Discovery of Key Platelet Protein Could Lead to New Class of Drugs

A new study has uncovered a key platelet protein that could pave the way for new drugs designed to treat thrombosis, or dangerous blood clots.

When it comes to the human circulatory system, platelets, which allow the blood to clot, represent something of a double-edged sword: too few and a person could bleed to death as a result of even a minor injury, but too many and a clot can form, potentially blocking blood flow through the veins and arteries.

Currently, several antithrombotic drugs are available; however, some cause bleeding, which is potentially problematic given that they are used to prevent thrombosis in patients undergoing heart surgery.

"There's still room for improvement, in terms of making an ideal drug that can block platelet function without initiating bleeding," lead author Stephen Holly, an assistant professor of biochemistry and biophysics at the University of North Carolina School of Medicine, said in a statement.

For this reason, Holly and his colleagues used a screening technique called activity-based protein profiling to examine the cardiovascular system. Designed to allow researchers to track the activities of proteins operating within a cell, the method is frequently used in cancer research.

First, the team pre-screened human platelets so as to narrow the field of drug-like compounds. From there they generated an activity-based protein profile using one of these compounds to single out proteins responsible for activation.

As a result, the researchers discovered "both novel inhibitors of platelet activation and a novel enzyme involved in platelet signaling," Holly said.

This discovery of the platelets' natural "on-off" switches could be used in the development of new drugs designed to prevent the formation of pathological blood clots, the researchers explain, saying that they plan on investigating the proteins' roles in animal models before possibly pursuing clinical human trials.

"I think we're at the start of an exciting journey of drug discovery for a new class of antithrombotic therapies," Holly said.