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News has come from Northwest Biotherapeutics (NWBO) on its DCVax® personalized immune therapies for solid tumor cancers. The information is an announcement that Phase 1/2 DCVax-Direct clinical trial for all inoperable solid tumor cancers, which is planned to begin within approximately the next sixty days, will not be blinded. This means that the results will be seen as the trial proceeds. Knowing that an efficacy endpoint is tumor shrinkage or elimination), this trial is expected to yield meaningful ongoing results by the second half of 2013.

This innovative clinical trial is approved by the FDA for all types of solid tumor cancers (i.e., cancers in any tissues of the body), and is configured to provide rapid results.

In the Phase 1 portion: The trial will test both safety and a variety of dosing regimes, and will do so in multiple different cancers - avoiding the need to conduct separate Phase I studies in each such cancer, as is usually the case. The trial will then go to Phase 2

The Phase 2 portion: Will test for efficacy, without the need for another FDA review. The endpoint for measuring efficacy in the Phase 2 portion of the trial will be tumor regression. This endpoint is a particularly important one clinically: once tumors are established, if they cannot be surgically removed, it is very difficult to stop their growth and their spread. This endpoint is important:

From a regulatory standpoint: it is the endpoint used most frequently for FDA-granted accelerated approval.

This endpoint is a rapid one: Typically, if tumor regression is going to occur, it is anticipated to be seen within a few months after treatment - far sooner than other clinical trial endpoints such as progression free survival (PFS) or overall survival (OS).

What happened in the preclinical studies: In multiple pre-clinical studies of various cancers in animals, direct injection of DCVax-Direct into some of the tumors in each animal resulted in complete clearance of all tumors in 80% to 100% of all of the animals in the various studies - both the tumors that were injected and the tumors that were not injected, indicating a systemic immune response. Further, the tumors were cleared relatively rapidly: within weeks after the DCVax-Direct injections.

Equally as important, sixty days after the tumors were cleared from the animals in these pre-clinical studies, the animals were re-injected with the same tumor cells that readily established tumors at the outset of the studies. This time, tumors failed to establish, indicating immune memory.

"Inoperable tumors represent a severe and widespread unmet medical need," commented Linda F. Powers, CEO of NW Bio. "Some types of cancer are inoperable even as to the primary tumors, and many others are inoperable due to the spread of metastases. DCVax-Direct can potentially offer an important new treatment option, which can address many or most of these situations. It is very exciting to contemplate the potential for seeing rapid results in our DCVax-Direct trial later this year, while we continue in parallel to execute our lead program: our international Phase 3 trial with DCVax-L for brain cancer."

Prohost Impressions

We are indeed speeding forward towards more realistic approach aimed at getting important life-saving drugs as quickly as possible to the clinic to be used on various diseases, not related to one organ at a time as it has been until now. As we reiterated in the past, we need an inventory of drugs that can be ready for use when sophisticated differential diagnostic procedures, including sequencing of the cancer genome and the patients' genomes indicate that a certain drug or approach to treatment has the chance to succeed in treating the disease. This is what personalized medicine is meant to be in the first place.

In the case of Northwest Biotherapeutics DCVax® personalized immune therapies for solid tumor cancers, the choice of this immunotherapeutic approach is based on FDA conviction from preclinical trials that DCVax® might succeed in inoperable solid tumors where all available drugs have failed. Helping also could be that the firm's hypothesis that led to the development of DCVax was convincing as compared to other approaches by other firms and research institutions. This hypothesis is based on that partial activation of dendritic cells, in a certain way and to a certain degree, producing dendritic cells that are able to perform both of the two key steps needed to mobilize an overall immune response attacking the cancer. Neither immature dendritic cells nor fully mature dendritic cells were able to do the same.

This is what translational medicine is all about, i.e., taking the molecules from basic science to the clinic "from bench to bedside" as fast as possible.

The reform that's taking place in the drug approval processes will save huge amounts of money, which has been unnecessarily spent by the firms at the expense of shareholders. Risk was inflated, considering that after years in development, the drug might fail, devastating the drug developing firms and investors in them.

Regardless of the results, this is good news. In the case of Northwest Biotherapeutics' DCVax, good news is that the trials will not be blinded and everybody will know the outcome as soon as in a couple of months.

Are we in the future? You bet we are and the best is yet to come.

We still own no shares in NWBO.

Disclosure: None

Source: Northwest Biotherapeutics: An Example Of Reform In The Drug Approval Process