LA JOLLA, Calif., March 4, 2013—Sanford-Burnham Medical Research Institute (Sanford-Burnham) and 60° Pharmaceuticals, LLC, have entered into a partnership to test furin, a human proteinase, as a drug target for the treatment of dengue fever, one of the most common infectious diseases in the tropics and subtropics. 60° Pharmaceuticals, a philanthropic-for-profit company focused on neglected and rare diseases, agreed to provide funding to Sanford-Burnham for the first phase of research to explore inhibitors of furin.
Since viral genomes are too small to encode every protein needed for their survival, they take advantage of proteins in their human hosts. For the dengue virus and numerous other viruses—such as West Nile, Ebola, or yellow fever—furin is a vital host protein. For this reason, these viruses can only attack human cells that produce furin. Scientists at Sanford-Burnham are now trying to find a way to inhibit furin, making it impossible for the dengue virus to exploit and "hijack" a host cell.
"We're working on a new small-molecule furin inhibitor. It's a challenge to design, but could solve the toxicity problem that plagues existing viral inhibitors," explains Alex Strongin, Ph.D., professor in Sanford-Burnham's Infectious and Inflammatory Disease Center.
During the first phase of the research partnership, Drs. Alex Strongin and Maurizio Pellecchia and their laboratories at Sanford-Burnham will identify the most promising lead candidate from multiple small-molecule compounds they have already synthesized and tested in cell-based tests. They will evaluate each compound's absorption and distribution in the body (pharmacokinetics), as well as toxicity and efficacy in animal models. These data will inform potential second-phase research, which will focus on further developing the lead compound for oral application.
"The partnership with Sanford-Burnham exemplifies our commitment to developing treatments for the world's most challenging and neglected diseases, such as dengue fever," adds Geoffrey Dow, CEO of 60° Pharmaceuticals. "Only a combined effort can help address these challenges and make a real difference in the lives of millions of people living in regions affected by the dengue virus. Our goal is to find a treatment that is affordable, safe in warm and humid climates, and easy to administer."
Successfully designing a small-molecule inhibitor of furin that is safe and effective could have implications far beyond dengue fever. Given the protein's prevalence and important role in the lifecycle of a variety of viruses, a potent inhibitor could eventually lead to treatment options for many more viral diseases, affecting hundreds of millions of people worldwide.
About Dengue Fever
Dengue fever is an infectious tropical disease caused by the dengue virus, which is transmitted by mosquitoes. According to the Centers for Disease Control and Prevention, more than one-third of the world's population is living in areas at risk for transmission of the virus. Dengue infection is a leading cause of illness and death in the tropics and subtropics and as many as 100 million people are infected every year. There are currently no vaccines to prevent infection with the virus and the most effective protective measures so far have been those that protect from mosquito bites. Data on the global economic impact of dengue fever are not available, but according to a 2012 study by 60° Pharmaceuticals, the global economic burden of dengue fever is at least $1.7 billion per year.
About Sanford-Burnham Medical Research Institute
Sanford-Burnham Medical Research Institute is dedicated to discovering the fundamental molecular causes of disease and devising the innovative therapies of tomorrow. The Institute consistently ranks among the top five organizations worldwide for its scientific impact in the fields of biology and biochemistry (defined by citations per publication) and currently ranks third in the nation in NIH funding among all laboratory-based research institutes. Sanford-Burnham utilizes a unique, collaborative approach to medical research and has established major research programs in cancer, neurodegeneration, diabetes, infectious and inflammatory, and childhood diseases. The Institute is especially known for its world-class capabilities in stem-cell research and drug-discovery technologies. Sanford-Burnham is a U.S.-based, nonprofit public benefit corporation, with operations in San Diego (La Jolla), Calif., and Orlando (Lake Nona), Fla. For more information, news, and events, please visit us at sanfordburnham.org.
About 60° Pharmaceuticals, LLC
60° Pharmaceuticals, LLC, is a new pharmaceutical company based in Washington, D.C. The company was formally launched in March 2012. The core business of the company is to discover, develop, and distribute new drugs for neglected diseases. 60° Pharmaceuticals is operating with a philanthropic-for-profit structure that will allow the company to invest in diseases with more modest markets than traditional pharmaceutical companies. The company's intellectual property and innovation strategy is based on a crowdsourcing platform. 60° Pharmaceuticals has been seeking partners to discover a new drug to treat dengue fever. Other neglected disease indications will follow in the future. For additional information, please visit 60degreespharma.com.