Monday, April 23, 2012

NOT in biotech's robust future

The main theme of this blog is that massive innovation currently underway in the biotech industry will improve health care globally and create exciting businesses. The hallmarks of this "molecular future" will be personalized medicine, genomics, and broad digitally-enabled technologies, such as DNA sequencing, arrays, and multiplexed assays.

But today - as a follow-on to last week's Top 10 Promising Cancer Drugs post - let's talk about one biotech 'innovation' that won't be powering growth or improving outcomes: cancer immunotherapy.

FierceBiotech collected the comments spawned by their original Top 10 article, and there was a surprising amount of positive commentary in support of cancer immunotherapies. (Sometimes referred to as vaccines.)

As bullish as I am on the "molecular future," I have the exact opposite feeling for cancer immunotherapy - partially due to my experience working at a cancer immunotherapy company in the late 1990's.

The concept of cancer immunotherapy is very appealing - train or otherwise get the patient's immune system to recognize and respond to a cancerous tumor as foreign, thereby empowering a non-toxic immune response. And, indeed, this has been shown to happen in various studies, dating back more than two decades.

An effective cancer immunotherapy could be extremely targeted with limited side effects. But the immune system is far, far more complex than anticipated and frankly humbling to most researchers. It is seductively simple to conclude that all one has to do is pick the right antigen to stimulate an antibody response (as in many immunological disease), but there are apparently many biological holes in this theory special to cancer. (For one, we're learning that cancer tumors are not homogeneous.)

Here's my rationale for immunotherapy skepticism:

1) There is a long track record of failure in this area.
2)  the core thesis is still lacking validation.
3) big pharma has virtually no investment in this research area
4) the regulatory approval path is not optimized for cancer vaccines.
5) cancer immunotherapy research is asset-intensive and effort-intensive. Pursuit of autologous therapies is particularly labor intensive. In contrast, 1 informaticist or 1 medicinal chemist is enough to launch a discovery/development program.
6) what little commercial effort in the area of cancer immunotherapy is being conducted by micro-cap companies. (I don't mean to denigrate any company successful enough to go public, but there is a load of history confirming that companies of this size and scale just don't have the assets/resources necessary to conduct pivotal research.)

In essence, cancer immunotherapy research has the challenge solving of incredibly complex disease biology using the an incredibly complex modality. In effect, cancer immunotherapy equals the complexity of cancer treatment squared, though with less pharma support.

Case in point: CEL-SCI (CVM). While spun out from the very credible Max Planck Institute and built on attractive scientific rationale, CEL-SCI has been chasing an immunotherapy solution ("Multikine") since 1983. Nearly 30 years later, CEL-SCI is a micro-cap, with a market valuation of ~$110M and likely insufficient resources to complete their Phase III trials of Multikine.

I really, really hope that CEL-SCI or the other cancer immunotherapy companies are ultimately successful, but my perspective is that there are more promising oncology technologies for pursuit and investment.

It should be pointed out, though, that there is an FDA-approved cancer immunotherapy - Dendreon's ProVenge for prostate cancer. Dendreon received FDA approval in 2010, but market response to date has been limited. It is unclear if the market's response to ProVenge is driven by technical skepticism, or by the complexities of creating and delivering an autologous vaccine.

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