Thematic Investing Series: Precision Medicine

Timely insights from portfolio managers and industry experts on key financial, economic and political issues.

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      By Will Glindmeyer, Ph.D.Equity Analyst, Growth Team, Eaton Vance Management

      Boston - The Human Genome Project, completed at the turn of the 21st century, was an enormous research study involving scientists and clinicians across the globe that detailed the first nearly complete sequence of human DNA. This genetic blueprint provided the foundation to understand how genes and proteins function and interact with each other, allowing us to begin to understand how various mutations and alterations of DNA impact our health. As we furthered our understanding of the different biological mechanisms that cause many diseases, scientists around the world have designed individualized medicines that take into account each patient's unique genetic variations - generally known as precision medicine.

      Declining genetic testing costs have transformed medicine

      Precision medicine-based approaches have now entered modern treatment paradigms and are often used in clinical practice. The ability to diagnose many diseases through genetic testing - then follow up with drugs that are particularly efficacious for certain mutations - has considerably expanded the range of therapies to include once-untreatable conditions that had been too difficult to target. This increased adoption, along with more efficient sequencing techniques, has led to a rapid decline in the cost of sequencing.


      Cancer treatment has evolved thanks to precision medicine

      Notable examples of precision medicine are found in the treatment of cancer. No longer limited to approaches such as chemotherapy and radiation therapy applied by trial and error, physicians can employ precision medicines targeted to the root cause of many tumors identified through genetic diagnostics - contributing to significantly better treatment outcomes and life expectancy.

      A noteworthy example is Keytruda, a therapy targeted toward PD-1 protein expression in many types of cancer. Its impact is most prominent in non-small cell lung cancer where it improved patient survival when added to chemotherapy regimens over chemotherapy alone in clinical trials.


      Next generation therapies may cure fatal diseases

      Cell and gene therapies are emerging fields in personalized medicine that use human cells or genetic material for therapeutic applications. For many rare diseases triggered by a single genetic mutation in a cell type or body tissue, research has delved into how we can correct, repair or replace DNA in that area. While older stem cell therapy treatments are relatively common in modern medical practice, the ability to target diseases with a therapeutic designed to treat a person's mutation allow far greater efficacy and safety.

      A major example is in the gene therapy Zolgensma, targeted for patients who are diagnosed for spinal muscular atrophy through genetic testing, which dramatically improves the survivability of infants versus standard of care.


      Government policy has facilitated the development of personalized medicine

      There have also been significant government policy changes to accommodate the emergence of precision medicines. The development of these medicines has been greatly aided by the Food and Drug Administration (FDA), which created a series of methods for accelerating these drugs to market. In addition, there has been a significant decrease in the number and size of clinical trials needed for approval. The 2015 Precision Medicine Initiative also prioritized research and development in the field, with a short-term goal of discovering new cancer treatments and a longer-term goal of expanding general disease knowledge.

      Creating a boom in the biotechnology industry

      Both large and small biotech companies have geared their in-house R&D activities toward precision medicine, with a corresponding increase in their funding in these areas. Furthermore, academic labs that had been working on their own programs have spun out companies or licensed their technologies to established firms. Many of the smaller companies that have demonstrated strong clinical data and gotten drugs approved have been acquired by the more established companies, creating a positive feedback loop.

      Bottom line: Improving our understanding of the genetic mechanisms behind a range of diseases has expanded therapeutic possibilities, leading to new opportunities for biotechnology companies and potentially better performance for investors in healthcare.