NorthWest Biotherapeutics Inc (NWBO)

[Rkmatters]

Thanks. I think regular approval will be a given, if AA doesn't pan out. Confirmation approval may be needed for rGBM. And yes, I think they will allow recurrent patients to get the vaccine before a confirmation trial is done.

You may not be aware of this but seizure medication interferes with chemo drug metabolism, and thus reduces the effect. If DCVax-L nGBM and rGBM crossover (placebo) both mean that patients are on lower doses, then that is meaningful clinical benefit.

Dr. Pazdur added that the magnitude of a therapy’s effect on an endpoint is an important consideration in regulatory decision-making. For example, a doubling of PFS would be a more compelling result than a 15% improvement.

And

Dr. Fine observed that high doses of steroids are a major cause of morbidity in patients with brain tumors. Steroid doses are determined empirically by attempting to find the lowest dose that optimizes the patient’s neurologic function. A nontoxic drug that stabilized the vasculature and enabled patients to take lower doses of steroids would provide clinical benefit even if it had no effect on the tumor itself. He asked how a trial of such an agent could be designed to reliably capture this benefit.

Dr. Pazdur said such a trial would have to convincingly demonstrate a beneficial effect on steroid doses and on the toxic side effects of steroids. Measuring such changes consistently in an unblinded trial could be challenging.