The largest contract ever awarded by a voluntary health organization for drug discovery was announced by the Cystic Fibrosis Foundation$30 million to Aurora Biosciences (San Diego, CA). Under terms of the five-year agreement, Aurora will identify and develop two to three new cystic fibrosis (CF) drug candidates ready for the preclinical and clinical research stages. The CF Foundation will pay approximately $30 million to Aurora including research support and early milestones. With completion of specific clinical milestone achievements, additional payments would be made, representing a potential investment by the foundation to Aurora of approximately $46.9 million.
While it is customary for foundations to fund academic research, this kind of arrangement is novel. Because of the relatively small number of individuals affected by CF, pharmaceutical companies have been unwilling to invest in early discovery efforts for CF drugs. The CF Foundation, therefore, decided to diversify and expand its drug discovery efforts with this project. This innovation will feed the foundation's drug development pipeline that will now stretch from drug discovery through every stage of evaluation.
Using Aurora's high-throughput screening technology, recently adapted for CF research, Aurora scientists will be able to screen hundreds of thousands of chemical compounds from Aurora's chemical library to see whether any of these compounds correct or modify defective CF cellular targets. The goal of the CF Foundation/Aurora project is to dramatically shorten the usual 14-year period it takes to bring a new drug to market and to reduce the development costs substantially.
Although this CF Foundation/Aurora project represents a substantial financial investment, the total cost of developing two to three new CF products would be far less than the $400$500 million often spent by industry to produce one single marketable drug. The new CF Foundation strategy for drug discovery is possible for two primary reasons, according to Robert J. Beall, president and CEO of the CF Foundation, "Obviously, new technologies have emerged. At the same time, several pieces of the CF molecular puzzle have been elucidated. Combining these two factors will greatly accelerate the CF drug discovery and development pipeline."
"The CF Foundation is launching an industry approach to drug discovery that is unrivaled by a voluntary health organization," said Stuart J.M. Collinson, chairman, CEO, and president of Aurora. "The important science that CFF has funded over the years in university labs and medical centers has created new opportunities for therapies. To convert these opportunities quickly and efficiently into compounds that can be tested in the clinic requires skill sets, technologies and expertise that may be beyond those in the basic research lab. These are the capabilities Aurora brings to this partnership."
A major effort of the project will be the search for a strategy to correct a defective protein in the cells lining the airways of people with CF; the genetic mutation (and its protein) were discovered in 1989 by a team of CF Foundation-supported scientists. The defective protein interferes with the proper movement of chloride and sodium across the cells of the airways producing a sticky mucus buildup in the lungs. Another treatment strategy will focus on finding novel agents to defeat chronic bacterial infections in CF lungsthe primary cause of death for those with CF.
Funds for the CF Foundation/Aurora drug discovery project are being raised by the CF Foundation's major gifts campaign, Building Bridges to a Cure, which includes donations from private and family foundations, as well as individuals. The drug discovery initiative was given a major boost in 1999 with a gift from the Bill and Melinda Gates Foundation that totaled $20 million.
For more information: The Cystic Fibrosis Foundation, 6931 Arlington Rd., Bethesda, MD 20814. Tel: 800-344-4823.
Edited by Laura DeFrancesco