are reporting 1 year survival rate for recurrent GBM of 92% compared with historical controls of 15%.  If that is true, this is amazing.  It was a small study but may lead to approval in Japan.

 There have been other trials that show statins and NSAIDS have helped brain tumors. This study says no (except Aspirin for grade 3 gliomas).  It is hard to prove one way or another with such conflicting information.  A randomized blinded trial would be the best way but that will never happen due to cost. The next best thing is for the registry to track these drugs - but record the dosage and length of time the drugs are used, then evaluate it over a large number of patients. 

 Medicare is going to reconsider it's decision to not cover Optune.  The meeting is going to be streamed online but you have to register (free) soon. You can not comment at this meeting but they will have a follow up meeting where you can tell Medicare how you feel on this topic. It is important to watch this to show the support of the brain tumor community.

 This refers to the Gamma Tiles that I wrote about a few weeks ago. They are implantable wafers that slowly release radiation to the tumor bed and were recently approved by the FDA.  We will have a webinar on this treatment in May during brain tumor awareness month!  Watch for the announcement as it gets closer.  

 An alternative to dexamethasone is badly needed, especially when using immunotherapies or Optune.  This may be one such alternative but the experiment was done in rats.  Need to wait until it is tested in people before using it.  

 This is another way to beat the edema from a brain tumor without using steroids. These researchers used an antibody that blocks the protein that causes the swelling in the first place!  Again - too early but this is something to keep an eye on!

 Impressive results in the article but the underlying poster (http://oncotelic.com/wp-content/uploads/AACR-JCA-Glioma-Finalv3.pdf) is a little on the confusing side.  They talk about "chemo naive recurrent glioma" but I would assume most patients would get Temodar as newly diagnosed. Where do they find those patients who did not use Temodar? There might be something special about them.  Then they go on to show survivals with and without previous Temodar and with and without subsequent Temodar.  The best group appears to be the control group and the conclusion is that this treatment is not inferior to Temodar.  This might just be that the amount of text on a poster is limited and we have to wait for the published results to completely dissect the trial.

 I posted this a while ago when it was presented at the SNO meeting but it was just published so I am posting it again. One of the more important papers of the year. They were able to double survival just by starting the pembrolizumab (Keytruda) before surgery instead of after surgery the way it was always tried in the past.  This is important enough to print out and save it - if you ever need a brain tumor surgery ask your doctors about starting Keytruda before the surgery. Show them the article.

This article intended to be a balanced article on the plusses and minuses of Optune.  Unfortunately it is loaded with errors and bias.  For example,

1. They say:   They did a survey between January 2015 and July 2015 and found only 41% of doctors had access to the device.  Optune was approved for newly diagnosed in July 2015.  So they did the survey before the device was available for newly diagnosed. (The recurrent data was not great). This article was published in 2019.  Optune is now available at just about every major medical center in the USA. Their website, optune.com says there are 700 certified treatment centers that offer Optune in the USA.  They should have repeated the survey but instead used an old one that made Optune look bad.

2. They harp on the fact that there was no sham device in the control group.  That would have been ridiculous.  Imagine having to shave your head twice a week and carry around the old version of the device (which was twice as heavy and bulky as the current version), and risk getting skin problems without a chance to benefit from it? I would not want to do that.  The endpoint was overall survival. Placebos cannot show such an increase in overall survival.  The Temodar studies did not use a placebo and nobody faulted the study for that. The Avastin trials did use a placebo but the placebo group did not do better than the treatment group (which means placebos do not help  glioblastomas). This is absolutely not an issue.

3. They say Optune is not covered by Medicare or many other insurance companies. The second part of that statement is not true.  Over 90% of non-medicare insurance companies pay for Optune. They go on to say Medicare has now agreed to reconsider coverage – which only happened a few weeks ago so there is no reason to be so far behind on the statistics. That statement was true in 2015 but not in 2019. Hopefully by the end of this year all insurances including Medicare will be paying for it.  In the meantime, Novocure has said they would not let a patient not get access to this treatment due to being unable to afford it. They very generously gave it for free or at a very reduced rate to those on Medicare or who did not have insurance that covered it. 

4. They keep talking about patients being reluctant to wear the device and shave their heads. This is too subjective. Of course patients would prefer not to shave heads or have to wear a device that constantly reminds them of the disease.   However, having to use a wheelchair, or being unable to speak, at a much sooner time are not great alternatives. It is a personal decision that the patient has to make.

5. Finally – they complain about the price. At least they do point out that other new treatments are way more expensive.  I doubt we will ever see a new brain tumor treatment priced less than Optune.  Optune is about half of the price of Avastin, and the trials showed a big increase in survival for Optune, and no increase in survival for Avastin (although they did show a nice improvement in progression free survival and patients feel better on it). I never heard the complaint that Avastin was too expensive.  Hopefully we will have a few new treatments approved in the next year or 2.  We have no idea what they will cost but to put it in perspective:  The cheapest gene therapy on the market (for other diseases) is $373,000 for the one treatment. Most expensive now is over $1 million for the one treatment. A new one that is under review might be $4 million for the 1 dose. As for vaccines, the only one approved in the USA (for prostate cancer) cost about $93,000 for a 6 week course of treatment.    The only way to bring these prices down to earth is a major change in regulatory policy – but that is for another day.

 This is a fascinating gene therapy. Follow the link in the article to the video explaining how it works. They have the ability to insert a gene into the cancer cells which can be controlled by an oral drug - which turns on the production of IL-12 in the infected cells.  Stopping the oral drug turns off the production of IL-12 and they can fine tune the amount of IL-12 produced by adjusting the oral dose of the drug.  IL-12 turns on the immune system and helps your body fight the cancer.  The next step is to add a checkpoint inhibitor, which have not worked that great with brain tumors but this gene therapy might be able to make the checkpoint inhibitors work for brain tumors!

Exciting results for this experimental treatment which is in clinical trials for recurrent GBM.  This is give  one time, using convection enhanced delivery.  May be worth considering if you have the correct marker: Type II interleukin-4 receptor (consisting of the IL4Rα  and IL13Rα 1)

 This is an amazing concept. They trick the tumor cells into leaving the brain. It was never tried in people yet but it did work in mice!

Will be keeping an eye on this.

 This drug is a new class called "immune transduction modifiers" and targets 3 important markers: STAT3, HIF1-a and c-Myc as well as JAK2..  Will keep an eye on this one. There is a clinical trial in Texas:   https://clinicaltrials.gov/ct2/show/NCT01904123

 This is a new type of radiation therapy which is implanted at the time of surgery. It is made up of biodegradable wafers. It has FDA clearance so it can be used now.  The current article talks about using it for recurrent meningiomas, but it can also be used for any primary or metastatic recurrent brain tumors.  Interesting concept. Check their website for more details.

 There is a drug in clinical trials that targets this marker in other cancers. This project will do preclinical testing which, if successful, will lead to a clinical trial of this drug in pediatric DIPG.   

This is probably the most important article about Optune.  It updates the data reported the prior year and shows a little better results.  If you can maintain over 90% compliance, the  % of patients alive at 5 years was 29.3% compared to 4.5% in the control group which was Temodar and whatever else the doctor and patient wanted to use.  This is the best survival rate ever reported in a large brain tumor trial and should finally put to rest any resistance to it's use.  At this point, Optune (plus Temodar)  should be considered the new standard of care.

 This is another case where the median overall survival is meaningless and could hide impressive results. Toca 511 and TocaFC is an experimental gene therapy.  Toca 511 is a virus that is injected one time. It infects only tumor cells (see https://tocagen.com/our-science/#platform_tech for information on how that magic happens. It is fascinating).  When this virus infects a tumor cell, it inserts a gene that codes for a protein called CD.  CD  can break down the drug Toca FC into a chemotherapy drug 5FU, but only in cells that had the gene inserted.  Toca FC is an oral drug which is a reformulation of an old drug approved for fungal infections.   After injecting the virus, you wait a few weeks for the virus to spread to most of the tumor cells, then start a round of the oral TocaFC.  This drug is harmless to the body except when it comes into contact with the CD protein, which converts the drug to 5FU which kills the nearby cells.  This not only kills the tumor cells but exposes the insides of the tumor cells to the immune system to trigger an immune response.  You then stop the TOCA FC for a few weeks to allow the virus to infect the remaining cells and repeat the process as long as is needed.

This article reports on the results. This is on 56 recurrent high grade glioma patients - which is medium sized - enough patients to see the trend. They used a comparison to historical controls. The Median overall survival  only moved up 2.9 months compared to historical controls (that would be very significant if it was against randomized controls) and the response rate was only 11.3%.   Just looking at those numbers, I would usually say it is interesting but needs more research before I would consider using it if I needed it. However, they then go on to say of those 11.3% responders, ALL had complete responses for an extended period of time.  This is very impressive. It is rare to get a single complete response in a trial this size, and many times we see a complete response that only last a few months.  Seeing about 6 patients our of 56 having a sustained complete response places this treatment among the best options for brain cancer patients.  

Disclaimer: The Musella Foundation was an early supporter of this treatment via grants, and Tocagen is a sponsor of our organization