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Personalized Medicine (also known as Theranostics) is the integration of therapeutics and diagnostics. Is the great promise of personalized medicine finally on the brink of acceptance and real recognition? We believe so, although it remains an uphill battle for several reasons. An article as recently as August 20, 2008 in the Wall Street Journal bares testament to this emerging trend in therapy,

Pharmacy-benefits manager Medco Health Solutions Inc. has entered into a partnership with the Food and Drug Administration to study the role of patients' genetic makeup in prescribing medications, a collaboration aimed at expanding the scientific evidence for "personalized medicine.

Medco said it will derive its reports from clinical settings, examining, for example, whether physicians will be willing to change dosages based on a genetic test result.

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But before exploring bumps in the road, what is personalized medicine, really? Over the past several years, our understanding and knowledge of the human genome, proteome, and metabolic pathways has grown enormously, giving rise to the understanding that a patient’s unique genotype may respond differently to a drug or therapy than what is perceived as the norm. Medicine has come to realize that all solid tumors are not alike even though they may be found in the same organ such as the breast. This specific and individual information can be used to tailor a patient’s treatment by either eliminating therapies that might not work at all or by altering the amount of drug or therapy that is administered, thus reducing side effects. Most drugs and devices are approved using large cohorts of patients and it is widely known that their response to the drug or therapy varies widely. It is estimated that most drugs only work, to some degree or another, in 60% of patients.

Plavix is a good example. As the second most prescribed drug in the world (25 million prescriptions in 2007 and $8.5 billion in revenues), Plavix is widely prescribed for patients that have experienced heart attack, stroke, or other vascular events and who are believed to be at high risk for a recurrent event caused by a blood clot. However, approximately 30% of patients taking Plavix do not metabolize the drug because of genetic variation thus receiving little or no benefit. Bristol-Myers Squibb (NYSE:BMY) co-markets Plavix with Sanofi-Aventis SA (NYSE:SNY) of France. As we stated in an article we wrote for our blog on January 8, 2009, we believe the FDA will mandate PCR (polymerase chain reaction) based tests to detect the genetic variations that renders Plavix ineffective.

Personalized medicine has for the most part has been dominated by large companies that have had measured success in developing and getting approved specific genetic tests. Some examples include Roche’s (OTCQX:RHHBY) AmpliChip that predicts a patient’s response to therapies; BRCA1/BRCA2 test for breast and ovarian cancer risk; Monogram’s (MGRM) Trofile for HIV tropism; Bayer’s (OTC:BYERF) Trugene HIV tests and DAKO’s Herceptin Hercep genotyping test.

But personalized medicine is now taking a much more varied path with more specialized companies playing a role. Some of these companies offer platform technologies upon which can be developed a family of diagnostic tests while others provide specialized diagnostic lab services upon which treatment decisions can be customized while still others are developing tests for personalized point of care testing. A few of the more notable examples are CombiMatrix (NASDAQ:CBMX), Rosetta Genomics (NASDAQ:ROSG), VirtualScopics (NASDAQ:VSCP), Clarient (CLRT), and OraSure Technologies (NASDAQ:OSUR).

  • CombiMatrix- The company is the world leader in DNA Array Diagnostics with more products available in the market than its competitors’ combined product offering. Through the development of proprietary technologies, products and services, CBMX is a highly diversified company that participates in several rapid growth markets including genetic analysis, molecular diagnostics, drug development, nanotechnology research, defense and homeland security.

    In addition to being the world leader in developing DNA Array Diagnostics, the company is utilizing these tests to rapidly expand its Diagnostic Reference Lab business. The company provides reference lab services for prenatal scanning, genetic abnormalities, autism, HER2 gene status for breast cancer, HemeScan for Chronic Lymphocytic Leukemia, and a prostate cancer screen for several genetic tumor markers that enable a more precise stratification of the risk profile of the patient.

    CBMX recently reported very positive preliminary data on its investigational Comprehensive Cancer Array (CCA) test showing it can non-invasively screen for the early detection of cancer. The study focused on prostate, colon, ovarian, breast, and lung cancers, as these five cancers comprise roughly 85% of all solid tumors in the United States. Preliminary data using serum from cancer-free patients and patients with cancer at various stages (stage 1 to stage 4) were presented. CombiMatrix used a comprehensive micro RNA (miRNA, a recently discovered type of nucleic acid) array built on its CustomArray(tm) platform to perform this study.

    The study concluded that the miRNA expression patterns, in blood, for patients with cancer (including early stage 1) were dramatically different from patients who were cancer-free. The resulting analysis indicated that a clear distinction could be made between patients with cancer and those without.

    We believe this will lead to a screening panel for the aforementioned cancers with the obvious benefit to the patient of earlier intervention than has been the current standard of care.

  • Rosetta Genomics- An Israeli company, Rosetta Genomics is a developmental stage company also involved in micro RNA, used both as diagnostics and therapeutics. The company reported its firs revenues recently but more importantly, the first-ever study, showing in-vivo systemic a demonstration of a micro RNA therapeutic in liver cancer, with a two-fold decrease in tumor-Mass and a dramatic suppression of the targeted micro RNA. The study was done in mice. Liver cancer is the third leading cause of cancer death and affects 19,000 new patients in the U.S. annually. Unfortunately, the five-year survival rate is only 10%.

The company provides three diagnostic tests based on miRNA technology. miRview(TM) mets - This test can accurately identify the
primary tumor site in patients presenting with metastatic cancer, as well in patients with Cancer of Unknown Primary (CUP). miRview(TM) squamous This test differentiates between two subtypes of non small cell lung cancer (NSCLC). This diagnosis is important as some NSCLC patients have demonstrated varying response patterns. miRview(TM) meso - This test differentiates mesothelioma, a cancer connected to asbestos exposure, from other carcinomas in the lung.

  • VirtualScopics- Presently, VirtualScopics is a contract research organization serving the pharmaceutical and biotech industries in a very unique manner. The company provides 3D analysis of solid tumor images with proprietary software that can detect very small changes in the size and shape of a tumor. The semi-automated systems measure and monitor disease progression and changes in biological processes over time. This process provides highly reproducible results, enabling us to measure minute changes in the structure and function of various biological structures and functions, both rapidly and with a high degree of confidence. Drug development time lines can be dramatically shortened by more quickly being able to determine if a drug candidate is effective in reducing tumor size.

    While VSCP is building a very nice business in this highly differentiated technology and now has relationships with 12 of the top 15 pharma/biotech companies in the world, this technology will eventually be used to determine if a specific drug is effective in treating a patient, a determination which can be made in as little as 24 to 48 hours after administration. The company also announced its involvement with a consortium of cardiovascular companies studying the ultrasound and MRI visualization of coronary plaques. These are examples of very attractive diagnostic opportunities that will result in more personalized medicine.

    VirtualScopics reach operating cash flow breakeven in the fourth quarter and announced a backlog of $27 million in revenues on a base of $7.1 million achieved in 2008.

  • Clarient- Clarient, Inc. operates as a diagnostics services company in the United States. It provides cellular assessment and cancer characterization to community pathologists, academic researchers and university hospitals, and bio pharmaceutical companies. The company provides a wide variety of diagnostic tests based on immunohistochemistry (IHC), flow cytometry, polymerase chain reaction (PCR), fluorescence in situ hybridization (FISH), and In situ hybridization (ISH).

The company also offers third party billing, client billing, and patient billing arrangements services, as well as provides cancer diagnostics and consultative services, ranging from technical laboratory services to professional interpretation. In addition, it provides commercial services to biopharmaceutical companies and other research organizations to assist their efforts, ranging from drug discovery to the development of directed diagnostics through clinical trials.

It has collaboration agreements with Natural Selection, Inc. to use genomic mathematic capability to supplement proteomic mathematic capability to help develop novel cancer markers, as well as with Prediction Sciences, Inc. to commercialize a novel breast cancer test. In January 2008, Clarient announced a strategic partnership with CombiMatrix to market and sell a novel genomics-based cancer test called HemeScan, a comprehensive test related to the treatment and care of chronic lymphocytic leukemia (CLL), among other cancers. The HemeScan test was developed by CombiMatrix.

  • OraSure Technologies- An established company, OraSure engages in the development, manufacture, and marketing of oral fluid specimen collection devices primarily in the United States and Europe. The company’s principal products include OraQuick ADVANCE HIV-1/2, a point-of-care test for antibodies to the human immunodeficiency virus (HIV) type 1 and type 2; OraSure, an oral fluid collection device for the detection of antibodies to HIV-1 in an oral fluid sample; and Intercept, which is an oral fluid collection device along with nine related immunoassays and used for oral fluid drugs of abuse testing.

But of most interest is the highly anticipated FDA approval for the first OTC HIV test to be released to the general public. This will make it extremely easy for anyone to test his or her HIV status, a truly important advance in personalized medicine.

So what are the impediments to a wider adoption of personalized medicine? We sense a reluctance on the part of big pharma to encourage personalized medicine. After, all pharmaceutical companies are most interested in developing “blockbuster” drugs, such as Plavix, and its doesn’t do their business very much good to admit that a drug doesn’t work in 30% of the population and oh by the way, there is a way to test if it is effective. What we believe will be the major drivers of personalized medicine in our opinion are a mounting body of knowledge that it produces better medical outcomes and a growing realization on the part of payers that in the long run, it will reduce the cost of care by eliminating unnecessary and ineffective treatment.

Disclosure: no positions

Source: Winning Stocks in the Age of Personalized Medicine