Wake Forest University School of Medicine’s Rare Inherited Kidney Disease team will enhance its collaboration with the Critical Path Institute (C-Path), an independent nonprofit established in 2005 as a public-private partnership in response to the FDA’s Critical Path Initiative.
As a result of a contract awarded by the FDA to C-Path’s Polycystic Kidney Disease Outcomes Consortium, the group will study an inherited kidney disease called autosomal dominant tubulointerstitial kidney disease (ADTKD), and the Wake Forest Rare Inherited Kidney Disease team will play a crucial role.
ADTKD is an uncommon condition caused by mutations. It is estimated to affect approximately 25,000 individuals in the U.S. ADTKD causes a form of inherited kidney disease that leads to the need for dialysis or transplantation at an average age of 45 years. The child of an affected parent has a 50% chance of being affected, so many family members may be affected.
Anthony Bleyer, M.D., professor of nephrology at Wake Forest University School of Medicine, along with the Wake Forest Rare Inherited Kidney Disease team, were instrumental in the identification of the genetic cause of these conditions. Wake Forest University School of Medicine has the only registry of patients with this disorder in the U.S.
There are no treatments for ADTKD, and the development of treatments is hindered by limited characterization of the disease and its risk factors.
C-Path, which has a high level of expertise in the analysis of disease progression data, will analyze data from the Wake Forest University School of Medicine registry to better understand risk factors and biomarkers for ADTKD. The medical school will also prospectively study disease progression.
“We are very excited about this grant and our collaboration with C-Path and see this as a tremendous opportunity to learn about ADTKD and help individuals with this disorder,” Bleyer said. “We will recruit patients from all over the U.S. who have ADTKD and will follow them over four years.”
Bleyer said a strength of the study is that individuals can undergo laboratory testing locally, and most individuals will not need to travel to Winston-Salem to participate in the study.
“C-Path has made great strides in its work in autosomal dominant polycystic kidney disease, and we are happy to be working with them on this new endeavor,” Bleyer said. “It is promising for patients with ADTKD, and we are thrilled that the FDA has provided our patients with hope and funding as we move toward finding a treatment for ADTKD.”
Patients who have or believe they have ADTKD are asked to contact kidney@wakehealth.edu so they can be part of this effort.