The Promise of Personalized Medicine

11/24/20: Even though most Americans will be celebrating the Thanksgiving holiday closer to home and with fewer family members this year, one thing we are thankful for are the COVID-19 vaccines that are about to be approved for distribution. Thanks to innovative new research, scientists were able to identify and attack the virus at the molecular level by replicating its RNA. This helped not only shorten the time from “lab to jab” to months from what once took years, but also improved efficacy: the COVID-19 vaccines are showing 90-plus percent effectiveness in warding off the virus. These vaccines share the same level of efficacy as the smallpox and measles vaccines, which were both medical breakthroughs. Personalized medicine tailors treatments to a patient’s unique molecular and genetic profile, allowing physicians to select therapies or treatment protocols based on a patient’s unique cellular attributes. The approach minimizes potential side effects while ensuring more successful outcomes.

The Human Genome Project represents one of the most important advances ever made in human health. Conceptualized in 1988, the project became a reality in 2001 when the human genome was published. From 1990 through 1993, scientists mapped humans’ genetic blueprint by deciphering the human genome in three ways: first, determining the order, or “sequence” of our DNA; second, by making a map that shows the locations of genes on major chromosomes; and third, by creating linkage maps, through which inherited traits can be tracked. Genome mapping is a medical breakthrough on the scale of the discovery of germ theory in the 19^th century, which enabled scientists to link diseases to specific organisms. This led to the 20^th century development of antibiotics to treat bacterial infections and vaccines to prevent viral infections, like polio.

Many industries have shifted toward personalization is a way to address client needs. Medicine, an industry based on volume and standards, remains in the early stages of personalization. Armed with immense amounts of statistical data and case studies, clinicians have tended to focus on averages and probabilities, creating therapies for the average patient with average symptoms. Treating the averages, however, can lead to unwanted side effects and disappointing outcomes. Personalized medicine, made possible by understanding how a person’s unique molecular and genetic profile makes them susceptible to certain diseases, increases the ability to predict effective treatments for individual patients.

Personalized medicine is transformative, enabling a shift from reactive treatments to preventative therapeutics. Genome sequencing allows doctors to identify mutations in a person’s DNA that are linked to a specific disease. People born with genetic mutations often don’t exhibit symptoms for many years after birth, so predictive testing could play an important role in developing proactive therapies and treatments. For example, women with specific gene variations are 85 per cent more likely to develop breast cancer. Early genetic testing can save lives by enabling doctors to offer patients preventative measures like increased disease monitoring or risk-reducing surgery. Gene profiling also reduces the risk of complications from organ transplants by calculating the probability of rejection prior to surgery.

Pharmacogenomic tests help predict which medicines and at which doses will be most effective and safest in treating a patient, eliminating the “trial-and-error” approach to prescribing medications.  According to the Personalized Medicine Coalition, more than 5 per cent of all hospital admissions are associated with adverse drug reactions. Many adverse reactions can be attributed to gene variations that cause medicines to metabolize at different rates, resulting in some patients metabolizing drugs faster or slower than average. Adverse drug reactions account for more than 100,000 deaths per year in the United States, representing the fourth leading cause of death, after heart disease, cancer and stroke, but well ahead of diabetes, pulmonary diseases and AIDS. Designing treatments customized for a patient’s genomic composition will lead to fewer adverse outcomes.

Cell-based gene therapies are also used to combat molecular pathways associated with disease. These therapies provide long-term benefits by altering a disease’s molecular pathways. Cell-based therapies may involve replacing, repairing or inactivating a specific disease-causing gene or by introducing a modified gene into a patient’s cells to help treat the disease. The most visible examples of gene therapies are today’s COVID-19 vaccines, which were developed by exploiting the virus’s unique RNA sequence.

Customized diagnoses and treatments will likely raise health care costs as the industry moves away from a one-size-fits-all framework. The good news is genome sequencing costs have cascaded. The cost of mapping a human genome fell from $100 million in 2001 to less than $1,000 today, according to the National Institute of Health’s Genome Research Institute. The decline far exceeded Moore’s Law for computing, which postulated costs halving every 18 months.

The number of personalized medicines has increased dramatically in the last 15 years. According to the FDA, there are 431 “personalized” drugs on the market today, with pharmacogenomic biomarkers in the drug’s label. That’s up from just five drugs in 2008. Data is critical to success, both in size and scope. In an information economy, companies with the ability to gather, manage and interpret data and transform their insights into disruptive treatments will play a leading role in the health care revolution.  The benefits of personalized medicine are in their infancy. Innovative companies that employ and refine genomic therapies will dominate their industry. Please look for our genomics white paper as part of our thematic investing white paper series. Talk to your Cresset advisor to learn more about our thematic investing strategies.