Passage of the 21st Century Cures Act and its Potential Implications on Biodefense

Last week, President Obama signed the 21st Century Cures Act into law.  The $6.3 billion bill establishes new rules that direct the United States Food and Drug Administration (FDA) to approve drugs and devices with greater urgency.  Specific to medical devices, the Act requires the FDA to establish a priority review program for “breakthrough” devices, or for devices that target diseases for which no FDA-cleared or approved alternatives are available.

A significant void in healthcare is the lack of FDA-cleared therapies to defend against infectious pathogens that emerge naturally or are created by man as agents of bioterrorism.  At present, U.S. citizens are naked against most of these threats.  As an example, only a small number of the 300+ viruses known to be infectious to man are addressed with an FDA-cleared drug or vaccine.

With the Cures Act now signed into law, the incoming Trump administration has an opportunity to advance the broad-spectrum medical countermeasure goal set forth by the U.S. Department of Health and Human Services (HHS) 2015 Public Health Emergency Medical Countermeasure Enterprise, otherwise known as PHEMCE.  If achieved, the PHEMCE broad-spectrum goal would spur the advancement of innovative therapeutics to defend against the many pathogens that are not addressed with traditional disease-specific drugs or vaccines approved by the FDA.

At Aethlon Medical, we are advancing our Hemopurifier® to fulfill the HHS broad-spectrum objective.  As a medical device, we believe the Hemopurifier is the first therapeutic candidate of its kind to be advanced in FDA-approved studies. Based on previous pre-clinical and human clinical outcomes, we also believe that our Hemopurifier is a leading broad-spectrum countermeasure (additional details at the end of this note) against virulent viral pathogens that remain beyond the reach of drug and vaccine therapies.

In the case of pathogen threats, there is often an assumption that sufficient government spending will yield drug and vaccine cures.  However, our government has spent an estimated $80 billion since 2001 on biodefense initiatives, yet just one of 13 viruses classified as “Category A” are addressed with an approved therapy.  “Category A” pathogens are biological threats that pose the highest risk to national security and public health.  In the meantime, a confluence of global warming, urban crowding and global travel are expected to fuel increased occurrences of pandemic outbreaks in the future.

Leveraging the Cures Act to advance the HHS broad-spectrum objective should be a priority initiative for the incoming administration, as historic attempts to align a disease-specific drug or vaccine with a pathogen threat have proven to be an immensely challenging feat that fails far more often than it succeeds. While a safe and effect vaccine would provide the greatest protection against a virulent pathogen, we recognize that it is statistically improbable for such a disease-specific countermeasure to be developed, proven effective, manufactured, and then delivered within a time frame necessary to combat an emerging life-threatening pathogen.   In the case of highly virulent pathogens, it is often not feasible to conduct controlled human studies that are necessary to demonstrate the benefit of a disease-specific drug or vaccine.  As pathogen outbreaks are also unpredictable, there is limited commercial incentive for researchers to develop disease-specific therapies prior to an outbreak.

Ebola and Zika virus are recent examples of pathogen threats that were known to be infectious to man for decades. Yet, when they suddenly emerged, no proven antiviral drug or vaccine was available.  The broad-spectrum goal of HHS offers to inspire therapeutic innovation that could overcome the challenge of aligning a therapy with each and every pathogen threat.  Instead of inhibiting the replication of a specific pathogen, broad-spectrum therapies would stimulate or protect the immune system’s ability to combat a wide range of pandemic infections.   In the treatment of cancer, similar strategies are already being advanced through the development of immuno-oncology devices and drugs.

Then, there is the issue of bioterrorism.  Last month, the President’s Council of Advisors on Science and Technology (PCAST), which consists of 18 scientists and policy experts in various disciplines, issued a letter to President Obama to recommend the implementation of new countermeasures to deal with artificially engineered pathogens as they have the potential to be even more dangerous than naturally occurring pandemic threats. PCAST was especially concerned with the emergence and availability of CRISPR gene-editing technology, which provides a platform to alter and enhance a pathogen to more lethal. The PCAST recommendation further reinforces the immense value that a broad-spectrum countermeasure could have in protecting U.S. citizens.

The recently published book “Pandemic: Tracking Contagions from Cholera to Ebola and Beyond,” cites a survey by Dr. Larry Brilliant in which 90 percent of epidemiologists predict that a pandemic event will sicken 1 billion individuals, kill up to 165 million, and trigger a global recession at some point in the next two generations.   While such a prediction may seem dire, an outbreak of similar magnitude did occur in the last century.  In 1918 and 1919, a virulent strain of influenza virus was estimated to have infected one-third of the world’s population and killed an approximate 50 million people.  Since that outbreak, the world population has grown from 1.8 billion to 7.4 billion individuals.

We applaud the passage of the 21st Century Cures Act and encourage the incoming administration to leverage the mandates of the Act to advance the HHS broad-spectrum vision before the next large-scale pandemic.