Replies to post #766914 on NorthWest Biotherapeutics Inc (NWBO)

[NotSure2]

IFR - case to case basis - only...

Ouch ....

Will that be enough to cover yearly expense of the company?

[Doc logic]

Chiugray,

I believe that Specials data was submitted along with scientific evidence that DC therapy, in general by nature and specifically as presented by NWBO with their activation process, is indeed tissue agnostic. The emphasis being placed on platform technology in various comments by regulators points directly to DCVax and other things in development. I actually suggested in a post a while back that the additional 700,000 pages could include documentation related to this since the proteomics data had been developed and revealed. Biomarker confirmation was also presented I believe by Dr. Linda Liau at one point and the proteomics by Dr. Bosch in a presentation shared on this board.
What this all points to is a BIG proactive move by MHRA and NHS to advance the tissue agnostic validation process. Who’da thunk it?; ). THIS would create an absolute undeniable top priority to get manufacturing up to speed (the bigger boat) not only for NWBO but MHRA, NHS, NICE, FDA, PEI and all the rest. This would also explain why there has been no delay or backlog with regard to this application because it has essentially been a rolling application waiting on Flaskworks development. I believe development is also taking into consideration compliance issues for all the various regulators even though not applied to yet. NICE has been patiently waiting for something right?; ).
There simply is no other practical way than Flaskworks to advance the concept of tissue agnostic usage without creating major problems for NWBO and regulators for not having availability ready upon approval. The way to head this off for the time being is by creating limited access which is what the NHS individual application process will help do as prioritization and distribution will take place with GBM and rGBM likely being placed at the forefront. The idea of franchising also fits into the idea of rapid deployment once Flaskworks is validated.
So what this looks like to me is similar to a Phase 4 trial. Approval for GBM and rGBM 1st with individually applied for access for all cancer types while confirmatory review of data is ongoing during the buildout and validation of Flaskworks. Best wishes.

[sharpie510]

No matter if the MHRA initial approval is for (A) new and recurrent glioblastoma, (B) grade 3 and 4 gliomas, or (C) any solid tumor cancer, demand is going to overwhelm the supply in the beginning, so it would make sense to provide DCVax-L using a benefit-based and/or treatment of last resort consideration. Likely the mesenchymal subgroup would be high on that list, as they tend to do the worst under standard of care but respond best to DCVax-L. So maybe many mesenchymal recurrent gbm patients will get the vaccine first, and recurrent patients arguably need the vaccine the soonest. Eventually, the pool of eligible patients that can have access will increase as Flaskworks automation kicks in. While some subgroups did better than others, all patients seem to benefit from the vaccine, so eventually all should be able to access it.

Dr. Keyoumars Ashkan at ASCO 2018, 51m17s:

We also have to work with the regulatory bodies, and we probably ought to do that now, right, not even waiting, because we want this to be available to all the patients. Again, while it’s very nice about these studies… yes, those patients who are in better prognostic groups are going to benefit more… but there is a benefit to all patients. We want this available to absolutely everybody.

Webcast: Dr. Marnix Bosch’s Presentation on DCVax® Personalized Dendritic Cell Vaccines from ASCO 2018

Dr. Linda Liau at the Neurosurgery Grand Rounds, 22m14s:

Interestingly, the patients who actually saw the most benefit from immunotherapy was in the mesenchymal subgroup and that's actually the poorer subgroup. Those are the ones that are idh1 wild type, MGMT methylated or non-methylated, it actually was in both populations, but those are the ones who typically have, a you know, median survival of about 14 to 16 months. So in that group, we, you know, the vaccine actually did seem to show a significant difference, and actually all our long-term survivors were in the mesenchymal subgroup. So there's something about that particular subgroup, um that you know, I think is, you know, inducing a better, uh perhaps, immune response for these patients and that's kind of what we've been studying for the last you know five or six years now.

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