The experimental drugs Onc201 and Onc206 work on this pathway.   We need to get them approved so we could use them in combinations.

 This is in a small group of patients but very promising!

 This experimental drug is available via expanded access through xCures (I am a paid consultant for xCures) https://clinicaltrials.gov/ct2/show/NCT04566393

 This shows the importance of having genetic testing done on your tumor.  The BRAF V600E mutation is rare, but when found, this drug combination is useful.  This mutation is more commonly found in kids with low grade brain tumors, but also sometimes in adult glioblastoma. Although approved for kids with low grade tumor, it can be used off label for adults and kids with high grade tumors when they have this mutation.

 This drives me crazy. About 20% of the patients have a major response and at least 1/3 of the patients are helped somewhat with onc-201 alone. This is for dmg and dipg, where no drug has ever shown a response before. The logical next step is to try the combinations we think will make it work for most patients. There are small trials underway to slowly test a few combinations. However, instead of concentrating on making it work better, they decide to waste years, lots of money, and kill patients with a literal sugar pill placebo controlled trial. Shows a complete lack of understanding of what is needed. Drugs like this should be fda approved so every patient can get easy access and we can learn how best to use it.

This is a fundraiser for brain tumor research. And lots of fun!   

 I love to see research on low grade gliomas.These tumors are usually ignored and excluded from most brain tumor trials. Although they do not discuss the numbers, it is great to see they met the endpoints.  

 We partner with the National Brain Tumor Society to advocate for issues that affect brian tumor families.    If you would like to help, read the attached notice and volunteer to go to Washington, DC to visit your congress people!   No experience needed. It is a lot of fun and it will make a difference!

 Impressive 5 year survival of 10% with Gliadel Wafers.  This has been approved in the USA for a long time - this is the long term results. They do not mention controls, but historically, the accepted 5 year survival is about 4%.  Doesn't sound like much by itself but it may buy time for other treatments to work, and possibly be useful in combination therapies.  

 They show a significant benefit when combining these 2 treatments in the lab.  Each had a strong effect by themselves, but the combination was much better.    As noted in the article, it should first be trested if AZD1152 gets to the tumor in the right concentration - might be a good combination to try in a phase 0 trial.

 Optune is finally available in France!   It is a shame that it took so long.  

 This is from our friends at Weil Cornell Medical Center in NYC.      These webinars are very informative and give you a chance to ask questions of the experts!

We partner with Cancer Commons to run our patient navigation program.   Any cancer patient can (and should) take advantage of the program!   We have had some remarkable outcomes, even when others have already given up hope. 

 Our program is open again. Hopefully it will remain open for a about a month before we run out of money again!  But don't count on that. If you think you could use help, apply as soon as possible!  Once accepted, you get 1 year to use the money, starting 3 months before you are approved and ending 9 months after approval. 

This article really doesn't seem to understand our situation when dealing with a malignant brain tumor.   Every patient that goes through our patient navigation program is given a vaccine as one of the suggested options.   There are a few clinical trials for vaccines, but they are very difficult to get into.  The vast majority of these patients can not get into these trials, and if they do get in, then they have to contend with a placebo control on most of them, so they might not even get the vaccine.   One of my friends started the foundation named in the article to help solve  this problem.  We had many patients going outside the USA to get access to vaccines, which is not only expensive but difficult for many brain tumor patients.   Ideally, this foundation would find a way to get funding for these patients but right now there is no funding other than requiring the patient to pay the costs.   Under FDA rules, unapproved treatments accessed under expanded  access can only charge the actual cost of the treatment. This was not meant to be  for profit. All of the available vaccines were evaluated and they selected the personalized neoantigen synthetic long peptide vaccine which was created at the Washing University School of Medicine.    Based on your molecular pathology report, a team of experts figure out which of the markers are most likely the drivers of the tumor, and create a unique vaccine just for your particular case.  This is an expensive process and undergoes a lot of quality control before a patient can get it.  

So it brings up an ethical dilemma:  Is it fair to provide a promising experimental treatment only to those that can afford it?   Or is it better not to do it at all?  Keep in mind this is no more expensive that most other treatments that are being used - the problem comes up because insurance usually does not pay this and it does pay for the other treatments.  So it is a problem with our system, not with the vaccine.  This is the reason I am supporting the Promising Pathway Act. Under the current system, the cost to take this vaccine into the standard (or even accelerated) FDA approval pathway would take at least 7-10 year and cost anywhere from $500 million to over a billion dollars.   Under the Promising Pathway Act, we could cut that down to maybe 2-4 years and $5 to $10 million.  Still expensive but much easier to raise than under the standard pathway. 

Bottom line: This is a promising treatment and it is better to have access than not have access, even if it cost a lot of money. We need to find a way to help patients pay treatments like this.

 The Musella Foundation gave a $50,000 grant back in 2010 to get this project going.  Used by itself, it has pretty good results in a phase 1 trial:3 patents (out of 43) had long term partial responses of 54,34 and 28 months after a single injection of the treatment.  They think combining it with a checkpoint inhibitor will make it work better, and it did in mice.  It cures GBMs in mice, but now they have to prove the combination helps in people. There is a clinical trial of this combination for recurrent malignant glioma going on at Duke.  See https://clinicaltrials.gov/ct2/show/NCT04547777 for details. The concept makes a lot of sense. Hope it pans out.

 These authors make an excellent point:  The clinical trials system needs to be updated.  We should create a standardized database - perhaps using clinicaltrials.gov - to hold the patient level data in a trial.  The control groups' de-identified data should be available to researchers to use for external contemporaneous control groups.  Most trials do not accrue completely - too many lives are wasted in a control group, when this database could eliminate or minimize the need for control groups.    The FDA should scrutinize clinical trial design and make suggestions at the start. In practice, they seem to rubber stamp trial design at the start, but then when it comes time for approval, they rip apart the trial design that they originally approved.   Perhaps trial designs should go before a board of neuro-oncologists which help transform the trial into a better design so we do not waste lives.

 We need more research into this.  Dr Virgina Stark Vance was the first neuro-oncologist to use Avastin for Glioblastomas.  It was scary using it for the first time because the blood vessels in a glioblastoma are messed up and there is a fear that Avastin might cause bleeding into the brain.  So she used 5mg/kg  which was 1/2 of the standard dose  used for Colon cancer at the time. She had remarkable results.   Then many trials were launched to confirm the activity that she saw.  Most of them used the 10mg/kg dose that was popular for Colon Cancer.  The results showed an increase in response rate and progression free survival but no increase in overall survival. However, Dr Vance continued to use the lower dose, which she feels has better outcomes. This study confirms that. 

  We have run out of funding and the copay program will remain closed until we get enough donations to the program to reopen.  

 I have been interested in this drug for a long time. I met the inventors of this drug and gave them a research grant back in 2013. I am very excited that the company opened an expanded access program for pediatric patients with Diffuse Midline Glioma and DIPGs.   There is a huge unmet need for these tumor types, and we badly need more treatment options.

As I mentioned many times before, I helped start xCures and am a paid consultant to xCures.   They are revolutionizing how expanded access works. In the past, most doctors did not like to participate in expanded access programs because of the extra time needed for paperwork and to work out logistics.   xCures takes over most of the paperwork and logistics now, and helps the doctors with the little paperwork that they have to do. 

In the past, expanded access programs did not help speed up drug approvals. Now, xCures monitors the patients in expanded access programs as a virtual trial, gathering real world evidence of how the drug works. Best part is programs like this accept most patients with the specified tumor types so we really get to know how the drug performs.  This is in contrast to clinical trials that pick the patients that would make the drug look best.   This real world evidence can (and has) been used to get FDA designations and as part of new drug applications to support the evidence from clinical trials.

And best of all, xCures makes it much easier for the patient.  First, our patient navigation program (Musella Foundation, Cancer Commons and xCures) can help find the best treatment options for each patient - including expanded access programs, then we do a lot of the work needed to actually enroll and get the drug.   

 xCures ran the first expanded access program for Onc-201, which was a big success, and is currently running another expanded access program - Ulixertinib, which is for any type of cancer (including braintumors) that has mutations in the MAPK pathway (KRAS, BRAF, NRAS, HRAS, MEK and ERK mutations).    They are also running the  Patient-Centric Platform Trial for Precision Oncology which is the basis of our patient navigation program!