Novel Screening Model for Adverse Drug Reactions

Market Overview:

The U.S. FDA Adverse Events Reporting System (FAERS) has reported steady, exponential increases in both serious patient outcomes and mortality over the last decade (Figure 1). Adverse drug reactions present a unique challenge because human drug metabolism varies dramatically among individuals, and animal models are not always predictive of human response to new drugs.

Clemson researchers have developed a novel screening tool for testing drug interaction with a variety of human gene polymorphisms. The cytochrome p450 gene family (CYP) is estimated to be involved in 75% of all metabolic reactions within the human body, and the CYP enzymes are the major player in drug metabolism. The new screening tool enables silencing (or knockdown) of many animal CYP genes simultaneously, by targeting an array of specific mRNA sequences. Because CYP gene number varies among animal species, this multi-gene knockdown enables replacement of an animal CYP gene family with the equivalent human genes—potentially making an animal model more predictive of human drug response.

For example, the technology might permit creation of off-the-shelf humanized mice for CYP gene variants. These animals may be compared to control animals as a screening tool during late-stage drug testing, permitting manufacturers to inform healthcare providers in advance of potential patient reactions. Apart from its use as a screening tool for providing personalized medicine, a CYP-silencing technology may also have merit as a candidate for antiviral, antibiotic, and gene therapy applications.

Applications:

· Personalized healthcare

· More predictive model than other animal models

· Screening tool for adverse drug reactions

· Development of antiviral and antibiotic drugs

· Gene therapy for metabolic diseases

Benefits:

· Permits healthcare providers to reduce preventable causes of death and injury

· Enables improved treatment efficacy

· May permit early detection of costly recalls within pharmaceutical industry

· Reduce cost of treatment by getting it right the first time

Inventors: William Baldwin