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Personalized Medicine through the Genome


Why is personalized medicine so important in the age of the human genome?


Starting with a definition of personalized medicine can be useful to understanding its broader implications for biotech investors.

 "The application of genomic and molecular data to better target the delivery of health care, facilitate the discovery and clinical testing of new products, and help determine a person's predisposition to a particular disease or condition."

Proposed Senate Bill S976, Sponsored by Senator Barack Obama

"The use of genetic susceptibility or pharmacogenetic testing to tailor an individual's preventative care or drug therapy."

Nature Genetics -

"A form of medicine that uses information about a person's genes, proteins, and environment to prevent diagnose, and treat disease."

National Cancer Institute -

What each of these slightly varied definitions makes clear is that the identification of genetic information will play a role in treating and preventing disease.  From an investor's point of view it's perhaps just as relevant to realize that genetic information has been driving the selection of promising drug candidates for some time now.  Only a handful of these genetic based treatment opportunities have come to market to date.  However, this is about to change. 

Investors need to understand that the fundamentals of the relationship between big pharma (long tied to a "one size fits all" approach of drug development) and the small to mid-sized biotech companies (often on the cutting edge of the genetic discovery/targeted-drug side of drug development) will be altered forever. 

There is immense opportunity in this intersection as big pharma seeks to replace aging drugs portfolios with the best available targeted candidates already well under way in clinical development.

The longer term goal

The long range goal of personalized medicine can best be described as identifying in a preventative way the susceptibility to a disease condition and acting in a preventative way to mitigate disease and even costs of patient care.  This longer term goal is perhaps much further from fruition than is practical for near term investors in many diseases.  However the pursuit of this goal is quite relevant for the nearer term, especially in oncology, as the goals of the long term vision are already beginning to be realized in some areas. 

The right drug for the right patient

Personalized medicine is about the right treatment for the right condition in the right patient. 

It is well known that oncology treatment in general benefits only a small percentage of patients receiving the treatment.  For example, many that receive surgical treatment to remove a tumor are at little to no risk of actually having a recurring cancer.  (note: A genetic determination of risk through tumor biopsy could have eliminated these patients from additional drug therapy.)  Many patients that do receive post operative chemotherapy will therefore actually have no clinical benefit because they were not actually at further risk. 

Others patients will have no benefit because the drug will not respond due to unknown (often genetic) reasons.  (note: A genetic determination could identify the "right drug" for these patients.) 

Finally there is the handful of patients for whom the therapy is actually working to save their lives.  However in the one size fits all approach these patients were beneficiaries as much by chance as by science.

Without genetic differentiation as a predictor we basically don't know "who is who" and treat 100 people to perhaps have clinical benefit to 5 to 20. 

If science can get to the point of treating "the right 20 people" with "the right treatment" that would be incredible.  Another 25 may benefit from something altogether different.  And 55 may not even need additional treatment at all.  The savings potential in the health care system is almost immeasurable.  But instead of lots of "blockbuster drugs" there are likely to be lots of "smart drugs".

An example of a potential patient population for a targeted therapy is shown below.


Personalized medicine is already here in limited doses

Several examples of personalized medicine can already be seen in breast cancer.  These include identifying genes that indicate susceptibility to breast cancer, identifying genes that predict whether metastatic potential is high or low, and identifying genes that indicate whether a particular drug will successfully treat the condition.  These advances in breast cancer have yet to be transferred to other therapies.  Much of the untapped investor potential in these areas simply involves moving methods already underway in breast cancer to areas such as colon, lung, and pancreatic cancers.    

Genentech's Herceptin falls into the category of personalized medicine.  Herceptin is effective when breast cancer patients are over expressing HER2 (Human Epidermal growth factor Receptor 2).  The drug Herceptin specifically inhibits the HER2 protein expressed in about 20 - 25% of breast cancer patients.  Part of the FDA label of the drug requires that patients be screened for HER2 before using the drug.

Herceptin represents a necessary sea change in pharmaceutical product development.  There is a fundamental change occurring that will deliver more effective drugs to a narrower set of patients ideally suited to receive the therapy.  While personalized medicine may seem like a new and "not ready" concept to some investors it is important to recognize that the era of smart drugs actually arrived years ago with the approval of Herceptin.

In our personalized Medicine portfolio we identify key enablers in the field that stand to prosper in personalized medicine.  We also highlight the opportunities in companies taking a proactive role in delivering effective personalized medicine "targeted drugs" from conception - through clinical testing - to the bedside.  This includes companies that are focusing on patching the genetic holes of therapies that fail in certain genetic subgroups of existing therapies.

The goal of our Personalized Medicine portfolio is to provide you with the information that transforms the era of "the right treatment for the right condition in the right patient" to gains for you in the form of "the right stocks" for personalized medicine through the genome.