"Instead, the real goal is to improve and extend the patient's life." Flipper
I agree with you and I'm sure the FDA agrees with you as well as all clinicians, oncologists, and of course patients. The problem is that using overall survival for a primary end point will be far more costly and time consuming for many cancer indications to be used in an early pI or even pII trials for a treatment that is new and unproven. Therefore, as you already know, the need for surrogate endpoints that are accepted to CORRELATE to OS advantage are used esp for AA approval. The problem with not accepting these surrogate endpoints for dc vaccines as not being appropriate (which may be true) is that CI and most likely other therapies are still using those surrogate endpoints successfully in early trials to justify a more expensive pIII to look for that correlation to OS advantage. This is what Direct needs to show today to justify to the world (and big pharma) that this treatment is worth partnering to do combo treatments. The paper that Ohbutz brought up for Dr. Coukos shows that combo treatments for dc vaccines can show ORR results using Recist and justify looking into it in a bigger pII/pIII trial.
Funny thing is that I believe it was Ohbutz who also brought up a paper/abstract from a Asian grp that did a randomized pII trial to show that Dc vaccines produce a long survival rate compared to control grp (not historical but an actual comparator grp) to prove to me that DC vaccines work in GBM. The funny part is that paper as I pointed it out to him showed that PFS data showed NO CORRELATION with that large OS survival advantage (that is the PFS had no difference between the two grps that had any SS). Now what if some grp were to tweak dc vaccines or any other cancer vaccines so that BOTH PFS/ORR AND OS results were measurable and SS. That would be the ideal result for any lab/company esp for an early pI study (sort of like what IMUC and NWBO did in their pI trials in GBM w/ phenomenal success- actually I shouldn't say IMUC or NWBO but the actual individual research grp who did the initial studies which in NWBO's case was Dr. Liau in UCLA) One of my concern that I brought up a while back is that when IMUC and NWBO both tried to scale up the lab production of DC to a commercial scale, something may have changed to alter the effectiveness of the dc vaccine to be significant enough to affect esp the PFS endpoint. But this is conjecture at this point and there are other more plausible reasons why IMUC's pII trial didn't replicate their pI results. AND I still believe dc vaccines work! But it is not a simple treatment which may be one reason why big pharma shies away from dc vaccines.
I really didn't want to bring any of this up again since it has been in the past discussed on this MB but Ohbutz just had to bring this issue up again. I'll just simply wait for the ASCO presentation to look over tonight. GL
