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This H3 K27M mutation is a marker for the worst prognostic group of brain tumors, and as far as I know, this is the first drug in clinical trials to target this mutation. Aside from being present in some of the worst GBMs, it is present in most DIPG tumors.
This is a gene therapy that is showing some impressive results in an early trial!
I love combinations like this. I believe that it might be impossible to cure GBMs with just a single approach.
This shows that the convection enhanced delivery methods have improved to the point where they can get the drug, on average, to over 75% of the tumor, and it should only get better with more experience. They compare this to the PRECISE trial where the drug only got to about 20% of the tumor.
Here is a link to the poster.
There is a phase 2 clinical trial going on now for this treatment, MDNA55, in patients with recurrent or progressive GBM.
Disclaimer: Medicenna Therapeutics is a sponsor of the Musella Foundation
I am a fan of this drug, which hopefully will be shown to work on unmethylated MGMT patients as well or better than Temodar works on methylated MGMT patients. However, this report is a little too early to tell. Hopefully will have more details at the next SNO meeting.
There is a dose response curve. (Which also should convince the naysayers that the device is effective) The better the compliance, the better the patients do. They have been saying 75% is the target compliance rate but the new data shows that 90% should be the target. Those patients who had a compliance rate of 90% or more, had a survival rate of 29.3% at 5 years, compared to the control group (temodar) which had a 4.5% survival rate, an almost 650% increase in survival rate.
With a compliance of 70-80%, the 5 year survival rate drops to 19.9%. Still much better than the control group... so don't stress too much if you can't hit the 90%, but try.
These results were disappointing. Last year, the data was presented that this combination worked very well. This year they are saying that although there is a a relatively large improvement in progression free survival (from 1.5 months with the Lomustine (CCNU) alone to 4.2 months with the combination of Lomustine and Bevacizumab (Avastin), there was little (less than a month) improvement in overall survival.
This showed no benefit of adding Gliadel when 5-ALA was used to guide the surgery. Surprising results.
A 29.3% 5 year survival - on a large number of patients in a randomized phase 3 trial is very impressive. This shows a dose-response effect, which proves that the concept actually works. When comparing the median survival, remember that this is from the time that the patients were randomized, not from diagnosis - the difference is almost 4 months. This means that the median survival is almost 29 months, which is a big step forward!
I went to an impressive presentation by Tocagen today at the SNO meeting! They have a few long term survivors who not only are survivors, but have been able to return to work and lead productive lives. The phase 3 trial opened yesterday.
Thanks to Cure Magazine, Novocure, Dr Sumrall, as well as my wife and kids - without whom I wouldn't have been able to do this!
I am proud to say that I am the patient advocate on the board for this program! This is a brilliant group of researchers who deserve this recognition and will put the grant money to great use to speed up the search for the cure!
Unfortunately, the combination did not do any better than Avastin alone
Impressive results. 20% complete response rate form a single injection of this drug, and no serious side effects. This is the type of treatment that should be approved immediately.
This is a new way to target brain tumors. We are proud to say the Musella Foundation was one of the sponsors of the research.
This blood (and spinal fluid) test can help diagnose and monitor the response to treatment for these devastating tumors. There are drugs under development which target the marker mentioned, so this blood test might be able to be used to determine which patients get these new drugs, without the need for biopsies. Amazing stuff. And we are proud to say we are one of the sponsors. Your donations at work!
This mutation is a bad prognostic sign, but they have a 15 year survivor on this medication.
I love this model. Combine it with our proposal to speed up access to experimental drugs and we may have a cure much sooner than we thought!
It is a good start, especially the part about tracking treatments after approval, but if they were serious about finding the cure they would incorporate earlier approvals as outlined here. That would cut the time and costs by an order of magnitude.
