By Jill Panetta, Chief Scientific Officer, PKD Foundation
Polycystic Kidney Disease (PKD) Awareness Week has been designated the week of June 18 to 24, 2012. This week allows the PKD Foundation to be able to fulfill part of its mission to people with PKD — to increase awareness of a disease which few know about and raise the understanding and the impact that PKD has on patients and their families.
Autosomal dominant polycystic kidney disease (ADPKD) is a hereditary systemic disorder that most notably affects the kidneys, causing bilateral renal cysts and loss of renal function. PKD is relentlessly progressive. As the disease develops, cysts appear on both kidneys and the kidneys themselves enlarge, often up to five times their normal size. Hypertension and pain are common. Patients usually become symptomatic after the age of thirty to forty when the latent disease begins to show itself. As the kidneys begin to fail, patients suffer life threatening cardiovascular disease, including aneurysms, strokes and heart attacks. For the half of PKD victims that develop kidney failure, dialysis and transplantation are the only available treatments.
Accelerating Treatments to Patients (ATP)
The Polycystic Kidney Disease Foundation’s mission says it best – discover treatments and a cure for PKD; improve the lives of those it affects. Continuous progress has been made toward these goals, thanks to the PKD Foundation’s efforts to harness the best minds in science, recruit the finest research talent and foster understanding of PKD within the clinical setting. The PKD Foundation’s significant and productive investments in basic PKD research are now ready for harvest. The field of PKD is ripe for a significant growth in translational research and development that will more effectively harness basic discoveries and spawn new therapies for PKD.
With so much at stake for people with polycystic kidney disease, the PKD Foundation has launched its Accelerating Treatments to Patients (ATP) program. ATP is a holistic integrated research and development initiative that was launched over 2 years ago. Each program within ATP touches one of the following areas: new discoveries in basic PKD science, the development of potential clinical candidates through a drug repurposing strategy, development of registration endpoints for clinical development, support of clinical trials through education and awareness programs and scientific meeting support.
The Foundation cannot do this work alone. We are committed to pursuing all promising avenues, leveraging assets and building significant collaborations and relationships. Through the Foundation’s tissue donation program, PKD patients who are undergoing a nephretomy have the opportunity to donate their kidney(s) to basic science research labs, thus allowing patients to make a difference and help move the research forward in the understanding of disease pathways and mechanisms involved in disease expression. Partnering with the biotech and pharmaceutical industries is critical to the success of the drug repurposing program and accelerating the development of treatments to patients. Another example of the Foundation bringing researchers together is the PKD Outcomes Consortium, a collaboration between the PKD Foundation, Critical Path Institute, members of the pharmaceutical industry, PKD investigators/clinicians, and the FDA with the goal to facilitate clinical trial development through the development of registration endpoints.
To learn more about the Foundation and its education and awareness programs, what it is doing to support research and find treatments for patients, and clinical trial opportunities, please visit www.pkdcure.org.
Mark your calendars for PKD Awareness Week June 18 to June 24 2012 and join us in our fight against this devastating disease.
Listen to a podcast with Gary Godsey, President and CEO of the PKD Foundation.