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Another new way to get treatments through the blood brain barrier!
This was also discussed at SNO last week. I do not think there is a major link between cell phones and brain cancer. At the presentation with the Central Brain Tumor Registry of the US, the question was asked and the response was that we are NOT seeing an increase in the incidence of brain tumors over the last 20 years. Since there has been a huge increase in cell phone use over that time, IF the cells phones were a major cause, we would see the increase in incidence.
Impressive results.. Especially the 6 month survival rate (for recurrent glioblastoma) of 93.8% compared to historical rate of 59%. It also did well with toxicity. This is a gene therapy. First the virus has to be injected - either intra-tumorally, at the time of surgery or IV (right now they are doing the IV study combined with intra-tumoral). Then they wait and give the virus time to infect the tumor cells where it inserts a gene that produces an enzyme that converts a safe, low toxicity antifungal drug into a toxic chemotherapy. . It only infects tumor cells and not normal cells. Visit their website to see how they made that possible! Then they give the oral anti-fungal drug which is relatively non-toxic. This drug crosses the blood brain barrier and is harmless to normal human cells, but when it gets to a cell that was infected and has the enzyme, this drug, 5fc gets converted to the chemotherapy drug 5fu, and it kills those cells. In theory it is the perfect magic bullet - killing all tumor cells with minimal side effects. In practice it doesn't kill all of the tumor cells, but those cells that aren't killed act as a reservoir for the virus and they wait another month and then repeat the 5FU. After a few cycles, most or all of the tumor cells should be gone. It worked well in animal models. Too early to tell how it will work in people but this interim data looks pretty good.
Celldex presented impressive data at the SNO meeting last week. They had a 3.4 month survival advantage with recurrent GBM patients. That should be a large enough benefit to get FDA approval. They will wait until they have the results on more patients, and if it hold up to this level of benefit, will ask FDA for approval. This would be a tremendous benefit as it is a simple vaccine with minimal side effects.
This was the most important brain tumor meeting in a long time. I am working on an article about the implications it will have, (should be ready in a day or 2) but here is an article by our medical writer, Stephen Western, on the most important news from the conference.
This is historic. As far as I know, and I have been involved with brain tumors for 22 years, this is the first time a GBM trial was ever stopped early because it actually worked! Optune – the new name for the FDA approved Novocure tumor treating field device, has finally shown what it can do. This was a large phase 3 randomized trial comparing the standard “Stupp Protocol” to the standard + the Optune device. There are many ways to look at the results (see the article for the rest) but keep in mind the numbers given are from the time the trial started, not from the date of diagnosis. The trial started around 3.8 months after diagnosis. There was an increase of about 50% in the number of patients alive at 2 years after starting treatment ( about 27.8 months after diagnosis) , from 29% in the Temodar group to 43% in the Optune group. Overall survival increased by about 3 months, from 16.6 to 19.9 months after trial started ( or about 20.4 to 23.7 after diagnosis).
Dr Stupp presented the data and then said "a new standard of care for patients with GBM has been established", and "A new cancer treatment modality has been born". He called it a paradigm shift.
The last time the standard of care changed was in 2005 when Dr Stupp presented his data on using Temodar at the same time as radiation, in addition to the old way of using Temodar. That resulted in a gain of 2.5 months, from 12.1 months to 14.6 months from diagnosis.
My opinion is that this is a big step forward, with very little downside. This is the first non-toxic therapy ever to succeed in a phase III trial for newly diagnosed cancer patients. I agree it should be the new standard of care. Of course a lot more work needs to be done but we have to go with the best we have at the time and this is it. I hope to see more trials combining other treatments with Optune – hopefully also with no or low toxicities. This brings us a step closer to our goal of a cure.
Talking to many of the doctors present at the meeting, there were still a few who said they wouldn’t use it. The main objection now is that it may interfere with clinical trials. I would suggest designing trials to allow for the use of Optune just as they had to do when the standard of care changed 9 years ago. I am a huge supporter of clinical trials but if there is a choice between using Optune and having minimal or no additional side effects with a known average benefit of a 50% better chance of living at least 2 years, vs. a clinical trial which has unknown side effect profile and unknown benefit in survival, I would probably choose the Optune.
If you decide that you want to try this treatment, ask your doctor about it but if they seem resistant to the idea, go to http://optune.com to find a doctor in your area that is trained to use it and get another opinion. This treatment has been FDA approved for recurrent glioblastoma, but it can be prescribed off label for newly diagnosed. Clearly the earlier you start it the better the outcome. Hopefully the FDA will approve it for newly diagnosed GBM soon!
Disclaimer: Novocure is a sponsor of the Musella Foundation but I have never personally received anything of value from them.
The NY Times picked up the story of the Optune (Novocure Device)! I love the response of the patient when told she could stop!
Sorry about that - the last blast had an incorrect link. Pass this one around please!
One of my favorite researchers - Dr Jim Olson - launched Project Violet, which raises money for his brain tumor research. A private donor pledged $1 for every view on this music video up to $100,000. Please pass this along to your friends!
This is so sad - they always run an excellent program and it is at a great location - in Florida - just when it is getting cold up here in the north. When I first got involved in brain tumors, I did not understand the value of conferences like this, but after going to one - you see how amazing they are. It is not just the education - we can get that from our websites. There is also the social aspect of interacting with other families going through the same thing, comparing notes and swapping tips and making friends. You get unprecedented access to question the top doctors in the field.
This project focuses on DIPG and has already found new biomarkers that might be able to be used as targets for new treatments.
This is an exciting treatment and the back story on how it got started is also very interesting. Google "NOAH Protocol" for details!
This is a report on the preclinical results of this new experimental treatment. They are running a phase 2 trial of it for GBM patients. See http://clinicaltrials.gov/ct2/show/NCT02014844 for details on the trial. CytRx is a new sponsor of our website.
This supplement publishes 4 research papers on the Novocure NovoTTf100-A system. The registry data which I mentioned a few weeks ago is officially published now. Other papers include an analysis of how patients respond over time. It shows that in half of the responders, the first MRI taken after starting treatment looked worse, then started looking better 5months later. The point is not to stop therapy too soon.
Exciting new trial. I love the concept of repurposing an approved drug for brain tumors, because IF it works, anyone can use it immediately off label and we do not need to wait years for FDA approval! I wish them luck.
This brings up an interesting ethical question: Is it OK to buy your way directly into a clinical trial? At first glance, I would say no, but then upon further consideration, the trial wouldn't be able to start for anyone if those first patients didn't pay the $2 million... so it is helping others. In the past, wealthy people have approached me and have asked for help in getting unapproved treatments. My response was always that it had to be done within a standard clinical trial. They would donate money to the Musella Foundation, and I would then find a researcher willing to design and run a clinical trial of the treatment we wanted, with liberal enough entry criteria that the donor would qualify. However, it had to be open to other patients as well. This has worked out pretty good and I do not think it has the moral issue of a patient paying directly to get into a trial. There is more oversight - our medical advisory board has to approve of the project, and we are able to watch and make sure the trial really opens for others, and the money is well spent and accounted for. This has resulted in new treatments that are now being used for many other patients.
This is a clever approach but not used in people yet. Something to keep an eye on.
This is another new option, but keep in mind it is 1 patient and only a very short follow up
This is always a fun event.
