Scientists Reviewing Results on Computer Screen

An RNA-based therapy is developing a way to stop advancement of life-threatening conditions — a biomedical research disruption that could change the industry altogether.

Innovation and disruption

Health Care Disruption: More Than Just Care and Payment Models

  • Rob Schile
  • 2/27/2019

When we think about change and disruption in health care, we often focus on ACOs, new bundled payments, and the latest reimbursement changes coming from The Centers for Medicare and Medicaid Services (CMS). Outside of care and payment models, our attention turns to new disruptors entering or expanding into health care, such as Amazon, Berkshire Hathaway, Walmart, and CVS.

But there is one area of change and disruption that seemingly receives little attention: biomedical research, and the long-term impact it can and undoubtedly will have on the delivery of health care. 

In August 2018, the Food and Drug Administration (FDA) approved a new, first of its kind RNA-based therapy called Onpattro (patisiran) that targets a rare, but often life-threatening, nerve disease called polyneuropathy. Outside of a few press releases, there seemed to be little fanfare.

Onpattro, and other similar new therapies under development, are capable of stopping the advancement of life-threatening conditions — once diagnosed, a patient’s course of treatment would be no more complicated than receiving periodic injections that would enable them to live normal lives.

Admittedly, this is a world in the distant future. But as scientific research continues to advance these therapies, perhaps that future is not as distant as one might think.

RNA therapies origin: The Human Genome Project

In order to understand the power and potential of these therapies, it’s important to take a step back and understand how they started.

The advancement of RNA therapies is rooted in the Human Genome Project, which in 2003 revealed for the first time ever the sequencing of genes in the human body. To break it down, a “genome” represents a living organism’s complete set of deoxyribonucleic acid, or DNA. This genome contains all of the information an organism needs to reproduce new cells and continue to live.

The Human Genome Project discovered how to sequence the DNA in the human body with a 99.9 percent accuracy rate. Think of the project results as a blueprint for the human body. Understanding this blueprint has enabled scientists to study and learn more about gene function in the human body, helping them to ultimately gain deeper insights into drivers of disease and understand why certain diseases respond or fail to respond to current-day treatments.

Our DNA carries the code for cell development. This code is then transferred to RNA (or ribonucleic acid), which exists in all living cells and is responsible for creating protein during new cell development. In other words, our DNA holds the message of our cell structure, and RNA serves as the carrier of this message as it goes throughout our body building new cells. If there are codes for the development of certain diseases, this code is transferred from our DNA to RNA.

Since discovering this connection, researchers have been able to experiment with different types of interventions in the messaging process to interrupt the creation of diseases as cells go through synthesis in our bodies. This interruption process is referred to as RNA interference (RNAi) therapies.

Onpattro shows the possibilities of RNA therapies

Onpattro (patisiran) is an infusion RNA therapy developed by researchers at Alnylam Pharmaceuticals, Inc. The therapy targets a rare life-threatening nerve condition known as hereditary transthyretin-mediated amyloidosis, or “hATTR.” According to The U.S. Food and Drug Administration (FDA), “It [hATTR] is characterized by the buildup of abnormal deposits of protein fibers called amyloid in the body's organs and tissues, interfering with their normal functioning. These protein deposits most frequently occur in the peripheral nervous system, which can result in a loss of sensation, pain, or immobility in the arms, legs, hands and feet. Amyloid deposits can also affect the functioning of the heart, kidneys, eyes and gastrointestinal tract.”

Until the development and approval of Onpattro, hATTR treatment focused on managing the symptoms of the disease, similar to many current treatments for complex life-threatening diseases. With Onpattro, however, the development of amyloid can be interrupted to ultimately reduce the accumulation in the body, thus preventing the harmful impact the disease has on critical organs and corresponding nerve damage.

When DNA sends the cell development message, Onpattro “attaches” to the RNA and interferes with (RNAi) the creation of the abnormal protein fibers. The remainder of the cell production process continues unharmed, allowing good proteins to develop, while impeding or halting the bad protein development.

The science that enables drugs like Onpattro to work is obviously more complicated than explained here, and is the result of decades of research and testing. However, the advancement of this science, and the potential impact it can have on disease formation, is nothing short of amazing. Wide scale application of these kinds of discoveries could mean life threatening diseases that once required complex surgical interventions, extended hospitalizations, and other expensive medical care could now be prevented in minutes with an injection in a physician’s office. This type of care delivery is obviously in the future, but many believe researchers are on the cusp of significant breakthroughs after decades of incremental progress. As these therapies progress, they will bring considerable disruption to health care as we know it today.

National Institute of Health makes connections

How quickly this science continues to develop, and the rate at which the FDA grants approval for additional therapies, remains to be seen. What is clear is the momentum behind it has been picking up steam for several years, and there is a significant amount of effort and coordination for new discoveries in place.

For example, the National Health Institute (NIH) has created the National Center for Advancing Translational Sciences (NCATS), which operates a state-of-the-art RNAi screening facility. According to NIH, some of the key barriers in advancing RNA therapies has been due to:

  • Lack of expertise to perform genome-wide RNAi screens
  • Lack of methodologies to interpret screening results accurately
  • Lack of a comprehensive data base that researchers can access for reference

The NIH’s NCATS serves as the conduit to address these issues and provide assistance to researchers every step of the way. Today, researchers have access to libraries of specific types of RNA therapies for more than 65,000 sequences that target more than 20,000 human genes. The ultimate goal is to expedite the potential usefulness of RNAi type therapies so that new treatments and cures for disease can be delivered to patients in need more rapidly.

How we can help

The FDA’s approval of Onpattro, and the promise that this drug and similar scientific discoveries have shown, illustrate the potential for sweeping change to health care as we know it today. Admittedly, it is difficult to plan for events that are still in the early phases of discovery, especially when there are significant near-term changes that health care providers need to be prepared for. However, it is important to not lose sight of advances in science as new breakthroughs are discovered.

As we continue to monitor, debate, and adjust for new care and payment models, we must not overlook how these types of discoveries could transform the care delivery system from management of symptoms of diseases to eliminating them.

Stay informed and up to date on these types of discoveries by connecting with CLA health care professionals. CLA’s integrated approach to research, coupled with our deep understanding of the health care industry, uniquely positions us to provide you with the critical insights to help you prepare for the future.

Discover more ways disruption is impacting health care by exploring our Health Care Innovations and Insights Blog HI2.