ST. LOUIS—The Critical Path Institute's PKD Consortium hasentered a collaboration with the scientists at software and consulting firmPharsight to develop quantitative models of kidney disease.
The collaboration is the latest step in the Critical PathInstitute (C-Path) PKD Consortium's quest to advance CDISC research datastandards and an imaging biomarker for autosomal dominant polycystic kidneydisease (ADPKD), which affects one in 500 Americans and more than 12 millionpeople worldwide.
The consortium is led by C-Path and funded through a grantfrom the PKD Foundation and philanthropic donations. Additionally, arepresentative from the U.S. Food and Drug Administration (FDA) serves as anactive advisor to the consortium. The consortium also includes four leadingacademic medical centers: Tufts University, the University of Colorado Denver,Emory University and the Mayo Clinic.
The primary goals of the PKD Consortium are to develop CDISCresearch data standards for PKD and to use clinical data from PKD patientscollected over many years through patient registries and clinical trials tosupport the FDA's qualification of an imaging biomarker, total kidney volume(TKV), as an endpoint in drug development trials.
Financial terms and other details of the deal areconfidential and have not been released.
According to Lisa Romero, communications and strategicoutreach manager for Critical Path Institute, the company "works with manycollaborators on its various consortia/projects and is looking forward to aproductive new partnership between its PKD Consortium and Pharsight."
"The PKD Consortium is very pleased about this significantpartnership to create and study quantitative models of disease progression inpatients with autosomal dominant polycystic kidney disease," she says, notinghow much need there is for a biomarker that will assess disease progressionearlier and make it easier to give patients the right kind of therapy at theright time.
"ADPKD is a debilitating genetic disease affecting more than600,000 Americans and 12 million people worldwide for which there is currentlyno known cure or effective treatment," explains Dr. Ronald Perrone, co-directorof the PKD Consortium and professor of Medicine at Tufts University School ofMedicine.
The timeline to develop a quantitative disease progressionmodel, Romero notes, depends on C-Path's plans and the plans of collaboratorsand pharmaceutical companies. She adds that the model provides an incentive forindustry to develop a treatment for this disease since there is an approvalpath.
"A qualified measure will encourage drug developmentorganizations to test potential new disease-modifying treatments for ADPKD,"she adds.
Dr. Eslie Dennis, executive director of the PKD Consortiumat C-Path, says the project leverages the power of collaboration.
"Traditional endpoints of renal function only show changesvery late in the course of the disease, making it difficult to assess theeffectiveness of new medications," Dennis says. "There is critical need for abiomarker that will assess disease progression at an earlier stage when patientsmay be more likely to respond to new therapies."
