THE Cystic Fibrosis Foundation has lined up yet another big gun in the proteomics field to zero in on potential protein biomarkers and therapeutic targets: After signing research collaborations with Proteome Systems and Affinium Pharmaceuticals, the drug discovery arm of the Cystic Fibrosis Foundation has now turned to Oxford Glycosciences as its third commercial partner in proteomics.
Cystic Fibrosis Foundation Therapeutics (CFFTI), the foundation’s non-profit drug discovery division, said last week that it will pay OGS as much as $5.5 million for its services in an effort to discover and validate serum biomarkers and potential therapeutics for cystic fibrosis and its associated pulmonary conditions.
The payments include an upfront technology access fee, research fees, and potential discovery and validation milestone fees.
This deal reflects CFFTI''s focus on discovering biomarkers for monitoring the condition of infants diagnosed with cystic fibrosis, and for assessing the efficacy of treatments for the disease during clinical trials. Currently the most widely-used methods for monitoring the progression of cystic fibrosis involve studying slow changes in pulmonary function.
“If we can find better biomarkers of efficacy of intervention, we think that would be a very important tool as we move forward in drug development,” said Robert Beall, CFFTI''s president and CEO.
Beall added that CFFTI plans to spend $13.5 million over the next four and a half years to fund proteomics research into cystic fibrosis. The foundation recently received a $20 million gift from the Gates Foundation and $25 million from the Marsico family, a spokesperson said.
While proteomics is not the only avenue that CFFTI has chosen to pursue its research, Beall said the foundation has alighted upon the discipline because of its potential for uncovering disease pathways, and for mapping out the interactions of the proteins involved in the disease, including the protein encoded by the CFTR gene, which is known to be associated with cystic fibrosis.
“It''s our frustration and our desire to find new targets,” he added. “You don''t know [what might work] until you try.”
To choose its partners in proteomics, Beall said CFFI entertained proposals from ten parties, held workshops to assess their scientific value, and chose six programs to fund. CFFTI has already announced programs with Proteome Systems, aimed at studying the proteins found in the lungs of patients with the disease, and with Affinium, aimed at studying the protein-protein interactions of the CFTR protein and other membrane proteins using affinity pull-down experiments.
The foundation chose to start its own drug discovery infrastructure in part because few pharma and biotech companies were willing to support basic research programs for the disease, Beall said. Cystic Fibrosis affects about 30,000 children and adults in the US.
In the OGS collaboration, the company will use its 2D gel electrophoresis and mass spectrometry platform to study samples provided by the foundation''s academic collaborator, Margaret Leigh, a professor of pediatrics and cystic fibrosis researcher at the University of North Carolina, Chapel Hill. CFFTI will have access to “everything we can do,” said Andrew Lyall, OGS'' director of proteome discovery.
Under the agreement, CFFTI retains exclusive rights to develop therapeutics for cystic fibrosis from any discoveries OGS makes, and OGS will have exclusive rights to develop databases, prognostic and diagnostic products, and to pursue therapeutic applications outside of cystic fibrosis. Both parties are also entitled to royalties from sales of products owned exclusively by the other. — JSM